A Randomized, Open-label Study of the Safety and Efficacy of Multi- Vessel Intracoronary Delivery of Allogeneic Cardiosphere-Derived Cells in Patients With Cardiomyopathy Secondary to Duchenne Muscular Dystrophy
Phase: PHASE2
Conditions: Duchenne Muscular Dystrophy, Cardiomyopathy
Sponsor: Capricor Inc.

C O N FI D E N TI A LI T Y S T A T E M E N T
T his d o c u me nt is c o nfi d e ntial a n d pr o prietar y t o Ca pric or, I nc. a n d its affiliates. Acce pta nc e of t his d o c u me nt 
c o nstit utes a gree me nt b y t he reci pie nt t hat n o u n p u blis he d i nf or mati o n c o ntai ne d herei n will b e re pr o d uce d, 
p u blis he d, or ot her wise disse mi nate d or discl ose d or use d wit h o ut t he pri or writte n a p pr o val of Ca pric or, I nc. 
a n d its affiliates, e xce pt t hat t his d o c u me nt ma y be discl ose d i n a n y me di u m t o a p pr o priate cli nical
i n vesti gat ors, I nstit uti o nal Re vie w B o ar ds, a n d ot hers directl y i n v ol ve d i n t he cli nical i n vesti gati o n t hat is t he 
s u bj ect of t his i nf or mati o n u n d er t he c o n diti o n t hat t he y kee p t he i nf or mati o n strictl y c o nfi d e ntial. 
Cli ni c al Tri al Pr ot o c ol: C A P- 1 0 0 2- D M D- 0 1 ( H O P E)
St u d y Title:
A Ra n d o mize d, O pe n-la bel St u d y of t he Safet y a n d Efficac y of M ulti-
Vess el I ntrac or o n ar y Deli ver y of All o ge nei c Car di os p here- D eri ve d 
Cells i n Patie nts wit h Car di o m y o pat h y Sec o n d ar y t o D uc he n ne 
M usc ular D ystr o p h y [ H O P E- D uc he n n e ( H alt car di o m y O Pat h y 
pr o gr Essi o n i n D uc he n n e)] 
St u d y N u m ber:
C A P- 1 0 0 2- D M D- 0 1
St u d y P h ase:
P hase II
Pr o d uct N a me:
C A P- 1 0 0 2 All o ge nei c Car di os p here- Deri ve d Cells
I N D N u m ber:
I n dic ati o n:
Car di o m y o pat h y i n D M D
I n v esti g at ors:
M ulti ce nter
St u d y Pri n ci pal I n vesti gat or:
, M D, M P H 
S p o ns or:
Ca pric or, I n c.
8 8 4 0 Wils hire Bl v d. 2 n d fl o or 
Be verl y Hills, C A  9 0 2 1 1 
S p o ns or C o nt act
& Me dic al 
M o nit or: 
, M D  
, Ca pric or, I n c.
Ori gi n al Pr ot oc ol:
0 1 Ma y 2 0 1 5
A me n d me nt 1. 0:
2 7 J a n uar y 2 0 1 6
A me n d me nt 2. 0:
2 9 Fe br u ar y 2 0 1 6
A me n d me nt 3. 0:
1 8 J ul y 2 0 1 6

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 2 of 1 1 1
S Y N O P SI S
S p o ns or:
Ca pric or, I n c.
N a me of Fi nis he d Pr o d uct:
C A P- 1 0 0 2 All o ge nei c Car di os p here- Deri ve d Cells
N a me of Acti ve I n g re die nt:
Car di os p here- D eri v e d Cells 
St u d y Title:
A Ra n d o mize d, O pe n-la bel St u d y of t he Safet y a n d Efficac y of M ulti- Vessel I ntrac or o nar y 
Deli ver y of All o ge n eic C ar di os p here- Deri ve d Cells i n Patie nts wit h Car di o m y o pat h y 
Sec o n dar y t o D u c he n ne M usc ular D ystr o p h y [ H O P E- D uc he n ne (H alt car di o m y O P at h y 
pr o gr Essi o n i n D uc he n ne )]
St u d y N u m ber:
C A P- 1 0 0 2- D M D- 0 1
St u d y P h ase: P hase II
Pri m ar y O bjecti ve(s):
T o assess t he safet y a n d t olera bilit y of C A P- 1 0 0 2 a d mi nistere d b y m ulti- vessel i ntrac or o nar y 
i nf usi o n i n s u bjects wit h car di o m y o pat h y se c o n dar y t o D u c he n n e m usc ular d ystr o p h y 
( D M D).
Sec o n d ar y O bjecti ve(s):
T o assess efficac y of C A P- 1 0 0 2 a d mi nistere d b y m ulti- vessel i ntrac or o nar y i nf usi o n i n 
s u bjects wit h car di o m y o pat h y sec o n dar y t o D M D b y sta bilizi n g or i m pr o vi n g car dia c 
str uct ure, b y e v al uati n g f u ncti o nal stat us, a n d b y assess me nt of q ualit y of life.
St u d y Desi g n:
Male s u bjects at least 1 2 years of a ge, wit h car di o m y o pat h y sec o n dar y t o D M D, a n d m eeti n g 
all i ncl usi o n a n d n o e x cl usi o n criteria, will be ra n d o mize d. Eac h s u bject t h at meets all 
eli gi bilit y criteria will be ra n d o mize d i n a 1: 1 ma n n er t o eit h er i n vesti gati o nal pr o d uct ( C A P-
1 0 0 2) or us ual care. 
If ra n d o mize d t o i n vesti gati o nal pr o d uct, a s u bject will recei ve a n i ntrac or o nar y i nf usi o n of 
C A P- 1 0 0 2 (i nte n d e d t otal d ose of 7 5 milli o n [ 7 5 M] cells) i n t hree c or o n ar y arteries 
s u p pl yi n g t he t hree maj or car di ac territ ories of t he left v e ntricle of t h e heart (a nteri or, lat eral, 
i nferi or/ p osteri or). All t hree maj or car di ac territ ories will be treate d (i nf use d) d uri n g a si n gle 
pr oce d ure i n a n o pe n-la b el fas hi o n. 
S u bjects ra n d o miz e d t o recei ve us u al care will n ot recei v e a n i nf usi o n.  T he y will c o nti n ue t o 
be care d f or a n d treate d i n w hate ver m a n ner t h e i n vesti gat or d ee ms m ost a p pr o priate f or t he 
s u bject o n a n o n g oi n g b asis, e xce pt t hat us u al care ca n n ot i ncl u de treat me nt wit h C A P- 1 0 0 2
or ot her i n vesti gati o nal treat me nts.

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 3 of 1 1 1
A p pr o xi matel y 2 4, a n d n ot m ore t h a n 3 0, s u bjects will be ra n d o mize d i nt o t h e st u d y i n t w o 
se q ue nti al e nr oll me nt gr o u ps. Safet y d ata fr o m Gr o u p 1 will u n der g o a Dat a S afet y 
M o nit ori n g B o ar d ( D S M B) re vi e w pri or t o i nitiati o n of e nr oll me nt f or Gr o u p 2. 
T he first 6- 8 ra n d o mize d s u bjects will c o m prise Gr o u p 1, a n d will i ncl u de a mi ni m u m of 3 
s u bjects c o m pleti n g i ntrac or o nar y i nf usi o n wit h C A P- 1 0 0 2.  T he D S M B will c o n d uct a 
re vie w of i nteri m safet y data t hr o u g h 7 2 h o urs p ost - Da y 0 f or at least 3 i nf use d s u bjects a n d 
f or at least 6 s u bjects o verall.  
E nr oll me nt of Gr o u p 2 will be gi n per D S M B rec o m me n dati o ns f oll o wi n g t h eir re vie w of t h e 
7 2 h o ur safet y d ata fr o m Gr o u p 1.  Gr o u p 2 will i ncl u de a p pr o xi matel y 1 8 s u bjects. 
Scree ni n g a n d ra n d o mizati o n will c o nti n ue u ntil at t otal of 1 2 s u bjects are i nf use d wit h 
C A P- 1 0 0 2 or 3 0 s u bjects are ra n d o mize d i nt o t he st u d y, w hic he ver c o mes first.  
  
  
  
S u bject i nf usi o ns will be sta g gere d b y at least t hree da ys t o all o w f or d etecti o n of ac ute 
pr oce d ure-relate d c o m plicati o ns s h o ul d t he y o cc ur. A d diti o nall y, s u bject i nf usi o ns i n Gr o u p 
2 will n ot be gi n u ntil at least t w o wee ks after t he fi nal i nf usi o n occ urs i n Gr o u p 1, t o all o w 
f or c o m pleti o n of t he Gr o u p 1 safet y data re vie w t hr o u g h 7 2 h o urs p ost D a y 0. 
N o scree ni n g pr oce d ures will ta ke place u ntil t h e s u bject is f ull y i nf or me d of t h e researc h a n d 
si g ns t he i nf or m e d c o nse nt or pr o vi des asse nt. S u bjects 1 8 ye ars of a ge or ol der m ust ha ve 
t he a bilit y t o pr o vi de i nf or me d c o nse nt a n d f oll o w u p wit h pr ot oc ol pr oce d ures. S u bjects at 
least 1 2 years of a ge b ut y o u n ger t ha n 1 8 years of a ge m ust ha ve t he a bilit y t o pr o vi de asse nt 

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 4 of 1 1 1 
al o n g wit h t he s u bject’s pare nt or g u ar dia n pr o vi di n g per missi o n f or st u d y partici p ati o n.
Ra n d o mizati o n will ta ke place wit hi n 3 0 da ys of t he first scree ni n g pr oce d ure. Aft er 
c o m pleti o n of t he scree ni n g pr o ce d ures, eli gi ble s u bjects ra n d o miz e d t o acti ve treat m e nt ar m 
will recei v e C A P- 1 0 0 2 a d mi nistere d via i ntrac or o nar y i nf usi o n o n Da y 0. Da y 0 f or eli gi ble 
s u bjects ra n d o mi ze d t o t he us ual care ar m will occ ur 7 da ys after t he d ate of ra n d o mi zati o n. 
All ra n d o mize d s u bjects will ha ve st u d y visits at Da y 3, Wee ks 2 a n d 6, a n d M o nt hs 3, 6 a n d 
1 2 p ost Da y 0. Ke y safet y ass ess m e nts s uc h as s u d de n u ne x pecte d deat h, a n d ot h er maj or 
a d verse car di ac e ve nts ( M A C E), will be assesse d f or all s u bjects, wit h t he e x ce pti o n of t h ose 
o nl y rel e va nt t o s u bjects assi g ne d t o t he acti ve tr eat m e nt ar m, s u c h as  n e w TI MI fl o w 0- 2 or 
TI MI m y o car dial perf usi o n gra de ( T M P G) 0- 2 ( persisti n g > 3 mi n utes after ter mi nati o n of 
cell i nf usi o n). Si nce C A P- 1 0 0 2 is gr o w n fr o m d o n ors u nrelate d t o reci pie nts, h u m oral a n d 
cell ular i m m u ne res p o ns es will als o be e val uat e d. 
D S M B O versi g ht:
T he Data a n d Safet y M o nit ori n g B oar d ( D S M B) will meet per c harter, at least se mia n n uall y t o 
re vie w a vaila ble data, a n d will c o n d uct a n i nteri m re vie w of s afet y d ata f or Gr o u p 1 s u bjects 
t hr o u g h 7 2 h o urs p ost- d a y 0.  T he y will re vie w b ot h safet y a n d efficac y data after all s u bjects 
ha ve c o m plete d 6 m o nt hs of f oll o w- u p.
St u d y P o p ul ati o n a n d R ati o n ale:
A p pr o xi matel y t w e nt y-f o ur ( 2 4), a n d n ot m ore t ha n 3 0, male s u bjects at least 1 2 years of a ge, 
wit h car di o m y o p at h y sec o n dar y t o D M D a n d left ve ntric ular s car as assess e d b y l ate 
ga d oli ni u m e n ha n ce me nt ( L G E) car dia c M RI i n at least 4 se g me nts, will be c o nsi dere d f or 
e nr oll me nt i n t he st u d y.
T his sa m ple size is a de q uate t o s u p p ort a n assess m e nt of safet y a n d 
t olera bilit y of i ntrac or o n ar y i nf usi o n of C A P- 1 0 0 2 i n t his disease p o p ulati o n, as well as a n 
e x pl orat or y ass ess m e nt of efficac y. E nr oll me nt will c o nti n ue u ntil 1 2 s u bjects ha ve 
c o m plete d i nf usi o n wit h C A P- 1 0 0 2 or 3 0 s u bjects ha ve b ee n ra n d o mize d, w hic he ver o cc urs 
first.
Car di o m y o pat h y is c urre ntl y t he lea di n g ca use of deat h i n D M D p atie nts ( S p ur ne y 2 0 1 1,
Passa ma n o, Ta glia et al. 2 0 1 2), ha vi n g rece ntl y s u p pla nte d res pirat or y ca us es as a r es ult of 
treat me nt i m pr o v e me nts f or t hat as pect of t he dis ease. Affli cte d pati e nts lac k a f u ncti o nal 
d ystr o p hi n pr otei n a n d e x perie nce pr o gressi ve m uscle wea k n ess starti n g at a n earl y a ge a n d 
ulti m atel y a n a b bre viate d lifes pa n. Gi ve n t he gl o b al a n d pr o gressi ve nat ure of t he disease, 
rarel y are t hes e patie nts c o nsi dere d f or h eart tr a ns pla nt, use of mec ha nical car diac assist 
de vices is lar gel y e x peri me ntal ( R ya n, J efferies et al. 2 0 1 4), a n d t h e effecti v e ness of t h e 
t hera pies i n use or i n d e vel o p me nt f or D M D o n t h e car di ac ma nifestati o ns of t he disease is 
ge n erall y a s ec o n d ar y c o nsi derati o n. C A P- 1 0 0 2 is i nte n de d t o tar get t he car di o m y o pat h y i n 
D M D as o p p ose d t o t he s keletal m y o p at h y. 

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 5 of 1 1 1
I ncl usi o n Criteri a:
Eac h s u bject m ust meet all of t he f oll o wi n g i ncl usi o n crit eria t o be ra n d o mi ze d i n t he st u d y: 
1.
Male s u bjects 1 8 years of a ge or ol d er m ust be a bl e t o pr o vi de i nf or me d c o nse nt a n d 
f oll o w u p wit h pr ot oc ol pr oce d ures. Male s u bjects at least 1 2 years of a g e b ut y o u n ger 
t ha n 1 8 years of a ge m ust be a ble t o pr o vi de ass e nt wit h pare nt or g uar di a n pr o vi di n g 
per missi o n f or st u d y p arti ci pati o n. O nl y male s u bj ects will be ra n d o mize d i nt o t his 
st u d y.
2.
D oc u me nte d dia g n osis of D uc he n ne M us c ular D ystr o p h y b y ge netic m utati o n a nal ysis. 
3.
Car di o m y o pat h y wit h left ve ntric ul ar scar b y L G E i n at least 4 se g me nts as assesse d b y 
c o ntrast-e n h a nce d M RI a n d E F > 3 5 % at t h e ti m e of scree ni n g.  
4.
Use of e vi de nce b ase d m e dical-t hera p y i n acc or d a nce wit h t he “ D M D Care 
C o nsi d erati o ns W or ki n g Gr o u p” g ui deli n es f or t he ma na ge me nt of D M D ( B us h b y, 
Fi n kel et al. 2 0 1 0a, B us h b y, Fi n kel et al. 2 0 1 0 b), f or at least t hree m o nt hs pri or t o 
si g ni n g t he c o nse nt f or m ( or, pr o vi di n g asse nt) or d oc u me nte d c o ntr ai n dicati o n or 
i nt olera nce or patie nt prefere nce.   
5.
S u bjects m ust be ta ki n g s yste mic gl uc o c ortic oi ds f or at least si x m o nt hs pri or t o 
scree ni n g. 
6.
S u bjects m ust be 1 2 years of a ge or ol d er at ti m e of scree ni n g
7.
S u bjects m ust be a p pr o priate ca n di d ates f or car dia c cat heterizati o n a n d i ntrac or o nar y 
i nf usi o n of C A P- 1 0 0 2, i n t h e j u d ge me nt of t he site’s i nter ve nti o nal car di ol o gist.
E xcl usi o n Criteri a:
S u bjects t h at m eet a n y of t h e f oll o wi n g e xcl usi o n criteria will n ot be per mitte d t o e nr oll i nt o 
t he st u d y: 
1.
T hera p y wit h i ntra ve n o us i n otr o pic or vas oacti v e me dicati o ns at t h e ti me of scree ni n g. 
2.
I n a bilit y t o u n der g o car di ac c at heterizati o n a n d/ or M RI wit h o ut ge neral a nest h esia.
3.
I m m u n ol o gic i nc o m pati bilit y wit h all a v aila ble M aster Cell Ba n ks ( M C Bs) b y si n gle-
a nti ge n b ea d ( S A B) s er u m a nti b o d y pr ofili n g.
4.
Pl a n ne d or li kel y maj or s ur ger y i n t he n e xt 1 2 m o nt hs after pla n ne d ra n d o mizati o n. 
5.
Left Ve ntric ul ar Assist D e vices ( L V A D) or t h ose s u bjects acti vel y i n t he pr ocess of 
ac q uiri n g a L V A D.
6.
C o ntrai n dicati o n t o car di ac M RI. 
7.
K n o w n h y p erse nsiti vit y t o c o ntrast a ge nts. 
8.
Esti mate d gl o mer ular filtrati o n rate ( G F R) < 6 0 m L/ mi n, as calc ulate d b y t he C K D- E PI 
c ystati n C e q u ati o n (I n ker, Sc h mi d et al. 2 0 1 2). 

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 6 of 1 1 1
9.
Acti ve i nfecti o n n ot res p o nsi ve t o treat m e nt.
1 0.
Acti ve s yst e mic aller gic reacti o n(s), c o n necti ve tiss ue disease or a ut oi m m u ne 
dis or der(s).
1 1.
Hist or y of car di ac t u m or or car di ac t u m or de m o nstrate d o n scree ni n g M RI.
1 2.
Hist or y of pre vi o us ste m cell t h era p y.
1 3.
Hist or y of us e of me dicati o ns liste d i n A p p e n di x 3 wit hi n 3 m o nt hs pri or t o si g ni n g t he 
I C F / Asse nt t hr o u g h c o m pleti o n of t he st u d y i nf usi o n. 
1 4.
K n o w n m o derat e-t o-se vere a ortic ste n osis/i ns ufficie nc y or se v ere mitral 
ste n osis/re g ur gitati o n.
1 5.
C urre nt acti v e alc o h ol or dr u g a b use. 
1 6.
K n o w n hist or y of H u m a n I m m u n o deficie nc y Vir us ( HI V) i nfecti o n.
1 7.
K n o w n hist or y of c hr o ni c viral he patitis.
1 8.
A b n or mal li ver f u ncti o n ( A L T/ A S T > 1 0 ti mes t he u p per refere nce ra n ge) a n d/ or 
wit h o ut a re versi ble, i de ntifia ble ca use.
1 9.
K n o w n h y p erse nsiti vit y t o b o vi ne pr o d ucts.
2 0.
K n o w n h y p erse nsiti vit y t o di met h yl s ulf o xi d e ( D M S O).
2 1.
U nc o ntr olle d dia betes ( H b A 1c > 9. 0).
2 2. I n a bilit y t o c o m pl y wit h pr ot oc ol-relat e d pr oce d ures, i ncl u di n g re q uire d st u d y visits.
2 3.
A n y c o n diti o n or ot h er reas o n t hat, i n t he o pi ni o n of t he I n vesti gat or or M e dical 
M o nit or, w o ul d re n der t h e s u bject u ns uita ble f or t he st u d y.
2 4.
C urre ntl y recei vi n g i n v esti gati o nal treat me nt o n a n ot her cli nical st u d y or e x pa n de d 
access pr ot o c ol, i ncl u di n g a n y of t he f oll o wi n g:
•
Recei ve d i n vesti gati o nal i nter ve nti o n wit hi n 3 0 da ys pri or t o ra n d o mizati o n
•
Treat m e nt a n d/ or a n i nc o m plete f oll o w- u p t o treat me nt wit h a n y i n vesti gati o nal cell 
base d t h era p y wit hi n 6 m o nt hs pri or t o ra n d o miz ati o n
•
Acti ve partici pati o n i n ot her res ear c h t h era p y f or car di o vasc ular re pair/re ge nerati o n

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 7 of 1 1 1
Test Pr o d u ct, D ose, a n d M o de of A d mi nistr ati o n:
C A P- 1 0 0 2 is a n i n vesti gati o nal pr o d uct c o nsisti n g of all o ge neic car di os p h ere- d eri ve d cells 
( C D Cs). Eac h d ose of C A P- 1 0 0 2 is s u p plie d as a c o nce ntrate d s us pe nsi o n of 2 5 milli o n 
C D Cs 
I n cases w h ere a d ose of 1 2. 5 milli o n C D Cs 
is utilize d, t he h alf d ose is a d mi nistere d i n a half v ol u me.
All s u bjects ( Gr o u ps 1 a n d 2) assi g n e d t o t he acti v e treat me nt ar m will recei ve a pr ot oc ol-
defi ne d t otal d ose of C A P- 1 0 0 2 cells i nf use d t o t he l eft v e ntricle car diac territ ories (a nteri or, 
lateral, i nferi or/ p osteri or). A si n gle ma xi m u m d ose a d mi nistrati o n of 2 5 M C A P- 1 0 0 2 cells or 
1 2. 5 M C A P- 1 0 0 2 cells will be d eli v ere d wit h a T E R U M O Fi necr oss T M M G g ui di n g cat h eter 
t o eac h car diac territ or y (i nte n de d t otal d ose t o all t hree territ ories is 7 5 M cells).
A d diti o nall y, a n i nter me diate was h s ol uti o n is a d mi nistere d t o eac h s u bject bet wee n b ol uses 
of C A P- 1 0 0 2. 
Us u al C are:
S u bjects ra n d o miz e d t o us ual care will recei ve o pti mal me dical t h era p y acc or di n g t o t he 
treat me nt pla n of t h eir pri mar y D M D d oct or a n d site i n vesti gat or, i nf or me d b y t h e e vi de nce 
base d m e dical-t hera p y i n acc or da nce wit h t he “ D M D Care C o nsi derati o ns W or ki n g Gr o u p” 
g ui deli nes f or t he m a na ge me nt of D M D ( B us h b y, Fi n kel et al. 2 0 1 0a, B us h b y, Fi n kel et al. 
2 0 1 0 b).  Us ual care s h o ul d n ot i ncl u de ot her i n vesti gati o nal t h era pies u ntil t he M o nt h 1 2 
st u d y visit is c o m plete d.  
D ur ati o n of F oll o w- U p:
All ra n d o mize d s u bjects will be f oll o w e d o ut t o o ne ye ar p ost- Da y 0. A n n u al f oll o w- u p 
p h o ne calls will be c o n d u cte d f or a n a d diti o nal f o ur years after t his ti me f or s u bjects t h at 
pr o vi de c o nse nt a n d/ or asse nt. T hese a n n u al f oll o w- u p p h o ne c alls will c o nti n ue i n c o ncert 
wit h a s u bject’s n or mal, a n n ual cli nical f oll o w- u p w hic h ma y i ncl u de a n n u al car dia c M RI 
w here cli nicall y i n dicate d.
D ue t o t he s mall size of t he trial p o p ulati o n, c o nti n ue d parti ci pati o n f or all s u bjects is cr ucial 
t o data q ualit y. T h eref ore, n o s u bject will be assesse d as “ L ost t o F oll o w- U p” u nless t he site 
has atte m pte d t o c o ntact t he s u bject ( or p are nt/ g uar dia n) b y p h o ne ( or e mail or ot her s u bject-
preferre d m et h o d of c o m m u nicati o n) o n at least 3 differe nt d a ys, pl us a n att e m pt t o c o ntact 
t he s u bject ( or pare nt/ g uar dia n) b y certifie d mail.

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 8 of 1 1 1
Effic ac y Assess me nts:
T he f oll o wi n g will be e val uate d as e x pl orat or y efficac y e n d p oi nts at si x- a n d t w el v e- m o nt h 
f oll o w- u p visits:
1.
Car diac Str uct ural: A bs ol ute a n d relati ve c ha n ge i n para meters meas ure d b y car diac 
M RI, i n cl u di n g:
a.
Left ve ntric ular E F ( %);
b.
Left ve ntric ular e n d- di ast olic v ol u me a n d e n d-s yst olic v ol u me;
c.
Left ve ntric ular str o ke v ol u m e;
d.
Re gi o nal left ve ntric ular f u ncti o n assess me nt;
e.
Left ve ntric ular  L G E e x press e d as a perce nt of left ve ntric ular mass;
f.
Left ve ntric ul ar L G E e x presse d i n gra ms;
g.
Left ve ntric ular via ble m ass e x presse d i n gra ms;
h.
Left ve ntric ular circ u mfere nti al strai n;
i.
N u m ber of left v e ntric ular se g me nts wit h L G E.  
2.
F u ncti o nal: S erial c ha n ges i n Perf or ma nce of U p p er Li m b ( P U L) s cale, s pir o metr y, a n d 
6- mi n ute wal k test ( 6 M W T) w he n dee me d a p pr o priate b y t he I n v esti gat or.
3.
Q ualit y of Life: C h a n ge i n Pe ds Q L ( Pe diatric Q u alit y of Life I n ve nt or y), i n cl u di n g t he 
car dia c m o d ule, a n d P O D CI A d ol esce nt Q uesti o n naire.
4.
Bi o mar kers: Oste o p o nti n, S T 2, I L- 1 0, a n d Galecti n- 3; f or s u bjects pr o vi di n g 
c o nse nt/ asse nt, a d diti o nal sa m ples f or e x pl orat or y a nal ys es will be c oll ecte d, arc hi ve d, 
a n d a vaila ble f or f ut ure t esti n g.
S afet y Assess me nts:
T he st u d y will m o nit or t he pr o p orti o n of s u bjects e x peri e nci n g a n y of t he f oll o wi n g e ve nts 
d uri n g or p ost i ntrac or o n ar y i nf usi o n deli ver y. F or c o m paris o n, t hese s a me e ve nts, as 
a p plica ble, will be m o nit ore d i n us ual care s u bjects o n or after Da y 0, usi n g 9: 0 0 a m o n Da y 
0 as t he refere nce ti me p oi nt:
1.
Ne w TI MI fl o w 0- 2 or TI MI m y o car di al perf usi o n gra de ( T M P G) 0- 2, n ot e d 
i m m e diatel y f oll o wi n g i ntrac or o n ar y i nf usi o n of C A P- 1 0 0 2 a n d persisti n g > 3 mi n after 
cell i nf usi o n, des pite i ntrac or o nar y vas o dil at or a d mi nistrati o n.
2.
S u d de n u ne x pecte d deat h wit hi n 7 2 h o urs of i ntrac or o nar y i nf usi o n defi ne d as occ urri n g 
wit hi n o ne h o ur of s y m pt o m o nset, or u n- wit n esse d deat h i n a p ers o n pre vi o usl y 
o bser ve d t o be w ell wit hi n t he prece di n g 2 4 h o urs wit h o ut a n i de ntifie d ca use.
3.
Maj or a d vers e car diac e v e nts ( M A C E) wit hi n 7 2 h o urs of i ntrac or o nar y i nf usi o n, 
i ncl u di n g deat h, n o n-fat al m y ocar dial i nfarcti o n a n d h os pit aliz ati o n f or car di o vasc ular 
e ve nt (i ncl u di n g heart fail ure h os pitalizati o ns). E vi de nce of m y ocar dial i nj ur y will be 
assess e d b y a r ule o ut MI car diac e nz y m e pr ot o c ol perf or me d f oll o wi n g a d mi nistrati o n 
of C A P- 1 0 0 2. Tr o p o ni n a n d C K- M B will be o btai ne d e ver y 8 h o urs ( ± 3 0 mi n utes) at 
mi ni m u m f or 2 0- 2 4 h o urs after C A P- 1 0 0 2 i nf usi o n.

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 9 of 1 1 1
T he st u d y will e val uate t h e f oll o wi n g d uri n g t h e si x a n d t wel ve m o nt h f oll o w- u p peri o d:
1.
S u d de n u ne x pecte d deat h defi n e d as o cc urri n g wit hi n o ne h o ur of s y m pt o m o nset, or 
u n wit nesse d deat h i n a p ers o n pre vi o usl y o bser v e d t o be well wit hi n t he prece di n g 2 4 
h o urs wit h o ut a n i de ntifie d ca use.
2.
Maj or a d vers e car diac e v e nts ( M A C E), i n cl u di n g deat h, n o n-fatal m y o car dial i nfarcti o n, 
h os pitalizati o n f or car di o vasc ular e ve nt (i ncl u di n g heart fail ure h os pit aliz ati o ns), 
e mer ge nc y r o o m treat me nt f or heart fail ure (i ncl u di n g o ut patie nt i nf usi o n), left 
ve ntric ul ar assist de vice or heart tr a ns pla nt.
3.
A n y h os pit aliz ati o n d ue t o a car di o v asc ular ca us e.
4.
A n y i nter-c urre nt car di o v asc ular ill ness w hic h pr ol o n gs h os pitalizati o n. 
5.
De vel o p me nt of, or a n i n creas e i n, t he fre q u e nc y of V T wit h d urati o n of 3 0 sec o n ds or 
l o n ger ascertai ne d b y c o nti n u o us car diac r h yt h m m o nit ori n g.
6.
De vel o p me nt of i ncreas e d a nti- H u m a n Le u k o c yt e A nti ge n ( H L A) a nti b o d y le vels wit h 
de vel o p me nt of se nsitizati o n t o H L A a nti ge ns s p ecific t o t he C A P- 1 0 0 2 C D C d o n or at 
i m m u n ol o gicall y si g nifica nt titers.
7.
Pea k c ha n ge fr o m b aseli ne i n tr o p o ni n a n d C K- M B le vels f oll o wi n g C A P- 1 0 0 2 i nf usi o n.
Us u al C are S u bjects – C A P- 1 0 0 2 Access P ost- St u d y:
If t he a v aila ble e vi de n ce s u g gests a n a p pr o priate ris k/ be nefit pr ofile f or t his pr o d uct, 
Ca pric or ma y ( wit h D S M B a gree me nt) o p e n a n o pe n-la bel e xte nsi o n pr ot oc ol t o ma ke C A P-
1 0 0 2 a vaila ble f or Us ual Care s u bjects after t he y c o m plete t h e 1 2- m o nt h st u d y ass ess me nt 
peri o d.  
St atistic al Met h o ds:
Ra n d o mizati o n: A ra n d o mizati o n all ocati o n al g orit h m will be a p plie d b y s u bject s o t h at t h e 
treat me nt all ocati o n of C A P- 1 0 0 2: place b o is 1: 1.
A nal ysis P o p ulati o ns:
•
I nte nt t o Tre at (I T T) P o p ul ati o n: All s u bjects ra n d o mize d. S u bjects will be s u m marize d 
a n d a nal yz e d per t h eir ra n d o mizati o n assi g n me nt, re gar dl ess of t he treat m e nt ( C A P- 1 0 0 2
or us ual care) act u all y recei v e d.
•
M o difie d I T T ( mI T T) P o p ul ati o n : S u bjects i n t he C A P- 1 0 0 2 treat me nt gr o u p w h o 
recei v e d t he I P, a n d all s u bjects i n t he us ual car e treat me nt gr o u p w h o re m ai n o n st u d y as 
of 9: 0 0 a m (s u bject’s ti me z o ne) o n Da y 0. S u bjects will be s u m marize d a n d a nal yze d p er 
t heir ra n d o miz ati o n assi g n me nt, re gar dl ess of t he treat m e nt ( C A P- 1 0 0 2 or us u al care) 
act uall y recei ve d.
•
Per Pr ot oc ol P o p ul ati o n : S u bjects wit h n o maj or pr ot oc ol vi olati o ns. T he list of s u bjects 
wit h maj or pr ot o c ol vi olati o ns will be c o m pile d pri or t o data base l o c k. 
•
S afety P o p ul ati o n : S u bjects ra n d o mi ze d t o C A P- 1 0 0 2 treat me nt w h o recei v e d t h e I P, a n d 
all s u bjects i n t he us ual care tr eat m e nt gr o u p w h o re m ai n o n st u d y as of 9: 0 0 a m 
(s u bject’s ti m e z o ne) o n Da y 0. S u bjects will be s u m marize d a n d a nal yz e d per treat me nt 

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 1 0 of 1 1 1
act uall y recei ve d, re gar dless of t heir ra n d o mizati o n assi g n m e nt.
Efficac y A nal yses:
T he pri mar y a nal ysis of efficac y will be perf or me d f or t he mI T T P o p ulati o n as defi n e d 
a b o ve. A d diti o nal a nal ys es will be defi ne d i n t he St atistical A nal ysis Pl a n ( S A P).
Car diac str uct ural res ults will be s u m marize d f or t he i nter ve nti o n a n d c o ntr ol gr o u ps 
f or eac h visit at w hic h t he y are p erf or me d. A bs ol ute a n d relati ve c ha n ges c o m pare d t o 
baseli n e will be s u m mari z e d usi n g descri pti ve statistics.
F u ncti o nal a n d m o bilit y val ues will be s u m m ariz e d usi n g descri pti ve statistics a n d 
c ha n ges fr o m baseli n e will be c o m pare d b et wee n gr o u ps.
I m pr o v e me nts i n q ualit y of life para meters will be liste d a n d s u m m ariz e d usi n g 
descri pti ve statistics a n d c o m p are d b et wee n gr o u ps.
Bi o mar ker res ults f or Ost e o p o nti n, S T 2, I L- 1 0, a n d Galecti n- 3 will be liste d a n d 
s u m marize d usi n g descri pti ve statistics.  
Safet y A nal ysis:
T he pri mar y a nal ysis of safet y will be perf or me d o n t he S afet y P o p ulati o n as defi ne d 
a b o ve. A d diti o nal a nal ys es will be defi ne d i n t he S A P. Safet y a n d t olera bilit y of 
i ntrac or o nar y i nf usi o n of C A P- 1 0 0 2 will be e val uate d b y t he occ urre nce of re d u cti o n 
i n TI MI fl o w a n d maj or car diac e ve nts, fre q u e nc y a n d se verit y of a d vers e e ve nts, a n d 
b y c h a n ges fr o m baseli ne i n la b orat or y ass ess me nts, vital si g ns, p h ysical 
e x a mi nati o n, a n d E C G.
Missi n g Dat a:
Ha n dli n g of missi n g data will be detaile d i n t he S A P.
D ate of O ri gi n al A p pr o ve d P r ot oc ol: 0 1 M a y 2 0 1 5
D ate of M ost R ece nt P r ot oc ol A me n d me nt (if a p pli c a bl e):
2 9 Fe br uar y 2 0 1 6
Pre p are d i n: Micr os oft
W or d 2 0 1 0

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 1 1 of 1 1 1
T A B L E O F C O N T E N T S
S Y N O P SI S ................................................................................................................................. 2
T A B L E O F C O N T E N T S ......................................................................................................... 1 1
LI S T O F I N- T E X T T A B L E S .................................................................................................. 1 5
LI S T O F I N- T E X T FI G U R E S................................................................................................. 1 5
LI S T O F A P P E N DI C E S .......................................................................................................... 1 5
LI S T O F A B B R E VI A TI O N S A N D D E FI NI TI O N S O F T E R M S .......................................... 1 6
1
I N T R O D U C TI O N ........................................................................................................... 2 1
.......... 2 2
.......... 2 3
.......... 2 3
.......... 2 4
.......... 2 5
..... 2 6
..... 2 6
..... 2 7
..... 2 9
..... 3 0
..... 3 2
..... 3 2
..... 3 3
..... 3 4
..... 3 4
..... 3 5
..... 3 5
..... 3 5
..... 3 7
..... 3 7
..... 3 7
..... 3 8
1. 3 
I nf or mati o n o n C A P- 1 0 0 2 ..................................................................................... 3 8
 
3 9
1. 5 
St u d y P o p ulati o n .................................................................................................... 4 0
1. 6 
C ar diac Ma g n etic Res o n a nce I m a gi n g .................................................................. 4 0
1. 7 
P ote ntial Ris ks a n d Be nefits .................................................................................. 4 1

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 1 2 of 1 1 1
2
S T U D Y O BJ E C TI V E S .................................................................................................... 4 3
2. 1 
S afet y O bjecti v e ..................................................................................................... 4 3
2. 2 
Efficac y O bjecti ve ................................................................................................. 4 3
3
I N V E S TI G A TI O N A L P L A N .......................................................................................... 4 4
3. 1  
O verall St u d y Desi g n a n d Pl a n .............................................................................. 4 4
3. 2 
E x pl orat or y Effi cac y E n d p oi nts ............................................................................. 4 6
3. 3 
S afet y M o nit ori n g .................................................................................................. 4 7
3. 4 
R ati o nale f or St u d y D esi g n a n d C o ntr ol Gr o u p ..................................................... 4 8
3. 5 
St u d y D urati o n a n d Dates ...................................................................................... 4 8
4
S T U D Y P O P U L A TI O N S E L E C TI O N ........................................................................... 4 9
4. 1 
St u d y P o p ulati o n .................................................................................................... 4 9
4. 2 
I n cl usi o n Criteria ................................................................................................... 4 9
4. 3 
E x cl usi o n Criteria .................................................................................................. 4 9
5
S T U D Y T R E A T M E N T( S) .............................................................................................. 5 2
5. 1 
I n v esti gati o nal Pr o d uct .......................................................................................... 5 2
 
5 2
5. 1. 2  
D S A Scree ni n g............................................................................................. 5 2
 
5 3
5. 2  
Us ual Care .............................................................................................................. 5 3
5. 2. 1 
C A P- 1 0 0 2 Access P ost- St u d y...................................................................... 5 3
5. 3 
R a n d o mi zati o n ....................................................................................................... 5 3
5. 4 
C o nc o mita nt T hera p y............................................................................................. 5 3
5. 5 
Pri or T hera p y E x cl usi o ns/ Pr o hi bite d Me dicati o ns ................................................ 5 4
5. 6 
Treat me nt C o m plia nce ........................................................................................... 5 4
 
5 4
 
5 4
5. 9 
I n v esti gati o nal Pr o d uct R ete nti o n at St u d y Site .................................................... 5 5
6
S T U D Y P R O C E D U R E S ................................................................................................. 5 6
6. 1 
I nf or me d C o nse nt................................................................................................... 5 6
6. 2  
M e dical Hist or y & C o n c o mita nt Me dicati o ns ....................................................... 5 7
6. 3 
C ar diac a n d Li mit e d P h ysical E x a mi nati o ns ......................................................... 5 7
6. 4  
Vital Si g ns .............................................................................................................. 5 7
6. 5 
Cli nical La b orat or y Tests ....................................................................................... 5 7
6. 5. 1 
La b orat or y Para meters ................................................................................. 5 7
6. 5. 2 
S a m ple C ollecti o n, St ora ge, a n d S hi p pi n g .................................................. 5 7
6. 6 
1 2- Lea d E C G ......................................................................................................... 5 8
6. 7 
C o nti n u o us Car diac R h yt h m M o nit ori n g............................................................... 5 8

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 1 3 of 1 1 1
6. 8  
M a g n etic Res o na nce I ma gi n g ................................................................................ 5 9
6. 8. 1 
Ci ne I m a gi n g Pr ot oc ol ................................................................................. 5 9
6. 8. 2  
D ela ye d C o ntrast- E n h a nce me nt Pr ot oc ol .................................................... 5 9
6. 8. 3 
I ma ge A nal ysis............................................................................................. 5 9
6. 8. 4  
Hist orical Car diac M RI ................................................................................ 5 9
6. 9 
Cli nical F u ncti o n a n d Q u alit y of Lif e Meas ures.................................................... 6 0
6. 1 0
I nf usi o n Pr oce d ure ................................................................................................. 6 0
6. 1 0. 1
C A P- 1 0 0 2 I nf usi o n ...................................................................................... 6 0
6. 1 0. 2
I nf usi o n Pr oce d ure St o p pi n g Criteria .......................................................... 6 1
7
S T U D Y A C TI VI TI E S ..................................................................................................... 6 3
7. 1 
S cree ni n g Visit ....................................................................................................... 6 3
7. 2 
R a n d o mi zati o n, Da y 0, a n d Da y 1 ......................................................................... 6 4
7. 3  
D a y 3 P h o ne Visit .................................................................................................. 6 5
7. 4  
W ee k 2 Visit ( Da y 1 4 ± 3 da ys) ............................................................................. 6 5
7. 5  
W ee k 6 Visit ( Da y 4 0 ± 7 da ys) ............................................................................. 6 6
7. 6  
M o nt h 3 Visit ( Da y 9 0 ± 7 da ys) ........................................................................... 6 6
7. 7  
M o nt h 6 Visit ( Da y 1 8 0 ± 1 4 da ys) ....................................................................... 6 7
7. 8  
M o nt h 1 2 Visit ( Da y 3 6 0 ± 3 0 da ys) ..................................................................... 6 8
7. 9  
A n n ual F oll o w- u p P h o ne Call ............................................................................... 6 8
7. 1 0
Re m o val of Patie nts fr o m t h e Trial or St u d y Dr u g ................................................ 6 8
7. 1 1
Earl y T er mi n ati o n Pr oce d ures ............................................................................... 6 9
7. 1 2
L ost t o F oll o w- U p .................................................................................................. 6 9
7. 1 3
A d verse E v e nts Assess me nts ................................................................................. 7 0
7. 1 3. 1
Perf or mi n g A d vers e E ve nts Assess me nts .................................................... 7 0
7. 1 3. 2
Ti mi n g .......................................................................................................... 7 1
7. 1 3. 3
Se verit y ........................................................................................................ 7 1
7. 1 3. 4
R elati o ns hi p ................................................................................................. 7 1
7. 1 3. 5
E x pecte d ness ................................................................................................ 7 2
7. 1 3. 6
Cli nical Si g nifica nce .................................................................................... 7 3
7. 1 3. 7
Cli nical La b orat or y A d v erse E ve nts ............................................................ 7 3
7. 1 3. 8
Seri o us A d verse E v e nts ............................................................................... 7 4
7. 1 3. 8. 1  
D efi niti o n ........................................................................................... 7 4
7. 1 3. 8. 2 
R e p orti n g Seri o us A d v erse E ve nts .................................................... 7 4
7. 1 3. 9
S p o ns or M o nit ori n g of A d vers e E ve nts ....................................................... 7 5
7. 1 3. 1 0
P ost- St u d y A d v erse E v e nts a n d Seri o us A d verse E ve nts ............................ 7 6
8
Q U A LI T Y C O N T R O L A N D A S S U R A N C E ................................................................. 7 6
9
P L A N N E D S T A TI S TI C A L M E T H O D S ........................................................................ 7 7
9. 1  
G e neral C o nsi derati o ns .......................................................................................... 7 7
9. 2  
D eter mi nati o n of Sa m ple Si ze ............................................................................... 7 7

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 1 4 of 1 1 1
9. 3  
A nal ysis P o p ulati o ns .............................................................................................. 7 7
9. 4 
Efficac y A nal ysis ................................................................................................... 7 7
9. 4. 1 
C ar diac M RI, M o bilit y, a n d Q ualit y of Life Para meters ............................. 7 7
9. 4. 2 
Bi o mar kers ................................................................................................... 7 8
9. 5 
S afet y A nal ysis ...................................................................................................... 7 8
9. 5. 1 
S afet y E n d p oi nts .......................................................................................... 7 8
9. 5. 2  
A d verse E v e nts ............................................................................................ 7 8
9. 5. 3 
La b orat or y E v al uati o ns ................................................................................ 7 8
9. 6  
Missi n g Data .......................................................................................................... 7 8
9. 7 
St u d y St o p pi n g R ules ............................................................................................. 7 8
1 0  I N D E P E N D E N T O V E R SI G H T C O M MI T T E E S ........................................................... 7 9
1 0. 1
Cli nical E ve nts C o m mittee ( C E C) ......................................................................... 7 9
1 0. 2
Data Safet y M o nit ori n g B oar d ( D S M B) ................................................................ 7 9
1 1  A D MI NI S T R A TI V E C O N SI D E R A TI O N S .................................................................... 8 0
1 1. 1
I n v esti gat ors a n d St u d y A d mi nistrati ve Str uct ure ................................................. 8 0
1 1. 2
I nstit uti o nal Re vie w B oar d (I R B) A p pr o v al .......................................................... 8 0
1 1. 3
Et hical C o n d uct of t h e St u d y ................................................................................. 8 1
1 1. 4
S u bject I nf or mati o n, C o nse nt, a n d Asse nt ............................................................. 8 1
1 1. 5
S u bject C o nfi d e nti alit y .......................................................................................... 8 1
1 1. 6
St u d y M o nit ori n g ................................................................................................... 8 2
1 1. 7
Case R e p ort F or ms a n d St u d y Rec or ds ................................................................. 8 2
1 1. 8
Pr ot oc ol Vi olati o ns/ D e viati o ns .............................................................................. 8 3
1 1. 9
Access t o S o urce D oc u m e ntati o n .......................................................................... 8 3
1 1. 1 0
Data Ge n erati o n a n d A nal ysis ............................................................................... 8 3
1 1. 1 1
Data Rete nti o n ....................................................................................................... 8 3
1 1. 1 2
Fi n a ncial Discl os ure............................................................................................... 8 4
1 1. 1 3
P u blicati o n, Use a n d Discl os ure P olic y ................................................................. 8 4
1 2  R E F E R E N C E LI S T ......................................................................................................... 8 5
A p pe n di x 1  
Sc he d ule of E ve nts ............................................................................. 8 9
A p pe n di x 2  
Cli nical La b orat or y T ests ................................................................... 9 1
A p pe n di x 3  
Pr o hi bit e d Pri or Me dicati o ns ............................................................. 9 2
A p pe n di x 4  
A me n d me nt 3 C ha n ges  ( Pr ot oc ol Versi o n 1 8 J ul y 2 0 1 6) ................ 9 7
A p pe n di x 5  
A me n d me nt 2 C ha n ges  ( Pr ot oc ol Versi o n 2 9 Fe br uar y 2 0 1 6) ......... 9 9
A p pe n di x 6  
A me n d me nt 1 C ha n ges  ( Pr ot oc ol Versi o n 2 7 J a n uar y 2 0 1 6) ......... 1 0 6
A p pe n di x 7  
S p o ns or Si g nat ure ............................................................................ 1 1 0
A p pe n di x 8  
I n v esti gat or’s Si g n at ure ................................................................... 1 1 1

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 1 5 of 1 1 1
LI S T O F I N- T E X T T A B L E S
Ta ble 1.
S u m mar y of E x p os ure b y St u d y ....................................................................... 3 2
Ta ble 2.
L ocal La b orat or y T esti n g, Da y 0 a n d D a y 1 ( C A P- 1 0 0 2 Ar m O nl y) ............... 5 8
Ta ble 3.
Per- Visit Mi ni m u m C o nti n u o us Car diac M o nit ori n g D urati o ns ...................... 5 9
LI S T O F I N- T E X T FI G U R E S
.... 2 3
.... 2 4
.... 2 5
.... 2 6
.... 2 8
.... 2 9
.... 3 1
.... 3 6
Fi g ure 9.
R elati o ns hi p bet wee n m y ocar dial fi br osis, ej ecti o n fracti o n, a n d a ge i n 
D M D patie nts .................................................................................................... 4 1
Fi g ure 1 0. Sc he matic of t h e st u d y d esi g n ........................................................................... 4 6
LI S T O F A P P E N DI C E S
A p pe n di x 1  
Sc he d ule of E ve nts ............................................................................. 8 9
A p pe n di x 2  
Cli nical La b orat or y T ests ................................................................... 9 1
A p pe n di x 3  
Pr o hi bit e d Pri or Me dicati o ns ............................................................. 9 2
A p pe n di x 4  
A me n d me nt 3 C ha n ges  ( Pr ot oc ol Versi o n 1 8 J ul y 2 0 1 6) ................ 9 7
A p pe n di x 5  
A me n d me nt 2 C ha n ges  ( Pr ot oc ol Versi o n 2 9 Fe br uar y 2 0 1 6) ......... 9 9
A p pe n di x 6  
A me n d me nt 1 C ha n ges  ( Pr ot oc ol Versi o n 2 7 J a n uar y 2 0 1 6) ......... 1 0 6
A p pe n di x 7  
S p o ns or Si g nat ure ............................................................................ 1 1 0
A p pe n di x 8  
I n v esti gat or’s Si g n at ure ................................................................... 1 1 1

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 1 6 of 1 1 1
LI S T O F A B B R E VI A TI O N S A N D D E FI NI TI O N S O F T E R M S
6 M W T
6- Mi n ute Wal k Test
µl
Micr oliter
A D C
A di p ose- Deri ve d Cell
A E
A d verse E ve nt
A F
Asse nt F or m
A L B
Al b u mi n
A L K- P
Al kali ne p h os p hatase
A L T
Ala ni ne a mi n otra nsferas e (s a me as S G P T) 
A MI
Ac ute M y ocar dial I nfarcti o n
A S T
As partate a mi n otra nsferase (sa me as S G O T) 
At m
At m os p heres
B P
Bl o o d Press ure
B M M N C 
B o ne Marr o w M o n o n uclear Cell
B U N
Bl o o d urea nitr o ge n
C
Celsi us
C C U
Criti cal ( or C or o nar y) Care U nit
C D C
Car di os p here Deri ve d Cell
C E C
Cli nical E ve nts C o m mittee
C F R
C o de of Fe deral Re g ulati o ns 
C H F
C o n gesti ve Heart Fail ure
C K- M B 
Creati n e p h os p h o ki nase M B is oe nz y me
C M V
C yt o me gal o vir us
C R F
Case Re p ort F or m
C R O
C o ntract Researc h Or ga nizati o n
C S
Cli ni call y Si g nifica nt
C S C
Car diac St e m Cell
C T
C o m p uterize d T o m o gra p h y
D C M
Dilate d Car di o m y o pat h y

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 1 7 of 1 1 1
D M D
D uc he n ne M usc ular D ystr o p h y
D M S O
Di met h yl S ulf o xi de
D S A
D o n or- S pecific A nti b o d y 
D S M B
Data a n d Safet y M o nit ori n g B oar d
E B V
E pstei n- Barr Vir us
E C G
Electr ocar di o gra m
E D C
El ectr o nic Dat a Ca pt ure
E D V
E n d- Diast olic V ol u me
E F
Ej ecti o n Fracti o n
E LI S p ot 
E nz y me- Li n ke d I m m u n os or be nt S p ot
E S R
E x pe dite d Safet y Re p ort
E S V
E n d- S yst olic V ol u me
F
Fa hre n heit
F D A
F o o d a n d Dr u g A d mi nistrati o n
G C P
G o o d Cli nical Practice 
G F R
Gl o mer ular Filtrati o n Rate 
H Bs A g
He patitis B S urface A nti ge n 
Hct
He mat ocrit
H C V
He patitis C Vir us
H F
Heart Fail ure
H g b
He m o gl o bi n
H b A 1c
Gl yc os yl ate d He m o gl o bi n
HI P A A
Healt h I ns ura nce P orta bilit y a n d Acc o u nta bilit y Act
HI V
H u ma n I m m u n o deficie nc y Vir us
H L A
H u ma n Le u k oc yte A nti ge n
H R
Heart Rate
I C D
I m pla nta ble Car di o verter Defi brill at or
I C F
I nf or me d C o nse nt F or m
I C H
I nter nati o nal C o nfere n ce o n Har m o nisati o n

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 1 8 of 1 1 1
I E C
I n de pe n de nt Et hics C o m mittee
I g G
I m m u n o gl o b uli n G 
I g M
I m m u n o gl o b uli n M
I L- 1 0
I nterle u ki n 1 0
I N D
I n vesti gati o nal Ne w Dr u g
I N R
I nter nati o nal N or mali ze d Rati o
I P
I n vesti gati o nal Pr o d uct
i P S Cs
I n d uce d pl uri p ote nt ste m cells
I R B
I nstit uti o nal Re vie w B oar d
I S
I nfarct Size
I T T
I nte nt-t o- Treat (ra n d o mize d p o p ulati o n)
K
P otassi u m
K g
Kil o gra m
L A D
Left A nteri or Desce n di n g 
L C X
Left Circ u mfle x
L D H
Lactate de h y dr o ge nase 
L G E
Late ga d oli ni u m e n ha nce me nt
L V
Left Ve ntricle
L V A D
Left Ve ntric ular Assist De vice 
L V E F
Left Ve ntric ular Ej ecti o n Fracti o n 
M
Milli o n
M A C E
Maj or A d verse Car diac E ve nt
M C B
Master cell ba n k
Mc g
Micr o gra m
M C H
Mea n c or p usc ular he m o gl o bi n
M C H C
Mea n c or p usc ular he m o gl o bi n c o nce ntrati o n
M C V
Mea n c or p usc ular v ol u me
Me d D R A  
M e dical Dicti o nar y f or Re g ulat or y Acti vities
M FI
Mea n Fl u oresce nce I nte nsit y

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 1 9 of 1 1 1
MI
M y ocar dial I nfarcti o n
Mi n
Mi n ute 
m L
Milliliter
M L H F Q  
Mi n nes ota Li vi n g wit h Heart Fail ure Q uesti o n naire
M m
Milli meters 
m m ol
Milli m ole
M o A
Mec ha nis m of Acti o n
M O P
Ma n ual of Pr oce d ures (??)
M RI
Ma g netic Res o na nce I ma gi n g
M S C
Mese nc h y mal St e m Cell
Na
S o di u m
N C S
N ot Cli nicall y Si g nifica nt
N H L BI
Nati o nal Heart, L u n g a n d Bl o o d I nstit ute
N T- pr o B N P  
N-ter mi nal pr o- h or m o ne brai n natri ureti c pe pti de
N Y H A
Ne w Y or k Heart Ass ociati o n
O C R
O x y ge n C o ns u m pti o n Rate
O T W
O ver T he Wire
P
Pr o ba bilit y
P CI
Perc uta ne o us C or o nar y I nter ve nti o n
Pe ds Q L 
Pe diatric Q ualit y of Life I n ve nt or y
P G A
Patie nt Gl o bal Assess me nt
P O D CI
Pe diatric O ut c o mes Data C ollecti o n I nstr u me nt
P R A
Pa nel Reacti ve A nti b o di es
P S P
Patie nt S pecific Pr o ba bilit y
P T T
Acti vate d Partial T hr o m b o pl asti n Ti me
P U L
Perf or ma nce of U p per Li m b
Q o L
Q ualit y of Life
R B C
Re d bl o o d cell
R C A
Ri g ht C or o nar y Arter y

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 2 0 of 1 1 1
R N A
Ri b o n ucleic Aci d
R R
Res pirati o n Rate
S A B
Si n gle-a nti ge n bea d
S A E
Seri o us A d verse E ve nt 
S A P
St atistical A nal ysis Pla n 
S D
Sta n dar d De vi ati o n
S E M
Sta n dar d Err or of t he Mea n
S F- 3 6
S h ort F or m ( healt h s ur ve y) - 3 6 q uesti o ns 
S G O T
Ser u m Gl uta mi c O xal oacetic Tra nsa mi nase 
S G P T
Ser u m Gl uta mi c P yr u vi c Tra nsa mi n ase 
S P R O
Seri o us Pr oce d ure Relate d O utc o me
S T 2
Car diac Stress Bi o mar ker (I L- 1 Rece pt or Fa mil y; bi n ds I L- 3 3)
T E A E
Treat me nt- E mer ge nt A d verse E ve nt
TI MI
T hr o m b ol ysis I n M y ocar dial I nfarcti o n
T M P G
TI MI M y ocar dial Perf usi o n Gra de
T nI
Tr o p o ni n I
U A
Uri nal ysis
V F
Ve ntric ular Fi brillati o n
V G E F
Vasc ular E n d ot helial Gr o wt h Fact or
V T
Ve ntric ular T ac h ycar dia
W B C
W hite Bl o o d Cell
W P AI: S H P
W or k Pr o d ucti vit y a n d Acti vit y I m pair me nt Q uesti o n naire: S pecific Healt h 
Pr o ble m

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 2 1 of 1 1 1
1
I N T R O D U C TI O N
D uc he n ne M us c ular D ystr o p h y ( D M D) is a p e diatri c o nset, ge netic disease i n w hi c h patie nts 
lac k d ystr o p hi n, a pr otei n critical f or sta bilizi n g m uscle cell me m bra nes. Affecti n g 1 i n 
3, 5 0 0- 5, 5 0 0 male birt hs, D M D acc o u nts f or 8 0 % of all cases of m usc ular d ystr o p h y. Patie nts 
e x perie nce pr o gressi ve s keletal m uscle wea k ness a n d l oss of a m b ulati o n b y a ge 1 5, e ve nt ual 
res pirat or y a n d car diac fail ure, a n d a n a b bre viate d lifes pa n a vera gi n g o nl y t hree deca d es 
( B us h b y, Fi n kel et al. 2 0 1 0a, B us h b y, Fi n kel et al. 2 0 1 0 b). Wit h c ortic oster oi ds a n d n o n-
i n vasi ve ve ntilati o n, heart fail ure has e m er ge d as t he m ai n ca use of deat h. Yet t h e 
o ver w h el mi n g maj orit y of cli nical trials i n D M D h a ve f oc us e d o n s keletal rat her t ha n c ar diac 
m uscle disease. Wit h o ut a n y f u ncti o nal d ystr o p hi n pr otei n, car di o m y oc yt es e x perie nce a 
disr u pti o n of me m bra ne i nte grit y, lea di n g t o a n o v erl o a d of i ntracell ular cal ci u m, l ea di n g t o 
car di o m y oc yte d eat h. Cell deat h is acc o m pa nie d b y l ocal i nfla m mati o n w hic h ser ves as a 
s o urce of o xi d ati v e stress f or t he re mai ni n g car di o m y oc ytes ( S hir o k o va a n d Ni g gli 2 0 1 3). 
T hese f oci of cell deat h are ofte n e x perie nce d b y p atie nts as micr oi nfar cts, a n d acc o m p a ni e d 
b y c h est pai n a n d t h e release of car dia c e nz y m es, cli nical hall mar ks of MI. T his a m plif yi n g 
pr ocess tri g gers pr o gressi ve scar tiss ue d e p ositi o n a n d lea ds e ve nt uall y t o h eart fail ure. 
I m pr o v e me nts i n t he treat me nt of res pirat or y m us cle disease h a ve ele vate d t h e i n e vit a ble 
car di o m y o pat h y t o t he lea di n g ca use of d eat h i n D M D patie nts ( S p ur ne y 2 0 1 1, Passa ma n o, 
Ta glia et al. 2 0 1 2). 
N o s pecific t hera pies c urre ntl y e xist t o treat D M D car di o m y o pat h y. Gi ve n t he gl o b al a n d 
pr o gressi ve nat ure of t h e dis ease, rarel y are t hese patie nts c o nsi dere d f or h eart tra ns pla nt, us e 
of mec ha nical car di ac assist de vices is lar gel y e x peri m e nt al ( R ya n, J efferies et al. 2 0 1 4), a n d 
t he effecti ve ness of t h e t h era pies i n use or i n de vel o p me nt f or D M D o n t he car diac 
ma nifestati o ns of t he disease is ge n erall y a s ec o n dar y c o nsi derati o n. Sta n dar d of care 
c ortic oster oi d use i n D M D patie nts, ai me d at pr ol o n gi n g a m b ulati o n, has b ee n s h o w n t o 
dela y t he pr o gressi o n of heart disease w he n its us e is i nitiate d pri or t o t he o nset of car di ac 
d ysf u n cti o n ( Mar k h a m, Ki n nett et al. 2 0 0 8). H o w e ver, defi niti ve e vi de nce f or t his a n d t he 
o pti m al ster oi d d osi n g re gi me n are still u n der i n vesti gati o n (e. g. N C T 0 1 6 0 3 4 0 7). T he use of 
a n gi ote nsi n-c o n verti n g e nz y m e i n hi bit ors, a n gi ote nsi n rece pt or bl oc k ers, a n d beta bl oc kers 
o n t o p of ster oi ds has bec o me sta n dar d t hera p y f or t hese patie nts o nce car di ac d ysf u ncti o n 
has set i n (Jefferies, Ei de m et al. 2 0 0 5, Alle n, Fla ni ga n et al. 2 0 1 3). H o w e v er, pr o gressi ve 
decli n e of car di ac f u ncti o n still occ urs i n t h e face of w hat ca n b e c o nsi dere d c urre nt ma xi mal 
me dical ma na ge m e nt ( H or, Maz ur et al. 2 0 1 1). M ost n o vel D M D t hera pies ha ve bee n 
a d va nce d i n de vel o p me nt o n t he b asis of s keletal m uscle effects, a n d h a ve a re d uce d or 
u nc haracterize d effect o n car diac m uscle. Of t h e d o ze ns of rece ntl y c o m plete d or acti ve 
D M D cli nical trials, ver y fe w ha v e a pri mar y car diac e n d p oi nt. 
C A P- 1 0 0 2 is a n i n vesti gati o nal pr o d uct c o nsisti n g of all o ge neic car di os p h ere- d eri ve d cells 
( C D Cs). 

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 2 2 of 1 1 1
T he H O P E- D u c he ne P h ase II st u d y will be t he first i n vesti gati o n of C A P- 1 0 0 2 i n male 
s u bjects wit h car di o m y o pat h y sec o n dar y t o D M D a n d t he first trial of t he pr o d uct n ot 
c o n d ucte d e x cl usi v el y i n a d ults. 
    

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 2 3 of 1 1 1

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 2 4 of 1 1 1

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 2 5 of 1 1 1

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 2 6 of 1 1 1

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 2 7 of 1 1 1

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 2 8 of 1 1 1

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 2 9 of 1 1 1

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 3 0 of 1 1 1

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 3 1 of 1 1 1

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 3 2 of 1 1 1

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 3 3 of 1 1 1

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 3 4 of 1 1 1

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 3 5 of 1 1 1

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 3 6 of 1 1 1

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 3 7 of 1 1 1

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 3 8 of 1 1 1
1. 3 
I nf or m ati o n o n C A P- 1 0 0 2
C A P- 1 0 0 2 C D Cs are gr o w n fr o m d o n ors u nrelat e d t o treat me nt reci pie nts. H u ma n Le u k oc yt e 
A nti ge n ( H L A) matc hi n g of t he d o n ors a n d t he s u bjects will n ot be perf or me d. S u bjects will 
be assesse d f or a nti- H L A a nti b o dies at scree ni n g. S u bjects w h o ca n be matc he d (i n t h e se ns e 
t hat n o D S As are prese nt t o o ne or m ore d o n ors) will be ra n d o mize d i nt o t he st u d y. S u bjects 
w h o are mis matc h e d a g ai nst all a vaila ble d o n ors, beca use t he y har b or o ne or m ore a nti- H L A 
a nti b o dies a gai nst eac h d o n or, will be scree n fail ures a n d n ot ra n d o mize d i nt o t his st u d y.  
All ra n d o mize d s u bjects will be m o nit ore d f or de vel o p me nt of i ncrease d a nti- H L A a nti b o d y 
le vels wit h de vel o p me nt of se nsitizati o n t o H L A a nti ge ns s p ecific t o t he C A P- 1 0 0 2 d o n or 
(i.e. D S As).
C A P- 1 0 0 2 is a d mi nistere d as a d ose of 2 5 milli o n cells 

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 3 9 of 1 1 1

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 4 0 of 1 1 1
1. 5 
St u d y P o p ul ati o n 
Car di o m y o pat h y is c urre ntl y t he lea di n g ca use of deat h i n D M D p atie nts ( S p ur ne y 2 0 1 1,
Passa ma n o, Ta glia et al. 2 0 1 2), ha vi n g rece ntl y s u p pla nte d res pirat or y ca us es as a r es ult of 
treat me nt i m pr o v e me nts f or t hat as pect of t he dis ease. Affli cte d pati e nts lac k a f u ncti o nal 
d ystr o p hi n pr otei n a n d e x perie nce pr o gressi ve m uscle wea k n ess starti n g at a n earl y a ge a n d 
ulti m atel y a n a b bre viate d lifes pa n. Gi ve n t he gl o b al a n d pr o gressi ve nat ure of t he disease, 
rarel y are t hes e patie nts c o nsi dere d f or h eart tr a ns pla nt, use of mec ha nical car diac assist 
de vices is lar gel y e x peri me ntal ( R ya n, J efferies et al. 2 0 1 4), a n d t h e effecti v e ness of t h e 
t hera pies i n use or i n d e vel o p me nt f or D M D o n t h e car di ac ma nifestati o ns of t he disease is 
ge n erall y a s ec o n d ar y c o nsi derati o n. C A P- 1 0 0 2 is i nte n de d t o tar get t he car di o m y o pat h y i n 
D M D as o p p ose d t o t he s keletal m y o p at h y. 
1. 6 
C ar di a c M a g n eti c R e s o n a n c e I m a gi n g 
M RI will be use d t o ass ess n u mer o us efficac y e n d p oi nts i n t h e H O P E- D uc he n ne st u d y. 
Car diac M RI is pr o vi n g t o ha ve gre at pr o g n ostic a n d dia g n ostic val u e f or D M D 
car di o m y o pat h y, a n d its use has re veale d s e veral hall mar ks of t h e dis eas e pre vi o usl y 
u n detecta ble b y sta n d ar d ec h ocar di o gra p h y. 
Circ u mfer
cc), assesse d via a n M RI t a g gi n g pr ot oc ol, has bee n s h o w n t o be 
a b n or mal i n y o u n g D M D patie nts wit h still n or mal ejecti o n fracti o n ( E F) a n d n o si g ns of 
m y ocar dial fi br osis, a n d t o w orse n wit h a ge a n d al o n gsi de a decli ne i n E F a n d t he e mer ge n ce 
of m y ocar dial fi br osis ( H or, Wa nsa p ura et al. 2 0 0 9). F urt her m ore, a rece ntl y c o m plete d tri al 
of a mi neral o c ortic oi d rece pt or a nta g o nist use d i n c o nj u ncti o n wit h ma xi mal me dical 
cc e x peri e nce d b y s u bjects wit h s o me de gree of 
m y ocar dial fi br osis pre-e xisti n g at baseli ne c o ul d be atte n uate d wit h i nter ve nti o n, a n d t hat 
cc were acc o m pa ni e d b y i m pr o ve me nts i n E F a n d prece de d b y re d ucti o ns 
i n t h e de gree of m y ocar dial fi br osis prese nt ( Ra m a n, H or et al. 2 0 1 4).  
M y ocar dial fi br osis, ass esse d as late ga d oli ni u m e n ha nce m e nt ( L G E) o n M RI a n d q u a ntifie d 
eit her o n t he basis of se g me ntal i n v ol ve me nt or i n a bs ol ute ter ms, has als o bee n s h o w n t o be 
pre vale nt i n D M D p atie nts, t o i n crease wit h a ge, a n d t o c orrelat e ne gati vel y wit h E F ( H or, 
Ta yl or et al. 2 0 1 3). D ata ill ustrati n g t his relati o ns hi p is s h o w n i n Fi g ure 9. 

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 4 1 of 1 1 1
Fi g ur e 9. 
R el ati o n s hi p b et w e e n m y o c ar di al fi br o si s, ej e cti o n fr a cti o n, a n d 
a g e i n D M D p ati e nt s
T he L V E F of L G E ne gati ve ( bl ue dia m o n ds) a n d L G E p ositi ve (re d s q uare) p atie nts are pl otte d a gai nst a ge 
d e m o nstrati n g L G E was ass o ciate d wit h ol d er a ge a n d l o wer L V E F. Fr o m ( H or, T a yl or et al. 2 0 1 3).
1. 7 
P ot e nti al Ri s k s a n d B e n efit s 
Ris ks ass o ciate d wit h a d mi nistrati o n of t he st u d y a ge nt are pri maril y relate d t o placi n g a 
g ui de wire d o w n t he c or o nar y art eries i n or der t o p ositi o n t he i nf usi o n cat h eter. T he ris k of 
c or o n ar y dissecti o n is esti mate d t o be less t ha n 1 % i n t his setti n g (J a nsse ns, D u b ois et al. 
2 0 0 6, Sc hac hi n ger, Er bs et al. 2 0 0 6, Ass m us, Leist ner et al. 2 0 1 4)  
Ot her ris ks of t h e i nf usi o n pr oce d ure i n cl u de t h ose ris ks t hat are p ossi ble wit h dia g n ostic 
c or o n ar y a n gi o gr a p h y. T hese i ncl u de ris ks rel ate d t o i nfecti o n, blee di n g, h y p ote nsi o n, pai n 
a n d  he mat o ma at t he art erial p u nct ure sit e ( 0. 4 %), arr h yt h mia ( 0. 3 8 %), se nsiti vit y or aller g y 
t o ra di o gra p hic c o ntrast ( 0. 3 7 %), e x p os ure t o ra di ati o n, t hr o m b oe m b olis m ( 0. 0 7 %), c o ntrast 
i n d uce d n e p hr o p at h y, m y ocar dial isc h e mia or i nfarcti o n ( 0. 0 5 %). O v erall, t he ris k of a n y 
c o m plicati o n is a p pr o xi matel y 1. 4 %, a n d f or deat h is less t ha n 0. 1 % ( N ot o, J o h ns o n et al. 
1 9 9 1). T hese re p orte d rates of c o m plicati o n i ncl u d e s u bjects wit h si g nifica nt car diac dis ease 
a n d ot her c o m or bi dities.  
C A P- 1 0 0 2 C D Cs are gr o w n fr o m d o nate d h u m a n m y ocar di al tiss ue a n d

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 4 2 of 1 1 1
C o ntrast d ye is use d f or t he a n gi o gra p h y a n d M RI. T here is a s mall ris k of a n aller gic 
reacti o n ca usi n g ras h, itc hi n g, hi ves or w h eezi n g, b ut ver y rarel y ( 0. 0 7 %) are reacti o ns se vere 
( Dill ma n, Ellis et al. 2 0 0 7).  S u bjects will be assesse d f or a n y pre vi o us all er gic reacti o n t o 
c o ntrast pri or t o pr o ce d ures utilizi n g d ye. A nti hista mi ne a n d ster oi d ma y b e use d t o pre-
me dicate s u bjects w h o h a ve a hist or y of s e nsiti vit y or aller g y, acc or di n g t o t he h os pital's
us ual practi ce. T here is als o a s mall ( 5 %) p ossi bilit y of d e vel o pi n g a b n or mal f u ncti o n of t h e 
ki d ne ys ( Katz ber g a n d Ne w h o use 2 0 1 0).  T his a b n or mal ki d ne y f u ncti o n is us uall y 
te m p orar y a n d less li kel y if t here is n o u n derl yi n g ki d ne y f u ncti o n a b n or malit y. A d diti o nall y, 
t he ki d ne y a b n or malit y is ge n erall y res ol ve d b y t h e nat ural eli mi nati o n of t he c o ntrast d ye 
fr o m t he s u bject’s s yste m. Creati ni ne cleara n ce or G F R will b e assesse d pri or t o tests 
re q uiri n g c o ntrast. C o ntrast s ol uti o n a n d ti mi n g of t he test ma y b e a dj uste d i n s u bjects wit h 
i m pair me nt of ki d ne y f u ncti o n. S u bjects will be cl osel y m o nit ore d f or a n y si g ns of aller gic 
reacti o n or decrease i n re nal f u ncti o n as well as a n y ot her si d e effects.

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 4 3 of 1 1 1
2
S T U D Y O B J E C TI V E S
2. 1
S af et y O bj e cti v e
T he safet y o bjecti v e of t h e H O P E- D u c he n ne tri al will be t o i n vesti gate t h e safet y a n d 
t olera bilit y of C A P- 1 0 0 2 a d mi nistere d b y m ulti- vessel i ntrac or o n ar y i nf usi o n i n s u bjects wit h 
car di o m y o pat h y sec o n dar y t o D u c he n n e m usc ular d ystr o p h y ( D M D).
2. 2
Effi c a c y O bj e cti v e
T he effi cac y o bj ecti ve of t he H O P E- D u c he n ne tri al will be t o i n vesti gate t he effi cac y of 
C A P- 1 0 0 2 a d mi nistere d b y m ulti- vessel i ntrac or o nar y i nf usi o n i n s u bjects wit h 
car di o m y o pat h y sec o n dar y t o D M D b y st a bilizi n g or i m pr o vi n g car di ac str uct ure, b y 
e val uati n g f u ncti o nal stat us, a n d b y assess me nt of q ualit y of life.

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 4 4 of 1 1 1
3
I N V E S TI G A TI O N A L P L A N
3. 1 
O v er all St u d y D e si g n a n d Pl a n 
Male s u bjects wit h car di o m y o pat h y sec o n dar y t o D M D meeti n g all i ncl usi o n a n d n o 
e x cl usi o n criteria will be ra n d o mize d. All s u bjects will be at least 1 2 years of a ge. T h e y will 
be ra n d o mize d i n a 1: 1 ma n ner t o eit h er i ntrac or o n ar y i nf usi o n of C A P- 1 0 0 2 i n t hree 
c or o n ar y arteries s u p pl yi n g t he t hree maj or car di ac territ ories of t h e left ve ntricle of t he heart 
(a nteri or, lat eral, i nferi or/ p osteri or) or us ual care. I n t h e acti ve treat me nt ar m, all t hree maj or 
car dia c territ ories will be treate d (i nf us e d) d uri n g a si n gle pr oce d ure i n a n o pe n-la bel fas hi o n.  
A p pr o xi matel y 2 4, a n d n ot m ore t h a n 3 0, s u bjects will be ra n d o mize d i nt o t h e st u d y, i n t w o 
se q ue nti al e nr oll me nt gr o u ps. Safet y d ata fr o m Gr o u p 1 will u n der g o a Dat a S afet y 
M o nit ori n g B o ar d ( D S M B) re vie w pri or t o i nitiati o n of e nr oll me nt f or Gr o u p 2. 
T he first 6- 8 ra n d o mize d s u bjects will c o m prise Gr o u p 1, a n d will i ncl u de a mi ni m u m of 3 
s u bjects c o m pleti n g i ntrac or o nar y i nf usi o n wit h C A P- 1 0 0 2.  T he D S M B will c o n d uct a 
re vie w of i nteri m safet y data t hr o u g h 7 2 h o urs p ost- Da y 0 f or at least 3 i nf use d s u bjects a n d 
f or at least 6 s u bjects o verall.  
E nr oll me nt of Gr o u p 2 will be gi n per D S M B rec o m me n dati o ns f oll o wi n g t heir re vie w of t he 
7 2 h o ur safet y d ata fr o m Gr o u p 1. Gr o u p 2 will i ncl u de a p pr o xi matel y 1 8 s u bjects. S cree ni n g 
a n d ra n d o mizati o n will c o nti n ue u ntil at t otal of 1 2 s u bjects are i nf us e d wit h C A P- 1 0 0 2 or 3 0 
s u bjects are ra n d o mize d i nt o t he st u d y, w hic he ver c o m es first. 
  
  

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 4 5 of 1 1 1
S u bjects ra n d o miz e d t o recei ve us u al care will c o nti n ue t o be car e d f or a n d treate d i n 
w hate ver m a n ner t h e i n vesti gat or d ee ms m ost a p pr o priate f or t h e s u bject o n a n o n g oi n g 
basis, a n d will recei v e n o i nf usi o n. 
S u bject i nf usi o ns will be sta g gere d b y at least t hree da ys t o all o w f or d etecti o n of ac ute 
pr oce d ure-relate d c o m plicati o ns s h o ul d t he y o cc ur. A d diti o nall y, s u bject i nf usi o ns i n 
Gr o u p 2 will n ot be gi n u ntil at least t w o wee ks aft er t he fi nal i nf usi o n occ urs i n Gr o u p 1, t o 
all o w f or t h e c o m pleti o n of t he Gr o u p 1 safet y dat a re vie w t hr o u g h 7 2 h o urs p ost Da y 0. 
N o scree ni n g pr oce d ures will ta ke place u ntil t h e s u bject is f ull y i nf or me d of t h e researc h a n d 
si g ns t he i nf or m e d c o nse nt or pr o vi des asse nt. S u bjects 1 8 ye ars of a ge or ol der m ust ha ve 
t he a bilit y t o pr o vi de i nf or me d c o nse nt a n d f oll o w- u p wit h pr ot oc ol pr oce d ures. S u bjects at 
least 1 2 years of a ge b ut y o u n ger t ha n 1 8 years of a ge m ust ha ve t he a bilit y t o pr o vi de asse nt 
al o n g wit h t he s u bject’s pare nt or g u ar dia n pr o vi di n g per missi o n f or st u d y partici p ati o n. 
Ra n d o mizati o n will ta ke place wit hi n 3 0 da ys of t he first scree ni n g pr oce d ure. Aft er 
c o m pleti o n of t he scree ni n g pr o ce d ures, eli gi ble s u bjects ra n d o miz e d t o acti ve treat m e nt ar m 
will recei v e C A P- 1 0 0 2 a d mi nistere d via i ntrac or o nar y i nf usi o n o n Da y 0. Da y 0 f or eli gi ble 
s u bjects ra n d o mi ze d t o t he us ual care ar m will occ ur 7 da ys after t he d ate of ra n d o mizati o n, 
a n d a refere nce ti me p oi nt of 9: 0 0 a m o n Da y 0 will be use d i n place of i nf usi o n ti me. All 
ra n d o mize d s u bjects will ha ve a f oll o w- u p t ele p h o ne call o n St u d y D a y 3, a n d st u d y visits at 
Wee ks 2 a n d 6, a n d at M o nt hs 3, 6 a n d 1 2 p ost Da y 0. K e y safet y assess me nts s uc h as 
s u d de n u ne x pecte d deat h, a n d ot h er maj or a d vers e car dia c e ve nts ( M A C E), will be assesse d 
f or all s u bjects, wit h t he e x ce pti o n of t h ose o nl y rele va nt t o s u bjects assi g n e d t o t he acti ve 
treat me nt ar m, s uc h as  n e w TI MI fl o w 0- 2 or TI MI m y ocar dial perf usi o n gra de ( T M P G) 0- 2 
( persisti n g > 3 mi n utes after ter mi nati o n of cell i nf usi o n). Si nce C A P- 1 0 0 2 is all o ge neic,
h u m oral a n d cell ular i m m u ne res p o nses will als o be e val uat e d.   
T he Data a n d Safet y M o nit ori n g B oar d ( D S M B) will meet per c harter, at least se mia n n uall y t o 
re vie w a vaila ble data, a n d will c o n d uct a n i nteri m re vie w of s afet y d ata f or Gr o u p 1 s u bjects 
t hr o u g h 7 2 h o urs p ost- d a y 0.  T he y will re vie w b ot h safet y a n d efficac y data after all s u bjects 
ha ve c o m plete d 6 m o nt hs of f oll o w- u p. 
Fi g ure 1 0 pr o vi d es a sc h e matic re prese ntati o n of t he st u d y d esi g n. F urt her details re gar di n g
s pecific st u d y visits ca n b e f o u n d i n A p pe n di x 1, Sc he d ule of E ve nts .

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 4 6 of 1 1 1
Fi g ur e 1 0.
S c h e m ati c of t h e st u d y d e si g n
3. 2
E x pl or at or y Effi c a c y E n d p oi nt s
E x pl orat or y efficac y e n d p oi nts will be e val uate d at t h e si x- a n d t wel ve- m o nt h f oll o w- u p
visits. T he e x pl orat or y efficac y e n d p oi nts i ncl u de:
1.
Car diac Str uct ural: A bs ol ute a n d relati ve c ha n ge i n para meters meas ure d b y car diac 
M RI, i n cl u di n g:
a.
Left ve ntric ular E F ( %);
b.
Left ve ntric ular e n d- di ast olic v ol u me a n d e n d-s yst olic v ol u me;
c.
Left ve ntric ular str o ke v ol u m e;
d.
Re gi o nal left ve ntric ular f u ncti o n assess me nt;
e.
Left ve ntric ular  L G E e x press e d as a perce nt of left ve ntric ular mass;
f.
Left ve ntric ul ar L G E e x presse d i n gra ms;
g.
Left ve ntric ular via ble m ass e x presse d i n gra ms;
h.
Left ve ntric ular circ u mfere nti al strai n;
i.
N u m ber of left v e ntric ular se g me nts wit h L G E.  
2.
F u ncti o nal: S erial c ha n ges i n Perf or ma nce of U p p er Li m b ( P U L) s cale, s pir o metr y,
a n d 6- mi n ute w al k test ( 6 M W T) w he n dee me d a p pr o priate b y t he I n v esti gat or.

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 4 7 of 1 1 1
3.
Q ualit y of Life: C h a n ge i n Pe ds Q L ( Pe diatric Q u alit y of Life I n ve nt or y), i n cl u di n g 
t he car diac m o d ule, a n d P O D CI A d oles ce nt Q u esti o n naire.
4.
Bi o mar kers: Oste o p o nti n, S T 2, I L- 1 0, a n d Galecti n- 3; f or s u bjects pr o vi di n g 
c o nse nt/ asse nt, a d diti o nal sa m ples f or e x pl orat or y a nal ys es will b e c ollecte d, 
arc hi v e d, a n d a vaila bl e f or f ut ure testi n g.
3. 3
S af et y M o nit ori n g
T he st u d y will m o nit or t he pr o p orti o n of s u bjects e x peri e nci n g a n y of t he f oll o wi n g e ve nts 
d uri n g or p ost i ntrac or o n ar y i nf usi o n deli v er y. F or c o m paris o n, t hese sa me e ve nts will be 
m o nit ore d i n us u al care s u bjects usi n g 9: 0 0 a m o n Da y 0 as t he refere n ce ti me p oi nt:
1.
Ne w TI MI fl o w 0- 2 or TI MI m y o car di al perf usi o n gra de ( T M P G) 0- 2, n ot e d 
i m m e diatel y f oll o wi n g i ntrac or o n ar y i nf usi o n of C A P- 1 0 0 2 a n d persisti n g > 3 mi n 
after cell i nf usi o n, des pite i ntrac or o n ar y vas o dilat or a d mi nistrati o n.
2.
S u d de n u ne x pecte d deat h wit hi n 7 2 h o urs of i ntrac or o nar y i nf usi o n defi ne d as 
occ urri n g wit hi n o ne h o ur of s y m pt o m o nset, or u n- wit nesse d deat h i n a p ers o n 
pre vi o usl y o bs er ve d t o b e w ell wit hi n t he prece di n g 2 4 h o urs wit h o ut a n i de ntifie d 
ca use.
3.
Maj or a d vers e car diac e v e nts ( M A C E) wit hi n 7 2 h o urs of i ntrac or o nar y i nf usi o n, 
i ncl u di n g deat h, n o n-fat al m y ocar dial i nfarcti o n a n d h os pit aliz ati o n f or 
car di o vasc ular e v e nt (i ncl u di n g heart fail ure h os pitalizati o ns). E vi de nce of 
m y ocar dial i nj ur y will be assesse d b y a r ul e o ut MI car di ac e nz y me pr ot oc ol 
perf or me d f oll o wi n g a d mi nistrati o n of C A P- 1 0 0 2. Tr o p o ni n a n d C K- M B will be 
o btai ne d e ver y 8 h o urs ( ± 3 0 mi n utes) at mi ni m u m f or 2 0- 2 4 h o urs after C A P- 1 0 0 2
i nf usi o n.
T he st u d y will e val uate t h e f oll o wi n g d uri n g t h e si x a n d t wel ve m o nt h f oll o w- u p peri o d:
1.
S u d de n u ne x pecte d deat h defi n e d as o cc urri n g wit hi n o ne h o ur of s y m pt o m o nset, or 
u n wit nesse d deat h i n a p ers o n pre vi o usl y o bser v e d t o be well wit hi n t he prece di n g 2 4 
h o urs wit h o ut a n i de ntifie d ca use.
2.
Maj or a d vers e car diac e v e nts ( M A C E), i n cl u di n g deat h, n o n-fatal m y o car dial 
i nfarcti o n, h os pitalizati o n f or car di o vasc ular e ve nt (i ncl u di n g heart fail ure 
h os pitalizati o ns), e mer ge nc y r o o m treat m e nt f or h eart fail ure (i ncl u di n g o ut p atie nt 
i nf usi o n), left ve ntric ul ar assist de vice or heart tra ns pla nt.
3.
A n y h os pit aliz ati o n d ue t o a car di o v asc ular ca us e.
4.
A n y i nter-c urre nt car di o v asc ular ill ness w hic h pr ol o n gs h os pitalizati o n. 
5.
De vel o p me nt of, or a n i n creas e i n t he fre q ue n c y of V T wit h d urati o n of 3 0 sec o n ds or 
l o n ger ascertai ne d b y c o nti n u o us car diac r h yt h m m o nit ori n g. 

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 4 8 of 1 1 1
6.
De vel o p me nt of i ncreas e d a nti- H u m a n Le u k o c yt e A nti ge n ( H L A) a nti b o d y le vels 
wit h de vel o p me nt of se nsiti zati o n t o H L A a nti ge ns s pecific t o t he C A P- 1 0 0 2 C D C 
d o n or at i m m u n ol o gicall y si g nifica nt titers.
7.
Pea k c ha n ge fr o m b aseli ne i n tr o p o ni n a n d C K- M B le vels f oll o wi n g C A P- 1 0 0 2
i nf usi o n.
3. 4
R ati o n al e f or St u d y D e si g n a n d C o ntr ol Gr o u p
Male s u bjects wit h car di o m y o pat h y sec o n dar y t o D M D are b ei n g recr uite d f or t his trial. 
D M D is a n x-li n ke d disease. Fe males o nl y de vel o p D M D w he n t h e y are h o m oz y g o us f or t he 
c o n diti o n. T his is s o e xtre mel y rare t hat t he c ha nce of recr uiti n g e n o u g h fe male s u bjects t o 
t his trial t o ma ke a n y s afet y c o ncl usi o ns ma kes t h e e x cl usi o n of fe males a p pr o priate i n t his 
sit u ati o n.
A n o pe n c o ntr ol gr o u p recei vi n g us u al care has b ee n c h ose n f or t his st u d y i n or der t o ha v e a 
clear c o m parat or f or s afet y efficac y. It was n ot felt reas o na ble t o h a ve c o ntr ol s u bjects 
u n der g o eit h er m ulti- vess el place b o i nf usi o n or a s ha m pr oce d ure. 
Car diac i n v ol v e me nt ca n start i n c hil d h o o d or a d olesce nce a n d pr o gress at a varia ble rate. 
W hile m ost a d ults wit h D M D ha ve si g nifica nt e vi de nce of car di ac i n v ol ve me nt, a si g nifica nt 
pr o p orti o n of a d olesce nts ha ve pr o gressi ve si g nifica nt car diac i n v ol ve me nt s u p p orti n g t he 
nee d t o i n vesti gate t he safet y a n d effecti ve ness of t his treat m e nt i n s u bjects at least 1 2 years 
of a ge. 
3. 5
St u d y D ur ati o n a n d D at e s
N o st u d y-relate d pr oce d ures will be perf or me d u ntil a s u bject pr o vi des c o ns e nt or asse nt 
al o n g wit h pare ntal or g u ar dia n per missi o n. After war ds, a si n gle s u bject’s parti ci pati o n is 
e x pecte d t o last a p pr o xi matel y t hirtee n m o nt hs fr o m t he ti m e of t he first scree ni n g pr oce d ure. 
T he st u d y will re q uire u p t o se ve n sc he d ule d visits t o t he st u d y site: Scree ni n g, I nf usi o n, 
Wee ks 2 a n d 6, a n d M o nt hs 3, 6, a n d 1 2. A n n ual f oll o w- u p p h o ne calls will be c o n d ucte d f or 
a n a d diti o nal f o ur years o nl y if t he s u bject pr o vi des se parate c o nse nt or asse nt. 
It is esti mate d t hat 1 2 m o nt hs of accr u al will be re q uire d t o e nr oll t he tar get sa m ple size f or 
t his P hase II st u d y. 

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 4 9 of 1 1 1
4
S T U D Y P O P U L A TI O N S E L E C TI O N
4. 1
St u d y P o p ul ati o n
A p pr o xi matel y t w e nt y-f o ur ( 2 4) male s u bjects wit h car di o m y o pat h y sec o n dar y t o D M D a n d
left ve ntric ular s car b y l ate ga d oli ni u m e n ha nce m e nt ( L G E) car dia c M RI i n at least 4 
se g me nts will be c o nsi dere d f or e nr oll me nt i n t he st u d y.
4. 2
I n cl u si o n Crit eri a
Eac h s u bject m ust meet all of t he f oll o wi n g i ncl usi o n crit eria t o be ra n d o mi ze d i n t he st u d y:
1.
Male s u bjects 1 8 years of a ge or ol d er m ust be a bl e t o pr o vi de i nf or me d c o nse nt 
a n d f oll o w u p wit h pr ot oc ol pr oce d ures. Male s u bjects at least 1 2 years of a ge 
b ut y o u n ger t h a n 1 8 years of a ge m ust be a ble t o pr o vi de asse nt wit h pare nt or 
g uar dia n pr o vi di n g per missi o n f or st u d y partici pati o n. O nl y male s u bjects will be 
ra n d o mize d i nt o t his st u d y.
2.
D oc u me nte d dia g n osis of D uc he n n e M us c ular D ystr o p h y b y ge neti c m utati o n 
a nal ysis.
3.
Car di o m y o pat h y wit h left ve ntric ul ar scar b y L G E i n at least 4 se g me nts as 
assess e d b y c o ntrast-e n h a nce d M RI, a n d E F > 3 5 % at t h e ti me of scree ni n g.
4.
Use of e vi de nce b ase d m e dical-t hera p y i n acc or d a nce wit h t he “ D M D Care 
C o nsi d erati o ns W or ki n g Gr o u p” g ui deli n es f or t he ma na ge me nt of D M D, f or at 
least t hree m o nt hs pri or t o si g ni n g t h e c o nse nt f or m ( or, pr o vi di n g asse nt) or 
d oc u me nte d c o ntr ai n dicati o n or i nt olera nce or p atie nt prefere n ce.
5.
S u bjects m ust be ta ki n g s yste mic gl uc o c ortic oi ds f or at least si x m o nt hs pri or t o 
scree ni n g.
6.
S u bjects m ust be 1 2 years of a ge or ol d er at scree ni n g.
7.
S u bjects m ust be a p pr o priate ca n di d ates f or car dia c cat heterizati o n a n d 
i ntrac or o nar y i nf usi o n of C A P- 1 0 0 2, i n t he j u d ge me nt of t he site’s i nter ve nti o nal
car di ol o gist.
4. 3
E x cl u si o n Crit eri a
S u bjects t h at m eet a n y of t h e f oll o wi n g e xcl usi o n criteria will n ot be per mitte d t o e nr oll i nt o 
t he st u d y: 
1.
T hera p y wit h i ntra ve n o us i n otr o pic or vas oacti ve me dicati o ns at t h e ti me of 
scree ni n g.
2.
I n a bilit y t o u n der g o car di ac c at heterizati o n a n d/ or M RI wit h o ut ge neral 
a nest h esia.

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 5 0 of 1 1 1
3.
I m m u n ol o gic i nc o m pati bilit y wit h all a v aila ble M aster C ell B a n ks ( M C Bs) b y 
si n gle-a nti ge n b ea d ( S A B) ser u m a nti b o d y pr ofili n g.
4.
Pl a n n e d or li kel y maj or s ur ger y i n t he ne xt 1 2 m o nt hs after pla n n e d 
ra n d o mizati o n.
5.
Left Ve ntric ul ar Assist D e vices ( L V A D) or t h ose s u bjects acti vel y i n t he pr ocess 
of ac q uiri n g a L V A D.
6.
C o ntrai n dicati o n t o car di ac M RI.
7.
K n o w n h y p erse nsiti vit y t o c o ntrast a ge nts.
8.
Esti mate d gl o mer ular filtrati o n rate ( G F R) < 6 0 m L/ mi n, as calc ulate d b y t he 
C K D- E PI c ystati n C e q u ati o n (I n ker, Sc h mi d et al. 2 0 1 2).
9.
Acti ve i nfecti o n n ot res p o nsi ve t o treat m e nt.
1 0.
Acti ve s yst e mic aller gic reacti o n(s), c o n necti ve tiss ue dis ease or a ut oi m m u ne 
dis or der(s).
1 1.
Hist or y of car di ac t u m or or car di ac t u m or de m o nstrate d o n scree ni n g M RI.
1 2.
Hist or y of pre vi o us ste m cell t h era p y.
1 3.
Hist or y of us e of me dicati o ns liste d i n A p p e n di x 3 wit hi n 3 m o nt hs pri or t o 
si g ni n g t he I C F / Ass e nt t hr o u g h c o m pleti o n of t he st u d y i nf usi o n. 
1 4.
K n o w n m o derat e-t o-se vere a ortic ste n osis/i ns ufficie nc y or se v ere mitral 
ste n osis/re g ur gitati o n.
1 5.
C urre nt acti v e alc o h ol or dr u g a b use. 
1 6.
K n o w n hist or y of H u m a n I m m u n o deficie nc y Vir us ( HI V) i nfecti o n.
1 7.
K n o w n hist or y of c hr o ni c viral he patitis.
1 8.
A b n or mal li ver f u ncti o n ( A L T/ A S T > 1 0 ti mes t he u p per refere nce ra n ge) a n d/ or 
st u dies wit h o ut a re versi ble, i de ntifia ble ca us e.
1 9.
K n o w n h y p erse nsiti vit y t o b o vi ne pr o d ucts.
2 0.
K n o w n h y p erse nsiti vit y t o di met h yl s ulf o xi de ( D M S O).
2 1.
U nc o ntr olle d dia betes ( H b A 1c > 9. 0).
2 2. I n a bilit y t o c o m pl y wit h pr ot oc ol-relat e d pr oce d ures, i ncl u di n g re q uire d st u d y 
visits.

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 5 1 of 1 1 1
2 3.
A n y c o n diti o n or ot h er reas o n t hat, i n t he o pi ni o n of t he I n vesti gat or or M e dical 
M o nit or, w o ul d re n der t h e s u bject u ns uita ble f or t he st u d y.
2 4.
C urre ntl y recei vi n g i n v esti gati o nal treat me nt o n a n ot her cli nical trial or 
e x pa n d e d access pr ot oc ol, i ncl u di n g a n y of t h e f oll o wi n g:
•
Recei ve d i n vesti gati o nal i nter ve nti o n wit hi n 3 0 da ys pri or t o ra n d o mizati o n
•
Treat m e nt a n d/ or a n i nc o m plete f oll o w- u p t o treat m e nt wit h a n y 
i n vesti gati o nal cell base d t h era p y wit hi n 6 m o nt hs pri or t o ra n d o mi zati o n
•
Acti ve partici pati o n i n ot her res ear c h t h era p y f or car di o vasc ular 
re pair/re ge nerati o n

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 5 2 of 1 1 1
5
S T U D Y T R E A T M E N T( S)
5. 1 
I n v e sti g ati o n al Pr o d u ct 
C A P- 1 0 0 2 is a n i n vesti gati o nal pr o d uct (I P) c o nsisti n g of all o ge neic car di os p here- d eri v e d 
cells ( C D Cs).
5. 1. 2  
D S A S cr e e ni n g
C A P- 1 0 0 2 C D Cs will b e gr o w n fr o m d o n ors u nrel ate d t o t he reci pie nts. All ra n d o mize d 
s u bjects will be m o nit ore d d uri n g t h e c o urse of t he st u d y f or de v el o p me nt of i ncrease d a nti-
h u ma n le u k oc yte a nti ge n ( H L A) a nti b o d y le v els wit h de vel o p me nt of se nsiti zati o n t o H L A 
a nti ge ns s p ecific t o t he C A P- 1 0 0 2 C D C d o n or at i m m u n ol o gicall y si g nifica nt titers ( b y s oli d 
p hase i m m u n oassa y). W hile t he H L A t y p e of t h e reci pie nt s u bject, d o n or a n d t he C A P- 1 0 0 2 
C D Cs will be teste d a n d k n o w n, pri maril y f or pr o d uct i de ntificati o n p ur p oses, H L A 
matc hi n g of t h e d o n or a n d t he reci pie nt s u bjects will n ot be perf or me d. I nst ea d, s u bjects wit h 
meas ura ble d o n or s pecifi c a nti b o dies a gai nst (as m eas ure d b y t he S A B ass a y d uri n g 
scree ni n g) d o n or s pecific H L A i n all a vaila ble C A P- 1 0 0 2 master cell ba n ks are e x cl u de d 
fr o m t he st u d y.  

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 5 3 of 1 1 1
5. 2 
U s u al C ar e 
Us ual care will i ncl u de a n y a p pr o priate treat me nt(s) per t h e i n vesti gat or’s discreti o n, e xce pt 
t hat us ual care ca n n ot i n cl u de treat m e nt wit h C A P- 1 0 0 2, ot her ste m cell treat me nts, or ot her 
i n vesti gati o nal t hera pies d uri n g t he s u bject’s 1 2 m o nt h st u d y peri o d.  
S u bjects ra n d o miz e d t o us ual care will recei ve o pti mal me dical t h era p y acc or di n g t o t he 
treat me nt pla n of t h eir pri mar y D M D d oct or a n d site i n vesti gat or, i nf or me d b y t h e e vi de nce 
base d m e dical-t hera p y i n acc or da nce wit h t he “ D M D Care C o nsi derati o ns W or ki n g Gr o u p” 
g ui deli nes f or t he m a na ge me nt of D M D ( B us h b y, Fi n kel et al. 2 0 1 0a, B us h b y, Fi n kel et al. 
2 0 1 0 b).  
5. 2. 1  
C A P- 1 0 0 2 A c c e s s P o st- St u d y
If t he a v aila ble e vi de n ce s u g gests a n a p pr o priate ris k/ be nefit pr ofile f or t his pr o d uct, 
Ca pric or ma y ( wit h D S M B a gree me nt) o p e n a n o pe n-la bel e xte nsi o n pr ot oc ol t o ma ke C A P-
1 0 0 2 a vaila ble f or Us ual Care s u bjects after t he y c o m plete t h e 1 2- m o nt h st u d y ass ess me nt 
peri o d.  
5. 3 
R a n d o mi z ati o n  
C o nse nte d, eli gi ble s u bjects will b e ra n d o mize d 1: 1 t o recei ve eit h er C A P- 1 0 0 2 or us ual 
care.
5. 4 
C o n c o mit a nt T h er a p y 
All me dicati o n t hera pies recei v e d fr o m t he ti me of si g ni n g c o nse nt will be c ollecte d
( be gi n ni n g wit h t he Scree ni n g visit). Me dic ati o ns i n use fr o m i nitiati o n of t h e i nf usi o n 
pr oce d ure f or acti v e t h era p y s u bjects, or 9a m o n D a y 0 f or us u al care s u bj ects,  are 
c o nsi dere d c o nc o mita nt t hera pies. C o nc o mita nt t h era pies will be rec or de d at eac h st u d y visit.  

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 5 4 of 1 1 1
5. 5 
Pri or T h er a p y E x cl u si o n s/ Pr o hi bit e d M e di c ati o n s
S u bjects w h o ha ve ha d a n y of t he f oll o wi n g pri or t h era pies will n ot b e eli gi ble t o partici pate 
i n t h e st u d y: 
T hera p y wit h i ntra ve n o us i n otr o pic or vas oacti v e me dicati o ns at t he ti me of scree ni n g 
( E xcl usi o n Crit eri o n 1)
Left Ve ntric ul ar Assist D e vices or acti vel y i n t he pr ocess of ac q uiri n g a L V A D
( E xcl usi o n Crit eri o n 5)
Pre vi o us ste m cell t hera p y ( E xcl usi o n Criteri o n 1 2)
Me dicati o ns liste d i n A p pe n di x 3 are pr o hi bite d wit hi n 3 m o nt hs pri or t o si g ni n g t he I C F / 
Asse nt t hr o u g h c o m pleti o n of t he st u d y i nf usi o n/ St u d y Da y 0. ( E xcl usi o n Crit eri a 1 3)
T he f oll o wi n g m e di cati o ns are pr o hi bite d fr o m 3 0 da ys pri or t o ra n d o mi zati o n, t hr o u g h t he 
M o nt h 1 2/ E arl y T er mi nati o n st u d y visit:
A n y ste m cell t hera pi es ot her t ha n C A P- 1 0 0 2 
A n y i n vesti gati o nal treat me nts ot her t h a n C A P- 1 0 0 2  
5. 6 
Tr e at m e nt C o m pli a n c e 
Treat m e nt i n H O P E- D uc he n ne is a o ne-ti m e pr oce d ure. T h eref ore, c o nti n ue d c o m plia nce 
wit h t h e i n vesti gati o nal pr o d uct is n ot a p plica ble. St u d y site p ers o n nel will d oc u m e nt 
c o m plia nce wit h a n y c o n c o mita nt me dicati o ns a n d st u d y f oll o w- u p pr o ce d ures at e ac h st u d y 
visit.
  

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 5 5 of 1 1 1
5. 9 
I n v e sti g ati o n al Pr o d u ct R et e nti o n at St u d y Sit e 
Si n ce pr o d uct will be deli vere d as- n ee d e d o n a per s u bject basis, t here will be n o pr o d uct t o 
retai n at t he site.  

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 5 6 of 1 1 1
6
S T U D Y P R O C E D U R E S
6. 1
I nf or m e d C o n s e nt
All s u bjects t h at are at least of 1 8 years of a g e m ust pr o vi d e writte n c o nse nt pri or t o 
perf or mi n g a n y st u d y-relate d pr oce d ure. All s u bjects t hat are at least 1 2 years of a ge, b ut less 
t ha n 1 8, m ust pr o vi de writte n asse nt al o n g wit h pare ntal or g uar dia n writt e n per missi o n pri or 
t o perf or mi n g a n y st u d y-relate d pr o ce d ure.  A n I nf or me d C o nse nt F or m (I C F) will be 
pr o vi de d t o a n a d ult s u bject f or t heir c o nsi derati o n. A n Asse nt F or m ( A F) will be pr o vi de d t o 
a n a d olesce nt s u bject a n d t he s u bject’s pare nt(s) or g uar di a n(s) f or t h eir c o nsi derati o n. Eac h 
f or m will c o ntai n all U nite d States fe d erall y re q uire d ele me nts, all I C H-re q uire d ele me nts, 
a n d Healt h I ns ura nce P orta bilit y a n d Acc o u nta bilit y A ct ( HI P A A) a ut h orizati o n i nf or mati o n 
i n la n g ua ge t h at is u n dersta n da ble t o t he s u bject a n d s u bject’s  pare nt(s) or g uar dia n(s), if 
a p plica ble. T h e pr ocess of o btai ni n g i nf or me d c o ns e nt or asse nt will be i n c o m pli a nce wit h 
all fe deral re g ulati o ns, I nter n ati o nal C o nfere nce o n Har m o nisati o n (I C H) re q uire me nts, a n d 
l ocal la ws.  
T he I n vesti gat or a n d/ or desi g n ee m ust disc uss t he st u d y wit h eac h s u bje ct a n d t h e s u bject’s 
pare nt(s) or g uar di a n(s), if a p pli ca ble. T h e dis c ussi o n will i ncl u de t h e nat ure, sc o pe, 
pr oce d ures, a n d p ossi ble c o nse q ue nces of t he s u bject's partici pati o n i n t he st u d y. T he I C F/ A F 
a n d disc ussi o n will be i n a f or m u n derst a n da ble t o t h e s u bject a n d t he s u bject’s pare nt(s) or 
g uar dia n(s). T he s u bject a n d s u bject’s pare nt(s) or g uar dia n(s) m ust b e gi v e n a de q uate ti m e 
t o re vie w t he I C F/ A F a n d as k q uesti o ns re gar di n g t he I P, pr o ce d ures, ris ks a n d ot h er 
treat me nt.  F or s u bjects at least 1 8 years of a ge, a n I C F m ust be si g n e d a n d date d b y t he 
s u bject a n d t he I n v esti gat or or desi g nee bef ore partici pati o n i n t h e st u d y. F or s u bjects t hat are 
at least 1 2 years of a ge, b ut less t ha n 1 8, a n A F m ust be si g ne d a n d date d b y t he s u bject, t he 
s u bject’s pare nt(s) or g u ar dia n(s), a n d t h e I n vesti gat or or desi g nee bef ore t h e s u bject ca n 
partici pate i n t he st u d y. T he s u bject will rec ei ve a c o p y of t he si g ne d a n d date d I C F, a n d t he 
ori gi nal will be retai ne d i n t he site st u d y files. T he s u bject a n d pare nt(s) or g uar dia n(s) will 
recei v e a c o p y of t h e si g ne d a n d date d A F, a n d t he ori gi n al will be retai n e d i n t he st u d y sit e 
files. T he I n vesti gat or or desi g nee will e m p hasize t o t h e s u bject a n d s u bject’s pare nt(s) or 
g uar dia n(s) t h at st u d y partici pati o n is e ntirel y v ol u ntar y a n d t hat c o ns e nt a n d/ or asse nt 
re gar di n g st u d y partici pati o n ma y be wit h dra w n at a n y ti me wit h o ut pe nalt y or l oss of 
be nefits t o w hic h t he s u bject is ot her wise e ntitl e d.
M o dificati o ns t o t he st u d y desi g n, a dj ust me nts t o t he met h o d of st u d y c o n d uct, or release of 
ne w s afet y i nf or mati o n ma y o cc ur d uri n g t h e c o urse of t he st u d y t hat ma y re q uire a n 
a me n de d I C F a n d/ or A F. T he a me n d e d I C F a n d/ or A F will c o ntai n all U nit e d States fe d erall y 
re q uire d ele me nts, all I C H-re q uire d ele me nts, a n d H ealt h I ns ura nce P orta bilit y a n d 
Acc o u nta bilit y Act ( HI P A A) a ut h orizati o n i nf or mati o n i n la n g ua ge t h at is u n dersta n da ble t o 
t he s u bject a n d s u bject’s pare nt(s) or g u ar di a n(s). T he site m ust use t he a m e n de d I C F a n d/ or 
A F f or all ne w s u bjects. All o n g oi n g s u bjects m ust re peat t he c o nse nt a n d/ or asse nt pr ocess 
wit h t h e I n v esti g at or or d esi g nee usi n g t he a me n d e d I C F a n d/ or A F. 
O n g oi n g s u bje cts t h at m at ure t o at least 1 8 years of a ge d uri n g t he c o urse of t he st u d y ma y b e 
re q uire d t o pr o vi de writte n i nf or me d c o ns e nt (i.e., I C F) de p e n di n g o n t he re q uire me nts of t he 
re vie wi n g I R B, as w ell as l ocal re g ulati o ns. 

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 5 7 of 1 1 1
6. 2
M e di c al Hi st or y & C o n c o mit a nt
M e di c ati o n s
T he ge neral a n d car diac-s pecific me dical hist ories (e. g., si g nifica nt pr oce d ures, me dicati o ns, 
t hera pies, aller gies) of s u bjects ra n d o mize d i n t he st u d y will be d oc u me nt e d as part of t h e 
Scree ni n g Visit a n d re vi e w e d d uri n g all s u bse q ue nt visits f or a n y m o dificati o ns (i.e., 
assess me nt f or p ote ntial a d vers e e ve nts).  G e neral a n d car dia c healt h, as w ell as c o nc o mita nt 
me dicati o ns, will be re vi e we d a n d ass esse d at eac h visit a n d ne w i nf or mati o n will be u p date d 
i n t h e s u bject’s st u d y d o c u me ntati o n.
6. 3
C ar di a c a n d Li mit e d P h y si c al E x a mi n ati o n s
A q ualifie d p h ysicia n, eit her t h e I n vesti gat or or S u b-I n vesti gat or, will perf or m a
car di o vasc ular p h ysical e x a mi nati o n at eac h st u d y visit.  
A li mit e d p h ysi cal e x a mi nati o n will be c o n d ucte d at t he scree ni n g visit a n d t he 1 2 M o nt h 
f oll o w- u p visit. 
Wei g ht a n d hei g ht will be c ollecte d at Scree ni n g, 6- m o nt h, a n d 1 2- m o nt h visits. Hei g ht al o ne 
will als o be assesse d at t h e 6- wee k a n d 3- m o nt h visits. If sta n di n g hei g ht ca n n ot be 
meas ure d, h ei g ht will be calc ul ate d usi n g a m eas ure me nt of ul na le n gt h ( G a ul d et al 2 0 0 4).
6. 4
Vit al Si g n s
Te m perat ure, heart rate (seate d), bl o o d press ure (s eate d) a n d res pir ati o n rat e will b e rec or de d 
at eac h visit.
6. 5
Cli ni c al L a b or at or y T e st s
6. 5. 1 
L a b or at or y P ar a m et er s
Cli nical la b orat or y tests will i ncl u de t h ose liste d i n 6. 1 0. 1 C A P- 1 0 0 2 I nf usi o n, A p pe n di x 1 
( Sc he d ule of E v e nts), a n d A p pe n di x 2 ( Cli nical La b orat or y T ests). S u bjects will be i n a 
seate d or s u pi ne p ositi o n d uri n g bl o o d c oll ecti o n.
6. 5. 2 
S a m pl e C oll e cti o n, St or a g e, a n d S hi p pi n g
All cli nical la b orat or y testi n g i n t his trial will b e perf or me d b y ce ntr al la b orat or y facilities, 
e x ce pt t h ose perf or m e d o n Da ys 0 a n d 1 f or s u bjects ra n d o mize d t o C A P- 1 0 0 2 o nl y. All 
sa m ples will be c ollecte d, pr ocesse d, st ore d a n d s hi p pe d (as a p plic a ble) acc or di n g t o 
i nstr ucti o ns i n t he Cli nical La b orat or y M a n ual f or t his trial.
F or s u bjects ra n d o mi ze d t o C A P- 1 0 0 2, t he cli nical sit e s h o ul d c oll ect a n d pr ocess sa m ples 
f or t h e f oll o wi n g tests i n acc or da nce wit h t heir i nstit uti o nal p olicies t o a p pr o priatel y m o nit or 
s u bject safet y pre a n d p ost i nf usi o n:

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 5 8 of 1 1 1
T a bl e 2. 
L o c al L a b or at or y T e sti n g, D a y 0 a n d D a y 1 ( C A P- 1 0 0 2 Ar m O nl y)
P re-I nf usi o n
P ost-I nf usi o n
Tr o p o ni n I
Tr o p o ni n I (seri al c ollecti o n)
C K- M B 
C K- M B (seri al c ollecti o n)
Ser u m C h e mistr y *
Ser u m C he mistr y *
He mat ol o g y *
He mat ol o g y *
Uri nal ysis *
Uri nal ysis *
P T T & I N R (if ta ki n g warfari n)
* See A p p e n di x 2 f or details
Ser u m f or t h e d o n or s p ecific a nti b o d y ( D S A) m o nit ori n g, H L A t y pi n g a n d bl o o d f or t h e 
E LI S p ot assa y will be c ollecte d a n d pr o cesse d at eac h sit e a n d t he n s hi p pe d t o a ce ntral c ore 
la b orat or y f or a n al ysis wit hi n 2 4 h o urs of o btai ni n g t he s peci me ns ( See La b orat or y Ma n u al 
f or s hi p pi n g i nstr ucti o ns). S u bjects will be m o nit ore d f or t h e de vel o p me nt of a cell ular
i m m u ne res p o nse usi n g t he E LI S p ot (e nz y me-li n k e d i m m u n os or be nt s p ot) assa y. T he
E LI S p ot assa y will be perf or me d at scree ni n g a n d at t h e
Wee k 6 st u d y visit.
Ser u m f or researc h p ur p oses ( bi o mar kers) will be c ollecte d a n d pr ocesse d at eac h site t he n 
s hi p pe d t o a ce ntral c ore la b t hat will t he n s hi p t o Ca pric or f or fi nal st ora ge a n d a nal ysis (if 
s u bject has pr o vi d e d c o nse nt).
F or s u bjects w h o pr o vi de c o nse nt a n d/ or ass e nt, ser u m f or cli nical researc h i nt o bi o mar kers 
will b e c ollecte d at e ver y sc he d ule d visit (e x cl u di n g i nf usi o n) a n d se nt t o t h e S p o ns or, or 
desi g nee, f or st ora ge a n d/ or a nal ysis (i ncl u di n g a n d f ut ure a n al ysis). Per t he C o de of Fe deral 
Re g ulati o ns ( 2 1 C F R 3 2 0. 3 8), s er u m sa m ples c oll ecte d fr o m st u d y s u bjects will be 
mai ntai ne d i n st ora ge f or at least fi ve ( 5) years f oll o wi n g t he date o n w hic h t he I N D 
a p plicati o n or s u p ple me ntal a p plicati o n is a p pr o ve d. If s uc h a p plicati o n or s u p ple me ntal 
a p plicati o n is n ot a p pr o v e d, ser u m sa m ples will b e mai ntai ne d i n st ora ge f or at least 5 years 
f oll o wi n g t h e dat e of c o m pleti o n of t h e cli nical researc h st u d y.
6. 6
1 2- L e a d E C G
Be gi n ni n g at t he scree ni n g visit, a 1 2- Lea d E C G will be perf or me d at e ver y st u d y visit t o 
assess f or r h yt h m dist ur b a nces a n d n e w m y ocar di al is c he mia or i nfarcti o n. S u bse q ue nt E C Gs 
will b e c o m pare d wit h t h e scree ni n g E C G f or a n y c ha n ges i n r h yt h m a n d c o n d ucti o n. All 
c ha n ges a n d a n y ass o ciate d cli nical si g nifica nce will be e val uate d a n d d o c u me nte d o n t he 
case re p ort f or ms.
6. 7
C o nti n u o u s C ar di a c R h yt h m M o nit ori n g
T he s p o ns or will pr o vi de a weara ble, wireless de vice f or c o nti n u o us car dia c r h yt h m 
m o nit ori n g. S u bjects will wear a m o nit ori n g de vi ce f or d efi ne d s h ort-ter m i nter vals, starti n g 
at scree ni n g, o n Da y 1, a n d at t he Wee k 6, M o nt h 6, a n d M o nt h 1 2 st u d y visits. Ta ble 3 
details t he c o nti n u o us m o nit ori n g d urati o n re q uire d at eac h st u d y visit. T h e m o nit ori n g is 

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 5 9 of 1 1 1
use d t o detect a n d rec or d arr h yt h mias. All c ha n ges a n d a n y ass ociate d cli nical si g nifi ca nce 
will b e e val u ate d a n d d oc u me nte d o n t he case re p ort f or m.
T a bl e 3. 
P er- Vi sit Mi ni m u m C o nti n u o u s C ar di a c M o nit ori n g D ur ati o n s
St u d y Visit
Mi ni m u m C o nti n u o us 
M o nit ori n g D ur ati o n
Scree ni n g
2 4 h o urs
Da y 1 
7 2 h o urs
Wee k 6
7 2 h o urs
M o nt h 6
7 da ys
M o nt h 1 2
7 2 h o urs
6. 8
M a g n eti c R e s o n a n c e I m a gi n g 
All s u bjects will u n der g o c o ntrast-e n h a nce d M RI at scree ni n g a n d t he M o nt h 6 a n d 1 2 st u d y 
visits ( or, Earl y T er mi nati o n Visit). It is a ntici pate d t hat t he d urati o n of eac h M RI sessi o n 
will b e 4 5- 6 0 mi n utes. Ac q uisiti o n para m eters a n d tec h ni q ues are s pecifie d i n t he M RI 
I ma gi n g
M a n ual. T he sit e- base d M RI t ec h n ol o gists will be trai ne d a n d certifie d i n t he 
c o m m o n ac q uisiti o n pr ot oc ol pri or t o i niti ati o n of e nr oll me nt at eac h site.
6. 8. 1  
Ci n e I m a gi n g Pr ot o c ol
A ci n e i m a gi n g pr ot oc ol will be utili ze d f or gl o bal ejecti o n fracti o n a n d L V v ol u mes a n d 
mass deter mi n ati o n. Ac q uisiti o n para meters a n d t ec h ni q ues are s pecifie d i n t he M RI I m a gi n g
Ma n ual. T he site- b ase d M RI tec h nicia ns will be trai ne d a n d certifie d i n t h e c o m m o n 
ac q uisiti o n pr ot oc ol pri or t o i nitiati o n of e nr oll me nt at eac h site.
6. 8. 2  
D el a y e d C o ntr a st- E n h a n c e m e nt Pr ot o c ol
S u bjects will u n der g o a sta n dar dize d ac q uisiti o n pr ot oc ol i ncl u di n g a n i ntra ve n o us i njecti o n 
of ga d oli ni u m c o ntrast as defi ne d i n t h e M RI I ma gi n g Ma n u al c o nsiste nt wit h i nstit uti o nal 
g ui deli nes. 
6. 8. 3 
I m a g e A n al y si s
All st u d y i ma ges a n d a p plica ble i ma gi n g d ata will be se nt t o a n d ce ntrall y rea d at a n 
i n de pe n de nt i m a gi n g c ore b y car di o v asc ular ra di ol o g y e x perts. M RI will be perf or me d o n 
sta n dar d p ha nt o ms f or q u alit y ass ura nce a n d t o detect a n y c h a n ges i n s yste m se nsiti vit y o ver 
ti m e.
6. 8. 4  
Hi st ori c al C ar di a c
M RI
If a s u bject h as a hist orical car diac
M RI sca n a vail a ble pri or t o Scree ni n g d ate, t he site 
s h o ul d re q uest t hat t his hist orical car diac
M RI be se nt t o t he i ma gi n g c ore l a b f or t his trial, t o 
p ote ntiall y deter mi ne t he rate of car diac diseas e pr o gressi o n at t he ti m e of s cree ni n g. If 

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 6 0 of 1 1 1
m ulti ple hist orical c M RIs are a vaila ble, t he m ost rece nt sca n t hat occ urre d at least 6 wee ks pri or 
t o scree ni n g s h o ul d be tra nsferre d.
6. 9 
Cli ni c al F u n cti o n a n d Q u alit y of Lif e M e a s ur e s  
At t he scree ni n g, 6 Wee ks, 3 M o nt hs, 6 M o nt hs, a n d 1 2 M o nt hs ( or Earl y Ter mi nati o n) st u d y 
visits, t he s u bject will be as ke d t o c o m plete t h e Perf or ma nce of U p p er Li m b ( P U L) 
assess me nt a n d s pir o metr y. B ot h t he s u bject a n d t h e pare nt/ g uar di a n will be as ke d t o 
c o m plete t he Pe diatric Q ualit y of Life I n ve nt or y ( Pe ds Q L) a n d t he P O D CI A d olesce nt 
Q uesti o n naire ( P O D CI). 
I n a d diti o n, s u bjects assesse d b y t he I n vesti gat or as ca pa ble of perf or mi n g t he Si x Mi n ute
Wal k Test ( 6 M W T) will d o s o at scree ni n g, 6 We e ks, 3 M o nt hs, 6 M o nt hs, a n d 1 2 M o nt hs 
( or, E arl y T er mi n ati o n) st u d y visits. S u bjects u na ble t o perf or m t he Si x Mi n ute Wal k Test at 
scree ni n g will n ot be re q uire d t o perf or m t he test at a n y s u bse q u e nt visit.   
6. 1 0
I nf u si o n Pr o c e d ur e
6. 1 0. 1
C A P- 1 0 0 2 I nf u si o n
E ver y atte m pt s h o ul d be ma de t o i nf use t h e t hree maj or car di ac territ ories of t he left v e ntricle 
(a nteri or territ or y, lateral territ or y, a n d i nferi or/ p osteri or territ or y).  A d diti o nall y, t h e i nf usi n g 
I n v esti gat or ma y alter t he i nf usi o n pla n d uri n g t h e i nf usi o n pr oce d ure w he n dee me d 
cli nicall y necessar y. T he pr oce d ure will be perf or m e d as f oll o ws: 

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 6 1 of 1 1 1
  
F oll o wi n g i ntrac or o n ar y i nf usi o n, s u bjects will be o bser ve d i n t he h os pital f or 2 0- 2 4 h o urs 
wit h c o nti n u o us car diac (tele metr y) m o nit ori n g. Tr o p o ni n a n d C K- M B l a b orat or y st u dies a n d 
vital si g ns (te m perat ure, H R, B P, a n d R R) will be m o nit ore d e ver y 8 h o urs ( ± 3 0 mi n utes) 
fr o m t he e n d of t he i nf usi o n. A t otal of t hree sets of car di ac e nz y mes will be e val uate d t o r ule 
o ut car diac i nj ur y d uri n g t he p ost-i nf usi o n h os pital sta y t hr o u g h disc h ar ge.  If t he s u bject is 
disc har ge d pri or t o 2 4 h o urs, car dia c e nz y m es will b e dra w n j ust pri or t o disc har ge. 
Pri or t o dis c har ge fr o m t he h os pital, a p h ysi cal assess me nt will be perf or me d w hic h will 
i ncl u de c hest pai n ass ess me nt, re vie w of l a b orat or y res ults, 1 2-lea d E C G, a n d arterial 
p u nct ure sit e as w ell as assess me nt of a d vers e e v e nts. I n t he a bse nce of seri o us a d verse 
e ve nts, s u bjects will be dis c har ge d p er sta n dar d of care practice.
6. 1 0. 2 
I nf u si o n Pr o c e d ur e St o p pi n g Crit eri a
T he o perat or will c o nti n u o usl y m o nit or f or t h e f oll o wi n g St o p pi n g Criteria. If a n y St o p pi n g 
Criteria are met, t he i nf usi n g I n vesti gat or m ust st o p t h e i nf usi o n a n d assess f or cli nical 
si g nifica nce. T h e i nf usi n g I n v esti gat or ma y c o nti n ue wit h t he i nf usi o n if, base d o n t h e 
i nf usi n g I n v esti gat or’s me dical j u d ge me nt a n d cli nical c o nte xt, n o a d diti o nal ris k is prese nt e d 
t o t h e s u bject.  
1.
De vel o p me nt of TI MI fl o w 0- 2 or TI MI m y o car dial perf usi o n gra d e ( T M P G) 0- 2 
persisti n g f or > 3 mi n utes after a c ycl e of i nf usi o n, des pit e a d mi nistrati o n of 
i ntrac or o nar y vas o dilat ors;  
2.
S ustai ne d h y p ote nsi o n u nres p o nsi v e t o fl ui ds a n d/ or atr o pi ne/ vas o press or 
a d mi nistrati o n;
3.
E vi de nce of cere br o v asc ular acci de nt;
4.
E vi de nce of car di ac ta m p o na de;
5.
He m o peri car di u m re q uiri n g pericar di oce ntesis; 

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 6 2 of 1 1 1
6.
S ustai ne d V T/ V F t hat re q uires car di o v ersi o n/ defi brill ati o n or a d mi nistrati o n of a n 
a ntiarr h yt h mic;
7.
E vi de nce of t hr o m b us i n t h e a orta t hat w as n ot pre vi o usl y see n;
8.
S us pect e d or c o nfir me d a ortic dissecti o n;
9.
S us pect e d or c o nfir me d c or o nar y dissecti o n; 
1 0. Ot her e vi de nce of ac ute m y ocar dial i nfarcti o n;
1 1. Ac ute o nset of ri g ors (s y m pt o ms i n dicati v e of ac ute se nsiti vit y or i nfecti o n).

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 6 3 of 1 1 1
7
S T U D Y A C TI VI TI E S
Refer t o A p p e n di x 1 f or t he S c he d ule of E v e nts f or t he c o n d uct of t his st u d y.
7. 1
S cr e e ni n g Vi sit 
Writte n i nf or me d c o nse nt or asse nt m ust be o btai ne d fr o m t he s u bject pri or t o perf or mi n g 
a n y st u d y-relate d t as ks. After w ar ds scree ni n g peri o d f or t hat s u bject will be gi n a n d t h e 
f oll o wi n g scree ni n g pr o ce d ures will be perf or me d ( n ot m ore t ha n 3 0 da ys pri or t o 
ra n d o mizati o n):
•
Re vie w of I ncl usi o n / E x cl usi o n Criteria ( Secti o n 4. 2 a n d Secti o n 4. 3)
•
C oll ecti o n of de m o gra p hic i nf or mati o n
•
Re vie w of me dical a n d car diac hist or y, a n d c o nc o mita nt me dicati o ns
•
Meas ure me nt of vital si g ns ( b o d y te m p erat ure, H R, B P, a n d R R), w ei g ht a n d hei g ht
•
1 2-lea d E C G
•
2 4- h o ur E C G via s p o ns or- pr o vi d e d c o nti n u o us car diac r h yt h m m o nit ori n g s yst e m
•
Car diac a n d li mit e d p h ysical e x a mi nati o n
•
Car diac M RI
•
Ve n o us bl o o d will be c ollecte d f or t h e f oll o wi n g:
-
Cli nical la b orat or y tests: ser u m c he mistr y, he mat ol o g y, a n d s er ol o g y ( H bs A g,
a nti- H C V, a nti- HI V 1/ 2), C ystati n C, Tr o p o ni n a n d C K- M B
-
Bi o mar kers: Oste o p o nti n, S T 2, I L- 1 0, a n d Gal ecti n- 3
-
E x pl orat or y bi o mar kers (if c o nse nt/asse nt pr o vi de d)
-
I m m u n oass a y: H L A, D S A, a n d E LI S P O T
-
See 6. 5 f or a d diti o nal i nf or mati o n o n cli nical la b orat or y testi n g.
•
A uri ne sa m ple will be c ollecte d f or uri n al ysis 
•
P U L Scale
•
6 M W T (if ca pa ble)
•
Pe diatric Q o L I n ve nt or y ( Pe ds Q L)
•
P O D CI
•
S pir o metr y
•
A d vers e E v e nt Assess me nt
•
S u b missi o n of hist orical car diac M RI
S u bjects m ust c o nti n ue t o meet all of t he i ncl usi o n a n d n o ne of t h e e x cl usi o n crit eria b ef ore 
pr ocee di n g t o ra n d o mizati o n. S u bjects w h o d o n ot pr ocee d t o ra n d o mizati o n f or a n y reas o n 
d uri n g scree ni n g will be wit h dra w n fr o m t he st u d y as s cre e n fail ures a n d will ha ve n o f urt h er 
f oll o w u p.

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 6 4 of 1 1 1
7. 2
R a n d o mi z ati o n, D a y 0, a n d D a y 1
S u bjects t h at c o nti n ue t o meet eli gi bilit y will be ra n d o miz e d ( 1: 1) t o eit her t he acti ve 
treat me nt ar m ( C A P- 1 0 0 2) or us u al care ar m. 
F or s u bjects t hat are ra n d o miz e d t o t he us ual care ar m, Da y 0 will be defi ne d as se ve n ( 7)
da ys after t h e date of ra n d o miz ati o n. F or s u bjects t hat are ra n d o mize d t o t he acti ve treat me nt 
ar m, Da y 0 will be defi ne d as t he date of t h e i ntrac or o nar y i nf usi o n of C A P- 1 0 0 2.
F or s u bjects r a n d o mize d t o C A P- 1 0 0 2 o nl y, t he i nf usi o n visit will i ncl u de tests a n d 
assess me nts as descri be d bel o w a n d i n acc or da nce wit h t he site’s sta n dar d o perati n g 
pr oce d ures.
Pre-I nf usi o n:
•
Eli gi bilit y Re vie w
•
Re vie w of me dical a n d car diac hist or y, a n d c o nc o mita nt me dicati o ns
•
Car diac p h ysical e x a mi nati o n
•
Meas ure me nt of vital si g ns (te m perat ure, H R, B P, a n d R R) 
•
1 2- Lea d E C G 
•
Ve n o us bl o o d will be c ollecte d f or t h e f oll o wi n g:
-
Cli nical la b orat or y tests: c he mistr y, h e mat ol o g y 
-
C o a g ulati o n if s u bject is ta ki n g w arfari n
•
Serial ve n o us c oll ecti o n f or car di ac e nz y mes ( Tr o p o ni n a n d C K- M B)
•
A uri ne sa m ple will be c ollecte d f or uri n al ysis
•
A d vers e e ve nt assess me nt
I nf usi o n
Refere n ce Se cti o n 6. 1 0 f or c o m plete i nf usi o n details
P ost-i nf usi o n ( D 0- D 1):
•
Serial c ollecti o n of vital si g ns (te m perat ure, H R, B P, a n d R R) e ver y 8 h o urs ( ± 3 0 
mi n utes) fr o m t he e n d of t he i nf usi o n. 
•
Car diac P h ysical E x a mi nati o n 
•
1 2-lea d E C G 
•
Serial ve n o us c oll ecti o n f or car di ac e nz y mes ( Tr o p o ni n a n d C K- M B)
•
Ve n o us bl o o d will be c ollecte d f or cli nical la b orat or y tests: c h e mistr y, 
he mat ol o g y, a n d uri n al ysis
F oll o wi n g i ntrac or o n ar y i nf usi o n, s u bjects will be o bser ve d i n t he h os pital f or 2 0- 2 4 h o urs 
wit h c o nti n u o us car diac (tele metr y) m o nit ori n g. Tr o p o ni n a n d C K- M B l a b orat or y st u dies 
will b e m o nit ore d e ver y 8 h o urs ( ± 3 0 mi n utes) fr o m t he e n d of t he i nf usi o n. A t otal of t hree 

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 6 5 of 1 1 1
sets of car di ac e nz y mes will be e val uate d t o r ule o ut car diac i nj ur y d uri n g t h e p ost-i nf usi o n 
h os pital sta y t hr o u g h dis c har ge.  If t he s u bject is disc har ge d pri or t o 2 4 h o urs, car diac 
e nz y mes will be dra w n j ust pri or t o disc har ge.
D ay 1 Pri or t o H os pit al Dis c h ar ge:
•
Re vie w of c o n c o mita nt me dicati o ns
•
A d vers e e ve nt assess me nt
•
P h ysical ass ess me nt (c hest pai n, i nf usi o n/arterial p u nct ure sit e(s))
•
Re vie w of la b res ults a n d 1 2-lea d E C G
Pri or t o dis c har ge fr o m t he h os pital, a p h ysi cal assess me nt will be perf or me d w hic h will 
i ncl u de c hest pai n ass ess me nt, re vie w of l a b orat or y res ults, 1 2-lea d E C G, a n d arterial 
p u nct ure sit e as w ell as assess me nt of a d vers e e v e nts. I n t he a bse nce of seri o us a d v erse 
e ve nts, s u bjects will be dis c har ge d p er sta n dar d of care practice.
D a y 1 f or A L L S U B J E C T S:
•
I niti ate 7 2- h o ur c o nti n u o us car diac r h yt h m m o nit ori n g usi n g s p o ns or-s u p plie d 
m o nit or s yste m (i n dicate d f or h o me use)
•
N o st u d y visit is re q uire d f or Da y 1, as s u bjects will recei ve pri or i nstr ucti o n o n 
use of t h e m o nit ori n g s yste m
7. 3
D a y 3 P h o n e Vi sit 
T he i n v esti gat or’s site staff will c o ntact t he s u bject ( or pare nt/ g u ar dia n) b y tele p h o ne o n St u d y 
Da y 3 ( 3 da ys p ost Da y 0) f or safet y f oll o w- u p. St u d y Da y 3 is 3 da ys p ost-i nf usi o n f or 
s u bjects ra n d o mize d t o C A P- 1 0 0 2.  F or s u bjects ra n d o mize d t o us ual care, St u d y Da y 3 is te n 
da ys aft er ra n d o mizati o n.
T he sit e will i n q uire as t o t he s u bj ect’s o verall healt h a n d w het her t he s u bject has ha d a n y 
a d verse e ve nts, es peciall y a n y re q uiri n g h os pitalizati o n or ot her me dical i nter ve nti o n, i n t he 
prece di n g 2 da ys (i.e., Da y 1 – Da y 3).  
If necessar y, t he p h o ne call ma y be c o n d ucte d o n St u d y Da ys 4, 5, or 6.
If t he site is u na ble t o reac h t he s u bject b y tele p h o ne, t he site s h o ul d m a ke t w o m ore atte m pts 
t o c o nt act t he s u bject, o ne eac h o n t he ne xt t w o da ys.
7. 4
W e e k 2 Vi sit ( D a y 1 4 ± 3 d a y s) 
S u bjects will ret ur n t o t he cli nical sit e a n d c o m plete t he f oll o wi n g pr oce d ures:
•
A d vers e e ve nt assess me nt
•
Re vie w of c o n c o mita nt me dicati o ns 
•
Meas ure me nt of vital si g ns (te m perat ure, H R, B P, a n d R R)

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 6 6 of 1 1 1
•
1 2- Lea d E C G
•
Car diac P h ysical E x a mi nati o n
•
Ve n o us bl o o d will be c ollecte d f or t h e f oll o wi n g:
-
Cli nical la b orat or y tests: ser u m c he mistr y, he mat ol o g y 
-
Car di ac e nz y mes: Tr o p o ni n a n d C K- M B
-
Bi o mar kers: Oste o p o nti n, S T 2, I L- 1 0, a n d Gal ecti n- 3
-
E x pl orat or y bi o mar kers (if c o nse nt/asse nt pr o vi de d)
-
I m m u n oass a y: D S A
•
A uri ne sa m ple will be c oll ecte d f or uri n al ysis
7. 5
W e e k 6 Vi sit ( D a y 4 0 ± 7 d a y s) 
S u bjects will ret ur n t o t he cli nical sit e a n d c o m plete t he f oll o wi n g pr oce d ures:
•
A d vers e e ve nt assess me nt
•
Re vie w of c o n c o mita nt me dicati o ns 
•
Meas ure me nt of vital si g ns (te m perat ure, H R, B P, a n d R R) a n d hei g ht
•
1 2- Lea d E C G
•
Car diac P h ysical E x a mi nati o n
•
7 2- h o ur c o nti n u o us car diac r h yt h m m o nit ori n g usi n g s p o ns or-s u p pli e d d e vi ce
•
Ve n o us bl o o d will be c ollecte d f or t h e f oll o wi n g:
-
Cli nical la b orat or y tests: ser u m c he mistr y, he mat ol o g y 
-
Bi o mar kers: Oste o p o nti n, S T 2, I L- 1 0, a n d Gal ecti n- 3
-
E x pl orat or y bi o mar kers (if c o nse nt/asse nt pr o vi de d)
-
I m m u n oass a y: D S A a n d E LI S p ot
•
A uri ne sa m ple will be c oll ecte d f or uri n al ysis
•
P U L Scale
•
6 M W T (if ca pa ble)
•
Pe diatric Q L I n ve nt or y
•
P O D CI
•
S pir o metr y
7. 6
M o nt h 3 Vi sit ( D a y 9 0 ± 7 d a y s)
•
S u bjects will ret ur n t o t he cli nical sit e a n d c o m plete t he f oll o wi n g pr oce d ures:
•
A d vers e e ve nt assess me nt
•
Re vie w of c o n c o mita nt me dicati o ns 
•
Meas ure me nt of vital si g ns (te m perat ure, H R, B P, a n d R R) a n d hei g ht
•
1 2- Lea d E C G
•
Car diac P h ysical E x a mi nati o n

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 6 7 of 1 1 1
•
Ve n o us bl o o d will be c ollecte d f or t h e f oll o wi n g:
-
Cli nical la b orat or y tests: ser u m c he mistr y, he mat ol o g y 
-
Bi o mar kers: Oste o p o nti n, S T 2, I L- 1 0, a n d Gal ecti n- 3
-
E x pl orat or y bi o mar kers (if c o nse nt/asse nt pr o vi de d)
-
I m m u n oass a y: D S A
•
A uri ne sa m ple will be c oll ecte d f or uri n al ysis
•
P U L Scale
•
6 M W T (if ca pa ble)
•
Pe diatric Q L I n ve nt or y
•
P O D CI
•
S pir o metr y
7. 7
M o nt h 6 Vi sit ( D a y 1 8 0 ± 1 4 d a y s) 
S u bjects will ret ur n t o t he cli nical sit e a n d c o m plete t he f oll o wi n g pr oce d ures:
•
A d vers e e ve nt assess me nt
•
Re vie w of c o n c o mita nt me dicati o ns 
•
Meas ure me nt of vital si g ns (te m perat ure, H R, B P, a n d R R), wei g ht a n d hei g ht
•
1 2- Lea d E C G
•
Car diac P h ysical E x a mi nati o n
•
Car diac M RI
•
Se ve n da y c o nti n u o us car diac r h yt h m m o nit ori n g usi n g s p o ns or-s u p plie d d e vice
•
Ve n o us bl o o d will be c ollecte d f or t h e f oll o wi n g:
-
Cli nical la b orat or y tests: ser u m c he mistr y, he mat ol o g y, a n d c ystati n C
-
Bi o mar kers: Oste o p o nti n, S T 2, I L- 1 0, a n d Gal ecti n- 3
-
E x pl orat or y bi o mar kers (if c o nse nt/asse nt pr o vi de d)
-
I m m u n oass a y: D S A
-
Car di ac e nz y mes: Tr o p o ni n a n d C K- M B
•
A uri ne sa m ple will be c oll ecte d f or uri n al ysis
•
P U L Scale
•
6 M W T (if ca pa ble)
•
Pe diatric Q L I n ve nt or y
•
P O D CI
•
S pir o metr y

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 6 8 of 1 1 1
7. 8
M o nt h 1 2 Vi sit ( D a y 3 6 0 ± 3 0 d a y s) 
S u bjects will ret ur n t o t he cli nical sit e a n d c o m plete t he f oll o wi n g pr oce d ures:
•
A d vers e e ve nt assess me nt
•
Re vie w of c o n c o mita nt me dicati o ns 
•
Meas ure me nt of vital si g ns (te m perat ure, H R, B P, a n d R R), wei g ht a n d hei g ht
•
1 2- Lea d E C G
•
Car diac a n d li mit e d p h ysical e x a mi nati o ns
•
Car diac M RI
•
7 2 h o ur c o nti n u o us car di ac r h yt h m m o nit ori n g usi n g s p o ns or-s u p pli e d d e vi ce
•
Ve n o us bl o o d will be c ollecte d f or t h e f oll o wi n g:
-
Cli nical la b orat or y tests: ser u m c he mistr y, he mat ol o g y, a n d c ystati n C
-
Bi o mar kers: Oste o p o nti n, S T 2, I L- 1 0, a n d Gal ecti n- 3
-
E x pl orat or y bi o mar kers (if c o nse nt/asse nt pr o vi de d)
-
I m m u n oass a y: D S A
-
Car di ac e nz y mes: Tr o p o ni n a n d C K- M B
•
A uri ne sa m ple will be c oll ecte d f or uri n al ysis
•
P U L Scale
•
6 M W T (if ca pa ble)
•
Pe diatric Q L I n ve nt or y
•
P O D CI
•
S pir o metr y
7. 9
A n n u al F oll o w- u p P h o n e C all 
F oll o wi n g a s u bject’s last site visit, a n n u al f oll o w- u p p h o ne calls will be c o n d ucte d f or a n 
a d diti o nal f o ur years f or s u bjects t hat pr o vi de c o nse nt a n d/ or asse nt. S u bjects will be c o nt acte d 
b y p h o ne a n n uall y ( ± 2 w ee ks) o n t he a n ni versar y of t heir Da y 0 t hr o u g h year 5 t o assess f or 
ac ute or pla n ne d pr o ce d ure a n d/ or a d missi o n f or car diac ca use as well as s ur vi val. T hes e 
a n n ual f oll o w- u p p h o ne calls will c o nti n ue i n c o ncert wit h a s u bject’s n or mal, a n n ual cli nical 
f oll o w- u p w hic h ma y i n cl u de a n n ual car diac M RI w here cli nicall y i n dicate d.
7. 1 0
R e m o v al of P ati e nt s fr o m t h e Tri al or St u d y Dr u g
T he I n vesti gat or ma y wit h dra w a s u bje ct fr o m partici pati o n i n t he st u d y f or a n y of t he 
f oll o wi n g reas o ns:
•
A si g nific a nt pr ot o c ol vi olati o n occ urs,
•
A n i nt olera ble a d v erse e ve nt o cc urs, t hat affects c o nti n uati o n of s u bject’s 
partici pati o n i n t h e st u d y,
•
T he S p o ns or or I n vesti gat or ter mi n ates t he st u d y, or
•
T he s u bject re q u ests t o be disc o nti n ue d fr o m t he st u d y.

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 6 9 of 1 1 1
If a s u bject wis h es t o disc o nti n ue st u d y partici pati o n s olel y f or t h e p ur p ose of e nr olli n g i n 
a n ot her cli nical trial or e x pa n de d access pr ot oc ol, t he i n vesti gat or s h o ul d ma ke e ver y atte m pt 
t o c o nti n ue t o c ollect f oll o w- u p me dical i nf or m ati o n f or t he s u bject per t his trial’s sc he d ule. 
T his s pecific cate g or y of pr ot oc ol vi olati o n (rec ei pt of pr o hi bite d me dicati o ns) will be 
ma na ge d as d efi ne d i n t h e S A P.
7. 1 1
E arl y T er mi n ati o n Pr o c e d ur e s
E ver y reas o na ble eff ort will be ma de t o retai n s u bjects i n t he st u d y. I n t he e ve nt t hat a s u bject 
m ust be wit h dra w n fr o m t he st u d y earl y, st u d y p ers o n nel s h o ul d atte m pt t o o btai n testi n g a n d
data re q uir e d f or t h e 1 2 M o nt h Visit (see Secti o n 7. 8) as well as t he reas o n f or wit h dra w al, if 
k n o w n. A Car diac M RI will be c o n d ucte d pr o vi d e d t h e s u bject’s m ost rece nt st u d y-relate d 
Car diac M RI w as c o n d ucte d at least t hree m o nt hs (i.e., 1 2 wee ks) pri or t o t he Earl y 
Ter mi n ati o n Visit.
If t he s u bject per mits, he or s he s h o ul d be c o ntacte d b y st u d y pers o n n el at t h e ti me of t h eir 
us ual st u d y visits (a n d a n n uall y t hr o u g h 5 years p ost-i nf usi o n), as descri b e d i n t h e pr ot oc ol.
7. 1 2
L o st t o F oll o w- U p
A s u bject ca n b e c o nsi dere d “l ost t o f oll o w- u p” if he misses a visit a n d st u d y pers o n nel are 
u na ble t o c o ntact hi m, or pare nt(s)/ g u ar dia n(s) as a p pli ca ble, i n a ti mel y ma n ner wit h a 
mi ni m u m of t hree d o c u me nte d p h o ne calls. If c o nt act wit h t he s u bject, or 
pare nt(s)/ g u ar dia n(s) as a p pli ca ble, is n ot acc o m plis he d, st u d y pers o n nel s h o ul d f oll o w- u p
wit h certifie d mail i n t h e f oll o wi n g ma n ner:
1.
Mail a certifie d letter wit h ret ur n recei pt re q uesti n g c o nt act a n d e x pressi n g c o ncer n f or 
t he s u bject’s well- b ei n g.
2. If t h e s u bject, or p are nt(s)/ g uar dia n(s) as a p plica ble, d oes n ot res p o n d wit hi n se ve n da ys, 
mail a n ot her certifie d letter wit h ret ur n recei pt st ati n g t hat t he s u bject’s parti ci pati o n i n 
t he st u d y has b ee n ter mi n ate d.
3. If a si g ne d maili n g recei pt f or t h e sec o n d letter is ret ur ne d, rec or d t he t er mi n ati o n date as 
t he date t he s u bject, or pare nt(s)/ g uar di a n(s) as a p plica ble, si g n e d it. Ot her wise, rec or d
t he ter mi nati o n date as t h e date t he sec o n d letter w as maile d.
If, des pite t he eff orts n ot e d a b o ve, it is necessar y t o classif y a s u bject as l ost t o f oll o w- u p,
t hat s u bject’s E n d of St u d y ( E O S) is t he date/ti m e of last c o ntact or k n o w n e ve nt (i n cl usi v e 
of st u d y visits, tele p h o ne c o ntacts, or ot her v erificati o n of s u bject s ur vi val or m ortalit y).

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 7 0 of 1 1 1
7. 1 3
A d v er s e E v e nt s A s s e s s m e nt s
7. 1 3. 1
P erf or mi n g A d v er s e E v e nt s A s s e s s m e nt s
A n A d verse E ve nt ( A E) is a n y u nt o w ar d me dic al occ urre nce i n a s u bject or cli nical 
i n vesti gati o n s u bject te m p orall y ass o ciate d wit h t he use of a m e dici nal pr o d uct, w het her or 
n ot c o nsi dere d relate d t o t he me dici nal pr o d uct. T he occ urre n ce d oes n ot n ecessaril y h a ve t o 
ha ve a ca usal relati o ns hi p wit h t his treat m e nt. A n A E ca n t h eref ore be a n y u nfa v ora bl e a n d 
u ni nte n de d si g n (i n cl u di n g a n a b n or mal la b orat or y fi n di n g, f or e xa m ple), s y m pt o m, or 
disease ( ne w or e xacer bate d) te m p orall y ass ociate d wit h t he use of a me dici nal pr o d uct, 
w het h er or n ot c o nsi dere d relate d t o t he m e dici n al pr o d uct.
E x a m ples of a n A E i ncl u de:
•
E x acer bati o n of a c hr o ni c or i nter mitte nt pre-e xisti n g c o n diti o n i ncl u di n g eit h er a n 
i ncreas e i n fre q u e nc y or i nte nsit y of t he c o n diti o n.
•
Si g nifica nt or u n e x pecte d w orse ni n g or e x acer bati o n of t he c o n diti o n/i n dicati o n 
u n der st u d y.
•
A ne w c o n diti o n detecte d or dia g n ose d after st u d y t hera p y a d mi nistrati o n e ve n 
t h o u g h it ma y ha ve b ee n prese nt pri or t o t he st art of t he st u d y.
•
Pre- or p ost-treat me nt e v e nts t hat occ ur as a res ult of pr ot oc ol- ma n date d pr oce d ures 
(e. g., i n vasi v e pr ot oc ol- d efi n e d pr oce d ures, m o dificati o n of a s u bject’s pre vi o us 
treat me nt re gi me n).
A n A E d oes n ot i n cl u de:
•
Me dical or s ur gical pr oce d ures (e. g., c ol o n osc o p y, bi o ps y). T h e me dical c o n diti o n 
t hat lea ds t o t he pr oc e d ure is a n A E.
•
S ocial or c o n ve nie n ce h os pital a d missi o ns w here a n u nt o war d me dical occ urre nce di d 
n ot occ ur.
•
Da y t o da y fl uct uati o ns of pre-e xisti n g disease or c o n diti o ns prese nt or det ecte d at t he 
start of t he st u d y t h at d o n ot w orse n.
•
T he disease/ dis or der bei n g st u die d or e x pecte d pr o gressi o n, si g ns, or s y m pt o ms of t he 
disease/ dis or der b ei n g st u die d u nless m ore se vere t ha n e x pecte d f or t he s u bject’s 
c o n diti o n.
T he I n vesti gat or will re vi e w all d oc u m e ntati o n (e. g., h os pital pr o gress n otes, la b orat or y, or 
dia g n ostic re p orts) relati ve t o t he e ve nt bei n g re p orte d. T he I n v esti gat or will t he n rec or d all 
rele va nt i nf or m ati o n re gar di n g a n A E/ S A E i nt o t he data s yste m. It is n ot acce pta ble f or t h e 
I n v esti gat or t o se n d p h ot oc o pies of t h e s u bjects’ m e dical rec or ds i n lie u of c o m pleti o n of t he 
a p pr o priate A E/ S A E pa ges. H o w e ver, t here ma y b e i nsta nces w h e n t he Me dical M o nit or (a n 
i n de pe n de nt p h ysicia n w h o is n ot partici pati n g i n t h e cli nical st u d y), re q u ests c o pies of 
me dical rec or ds f or certai n cases. I n t his i nsta nce, all s u bject i de ntifiers will be bli n de d o n 
t he c o pies of t h e m e di cal rec or ds pri or t o s u b missi o n t o t he Me dical M o nit or.

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 7 1 of 1 1 1
T he I n vesti gat or will atte m pt t o esta blis h a dia g n osis of t he e ve nt base d o n si g ns, s y m pt o ms, 
a n d/ or ot her cli nical i nf or mati o n. I n s u c h cases, t he dia g n osis s h o ul d be d oc u me nte d as t he 
A E/ S A E a n d n ot t he i n di vi d ual si g ns a n d s y m pt o ms.
7. 1 3. 2
Ti mi n g
A d vers e e ve nt assess me nt a n d d oc u me ntati o n will occ ur at e ver y st u d y visit fr o m s cree ni n g 
t hr o u g h o ut t h e st u d y. O n t he da y of treat me nt a d mi nistrati o n, a s u bject’s me dical c o m plai nt 
will b e c o nsi dere d t o be a treat me nt-e mer ge nt a d v erse e ve nt if t he e ve nt occ urs a n y ti me after 
t he first e x p os ure of i n vesti gati o nal pr o d uct at t he be gi n ni n g of t he cat heterizati o n f or t he 
i nf usi o n of C A P- 1 0 0 2.  F or s u bjects recei vi n g us ual care, a n e v e nt is c o nsi dere d tr eat m e nt-
e mer ge nt if it occ urs o n or after 9: 0 0 a m (i n t h e s u bject’s ti me z o ne) o n D a y 0.
7. 1 3. 3
S e v erit y
T he I n vesti gat or will ma ke a n assess me nt of i nte nsit y f or eac h A E a n d S A E re p orte d d uri n g 
t he st u d y. T he ass ess me nt will be base d o n t h e I n v esti gat or’s cli nical j u d g m e nt. T he i nte nsit y 
of eac h A E a n d S A E will be assi g ne d t o o n e of t he f oll o wi n g cate g ories:
Mil d:
A n e ve nt t hat is easil y t olerate d b y t he s u bject, ca usi n g mi ni mal disc o mf ort 
a n d   n ot i nterferi n g wit h e ver y da y acti vities.
M o der ate:
A n e ve nt t hat is s ufficie ntl y disc o mf orti n g t o i nterfere wit h n or mal e ver y d a y 
acti vities.
Se vere:
A n e ve nt t hat pre ve nts n or mal e ver y da y acti viti es.
A n A E t hat is assesse d as se vere will n ot be c o nf use d wit h a n S A E. Se verit y is a cate g or y 
utili ze d f or rati n g t he i nte nsit y of a n e v e nt; a n d b ot h A Es a n d S A Es ca n b e assesse d as 
se vere. A n e ve nt is descri be d as ‘seri o us’ w he n it meets o ne of t he pre- d efi ne d o utc o mes as 
descri b e d i n t he Se cti o n 7. 1 3. 8 Seri o us A d v erse E ve nts.
7. 1 3. 4
R el ati o n s hi p
T he I n vesti gat or is o bli gate d t o assess t he relati o ns hi p bet w ee n st u d y t hera p y a n d t h e 
occ urre nce of eac h A E/ S A E. T he I n vesti gat or will use cli nical j u d g me nt t o deter mi ne if t here 
is a reas o na bl e p ossi bilit y t hat t he p h ar mac ol o gical acti o n of t he st u d y a ge nt, or t he i nf usi o n 
pr oce d ure itself, was res p o nsi ble f or t h e A E/ S A E bei n g re p orte d. Alter nati ve ca uses s u c h as 
nat ural hist or y of t he u n d erl yi n g diseases, c o n c o mita nt t hera p y, ot her ris k fact ors, a n d t he 
te m p oral relati o ns hi p of t he e ve nt t o t he st u d y a ge nt will be c o nsi dere d a n d i n vesti gate d. T h e 
I n v esti gat or will als o c o ns ult t h e I n vesti gat or’s Br oc h ure a n d/ or Pr o d uct I nf or mati o n, f or 
mar kete d pr o d ucts, i n t he deter mi nati o n of his/ her assess me nt.
All A E/ S A E t hat occ ur d uri n g t he c o urse of t he cli nical st u d y will be e val uate d. F or eac h 
A E/ S A E, t he i n vesti gat or will ma ke a deter mi n ati o n of relate d n ess t o t he i n vesti gati o nal 
pr o d uct a n d, se p aratel y, assess relate d ness t o t he pr oce d ure.  Eac h will be defi ne d acc or di n g 
t o o ne of t he f oll o wi n g cate g ories:

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 7 2 of 1 1 1
Defi nite:
T he A E/ S A E is clearl y relate d t o t he i n vesti gati o n al pr o d uct or pr o ce d ure.
Pr o b a bl e:
T he A E/ S A E is li kel y relate d t o t he i n vesti gati o nal pr o d uct or pr oce d ure.
P ossi ble:
T he A E/ S A E ma y be relate d t o t he i n vesti gati o nal pr o d uct or pr o ce d ure. All 
A Es a n d S A Es t hat occ ur wit hi n 2 4 h o urs of i nter v e nti o n will, beca use of t h e
te m p oral relati o ns hi p, be c o nsi dere d as at least p ossi bl y relate d t o t h e 
pr oce d ure a n d will be re p orte d t o t he D S M B.
U nli k el y:
T he A E/ S A E is d o u btf ull y relate d t o t he i n vesti gati o nal pr o d uct or 
pr oce d ure.
U nrel ate d: T he A E/ S A E is clearl y N O T relate d t o t he i n vesti gati o nal pr o d uct or 
pr oce d ure.
T here ma y be sit uati o ns w he n a n S A E has o cc urre d a n d t he I n v esti gat or h as mi ni mal 
i nf or mati o n t o i ncl u de i n t he i niti al re p ort. H o we ver, it is ver y i m p orta nt t hat t he I n vesti gat or
ma ke a n assess me nt of ca usalit y
7. 1 3. 5
E x p e ct e d n e s s
T he f oll o wi n g a d vers e e v e nts are c o nsi dere d t o be e x pecte d as a res ult of t he cat het erizati o n 
a n d i nf usi o n pr oce d ure:
•
S o me pre mat ure ve ntric ular c o ntracti o ns, n o n-s ust ai ne d ve ntric ul ar tac h yc ar dia ( < 3 0 
sec d urati o n)
•
H y p ote nsi o n ( S yst olic Bl o o d Press ure < 8 0 m m H g) a n d/ or bra d ycar dia ( Heart R ate 
< 5 0 beats/ mi n) t hat res p o n ds ra pi dl y t o fl ui d, atr o pi ne a n d p ossi bl y vas o press or 
a d mi nistrati o n, if re q uir e d
•
Re d ucti o n of c or o nar y bl o o d fl o w, TI MI fl o w gra de I or II, persisti n g < 3 mi n after 
ball o o n deflati o n, d uri n g cell i nf usi o n
•
A n gi na, d urati o n < 1 0 mi n d uri n g cell i nf usi o n
•
S T ele vati o ns, d urati o n < 3 mi n d uri n g cell i nf usi o n, tr o p o ni n ele vati o ns a b o ve t h e 9 9 t h
perce ntile b ut n ot m ore t ha n 3 x, at a n y ti me d uri n g t h e first 2 4 h o urs after cell 
i nf usi o n
•
Dissecti o n of o ne or m ore of t h e i nf use d arteri es
•
Blee di n g, br uisi n g a n d/ or he mat o ma f or mati o n at t h e sit e of vasc ular access f or t he 
cell i nf usi o n pr oce d ure, at a n y ti me wit hi n t he first 2 4 h o urs after cell i nf usi o n
All a d verse e ve nts e x pecte d or ot her wise, s h o ul d be re p ort e d i n t he s u bject data. Seri o us 
A d vers e E v e nts ( S A Es) s h o ul d be re p orte d t o t he S p o ns or wit hi n 2 4 h o urs of t he site lear ni n g 
of t he e ve nt.

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 7 3 of 1 1 1
7. 1 3. 6
Cli ni c al Si g nifi c a n c e
A n a b n or mal la b val u e or test res ult s h o ul d be dee me d cli nicall y si g nifica nt if eit h er of t he 
f oll o wi n g c o n diti o ns is met:
•
T he a b n or malit y s u g gests a disease a n d/ or or ga n t o xicit y t hat is ne w or h as w orse ne d 
fr o m baseli n e.
•
T he a b n or malit y is of a d e gree t h at re q uires a d diti o nal acti v e ma n a ge me nt, e. g., 
c ha n ge of d os e, dis c o nti n uati o n of a dr u g, cl ose o bser vati o n, m ore fre q ue nt f oll o w- u p
assess me nts, or f urt h er di a g n ostic i n vesti gati o n.
A q ualifie d p h ysicia n at t he i n vesti gati ve site, eit h er t h e I n v esti gat or or a S u b-I n vesti gat or, 
will re vie w all st u d y-relate d la b orat or y a n d ot h er t est res ults. T he re vie wi n g I n v esti g at or will 
use his/ her me dical j u d g me nt t o classif y a n d d o c u me nt a n y res ults t h at are o utsi de of t he 
n or mal res ults ra n ge. T he I n v esti gat or will i n dicate w het her eac h a b n or mal res ult is cli nicall y 
si g nifica nt ( C S) or n ot cli nicall y si g nifica nt ( N C S) o n t h e s o urce d o c u me nt a n d case re p ort 
f or m.
O nce t he I n v esti gat or dee ms a n a b n or mal val ue t o be cli nicall y si g nifi ca nt ( C S), he or s he 
will d eter mi ne w het her t here is a cli nical e xa m fi n di n g or s y m pt o m ( n e w or pre-e xisti n g) t hat
e x plai ns t he a b n or mal val ue. A pr o gress n ote s u m marizi n g t he fi n di n gs, i ncl u di n g t h e 
reas o n(s) w h y t h e res ults are dee me d C S will be writte n t o pr o vi de d oc u me ntati o n f or a n 
a d verse e ve nt re p ort. If t he I n vesti gat or is a ble t o pr o vi de a differe ntial dia g n osis f or t h e C S 
res ult, he or s he s h o ul d d escri be t h e A E acc or di n gl y, e. g., uri nar y tract i nfecti o n or s us pecte d 
a ne mia. I n t he a bse nce of a n ass ociate d cli nical si g n or s y m pt o m, a n d if o nl y a si n gle v al ue is 
dee me d C S, list t he a b n or mal val ue itself as t he A E, e. g., ele vate d p otassi u m or decrease d 
calci u m. As m ore i nf or m ati o n a b o ut t h e a b n or mal val ue is k n o w n t hat pr o vi des a dia g n osis, 
t he A E s h o ul d be u p date d i n t he electr o nic d ata s yste m t o reflect t h e di a g n osis.
7. 1 3. 7
Cli ni c al L a b or at or y A d v er s e E v e nt s
A b n or mal la b orat or y fi n di n gs (e. g. cli nical c he mistr y, he mat ol o g y, a n d uri n al ysis) t hat are 
j u d ge d b y t he I n vesti gat or as cli nicall y si g nifica nt ( C S) will be d oc u me nte d as d escri be d i n 
Secti o n 7. 1 3. 1 a n d will be rec or d e d as A Es or S A Es if t h e y meet t h e defi niti o n of a n A E as 
defi ne d i n Secti o n 7. 1 3. 1 or S A E, as defi n e d i n Secti o n 7. 1 3. 8.
Cli nicall y si g nifica nt a b n or mal la b orat or y fi n di n gs t h at are detecte d d uri n g t he st u d y or are 
prese nt at scree ni n g a n d si g nifica ntl y w orse n f oll o wi n g t he start of t he st u d y will be re p orte d 
as A Es or S A Es. H o w e ver, cli nicall y si g nifica nt a b n or mal la b orat or y fi n di n gs t h at are 
ass ociate d wit h t he disease bei n g st u die d, u nless j u d ge d b y t h e I n vesti gat or as m ore se vere 
t ha n e x pecte d f or t he s u bject’s c o n diti o n, or t hat are pres e nt or detecte d at t he start of t h e 
st u d y b ut d o n ot w orse n, will n ot be re p orte d as A Es or S A Es.
T he I n vesti gat or will e xercise me dic al j u d g me nt i n deci di n g w het her a b n or mal la b orat or y 
val ues are cli nicall y si g nifica nt.

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 7 4 of 1 1 1
7. 1 3. 8
S eri o u s A d v er s e E v e nt s
7. 1 3. 8. 1  
D efi niti o n
A seri o us a d verse e v e nt ( S A E) is defi n e d b y fe deral re g ulati o n as a n y A E o cc urri n g at a n y 
d ose t hat res ults i n a n y of t he f oll o wi n g o ut c o mes: deat h, life-t hreate ni n g A E, h os pitalizati o n 
or pr ol o n gati o n of e xisti n g h os pitalizati o n, a persiste nt or si g nific a nt disa bilit y/i n ca pacit y, or 
a c o n ge nital a n o mal y/ birt h defect.
I m p orta nt me dical e v e nts t hat ma y n ot res ult i n deat h, be life t hreate ni n g, or re q uire 
h os pitalizati o n ma y b e c o nsi dere d a n S A E w he n, base d u p o n a p pr o priate me dical j u d g me nt, 
t he y m a y je o par diz e t h e s u bject a n d ma y re q uire me dical or s ur gical i nter ve nti o n t o pre ve nt 
o ne of t h e o utc o mes liste d i n t his defi niti o n. E xa m ples of s uc h me dical e v e nts i ncl u de 
aller gic br o n c h os pas m re q uiri n g i nte nsi ve treat m e nt i n a n e mer ge n c y r o o m or at h o me, bl o o d 
d yscrasias or c o n v ulsi o ns t hat d o n ot res ult i n i n patie nt h os pitalizati o n, or t he de vel o p me nt of 
dr u g d e pe n de nc y or dr u g a b use.
C o m plicati o ns t hat occ ur d uri n g h os pitalizati o n are A Es.  If a c o m pli cati o n pr ol o n gs 
h os pitalizati o n or f ulfills a n y ot h er seri o us criteria, t h e e ve nt is seri o us.  H os pitalizati o n f or 
electi ve treat me nt of a pre-e xisti n g c o n diti o n t h at di d n ot w orse n fr o m b aseli ne is n ot
c o nsi dere d t o be a n A E.
T he ter m disa bilit y mea ns a s u bsta ntial disr u pti o n of a pers o n’s a bilit y t o c o n d uct n or mal life 
f u ncti o ns.  T his defi niti o n is n ot i nte n de d t o i ncl u de e x perie nces of relati vel y mi n or me dical 
si g nifica nce s u c h as u nc o m plicate d hea dac he, na usea, v o miti n g, diarr hea, i nfl ue nza, 
acci d e nt al tra u ma (i. e., s prai n e d a n kle) t hat ma y i nterfere or pre ve nt e ver y da y life f u ncti o ns 
b ut d o n ot c o nstit ute a s u bsta ntial disr u pti o n.
7. 1 3. 8. 2  
R e p orti n g S eri o u s A d v er s e E v e nt s
All S A Es, i ncl u di n g a n y fatal or life-t hreat e ni n g e ve nt, m ust be re p orte d wit hi n 2 4 h o urs 
fr o m t he ti me t he I n v esti gat or is ma de a ware of t h e e ve nt. If t he I n vesti gat or d oes n ot h a ve 
all i nf or mati o n re gar di n g a n S A E, he/s he will n ot wait t o recei ve a d diti o nal i nf or mati o n 
bef ore rec or di n g t h e e ve nt i n t he d ata s yste m a n d c o m pleti n g as m uc h i nf or mati o n k n o w n at 
t he ti me of t he s u b missi o n. 
T o re p ort t h e e v e nt, a n S A E fr o m m ust be c o m plete d a n d s u b mitte d t o t h e data ma na ge me nt 
gr o u p wit hi n t h e a p pr o priate re p orti n g ti meli nes as f urt her descri be d i n t he st u d y ma n ual. 
A d diti o nall y, t h e Me dical M o nit or s h o ul d be c o ntacte d a n d i nf or me d of t h e e ve nt, prefera bl y 
b y fa x, as f oll o ws:
U nite d Bi o S o urce C or p orati o n
P h o ne: 1- 8 0 0- 8 2 9- 6 1 7 0
Fa x: 1- 8 7 7- 2 0 0- 2 9 4 5
E mail: Ca pric or Safet y @ u nite d bi os o urce.c o m

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 7 5 of 1 1 1
T he Me dical M o nit or will re vie w all i nf or m ati o n pr o vi de d a n d c o nta ct t h e I n vesti gat or f or 
a d diti o nal i nf or mati o n, as necessar y.
Per I C H E 2 A a n d 2 1 C F R 3 1 2, re g ul at or y a ge ncies will be n otifie d b y t h e S p o ns or b y 
tele p h o ne or fa x of a n y u ne x pecte d, S A E p ote ntiall y ass o ciate d wit h treat me nt as s o o n as 
p ossi ble, b ut i n n o e ve nt later t h a n 1 5 cale n dar da ys after t he S p o ns or’s i nitial recei pt of t he 
i nf or mati o n; fatal or life-t hreate ni n g u ne x pecte d S A Es ass ociate d wit h treat me nt will be 
re p orte d t o re g ulat ors wit hi n 7 cale n dar da ys. F oll o w- u p i nf or mati o n will be pr o vi de d as 
re q uire d.
All A Es a n d S A Es d oc u me nte d at a pre vi o us visit/ c o ntact t hat are desi g nate d as o n g oi n g will 
be re vie w e d at s u bse q u e nt visits/c o ntacts.
All A Es (i ncl u di n g S A Es) will be f oll o w e d u ntil res ol uti o n, u ntil n o f urt her c ha n ges i n t he 
e ve nt are e x pecte d (i.e. t he p oi nt at w hic h a s u bject e x perie nci n g a n a d v ers e e ve nt is treate d 
s uccessf ull y a n d sta bilize d e ve n t h o u g h t he y ma y c o nti n ue t o e x perie nce s e q uelae) as 
c o nfir me d b y t he I n v esti gat or, u ntil t he s u bject is l ost t o f oll o w- u p, or u ntil t he s u bject’s 1 2 
M o nt h Visit. S u bjects will be c o ntacte d b y p h o n e a n n uall y t hr o u g h year 5 p ost-i nf usi o n t o 
assess f or ac ute or pl a n ne d pr oce d ure a n d/ or a d missi o n f or car di ac c a use as well as s ur vi val. 
If a s u bject dies d uri n g p artici pati o n i n t he st u d y or d uri n g a rec o g nize d f oll o w- u p peri o d, t he 
Me dical M o nit or will be pr o vi de d wit h a c o p y of a n y p ost- m orte m fi n di n gs, i ncl u di n g 
hist o pat h ol o g y.
Ne w or u p d ate d i nf or mati o n will be rec or d e d b y m o dif yi n g t he A E f or ms i n t he data s yst e m.
T he I n vesti gat or will pr o m ptl y re p ort all S A Es wit hi n t h e ti mefra mes s pecifie d i n t his 
pr ot oc ol. T he I n vesti gat or will c o m pl y wit h t he a p plica ble l ocal re g ulat or y re q uire me nts 
relate d t o re p orti n g of S A Es t o his or her I nstit uti o nal Re vie w B oar d (I R B) or I n de pe n de nt 
Et hics C o m mittee (I E C).
T his pr ot oc ol is bei n g fil e d u n der a n I n v esti gati o nal Ne w Dr u g (I N D) a p pli cati o n wit h t he 
F D A i n t he U S as w ell as wit h ot her i nter n ati o nal re g ul at or y a ge ncies.  A gi ve n S A E ma y 
q ualif y f or a n E x pe dite d Safet y R e p ort ( E S R) if t h e S A E is b ot h attri b uta ble t o st u d y t hera p y 
a n d u ne x pecte d. I n t his case, all I n vesti gat ors parti ci pati n g i n t he st u d y will recei v e t h e E S R. 
T he p ur p ose of t h e E S R is t o f ulfill s pecific re g ulat or y a n d G o o d Cli nical Practi ce ( G C P) 
re q uire me nts re gar di n g t he pr o d uct u n der i n vesti gati o n.
7. 1 3. 9
S p o n s or M o nit ori n g of A d v er s e E v e nt s
T he f oll o wi n g list s u m marizes t he S p o ns or’s r ole i n m o nit ori n g A E/ S A Es:
•
All S A Es will be re vie we d b y t h e S p o ns or’s
Me di cal M o nit or or desi g nee wit hi n 1 
b usi ness da y of recei vi n g t he seri o us a d verse e ve nt f or m fr o m t he cli nical ce nter.
•
If t he Me dical M o nit or re q uires a d diti o nal i nf or mati o n t o ma ke his/ her assess me nt, 
t he cli nical ce nters will h a ve 2 b usi ness da ys t o res p o n d t o t h e re q uest f or a d diti o nal 
i nf or mati o n.

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 7 6 of 1 1 1
7. 1 3. 1 0
P o st- St u d y A d v er s e E v e nt s a n d S eri o u s A d v er s e E v e nt s
T he I n vesti gat or s h o ul d n otif y t he Me di cal M o nit or of a n y d eat h or S A E o cc urri n g at a n y 
ti me after a s u bject has c o m plete d or ter mi n ate d a cli nical trial, w he n s uc h deat h or S A E ma y 
reas o na bl y b e relate d t o t he st u d y t hera p y use d i n t his i n vesti gati o nal trial. I n v esti gat ors are 
n ot o bli gate d t o acti vel y see k A Es fr o m f or m er st u d y partici pa nts.
8
Q U A LI T Y C O N T R O L A N D A S S U R A N C E
Data will be e ntere d usi n g a v ali d ate d data s yst e m. A d etaile d descri pti o n of t h e data 
c ollecti o n t o ols a n d data ma na ge me nt pr ocess ca n be f o u n d i n t h e case re p ort f or m
c o m pl eti o n g ui deli nes a n d ot h er data ma n a ge me nt materials t hat will be pr o vi de d t o eac h 
site.  T he sit e m ust e ns ure a p pr o priate s o urce d ata d oc u me ntati o n. T he I n v esti gat or or 
desi g nee m ust e nter all re q uire d s u bject data i n t he data s yst e m i n a ti mel y fas hi o n ( wit hi n 3 
wee ks of st u d y visit) a n d a n e x pla nati o n m ust be d oc u me nte d f or a n y missi n g data.
A site i niti ati o n visit will be c o n d ucte d at eac h site parti ci pati n g i n t he st u d y.  D uri n g t his 
visit, t here will be a re vie w of t h e st u d y pr ot oc ol, st u d y pr oce d ures, t he case re p ort f or ms, t he 
data c ollecti o n a n d s u b missi o n pr ocess, a n d t he sit e’s re g ulat or y o bli g ati o ns as well as a n y 
s pecial pr oce d ures wit h t he ce nter pers o n nel. A re prese ntati ve fr o m t he S p o ns or, or desi g nee, 
will lea d t he sit e i nitiati o n visits. All trai ni n g will be d oc u me nte d. A f or mal, ce ntraliz e d 
i n vesti gat or m eeti n g m a y be hel d pri or t o i nitiati o n of t he st u d y, b ut will n ot re m o ve t h e 
re q uire me nt f or c o n d u cti n g a sit e i nitiati o n visit at eac h p arti ci pati n g site. 

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 7 7 of 1 1 1
9
P L A N N E D S T A TI S TI C A L
M E T H O D S
9. 1
G e n er al C o n si d er ati o n s
A n y c ha n ges m a de t o t he a nal ysis of t h e data n ot state d i n t he pr ot oc ol will be descri b e d i n 
t he Statistical A nal ysis Pl a n.
9. 2
D et er mi n ati o n of S a m pl e Si z e
T his sa m ple size is a de q uate t o s u p p ort a n assess m e nt of safet y a n d t olera bilit y of 
i ntrac or o nar y i nf usi o n of C A P- 1 0 0 2 i n t his dis ease p o p ulati o n, as well as a n e x pl orat or y 
assess me nt of efficac y.
9. 3
A n al y si s P o p ul ati o n s 
•
I nte nt t o Treat (I T T) P o p ulati o n: All s u bjects ra n d o mize d. S u bjects will be s u m mariz e d 
a n d a nal yz e d per t h eir ra n d o mizati o n assi g n me nt, re gar dl ess of t he treat m e nt ( C A P- 1 0 0 2
or us ual care) act u all y recei v e d.
•
M o difie d I T T ( mI T T) P o p ulati o n: S u bjects i n t he C A P- 1 0 0 2 treat m e nt gr o u p w h o 
recei v e d t he I P, a n d all s u bjects i n t he us ual car e treat me nt gr o u p w h o re m ai n o n st u d y as 
of 9: 0 0 a m (s u bject’s ti me z o ne) o n Da y 0. S u bjects will be s u m marize d a n d a nal yze d p er 
t heir ra n d o miz ati o n assi g n me nt, re gar dl ess of t he treat m e nt ( C A P- 1 0 0 2 or us u al care) 
act uall y recei ve d.
•
Per Pr ot oc ol P o p ulati o n: S u bjects wit h n o maj or pr ot oc ol vi olati o ns. T he list of s u bjects 
wit h maj or pr ot o c ol vi olati o ns will be c o m pile d pri or t o data base l o c k. 
•
Safet y P o p ulati o n: S u bjects ra n d o mi ze d t o C A P- 1 0 0 2 treat me nt w h o recei v e d t he I P, a n d 
all s u bjects i n t he us ual care tr eat m e nt gr o u p w h o re m ai n o n st u d y as of 9: 0 0 a m 
(s u bject’s ti m e z o ne) o n Da y 0. S u bjects will be s u m marize d a n d a nal yz e d per treat me nt 
act uall y recei ve d, re gar dless of t heir ra n d o mizati o n assi g n m e nt.
9. 4
Effi c a c y A n al y si s
T he pri mar y a nal ysis of efficac y will be perf or me d f or t he mI T T P o p ulati o n as defi n e d 
a b o ve. A d diti o nal a nal ys es will be defi ne d i n t he S A P.
9. 4. 1  
C ar di a c M RI,
M o bilit y, a n d Q u alit y of Lif e P ar a m et er s
Car diac str uct ural res ults will be s u m marize d f or t he i nter ve nti o n a n d c o ntr ol gr o u ps f or eac h 
visit at w hic h t he y are p erf or me d. A bs ol ute a n d relati ve c ha n ges c o m pare d t o baseli n e will 
be s u m mariz e d usi n g descri pti ve statistics.
F u ncti o nal a n d m o bilit y val ues will be s u m m ariz e d usi n g descri pti ve statistics a n d c ha n ges 
fr o m baseli ne will b e c o m pare d bet w ee n gr o u ps.
I m pr o v e me nts i n q ualit y of life para meters will be liste d a n d s u m m ariz e d usi n g descri pti ve 
statisti cs a n d c o m pare d b et wee n gr o u ps.

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 7 8 of 1 1 1
9. 4. 2 
Bi o m ar k er s 
Bi o mar ker res ults f or Ost e o p o nti n, S T 2, I L- 1 0, a n d Galecti n- 3 will be liste d a n d s u m marize d 
usi n g descri pti v e statistics.  
9. 5
S af et y A n al y si s 
T he pri mar y a nal ysis of safet y will be perf or me d o n t he S afet y P o p ulati o n as defi ne d a b o v e. 
A d diti o nal a nal ys es will be defi ne d i n t he S A P.
9. 5. 1 
S af et y E n d p oi nt s
Safet y a n d t olera bilit y of i ntrac or o nar y i nf usi o n of C A P- 1 0 0 2 will be e val uate d b y t he 
occ urre nce of re d ucti o n i n TI MI fl o w a n d m aj or car diac e ve nts, fre q ue nc y a n d se verit y of 
a d verse e ve nts, a n d b y c ha n ges fr o m b aseli ne i n la b orat or y assess me nts, vital si g ns, p h ysical 
e x a mi nati o n, a n d E C G.
9. 5. 2 
A d v er s e E v e nt s 
A d vers e e ve nts will be c o de d usi n g t he M e d D R A dicti o nar y a n d ta b ulate d. 
Treat m e nt- e mer ge nt a d v erse e v e nts ( T E A Es) are defi n e d as a n y A E t hat st arte d after t h e first 
e x p os ure of i n vesti gati o n al pr o d uct or starte d pri or t o t he first e x p os ure b ut i ncreas e d i n 
se verit y or fre q ue n c y aft er i nf usi o n. F or t h os e s u bjects ra n d o mize d t o us ual care, T E A Es are
defi ne d as a n y a d verse e ve nt be gi n ni n g o n or after 9: 0 0 a m (s u bject’s ti me z o ne) o n Da y 0 or
t hat i ncreas e d i n se verit y or fre q u e nc y after t h at ti me. T he i n ci de nce of T E A Es will be 
prese nte d b y s yste m or ga n class a n d preferre d ter m. A d verse e ve nts will als o be s u m marize d 
b y se v erit y a n d relati o ns hi p t o t he i n vesti gati o nal pr o d uct a n d pr oce d ure.
T he i nci d e nce of T E A Es l ea di n g t o wit h dra wal fr o m t h e st u d y will be prese nte d. 
Listi n gs of a n y seri o us a d verse e ve nts ( S A Es), deat hs, a n d A Es or a b n or mal la b orat or y 
val ues lea di n g t o disc o nti n uati o n of a s u bject fr o m t he st u d y will be prese nt e d.  
9. 5. 3 
L a b or at or y E v al u ati o n s 
La b orat or y v al ues at eac h c oll ecti o n ti me will b e liste d a n d s u m m ariz e d usi n g descri pti ve 
statisti cs. E ac h p ost-treat me nt la b orat or y val u e will be c o m pare d t o bas eli n e (last val ue pri or 
t o starti n g treat m e nt).  
9. 6
Mi s si n g D at a 
Ha n dli n g of missi n g data will be detaile d i n t he S A P. 
9. 7
St u d y St o p pi n g R ul e s
T he st u d y will be h alte d if c o nti n uati o n re prese nts a n u nacce pta ble ris k/ b e nefit pr ofile f or 
st u d y s u bjects, as d eter mi n e d b y t he s p o ns or, t he D S M B, or a p plic a ble re g ulat or y a ut h oriti es.
St u d y st o p pi n g r ules, fi n aliz e d b y c o nse ns us of t h e D S M B, are detaile d i n t he D S M B 
C harter.

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 7 9 of 1 1 1
1 0 I N D E P E N D E N T O V E R SI G H T C O M MI T T E E S
1 0. 1
Cli ni c al E v e nt s C o m mitt e e ( C E C)
T he c har ge of t h e Cli nical E ve nts C o m mittee ( C E C) is t o re vie w s o urce d o c u me nts a n d t o 
a dj u dicate (a) all pri mar y a n d sec o n dar y safet y e n d p oi nt e ve nts, ( b) all seri o us, all pr ot o c ol-
defi ne d, a n d i nter ve nti o n-relate d a d v ers e e v e nts, t he s e verit y a n d relate d n ess of t he a d vers e 
e ve nts, a n d (c) t he ca uses of m ortalit y e v e nts. T he i n di vi d uals w h o will ser ve o n t h e 
c o m mitt ee are u n affili ate d wit h t he cli nical site a n d t he st u d y dr u g ma n ufact urer ( Ca pric or, 
I n c.), a n d will be a p p oi nte d b y t h e S p o ns or.  T he c o m mittee will c o nsist of, at least, a heart 
fail ure car di ol o gist a n d i nter v e nti o nal car di ol o gists.  A d diti o nal e x perts i n i m m u n ol o g y will 
be c o ns ulta nts t o t he c o m mittee as necess ar y. T he C E C will m eet e ver y 6 m o nt hs or as 
nee de d t o a dj u dicate a d v erse e v e nts a n d o utc o mes data f or eac h s u bject ra n d o miz e d.
1 0. 2
D at a S af et y M o nit ori n g B o ar d ( D S M B) 
T o meet t h e st u d y's et hical res p o nsi bilit y t o its s u bjects, a n i n de pe n d e nt D S M B will m o nit or 
res ults d uri n g t he st u d y. T he b oar d c o nsists of p h ysicia ns a n d bi ostatisticia n(s), w h o ha v e n o 
f or mal i n v ol ve me nt or c o nflict of i nterest wit h t he s u bjects, ma n ufact urer ( Ca pric or, I n c.), 
t he i n vesti gat ors, or t he cli nical sites, a n d will be a p p oi nte d b y t he s p o ns or. T he D S M B will 
act i n a se ni or a d vis or y ca pacit y t o t he s p o ns or re gar di n g d ata a n d s afet y m atters t hr o u g h o ut 
t he d urati o n of t h e st u d y. T he b oar d will meet o n a peri o dic basis acc or di n g t o t he D S M B
c harter t o m o nit or t he a v aila ble i nf or mati o n re gar di n g safet y, efficac y, a n d q ualit y of trial 
c o n d uct. As n ote d i n Secti o n 3. 1, t he D S M B will re vie w t he 7 2 h o ur safet y data f or Gr o u p 1,
t o deter mi ne w het her e nr oll me nt f or Gr o u p 2 ma y i niti ate. I n a d diti o n, t h e D S M B will 
re vie w i nteri m s u m mar y res ults of t h e acc u m ulati n g dat a fr o m t he Cli nical E ve nts
C o m mittee e ver y 6 m o nt hs.  T he y will c o m m u nicate t heir fi n di n gs directl y wit h t he s p o ns or.  
T he cli nical ce nt ers will ha ve n o c o ntact wit h t he me m bers of D S M B a n d n o v oti n g me m ber 
of t he c o m mittee ma y partici pate i n t he st u d y as a n i n vesti gat or.

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 8 0 of 1 1 1
1 1
A D MI NI S T R A TI V E C O N SI D E R A TI O N S
1 1. 1 I n v e sti g at or s a n d St u d y A d mi ni str ati v e Str u ct ur e 
A c o m plete list of parti ci pati n g I n v esti gat ors a n d i nstit uti o ns will be mai ntai ne d b y t he 
S p o ns or a n d will be p oste d at htt p:/ w w w.cli nicaltrials. g o v i n t he listi n g f or t his st u d y.
S h o ul d a q uesti o n aris e d uri n g t h e st u d y fr o m t he I n v esti gat or or st u d y staff, t he staff ma y 
c o ntact t he S p o ns or staff usi n g t he c urre nt st u d y c o ntact i nf or mati o n as pr o vi de d o n 
htt p:/ w w w.cli nicaltrials. g o v i n t he listi n g f or t his st u d y.
C o ntact i nf or mati o n f or t he M e dical M o nit or f or t he st u d y is pr o vi de d b el o w: 
S p o ns or Me dical M o nit or: 
, M D   
, Ca pric or I n c.
St u d y M o nit ors ( Cli nical R esearc h Ass ociates) will als o be assi g ne d t o t his st u d y a n d c o ntact 
i nf or mati o n will be pr o vi de d t o t he sites t hr o u g h o ut t he st u d y. 
C o ntract researc h or ga ni zati o ns ( C R Os) will be use d, at t h e S p o ns or’s directi o n, f or site 
m o nit ori n g, data ma n a ge me nt, a nal ysis a n d/ or re p orti n g f or t he st u d y.  A c ore i m a gi n g gr o u p 
will b e use d t o ce ntrall y rea d all M RIs a n d a c ore l a b orat or y will be use d t o a nal yze s a m ples 
f or H L A t y pi n g, D S A m o nit ori n g a n d cell ular i m m u ne res p o nse ( E LI S p ot). C o ntact 
i nf or mati o n f or t hese gr o u ps will be pr o vi de d t o t he sites pri or t o t he st art of t h e st u d y. 
I n or der t o e ns ure q u alit y a n d c o nti n ua nc e of patie nt care, it is hi g hl y rec o m me n de d t h at t h e 
s u bject i nf or m his/ her pri m ar y care p h ysicia n of his parti ci pati o n i n t he st u d y 
1 1. 2 I n stit uti o n al R e vi e w B o ar d (I R B) A p pr o v al 
It is t he I n v esti gat ors’ res p o nsi bilit y t o e ns ure t h at t his pr ot oc ol is re vie we d a n d a p pr o ve d b y 
t he a p pr o priat e l ocal I nstit uti o nal Re vie w B oar d (I R B) pri or t o i nitiati n g t his st u d y. T he 
c o m p ositi o n a n d c o n d uct of t his c o m mitt ee m ust c o nf or m t o t he U nite d States C F R a n d 
I C H E 6.
T he I R B m ust als o re vi e w a n d a p pr o v e t h e sit e’s i nf or me d c o nse nt f or m (I C F), asse nt f or m 
( A F), a n d ot her writte n i nf or mati o n pr o vi de d t o t he s u bject a n d all a d v ertis e me nts t hat ma y 
be use d f or s u bject recr uit me nt. 
If it is necessar y t o a me n d t h e pr ot oc ol, t he I C F, or A F d uri n g t he st u d y, t h e I n vesti gat or will 
be res p o nsi ble f or e ns uri n g t hat t he I R B re vie ws a n d a p pr o ves t hes e a me n d e d d oc u me nts. A n 
I R B a p pr o v al of t he a m e n de d pr ot o c ol a n d/ or I C F/ A F m ust be o btai ne d i n writi n g bef ore 
i m ple me ntati o n of t he a me n de d pr oce d ures a n d bef ore n e w s u bjects c o ns e nt/ pr o vi de asse nt 
t o partici pate i n t he st u d y usi n g t he a me n d e d versi o n of t he I C F/ A F. 

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 8 1 of 1 1 1
1 1. 3
Et hi c al C o n d u ct of t h e St u d y
T his st u d y will be c o n d ucte d i n acc or da n ce wit h G o o d Cli nical Practice ( G C P) re q uire me nts 
descri b e d i n t he c urre nt re visi o n of I nter nati o nal C o nfere nce o n Har m o nisati o n of Tec h nic al 
Re q uire me nts of P h ar m ace uticals f or H u ma n Use (I C H) G ui d eli n es a n d all a p plica ble 
re g ulati o ns, i n cl u di n g c urre nt U nite d States C o de of Fe d eral Re g ulati o ns ( C F R), Title 2 1, 
Parts 1 1, 5 0, 5 4, 5 6, a n d 3 1 2 a n d Title 4 5, Part 1 6 4. C o m plia nce wit h t hese re g ulati o ns a n d 
g ui deli nes als o c o nstit utes c o m plia nce wit h t he et hical pri nci ples descri b e d i n t he c urre nt 
re visi o n of t he D eclarati o n of Helsi n ki. T his st u d y will als o be carrie d o ut i n acc or da nce wit h 
l ocal le gal re q uire me nts.
1 1. 4
S u bj e ct I nf or m ati o n, C o n s e nt, a n d A s s e nt
Bef ore bei n g a d mitt e d t o t he cli nical st u d y, all s u bjects m ust c o nse nt i n writi n g t o parti ci pat e. 
A n I C F will be gi v e n t o eac h s u bject, w hic h will c o ntai n all U nite d States fe derall y re q uire d 
ele me nts, all I C H-re q uire d ele me nts, a n d Healt h I ns ura nce P ort a bilit y a n d Acc o u nta bilit y
Act ( HI P A A) a ut h oriz ati o n i nf or mati o n i n la n g u a ge t h at is u n dersta n da ble t o t he s u bject.
T he pr ocess of o btai ni n g t h e i nf or me d c o nse nt a n d/ or asse nt will be i n c o m plia nce wit h all 
fe deral re g ulati o ns, I C H re q uire me nts, a n d l o cal l a ws.
T he I n vesti gat or will re vi e w t he st u d y wit h eac h s u bject, a n d as a p pli ca ble pare nt(s) a n d/ or 
g uar dia n(s). T he re vie w will i ncl u de t he nat ure, s c o pe, pr oce d ures, a n d p ossi ble 
c o nse q u e nces of t he s u bj ect's partici pati o n i n t h e st u d y. T he I C F a n d/ or A F a n d re vie w will 
be i n a f or m u n dersta n da ble t o t he s u bject. T he I n vesti gat or or d esi g n ee a n d t he s u bject, a n d 
as a p plica ble t he p are nt(s) a n d/ or g uar di a n(s), m ust b ot h si g n a n d date t he I C F a n d/ or A F 
after re vi e w a n d bef ore t he s u bject ca n p arti ci pate i n t h e st u d y. T he s u bje ct, a n d as a p pli ca bl e 
t he pare nt(s) or g uar dia n(s) will recei ve a c o p y of t he si g ne d a n d d ate d f or m, a n d t h e ori gi nal 
will b e retai ne d i n t h e sit e st u d y files. T h e I n vesti gat or or his/ her desi g nee will e m p hasiz e t o 
t he s u bject , a n d as a p plica ble pare nt(s) or g uar di a n(s), t hat st u d y partici pati o n is e ntirel y 
v ol u ntar y a n d t hat c o nse nt re gar di n g st u d y partici pati o n ma y b e wit h dra w n at a n y ti m e 
wit h o ut p e nalt y or l oss of be nefits t o w hic h t he s u bject is ot her wise e ntitle d.
If t he I C F a n d/ or A F is a me n de d d uri n g t he st u d y, t he I n v esti gat or m ust f oll o w all a p plica ble 
re g ulat or y re q uire me nts pertai ni n g t o a p pr o val of t he a me n de d I C F a n d/ or A F b y t he 
I R B/I E C. T he site m ust use t he a me n d e d c o nse nt/asse nt f or m f or all ne w s u bjects a n d re peat 
t he c o nse nt pr ocess wit h t h e a me n de d I C F/ A F f or a n y o n g oi n g s u bjects
1 1. 5
S u bj e ct C o nfi d e nti alit y
S u bjects’ na mes will re mai n c o nfi de ntial; o nl y s u bject n u m b er a n d birt h date will be 
rec or d e d i n t he data base. If t he s u bject na me a p p ears o n a n y ot h er d o c u me nt c oll ecte d (e. g., 
h os pital disc har ge s u m mar y), t he n a me will be o blit erate d bef ore t he d oc u me nt is 
tra ns mitte d. All st u d y fi n di n gs will be st ore d i n electr o nic data bases. T he s u bjects will gi v e 
e x plicit per missi o n f or re prese ntati ves of t he S p o ns or, re g ulat or y a ut h orities, a n d t he I R B t o 
i ns pect t heir me dical rec or ds t o verif y t he i nf or mati o n c ollecte d.

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 8 2 of 1 1 1
S u bjects will be i nf or me d t hat all pers o nal i nf or mati o n ma de a vaila bl e f or i ns pecti o n will be 
ha n dle d i n t he strictest c o nfi de nce a n d i n acc or da n ce wit h all st ate, l ocal, a n d fe deral data 
pr otecti o n/ pri v ac y la ws, i ncl u di n g, wit h o ut li mitati o n, t he HI P A A.
All partici pa nts i n t he st u d y will pr o vi d e writte n a ut h orizati o n t o discl ose pri vate healt h 
i nf or mati o n eit h er as a part of t he writte n I C F or as a se parate a ut h orizati o n f or m.  T he 
a ut h orizati o n will c o ntai n all re q uire d ele me nts s pecifie d b y 4 5 C F R 1 6 4 a n d I C H E 6 as 
a p plica ble, a n d will c o ntai n a wai ver of s u bject access t o st u d y-relate d pri v ate healt h 
i nf or mati o n u ntil t h e c o ncl usi o n of t he cli nical st u d y. T he a ut h orizati o n will re mai n vali d a n d 
i n f ull f orce a n d effect u ntil t he first t o occ ur of ( 1) t he e x pirati o n of 2 years after t h e st u d y 
t hera p y is a p pr o v e d f or t he i n dicati o n bei n g st u die d, or ( 2) t he e x pirati o n of 2 years after t h e 
researc h pr o gra m is disc o nti n ue d. I n di vi d ual s u bject me dical i nf or mati o n o btai n e d d uri n g 
t his st u d y is c o nfi de ntial a n d its discl os ure t o t hir d parti es ( ot her t h a n t h ose me nti o ne d i n t his 
secti o n) is strictl y pr o hi bite d. I n a d diti o n, me dical i nf or mati o n o btai ne d d uri n g t his st u d y
ma y b e pr o vi d e d t o t he s u bject's pers o nal p h ysicia n or t o ot her a p pr o priate me dical pers o n n el 
w he n re q uir e d i n c o n necti o n wit h t he s u bject's c o nti n ue d healt h a n d welf are.
T he S p o ns or will mai ntai n a pers o nal s u bje ct i d e ntificati o n list (s u bject a n d treat me nt 
n u m bers wit h t he c orres p o n di n g s u bject na mes) t o e na ble rec or ds t o be i d e ntifie d.
1 1. 6
St u d y M o nit ori n g
T he S p o ns or will pr o vi de st u d y m o nit ors a n d will d eter mi ne t he fre q ue nc y of m o nit ori n g
visits pri maril y base d o n i n di vi d ual site e nr oll me nt a n d site c o m plia nce wit h pr ot oc ol-
dictate d pr oce d ures, wit h visits ge nerall y occ urri n g o nce e ver y 2- 4 m o nt hs.  All sites will be 
m o nit ore d s h ortl y after t he first s u bject at t h e sit e c o m pletes t he I nf usi o n Visit. A d diti o nall y, 
st u d y m o nit ors will c o nti n u o usl y re vie w t he c o m pleti o n of st u d y d oc u me nts, data a n d
a d verse e ve nt re p orti n g i n a ti mel y ma n n er.
1 1. 7
C a s e R e p ort F or m s a n d St u d y R e c or d s
All case re p ort f or ms ( C R F) will be c o m plete d i n acc or da nce wit h G C P g ui d eli n es a n d as 
s o o n as p ossi ble after eac h cli nical trial visit. A data ca pt ure s yste m will be utilize d t o rec or d 
all of t he pr ot oc ol re q uire d i nf or mati o n t o be re p orte d t o t he S p o ns or o n eac h trial s u bject. 
St u d y p ers o n nel fr o m eac h site will be trai ne d i n h o w t o access a n d us e t he data s yste m t o 
e nter a n d tra ns mit data f or t he st u d y. St u d y p ers o n nel will be gi v e n a “si g n i n” a n d p ass w or d 
u ni q ue t o t he m w hic h will n ot be use d b y a n y ot her pers o n nel.  St u d y pers o n nel will be 
trai ne d re gar di n g pr o p er c orrecti o n of d ata e ntries i n t he data s yst e m.
St u d y p ers o n nel are res p o nsi ble t o e ns ure t hat all e ntries are acc urate, le gi ble a n d verifi a ble 
wit h t h e s o urce dat a i n t he me dical rec or d. A s o urce d oc u me nt is defi ne d as t he place i n t h e 
me dical rec or d w here a gi ve n data p oi nt first a p pears. 

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 8 3 of 1 1 1
1 1. 8
Pr ot o c ol Vi ol ati o n s/ D e vi ati o n s
As t he y o cc ur, pr ot oc ol vi olati o ns (i.e., e ve nts t hat ha ve p ote ntial t o i m pact i nter pretati o n of 
data f or i nte n d e d p o p ulati o n, s uc h as vi olati o ns of i n cl usi o n/e x cl usi o n crit eria) a n d de viati o ns 
(i.e., variati o ns fr o m pr ot o c ol-s pecifie d pr o cess es u nli kel y t o i m pact i nter pretati o n of data f or 
i nte n de d p o p ulati o n) will be d oc u me nte d at eac h sit e a n d re p ort e d t o t he S p o ns or. U p o n 
detecti o n of a vi olati o n, t he S p o ns or will e ns ure re p orti n g t o t he pr o p er l ocal a n d fe deral 
re g ulat or y a ut h orities i n acc or da nce wit h all a p pli ca ble f e deral a n d l ocal re g ulati o ns. T he 
S p o ns or will deter mi ne t he c o urse of acti o n base d o n t he se verit y of t he d e viati o n or 
vi olati o n. T hese ma y i ncl u de b ut are n ot li mite d t o, wit h dra wal of t he s u bje ct, a d diti o nal 
trai ni n g at t he site, a d diti o nal site m o nit ori n g, etc.  I n a d diti o n, t h e me dical m o nit or a n d
statisti cia n will re vie w t h e circ u msta n ces of eac h vi olati o n (i n a bli n de d fas hi o n) t o deter mi ne 
w het h er t h e d ata ca n reas o na bl y b e i ncl u de d i n t he s u bject data i n t h e fi nal st u d y a n al ysis.
1 1. 9
A c c e s s t o S o ur c e D o c u m e nt ati o n
T he I n vesti gat or, S p o ns or’s m o nit or a n d site cli nical st u d y p ers o n nel will be a ble t o access 
t he s o urce d oc u me ntati o n. De-i de ntifie d s o urce d o c u me ntati o n ma y be accesse d b y t he
S p o ns or (e. g., i n e val uati o n of a n S A E). I n a d diti o n, t he st u d y site m ust all o w trial-relat e d
m o nit ori n g a u dits, I R B re vi e w, a n d re g ul at or y i ns pecti o n(s), wit h direct access t o s o urce dat a 
a n d d oc u me nts.
1 1. 1 0 D at a G e n er ati o n a n d A n al y si s
C o ntract researc h or ga ni zati o ns ( C R Os) will pr o vi de data ma na ge me nt a n d a nal ysis a n d 
re p orti n g ser vices t o t he S p o ns or.  A vali d ate d data s yste m pr o prietar y t o t h e C R O will be 
utili ze d t o c ollect data a n d ge nerate a dat a base. St u d y sites will be res p o nsi ble f or e nteri n g 
data c ollecte d at t he site. E xter nal data s o urces (i.e., ce ntral la bs a n d c ore i ma gi n g ce nter) are 
e x pecte d eit h er t o pr o vi d e data sets f or electr o nic u pl oa d i nt o t he data base or e nter t h e 
a p pr o priate d ata directl y i nt o t he s yste m.  Data q u eries will b e ge n erate d b y t he d ata s yste m, 
C R O data ma na gers, a n d st u d y m o nit ors, t h e n f or war de d t o t h e sit es f or res ol uti o n.  
Rete nti o n of Data
1 1. 1 1 D at a R et e nti o n
I n acc or da nce wit h 2 1 C F R 3 1 2. 6 2, a n I n vesti gat or partici pati n g i n t his st u d y s hall retai n 
rec or ds, i ncl u di n g t he cas e re p ort f or ms a n d s u p p orti n g data i n cl u di n g si g ne d a n d date d 
c o nse nt f or ms, a n d me di cal rec or ds, pr o gr ess n otes of t h e p h ysi cia n, t h e i n di vi d ual's h os pital 
c hart(s), a n d t he n urses' n otes. Rec or ds are re q uire d t o be mai ntai ne d f or a peri o d of 2 years 
f oll o wi n g t h e date t he mar keti n g a p plicati o n is a p pr o ve d or, if n o a p plicati o n is t o be file d or 
if t h e a p plicati o n is n ot a p pr o ve d b y t he F D A, u ntil 2 years after t h e i n vesti gati o n is 
disc o nti n ue d a n d F D A is n otifie d. I n a n y case, t h e S p o ns or s h o ul d be n otifie d pri or t o t h e 
destr ucti o n of a n y st u d y rec or ds.

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 8 4 of 1 1 1
1 1. 1 2 Fi n a n ci al Di s cl o s ur e
I n acc or da nce wit h F D A re g ulat or y re q uire me nts, 2 1 C F R 5 4. 4, t h e Cli nical I n vesti gat ors 
a n d S u b-I n vesti gat ors at eac h sit e will be re q uire d t o c o m plete a fi na ncial discl os ure f or m
pr o vi de d b y t he S p o ns or pri or t o parti ci pati o n i n t he st u d y. E ac h Cli nical I n vesti gat or/ S u b-
I n v esti gat or s hall pr o vi de t o t he S p o ns or of t he st u d y s ufficie nt acc urate fi n a ncial 
i nf or mati o n t o all o w t he S p o ns or t o s u b mit c o m plete a n d acc urate certificati o n or discl os ure 
state me nts ( F or ms 3 4 5 4 a n d/ or 3 4 5 5) as re q uire d b y t he F D A re g ulati o ns. E ac h Cli nical 
I n v esti gat or/ S u b-I n vesti gat or s hall pr o m ptl y u p d ate t his i nf or mati o n if a n y rele va nt c ha n ges 
occ ur i n t h e c o urse of t he st u d y or f or 1 year f oll o wi n g c o m pleti o n of t he st u d y
1 1. 1 3 P u bli c ati o n, U s e a n d Di s cl o s ur e P oli c y
U nless t he S p o ns or has gi v e n its writte n c o nse nt i n a d va nce or u nless ot her wis e pr o vi de d i n 
t he Cli nical Trial A gree me nt e xec ute d b y t h e parties wit h res pect t o t he st u d y, neit her t he 
I nstit uti o n w here t h e st u d y is bei n g c o n d ucte d n or a n y Cli nical I n vesti gat or/ S u b-I n v esti gat or 
or st u d y pers o n nel ma y p u blis h S p o ns or c o nfi de ntial i nf or mati o n or st u d y d ata, discl ose 
S p o ns or c o nfi de ntial i nf or mati o n or st u d y d ata t o a n y t hir d part y, or use S p o ns or c o nfi de ntial 
i nf or mati o n or st u d y d ata f or a n y p ur p os e ot h er t h a n t h e perf or ma nce of t h e st u d y.

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 8 5 of 1 1 1
1 2
R E F E R E N C E LI S T
•
Alle n, H. D., K. M. Fl a ni ga n, P. T. T hr us h, I. D v orc hi k, H. Yi n, C. Ca nter, A. M. 
C o n n oll y, M. Parris h, C. M. Mc D o nal d, E. Bra u nli n, S. D. C ola n, J. Da y, B. Darras a n d 
J. R. M e n dell ( 2 0 1 3). " A ra n d o mize d, d o u ble- bli n d trial of lisi n o pril a n d l osarta n f or t h e 
treat me nt of car di o m y o p at h y i n d uc h e n ne m usc ul ar d ystr o p h y." P L o S C urr 5 .
•
Ass m us, B., D. M. Leist n er, V. Sc hac hi n ger, S. Er bs, A. Elsass er, W. Ha ber b osc h, R. 
Ha m brec ht, D. Se d di n g, J. Y u, R. C orti, D. G. M at he y, C. Bart h, C. Ma yer- We hrstei n, I. 
B urc k, T. S uesel bec k, T. Dill, C. W. Ha m m, T. T o n n, S. Di m meler, A. M. Zei h er a n d R.-
A. S. Gr o u p ( 2 0 1 4). " L o n g-ter m cli nical o utc o me aft er i ntrac or o n ar y a p pli cati o n of b o ne 
marr o w- d eri ve d m o n o n u clear cells f or ac ute m y o car dial i nfarcti o n: mi grat or y ca pacit y of 
a d mi nistere d cells deter mi nes e ve nt-free s ur vi v al." E ur H eart J 3 5 ( 1 9): 1 2 7 5- 1 2 8 3. 
•
B us h b y, K., R. Fi n kel, D. J. Bir n kra nt, L. E. Cas e, P. R. Cle me ns, L. Cri p e, A. Ka ul, K. 
Ki n nett, C. M c D o nal d, S. Pa n d ya, J. P o ys k y, F. S ha pir o, J. T o mezs k o, C. C o nsta nti n a n d 
D. M. D. C. C. W. Gr o u p ( 2 0 1 0a ). " Dia g n osis a n d ma na ge me nt of D u c he n n e m usc ular 
d ystr o p h y, part 1: dia g n osis, a n d p har mac ol o gical a n d ps yc h os ocial m a na ge m e nt." 
La n cet N e ur ol 9 ( 1): 7 7- 9 3. 
•
B us h b y, K., R. Fi n kel, D. J. Bir n kra nt, L. E. Cas e, P. R. Cle me ns, L. Cri p e, A. Ka ul, K. 
Ki n nett, C. M c D o nal d, S. Pa n d ya, J. P o ys k y, F. S ha pir o, J. T o mezs k o, C. C o nsta nti n a n d 
D. M. D. C. C. W. Gr o u p ( 2 0 1 0 b ). " Dia g n osis a n d ma na ge me nt of D u c he n n e m usc ular 
d ystr o p h y, part 2: i m ple me ntati o n of m ulti dis ci pli n ar y care." La n cet Ne ur ol 9 ( 2): 1 7 7-
1 8 9.
•
Dill m a n, J. R., J. H. Ellis, R. H. C o ha n, P. J. Str o use a n d S. C. J a n ( 2 0 0 7). " Fre q u e nc y 
a n d se verit y of ac ute aller gic-li ke reacti o ns t o ga d oli ni u m-c o ntai ni n g i. v. c o ntrast me dia 
i n c hil dre n a n d a d ults." AJ R. A merica n j o ur nal of r oe nt ge n ol o g y 1 8 9 ( 6): 1 5 3 3- 1 5 3 8. 
•
Ga ul d L. M., Ka p pers J., Carli n J. B., R o berts o n C. F. Hei g ht pre dicti o n fr o m ul na le n gt h. 
( 2 0 0 4). D e v M e d C hil d Ne ur ol. 2 0 0 4 J ul; 4 6( 7): 4 7 5- 8 0. 
htt p:// w w w. nc bi. nl m. ni h. g o v/ p u b me d/ 1 5 2 3 0 4 6 1

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 8 6 of 1 1 1
•
H or, K. N., W. Maz ur, M. D. Ta yl or, H. R. Al- K hali di, L. H. Cri pe, J. L. J efferies, S. V. 
Ra ma n, E. S. C h u n g, K. J. Ki n nett, K. Willia ms, W. M. G ottlie bs o n a n d D. W. Be ns o n 
( 2 0 1 1). " Effects of ster oi ds a n d a n gi ote nsi n c o n verti n g e nz y m e i n hi biti o n o n 
circ u mfere nti al strai n i n b o ys wit h D uc h e n n e m us c ular d ystr o p h y: a cr oss-secti o nal a n d 
l o n git u di nal st u d y utili zi n g car di o vasc ular m a g n etic res o na nce." J Car di o vasc Ma g n 
Res o n 1 3 : 6 0. 
•
H or, K. N., M. D. T a yl or, H. R. Al- K hali di, L. H. Cri pe, S. V. Ra ma n, J. L. J efferies, R. 
O' D o n n ell, D. W. Be ns o n a n d W. Maz ur ( 2 0 1 3). " Pre vale nce a n d distri b uti o n of late 
ga d oli ni u m e n ha n ce me nt i n a lar ge p o p ulati o n of p atie nts wit h D uc he n ne m usc ular 
d ystr o p h y: effect of a ge a n d left ve ntric ular s yst oli c f u n cti o n." J Car di o vasc Ma g n Res o n 
1 5 : 1 0 7. 
•
H or, K. N., J. Wa nsa p ura, L. W. Mar k h a m, W. Maz ur, L. H. Cri pe, R. Flec k, D. W. 
Be ns o n a n d W. M. G ottlie bs o n ( 2 0 0 9). " Circ u mfere nti al strai n a nal ysis i de ntifies strata 
of car di o m y o pat h y i n D u c he n ne m usc ular d ystr o p h y: a car diac ma g n etic res o na nce 
ta g gi n g st u d y." J A m C oll Car di ol 5 3 ( 1 4): 1 2 0 4- 1 2 1 0. 
•
I n k er, L. A., C. H. Sc h mi d, H. Ti g hi o uart, J. H. Ec kfel dt, H. I. Fel d ma n, T. Gree n e, J. W. 
K use k, J. Ma nzi, F. Va n Le nt e, Y. L. Z ha n g, J. C ores h, A. S. Le ve y a n d C.- E. 
I n v esti gat ors ( 2 0 1 2). " Esti mati n g gl o mer ular filtrati o n rate fr o m ser u m creati ni ne a n d 
c ystati n C." N E n gl J Me d 3 6 7 ( 1): 2 0- 2 9. 
•
J a nsse ns, S., C. D u b ois, J. B o gaert, K. T he u nisse n, C. Der o ose, W. Des met, M. Kala ntzi, 
L. H er b ots, P. Si n nae ve, J. De ns, J. Maerte ns, F. Ra de ma kers, S. D y mar k o ws ki, O. 
G he yse ns, J. Va n Cl ee m p ut, G. B or ma ns, J. N u yts, A. Bel m a ns, L. M ortel ma ns, M. 
B o o gaerts a n d F. Va n d e Werf ( 2 0 0 6). " A ut ol o g o us b o ne marr o w- deri v e d ste m-cell 
tra nsfer i n p atie nts wit h S T-se g m e nt ele vati o n m y o car dial i nfarcti o n: d o u ble- bli n d, 
ra n d o mise d c o ntr olle d tri al." La n cet 3 6 7 ( 9 5 0 5): 1 1 3- 1 2 1. 
•
J efferies, J. L., B. W. Ei d e m, J. W. Bel m o nt, W. J. Crai ge n, S. M. W are, S. D. Fer n bac h, 
S. R. Neis h, E. O. S mit h a n d J. A. T o w bi n ( 2 0 0 5). " Ge n etic pre di ct ors a n d re m o deli n g of 
dilate d car di o m y o pat h y i n m usc ular d ystr o p h y." Circ ulati o n 1 1 2 ( 1 8): 2 7 9 9- 2 8 0 4. 

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 8 7 of 1 1 1
•
Katz ber g, R. W. a n d J. H. Ne w h o use ( 2 0 1 0). "I ntra ve n o us c o ntrast me di u m-i n d uce d 
ne p hr ot o xicit y: is t he m e dical ris k reall y as great as we ha ve c o me t o belie ve?" 
Ra di ol o g y 2 5 6 ( 1): 2 1- 2 8. 
•
K o wal, J., M. T kac h a n d C. T her y ( 2 0 1 4). " Bi o ge nesis a n d secreti o n of e x os o mes." C urr 
O pi n Cell Bi ol 2 9 C : 1 1 6- 1 2 5. 
•
Malliaras, K., T. S. Li, D. L ut hri n ger, J. T err o vitis, K. C he n g, T. C h a kra v art y, G. G ala n g, 
Y. Z h a n g, F. Sc h o e n h off, J. Va n E y k, L. Mar ba n a n d E. Mar ba n ( 2 0 1 2). " Safet y a n d 
effic ac y of all o ge neic cell t hera p y i n i nfarcte d rats tra ns pla nte d wit h mis m atc he d 
car di os p here- deri ve d cells." Circ ulati o n 1 2 5 ( 1): 1 0 0- 1 1 2. 
•
Mar k h a m, L. W., K. Ki n nett, B. L. W o n g, D. W o o dr o w Be ns o n a n d L. H. Cri pe ( 2 0 0 8). 
" C ortic oster oi d treat me nt retar ds de vel o p me nt of v e ntric ular d ysf u n cti o n i n D uc he n ne 
m usc ular d ystr o p h y." Ne ur o m usc ul Dis or d 1 8 ( 5): 3 6 5- 3 7 0. 
•
N ot o, T. J., Jr., L. W. J o h ns o n, R. Kr o ne, W. F. Wea ver, D. A. Cl ar k, J. R. Kra m er, Jr. 
a n d G. W. Vetr o vec ( 1 9 9 1). " Car diac cat heterizati o n 1 9 9 0: a re p ort of t he Re gistr y of t h e 
S ociet y f or Car diac A n gi o gr a p h y a n d I nt er ve nti o ns ( S C A &I)." Cat heterizati o n a n d
car di o vasc ular dia g n osis 2 4 ( 2): 7 5- 8 3. 

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 8 8 of 1 1 1
•
Passa ma n o, L., A. Ta glia, A. Palla di n o, E. Vi g gia n o, P. D' A m br osi o, M. Sc utifer o, M. 
R osaria Ceci o, V. T orre, D. E. L. F, E. Pi cill o, O. P aciell o, G. Pil us o, G. Ni gr o a n d L. 
P olita n o ( 2 0 1 2). "I m pr o v e me nt of s ur vi v al i n D uc he n ne M usc ular D ystr o p h y: 
retr os pecti v e a nal ysis of 8 3 5 patie nts." A cta M y ol 3 1 ( 2): 1 2 1- 1 2 5. 
•
Ra ma n, S. V., K. N. H or, W. Maz ur, N. J. Hal n o n, J. T. Kissel, X. He, T. Tra n, S. S mart, 
B. Mc Cart h y, M. D. T a yl or, J. L. J efferies, J. A. Rafael- F ort n e y, J. L o we, S. L. R o ble a n d 
L. H. Cri p e ( 2 0 1 4). " E plere n o ne f or earl y car di o m y o pat h y i n D u c he n ne m usc ular 
d ystr o p h y: a ra n d o mise d, d o u ble- bli n d, place b o-c o ntr olle d trial." La ncet N e ur ol.
•
R ya n, T. D., J. L. J efferies, H. Sa w na ni, B. L. W o n g, A. G ar d n er, M. Del C orral, A. L orts 
a n d D. L. M oral es ( 2 0 1 4). "I m pla ntati o n of t he H eart M ate II a n d H eart Ware left 
ve ntric ul ar assist de vices i n patie nts wit h d uc he n n e m usc ular d ystr o p h y: less o ns lear ne d 
fr o m t he first a p plicati o ns." A S AI O J 6 0 ( 2): 2 4 6- 2 4 8. 
•
Sc hac hi n ger, V., S. Er bs, A. Els asser, W. Ha ber b osc h, R. Ha m brec ht, H. H olsc her m a n n, 
J. Y u, R. C orti, D. G. Mat he y, C. W. H a m m, T. S usel bec k, B. Ass m us, T. T o n n, S. 
Di m meler a n d A. M. Zei her ( 2 0 0 6). "I ntrac or o nar y b o ne m arr o w- deri v e d pr o g e nit or cells 
i n ac ute m y ocar dial i nfarcti o n." T he N e w E n gla n d j o ur nal of me dici ne 3 5 5 ( 1 2): 1 2 1 0-
1 2 2 1.
•
S hir o k o va, N. a n d E. Ni g gli ( 2 0 1 3). " Car di ac p he n ot y pe of D uc he n ne M usc ular 
D ystr o p h y: i nsi g hts fr o m cell ular st u dies." J M ol Cell Car di ol 5 8 : 2 1 7- 2 2 4. 
•
S p ur ne y, C. F. ( 2 0 1 1). " C ar di o m y o pat h y of D uc h e n ne m usc ular d ystr o p h y: c urre nt 
u n dersta n di n g a n d f ut ure directi o ns." M uscl e Ner ve 4 4 ( 1): 8- 1 9. 
•

C A P -1 0 0 2 All o ge neic Car di os p here -Deri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 8 9 of 1 1 1
A p p e n di x 1
S c h e d ul e of E v e nt s
Pr oce d ure / E ve nt
Scree ni n g
I nf usi o n 
( C A P- 1 0 0 2 
o nl y) 1
D a y 1 
( H o me)
D a y 3 
( P h o ne)
Wee k 2
Wee k 6
M o nt h 3
M o nt h 6
M o nt h 
1 2
E arl y 
Ter mi n ati o n 2
St u d y Da y
pri or t o 
ra n d o mizati o n
Da y 0
Da y 1 3
Da y 3 
( + 3)
Da y 1 4 
( ± 3)
Da y 4 0 
( ± 7)
Da y 9 0 
( ± 7)
Da y 1 8 0 
( ± 1 4)
Da y 3 6 0 
( ± 3 0)
As Nee de d
P R E
P O S T
I nf or me d C o nse nt
X
Eli gi bilit y Ass ess me nt
X
X
De m o gra p hics
X
M e dical Hist or y
X
X
Car diac Hist or y
X
X
C o nc o mita nt Me dicati o ns
X
X
X
X
X
X
X
X
X
Vital Si g ns
X
X
X
X
X
X
X
X
X
Wei g ht 
X
X
X
X
Hei g ht 4
X
X
X
X
X
X
1 2- Lea d E C G
X
X
X
X
X
X
X
X
X
Li mite d P h ysical E xa mi n ati o n 
X
X
X
Car diac P h ysical E x a mi n ati o n
X
X
X
X
X
X
X
X
X
Car diac M RI
X
X
X
X
C o nti n u o us car diac r h yt h m 
m o nit ori n g
X
X
X
X
X
X
Cli nical La b orat or y Tests 
(i n cl u d es C he mistr y, 
He mat ol o g y, Uri nal ysis)
X
X
X
X
X
X
X
X
X
Ser ol o g y 5
X
Oste o p o nti n
X
X
X
X
X
X
X
S T 2 
X
X
X
X
X
X
X
I L- 1 0
X
X
X
X
X
X
X
Gal ecti n- 3
X
X
X
X
X
X
X
E x pl orat or y Bi o mar k ers 
X
X
X
X
X
X
X

C A P -1 0 0 2 All o ge neic Car di os p here -Deri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y 2 0 1 6
C o nfi d e ntial
P a ge 9 0 of 1 1 1
Pr oce d ure / E ve nt
Scree ni n g
I nf usi o n 
( C A P- 1 0 0 2 
o nl y) 1
D a y 1 
( H o me)
D a y 3 
( P h o ne)
Wee k 2
Wee k 6
M o nt h 3
M o nt h 6
M o nt h 
1 2
E arl y 
Ter mi n ati o n 2
St u d y Da y
pri or t o 
ra n d o mizati o n
Da y 0
Da y 1 3
Da y 3 
( + 3)
Da y 1 4 
( ± 3)
Da y 4 0 
( ± 7)
Da y 9 0 
( ± 7)
Da y 1 8 0 
( ± 1 4)
Da y 3 6 0 
( ± 3 0)
As Nee de d
P R E
P O S T
H L A
X
D S A
X
X
X
X
X
X
X
E LI S p ot
X
X
P U L scale
X
X
X
X
X
X
6 M W T
X
X
X
X
X
X
P e diatric Q L I n ve nt or y
X
X
X
X
X
X
P O D CI
X
X
X
X
X
X
S pir o metr y
X
X
X
X
X
X
C o a g ulati o n
X
Ser u m Tr o p o ni n & C K- M B
X
X
X
X
X
X
X
I ntrac or o n ar y I nf usi o n
X
A d verse E ve nt Assess me nt
X
X
X
X
X
X
X
X
X
X
1 Da y 0 is t he i nf usi o n date f or s u bjects ra n d o mi ze d t o C A P- 1 0 0 2. All it e ms liste d f or I nf usi o n visit a n d assess me nts o n Da y 0 are o nl y a p plica ble 
f or s u bjects ra n d o mize d t o C A P- 1 0 0 2. S u bj ects ra n d o mi ze d t o Us ual Care d o n ot ha ve a Da y 0 visit.
2 Earl y Ter mi nati o n Visit pr oce d ures t o be c o m plete d at ti me of wit h dra wal, s h o ul d s u bj ect be wit h dra w n bef ore M o nt h 1 2 Visit. . A Car diac M RI 
will be c o n d u cte d pr o vi de d t he s u bj ect’s m ost rece nt st u d y-relate d Car diac M RI was c o n d ucte d at least t hree m o nt hs (i.e., 1 2 wee ks) pri or t o t he 
Earl y T er mi nati o n Visit.
3 F or eli gi ble s u bjects ra n d o mize d t o t h e us u al care ar m, Da y 1 is esta blis he d as 8 da ys after t he date of ra n d o mizati o n, wit h o ut a n act ual Da y 1
visit.
All st u d y s u bjects i nitiat e 7 2- h o ur c o nti n u o us car di ac r h yt h m m o nit ori n g o n Da y 1.
4 If sta n di n g hei g ht ca n n ot be meas ure d, h ei g ht will be calc ulate d usi n g a meas ure me nt of ul na le n gt h ( Ga ul d et al 2 0 0 4).
5 Ser ol o g y i ncl u des HI V a n d he patitis testi n g

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y, 2 0 1 6
C o nfi d e ntial
P a ge 9 1 of 1 1 1
A p p e n di x 2  
Cli ni c al L a b or at or y T e st s
Cli nical la b orat or y tests are t o i ncl u de t he f oll o wi n g.  All s u bjects will ha ve t he sa me 
sa m ples dra w n a n d t he sa me ass ess me nts perf or me d at eac h st u d y visit, e xce pt at D 0 a n d D 1 
w hic h are n ot st u d y visits f or s u bjects ra n d o mize d t o us ual care.
Ser u m C he mistry
A L T ( S G P T)
A S T ( S G O T) 
B U N
Creati ni ne
e G F R ( C K D- E PI C ystati n C [ 2 0 1 2])
Gl uc ose
Lactate de h y dr o ge nase ( L D H)
S o di u m 
P otassi u m
C hl ori de
Bilir u bi n
Al b u mi n
He m at ol o gy
W B C
R B C
He m o gl o bi n
He mat o crit
Platelet c o u nt
W B C (t otal a n d differe ntial c o u nts)
Ser ol o gy
He patitis B s urface a nti ge n ( H Bs A g) 
He patitis C Vir us a nti b o d y (a nti- H C V)
H u ma n I m m u n o deficie nc y Vir us a nti b o d y 
( HI V) 
A d diti o n al
H b A 1c
Tr o p o ni n I
C K- M B
C ystati n C
Uri n al ysis
A p peara nce
Bilir u bi n
C ol or 
Nitrite
Occ ult bl o o d 
p H
Pr otei n
S pecific gra vit y
Gl uc ose
K et o nes
Mi cr osc o pic e xa mi n ati o n of se di me nt
C o a g ul ati o n
I nf usi o n Visit o nly f or s u bj ects t aki n g w arf ari n
P T T
I N R
I m m u n o ass a y L a b or at ories
D S A
H L A
E LI S p ot
Bi o m arkers
Galecti n- 3
I L- 1 0
Oste o p o nti n
S T 2
E x pl orat or y Bi o mar kers ( if c o nse nt/ asse nt 
pr ovi de d )
All st u d y s peci me ns f or t his trial will be e x a mi ne d b y ce ntral la b orat or y facilities, e xce pt as 
necessar y o n Da y 0 a n d Da y 1 f or s u bjects u n der g oi n g i nf usi o n wit h C A P- 1 0 0 2.

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y, 2 0 1 6
C o nfi d e ntial
P a ge 9 2 of 1 1 1
A p p e n di x 3 
Pr o hi bit e d Pri or
M e di c ati o n s
As t he y m a y i nterfere wit h t h e f u ncti o n of C D Cs, t he f oll o wi n g me di cati o ns are pr o hi bite d 
fr o m t hree m o nt hs pri or t o t he scree ni n g visit, t hr o u g h St u d y Da y 0
G E N E RI C N A M E
T R A D E N A M E
A baca vir
E pzic o m ®
A baca vir
Zia ge n ®
A baca vir s ulfate, La mi v u di ne, a n d Zi d o v u di ne 
Trizi vir ®
A dali m u ma b
H u mira ®
Al desle u ki n
Pr ole u ki n ®
Altreta mi ne
He x ale n ®
A mi n o gl utet hi mi de
C yt a dre n ®
A na ki nra
Ki neret ®
A nastr oz ole
Ari mi de x ®
As para gi nas e
Els par ®
Ataza na vir s ulfate
Re yat a x ®
Azat hi o pri ne
I m ura n ®
Azat hi o pri ne
Azasa n
Basili xi ma b
Si m ulect ®
Ble o m yci n
Ble n o x a ne ®
B us ulfa n
M ylera n ®
Ca pecita bi ne
Xel o da ®
Car b o plati n
Para plati n ®
Car m usti ne
Bi C N U ® ( B C N U)
Cert oliz u ma b pe g ol
Ci mzia ®
C hl ora m b ucil
Le u k era n ®
Ci d of o vir
Visti de ®
Cis plati n
Pl ati n ol ®
Cla dri bi n e
Le ust ati n ®
C ycl o p h os p ha mi de
C yt o x a n ®
C ycl o p h os p ha mi de
Ne osar ®
C ycl os p ori ne
G e n graf ®
C ycl os p ori ne
N e oral ®
C ycl os p ori ne
S a n di m m u ne ®
C yt ara bi ne
C yt ara bi ne
C yt ara bi ne
De p o C yt ®
Dacar bazi ne
D TI C- D o m e ®
Dacliz u ma b
Ze n o p a x ®

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y, 2 0 1 6
C o nfi d e ntial
P a ge 9 3 of 1 1 1
G E N E RI C N A M E
T R A D E N A M E
Dacti n o m yci n
C os me ga n ®
Dar u na vir
Prezista ®
Dasati ni b
S pr yc el ®
Da u n or u bici n
Cer u bi di ne ®
Dela vir di ne
Rescri pt or ®
De nile u ki n diftit o x
O nta k ®
Di da n osi ne
Vi de x ®
D ocet a xel
Ta x otere ®
D ocet a xel
D ocefrez ®
D o x or u bici n
A dria m yci n ®
D o x or u bici n
D o xil ®
Efa vire nz
S usti va ®
Efa vire nz/e mtricita bi ne/t e n of o vir dis o pr o xil f u marate
Atri pla ®
E mtricita bi ne
E mtri va ®
E mtricita bi ne/te n of o vir dis o pr o xil f u marate
Tr u v a da ®
E nf u virti de
F uzei o n ®
E pir u bici n
Elle nce ®
Erl oti ni b
Tarce va ®
Estra m usti ne
E mc yt ®
Eta nerc e pt
E n brel ®
Et o p osi d e
Et o p o p h os ®
Et o p osi d e
Ve Pesi d ®
Etra viri ne
I ntele nce ®
E ver oli m us
Afi nit or ®
E ver oli m us
Z ortress ®
E x e mesta ne
Ar o masi n ®
Fl o x uri di n e
Fl u or o de o x y uri di ne ®
Fl o x uri di n e
A nc o b o n ®
Fl u dara bi n e
Of orta ®
Fl u dara bi n e
Fl u dara ®
F osa m pre na vir calci u m
Le xi va ®
Ge mcita bi ne
Ge mzar
G oli m u ma b i njecti o n
Si m p o ni
H y dr o x yc hl or o q ui ne
Pla q ue nil ®
H y dr o x y urea
Dr o xia ®
H y dr o x y urea
H y drea ®
I d ar u bici n
I d a m yci n ®
If os p ha mi de
Ife x ®

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y, 2 0 1 6
C o nfi d e ntial
P a ge 9 4 of 1 1 1
G E N E RI C N A M E
T R A D E N A M E
I mati ni b mes ylate
Glee v ac
I n di na vir
Cri xi va n ®
I nfli xi m a b
Re mica de ®
I nterfer o n alfa- 2a
R ofer o n- A ®
I nterfer o n alfa- 2 b
I ntr o n- A ®
I nterfer o n alfac o n- 1)
I nfer ge ®
I nterfer o n beta- 1a
A v o ne x ®
I nterfer o n beta- 1 b
Betas er o n ®
I nterfer o n beta- 1 b
E xta via ®
I nterfer o n beta-l b
Betas er o n ®
I nterfer o n ga m m a- 1 b
Acti m m u ne ®
I nterle u ki n- 2
Pr ole u ki n
Iri n ot eca n
Ca m pt osar
La mi v u di ne
E pi vir ®
La mi v u di ne & Zi d o v u di ne
C o m bi vir ®
La nre oti de
S o mat uli ne ® De p ot
La p ati ni b
T y ker b ®
Lefl u n o mi d e
Ara va ®
Le n ali d o mi de
Re vli mi d
Letr oz ole
Fe mara ®
Le u pr oli de
L u pr o n ®
Le u pr oli de
Eli gar d ®
L o m usti ne
Cee N u ® ( C C N U)
Mara vir oc
Selze ntr y
Mec hl oret ha mi ne H CI
M ustar ge n ®
Me gestr ol
Me gace ®
Mel p hala n
Al kera n ®
Merca pt o p uri n e
P uri net h ol ®
Met h otre x ate
R he u matre x ®
Met h otre x ate
Tre x all ™
Mit o m yci n
M uta m yci n ®
Mit ota ne
L ys o dre n ®
Mit o x a ntr o ne
N o va ntr o n e ®
M ur o m o na b- C D 3
Ort h ocl o ne- O K T 3
M yc o p h e n olate
Cell Ce pt ®
Nelfi na vir
Virace pt ®
Ne vira pi n e
Vira m u ne ®
Nil oti ni b
Tasi g n a ®

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y, 2 0 1 6
C o nfi d e ntial
P a ge 9 5 of 1 1 1
G E N E RI C N A M E
T R A D E N A M E
Octre oti d e acetat e
Sa n d ostati n ®
Paclita x el
Ta x ol ®
Paclita x el
A bra xa ne ®
Paz o pa ni b
V otrie nt ®
Pe gas p ar gase
O ncas par ®
Pe gi nterfer o n alfa- 2a
Pe gas ys ®
Pe gi nterfer o n alfa- 2 b
Pe g-I ntr o n ®
Pe nicilla mi ne
C u pri mi ne ®
Pr ocar bazi ne
Mat ula ne ®
Ralte gra vir
Ise ntress ®
Ri b a viri n
C o pe g us ®
Ril o nace p
Arcal yst ™
Rit o na vir
N or vir ®
Rit o na vir/ L o pi n o vir
Kaletra ®
Rit u xi ma b
Rit u x a n ®
Sa q ui na vir
I n virase ®
Sar gra m osti m
Le u ki n e ®
Sir oli m us
Ra pa m u ne ®
S orafe ni b
N e xa var ®
Sta v u di ne
Zerit ®
Stre pt o z oci n
Za n os ar ®
S ulfasalazi ne
A z ulfi di ne E N-ta bs ®
S u niti ni b malate
S ute nt ®
Tacr oli m us
Pr o graf ®
Ta m o xife n citrate
N ol va de x
Te m oz ol o mi d e
Te m o dar ®
Te msir oli m us i njecti o n
T orisel ®
Te ni p osi de
V u m o n ®
Te n of o vir dis o pr o xil f u marat e
Virea d ®
T hali d o mi de
T hal o mi d ®
T hi o g ua ni ne
T a bl oi d ®
T hi ote pa
T hi ote pa
Ti pra na vir
A pti v us ®
T ociliz u ma b
Acte mra ®
T o p oteca n
H yca mti n ®
T ore mife ne citrate
Farest o n ®
Trast u z u ma b
Herce pti n ®
Treti n oi n
Vesa n oi d ®

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y, 2 0 1 6
C o nfi d e ntial
P a ge 9 6 of 1 1 1
G E N E RI C N A M E
T R A D E N A M E
Valr u bici n
Valstar ®
Vi n blasti ne
Vi n blasti ne
Vi ncristi ne
Vi ncristi ne
Vi n orel bi n e
Na vel bi n e ®
V ori n ostat
Z oli nza ®
Zal cit a bi n e
Hi vi d ®
Zi d o v u di n e
Retr o vir ®

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y, 2 0 1 6
C o nfi d e ntial
P a ge 9 7 of 1 1 1
A p p e n di x 4  
A m e n d m e nt 3 C h a n g e s 
( Pr ot o c ol V er si o n 1 8 J ul y 2 0 1 6)
T he f oll o wi n g t a ble pres e nts a c o m plete list of c o nte nt c ha n ges i n t his pr ot oc ol a me n d me nt. 
T o a v oi d re d u n da n c y, t he “secti o ns affect e d” c ol u m n d oes n ot list t he pr ot oc ol s y n o psis as a n 
affecte d secti o n. 
Descri pti o n of C h a n ge
R ati o n ale/ J ustific ati o n
Secti o ns Affecte d
St u d y Assess me nts
“ 2 4- h o ur a m b ulat or y E C G 
m o nit ori n g” is re place d b y 
“c o nti n u o us car diac r h yt h m
m o nit ori n g” f or 7 da ys at 
M o nt h 6, a n d f or 7 2 h o urs at 
Da y 1, Wee k 6, a n d M o nt h 1 2.
T he pr ot o c ol alrea d y pr o vi de d f or n o n-
i n vasi ve car diac e ve nt m o nit ori n g at 
t h ese ti me p oi nts as a ke y safet y 
assess me nt. T he d urati o n f or eac h of 
t h ese m o nit ori n g assess me nts is bei n g 
e xte n de d t o pr o vi d e a d diti o nal 
i nf or mati o n o n t he fre q ue nc y a n d 
i nte nsit y of arr h yt h mi as i n D M D 
patie nts wit h car di o m y o pat h y.  
S y n o psis
3. 3 Safet y M o nit ori n g
6. 7 P ort a ble E C G 
M o nit or
7. 1 Scree ni n g Visit
7. 2 Ra n d o mi zati o n 
a n d Da y 0
7. 5 Wee k 6 Visit
7. 7 M o nt h 6 Visit
7. 8 M o nt h 1 2 Visit
A p pe n di x 1 Sc he d ule 
of E ve nts
“a m b ulat or y E C G m o nit ori n g 
via H olter M o nit or or ZI O ®
Patc h” is re place d b y “s p o ns or-
s u p pli e d c o nti n u o us car diac 
r h yt h m m o nit ori n g”.
Pre vi o usl y, t he a m b ul at or y E C G 
m o nit ori n g s yste m was deter mi n e d b y 
t h e sta n dar d of care at t he st u d y site.  
Wit h t he decisi o n t o e xte n d t he d urati o n 
of t he car diac m o nit ori n g assess me nts, 
t h e s p o ns or will pr o vi de m o nit ori n g 
de vices t o e ns ure c o nsist e nc y of 
assess me nts at all st u d y sites.
Cl arific ati o ns/ C orrecti o ns
E ve nts “ P ost-I nf usi o n” are 
n o w di vi d e d t o “ P ost-I nf usi o n 
( D 0- D 1)” vers us “ Pre- H os pit al 
Disc har ge ( D 1)”
T his e dit orial c ha n ge is ma de t o clarif y 
t h e se parati o n of i n- h os pital E C G 
m o nit ori n g o n Da y 0 (f or C A P- 1 0 0 2
patie nts o nl y) vers us 7 2- h o ur 
c o nti n u o us car diac r h yt h m m o nit ori n g 
o n Da y 1 (f or all patie nts).
7. 2 Ra n d o mi zati o n 
a n d Da y 0
T otal d ose recei ve d is 6 2. 5 M 
cells, n ot 6 7. 5 M cells as state d 
pre vi o usl y, f or s pecial cases 
w here i nf usi o n of 1 2. 5 M cells 
f or t hat car diac territ or y is 
warra nte d rat her t ha n t h e us ual 
d ose of 2 5 M cells. 
C orrecti o n t o arit h meti c err or i n pri or 
versi o ns of t h e pr ot oc ol.  I n cases 
w here t he i nf usi n g I n vesti gat or d ee ms 
t h at o ne of t he t hree c or o nar y arteries 
s u p pli es less t ha n 3 0 % of t he left 
ve ntric ular m y ocar di u m, t he i nf usi n g 
I n vesti gat or ma y c h o ose t o i nf use o nl y 
1 2. 5 M cells i nt o t hat c or o nar y arter y or 
arteries.
S y n o psis
3. 1 O verall St u d y 
Desi g n a n d Pla n
5. 1. 1 C A P- 1 0 0 2
D osi n g C o nsi derati o ns

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y, 2 0 1 6
C o nfi d e ntial
P a ge 9 8 of 1 1 1
Descri pti o n of C h a n ge
R ati o n ale/ J ustific ati o n
Secti o ns Affecte d
T e xt a d de d t o clarif y t h e 
ti mi n g of serial c ollecti o n of 
vit al si g ns p ost-i nf usi o n.
Pre vi o usl y, Secti o n 7. 2 s pecifie d " P ost-
i nf usi o n: seri al c ollecti o n of vit al 
si g ns (t e m p, H R, B P, R R)" b ut di d n ot 
s pecif y ti mi n g.  Te xt n o w a d de d t o 
clarif y as "e ver y 8 h o urs +/- 3 0 mi n utes 
fr o m t he e n d of i nf usi o n", c o nsiste nt 
wit h e xisti n g C R F c o m pleti o n 
g ui d eli n es a n d site trai ni n g d oc u me nts.
secti o ns 6. 1 0. 1 C A P-
1 0 0 2 I nf usi o n
7. 2 Ra n d o mi zati o n 
a n d Da y 0
A p pe n di x 1 Sc he d ule 
of E ve nts

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y, 2 0 1 6
C o nfi d e ntial
P a ge 9 9 of 1 1 1
A p p e n di x 5  
A m e n d m e nt 2 C h a n g e s 
( Pr ot o c ol V er si o n 2 9 F e br u ar y 2 0 1 6)
T he f oll o wi n g t a ble pres e nts a c o m plete list of c o nte nt c ha n ges i n t his pr ot oc ol a me n d me nt. 
T o a v oi d re d u n da n c y, t he “secti o ns affect e d” c ol u m n d oes n ot list t he pr ot oc ol s y n o psis as a n 
affecte d secti o n. F or c ha n ges s h o w n v er bati m, delete d te x t is s h o w n i n stri ket hr o u g h f o nt,
a n d ne w text is s h o w n i n re d it alicize d f o nt.
Descri pti o n of C h a n ge
R ati o n ale/ J ustific ati o n
Secti o ns Affecte d
M o dific ati o ns t o Eli gi bilit y a n d E a rl y Ter mi n ati o n Criteri a
Re m o val of i ncl usi o n criteri o n 
re q uiri n g s u bject b o d y wei g ht 
> 5 0 k g.
T he ori gi n al b o d y wei g ht criteri o n was 
a n ar bitrar y t hres h ol d i nte n d e d t o 
e xcl u de s u bjects t o o s mall t o safel y 
u n der g o car diac cat heteri zati o n. 
H o we ver, m ore a p pr o priate criteria 
e xist t o pr o vi de mea ni n gf ul pre dict ors 
of safet y, wit h o ut e xcl u di n g s u bjects 
w h o w o ul d be ot her wise eli gi ble f or t he 
trial.
I ncl usi o n Criteri o n 6:
3. 1 O verall St u d y 
Desi g n a n d Pla n
4. 2 I ncl usi o n Crit eria
Ne w i ncl usi o n criteri o n: 
S u bjects m ust be a p pr o pri at e 
c a n di d ates f or c ar di a c 
c at heteriz ati o n a n d 
i ntr ac or o n ary i nf usi o n of C A P-
1 0 0 2, i n t he j u d ge me nt of t he 
site’s i nt erve nti o n al 
c ar di ol o gist.
T his crit eri o n is a d de d as a m ore 
mea ni n gf ul safe g uar d t ha n t h e pri or 
i n cl usi o n criteri o n of 5 0 k g mi ni m u m 
b o d y wei g ht.
I ncl usi o n Criteri o n 7: 
3. 1 O verall St u d y 
Desi g n a n d Pla n
4. 2 I ncl usi o n Crit eria
E dit orial clarificati o n:
A si n gl e, c o m p o u n d i ncl usi o n 
crit eri o n ( # 4) is s e parate d t o 
t w o disti nct ite ms ( # 4 a n d # 5), 
as s h o w n at ri g ht:
4. Use of e vi d e nce base d me dical-
t h era p y i n acc or da nce wit h t h e “ D M D 
Care C o nsi derati o ns W or ki n g Gr o u p” 
g ui d eli n es f or t h e ma na ge me nt of 
D M D, f or at l east t hree m o nt hs pri or t o 
si g ni n g t h e c o nse nt f or m ( or, pr o vi di n g 
asse nt) or d oc u me nte d c o ntrai n di cati o n 
or i nt olera nce or patie nt prefere nce. 
A d diti o nall y, 
5. S u bjects m ust be ta ki n g s yste mi c 
gl u c oc ortic oi ds f or at least si x m o nt hs 
pri or t o scree ni n g
I ncl usi o n Criteria 4, 5: 
4. 2 I ncl usi o n Crit eria
Ne w E xcl usi o n Criteri o n:  
C urre ntly receivi n g 
i n vesti g ati o n al tre at me nt o n 
a n ot h er cli ni c al tri al or 
ex p a n de d access pr ot o c ol.
Treat me nt wit h ot her i n vesti gati o nal 
a ge nts w o ul d c o nf o u n d data fr o m b ot h 
trials.
E xcl usi o n Crit eri o n 
2 4: 
4. 3 E xcl usi o n Criteri a

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y, 2 0 1 6
C o nfi d e ntial
P a ge 1 0 0 of 1 1 1
Descri pti o n of C h a n ge
R ati o n ale/ J ustific ati o n
Secti o ns Affecte d
M o dific ati o ns t o Eli gi bilit y a n d E a rl y Ter mi n ati o n Criteri a (c o nti n ue d)
Cl arifi cati o n of E xcl usi o n 
Criteri o n 8 as f oll o ws: 
Esti mate d gl o mer ular filtrati o n 
rate ( G F R) < 6 0 m L/ mi n , as 
c alc ul at e d by t he C K D- E PI 
cyst ati n C e q u ati o n (I nker et al 
N EJ M 2 0 1 2) .
Cl arifi cati o n t o e ns ure c o nsiste nt 
assess me nt f or all s u bj ects 
E xcl usi o n Crit eri o n 8: 
4. 3 E xcl usi o n Criteri a
A p pe n di x 2 Cli nical 
La b orat or y T ests
Delete d state me nt:
S u bj ects ra n d o mize d t o C A P-
1 0 0 2 b ut n ot i nf use d f or a n y 
reas o n will be as ke d t o 
c o m plete t h e Earl y 
T er mi nati o n Visit. 
Ra n d o mi ze d s u bj ects will n ot be 
re place d. T his is c o nsist e nt wit h t h e 
a nal ysis p o p ulati o ns defi ne d i n t his 
a me n d me nt a n d f urt her detaile d i n t he 
S A P.
3. 1 O verall St u d y 
Desi g n a n d Pla n
Us u al C a re
Ne w secti o n defi ni n g “ us ual 
care”.
Pr ot oc ol di d n ot pre vi o usl y defi ne 
treat me nt a vaila ble t o s u bj ects 
ra n d o mize d t o recei ve us ual care.
5. 2 Us ual Care
Sti p ulati o n t hat, if 
rec o m me n de d b y t he D S M B, 
c o ntr ol s u bj ects c o m pleti n g 1 2
m o nt hs f oll o w- u p wit h us ual 
care ma y be eli gi ble t o recei ve 
C A P- 1 0 0 2 i n a n o pe n-la bel 
e xte nsi o n st u d y.
O pe n-l a bel access t o C A P- 1 0 0 2 after 
i n di vi d ual s u bjects c o m plete t he 
1 2- m o nt h assess me nt ma y e nc o ura ge
s u bj ect c o m pli a nce, f or t h e s u bj ects 
ra n d o mize d t o us ual care. T his access 
w o ul d n ot i m pact t h e i nte grit y of 
assess me nts f or t h e pri mar y a n d 
sec o n dar y st u d y o bj ecti ves, as all of 
t h ese are base d o n o bser vati o ns 
c ollecte d wit hi n 1 2 m o nt hs o n st u d y.
5. 2 Us ual Care
C o nc o mit a nt/ Pr o hi bite d Me dic ati o ns
Ot her i n vesti gati o nal t hera pi es 
are n o w pr o hi bite d fr o m 3 0 
da ys pre-ra n d o mizati o n 
t hr o u g h t he M o nt h 1 2 st u d y 
visit.
T his c ha n ge is necessar y t o pre ve nt 
c o nf o u n di n g of trial dat a.
3. 4 Rati o nal e f or 
St u d y Desi g n a n d 
C o ntr ol Gr o u p
5. 2 Us ual Care
5. 5 Pri or T hera p y 
E xcl usi o ns/ Pr o hi bite d 
Me dicati o ns
I n vesti gati o nal treat me nts a n d 
st e m cell t hera pi es are ne wl y 
liste d as pr o hi bit e d 
me dicati o ns.
T hese me dicati o ns are pr o hi bite d o n 
st u d y t o a v oi d c o nf o u n di n g st u d y data.
5. 2 Us ual Care
5. 5 Pri or T hera p y 
E xcl usi o ns/ Pr o hi bite d 
Me dicati o ns

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y, 2 0 1 6
C o nfi d e ntial
P a ge 1 0 1 of 1 1 1
Descri pti o n of C h a n ge
R ati o n ale/ J ustific ati o n
Secti o ns Affecte d
C o nc o mit a nt/ Pr o hi bite d Me dic ati o ns (c o nti n ue d)
C o nc o mit a nt t hera pies 
re defi ne d as t h ose recei ve d o n 
or after Da y 0 ( pre vi o usl y were 
ti e d t o scree ni n g a n d/ or 
i nf or me d c o nse nt).
Da y 0 is first da y of pr o d uct e x p os ure. 
5. 4 C o nc o mit a nt 
T hera p y
N ote t hat s u bjects s h o ul d n ot 
be re m o ve d fr o m t h e trial 
s ol el y f or use of pr o hi bite d 
me dicati o ns (i ncl u di n g ot her 
i n vesti gati o nal t hera pies). 
A nal ysis of dat a fr o m s u bj ects 
wit h t his class of si g nifica nt 
pr ot oc ol vi olati o n will be 
ma na ge d as detaile d i n t he 
S A P.
St u d y treat me nt i n t his trial c o nsists of 
o nl y a si n gle i nf usi o n f or s u bj ects 
ra n d o mize d t o C A P- 1 0 0 2, a n d o nl y 
us ual care f or ot her s u bj ects. Treat me nt 
wit h pr o hi bite d me dicati o ns (s uc h as 
ot h er i n vesti gati o nal t hera pies) 
t h eref ore is hi g hl y u nli kel y t o prese nt 
a n y s pecial safet y ris k f or t he s u bjects, 
alt h o u g h it i m pacts t he i nt e grit y of t he 
st u d y data. Gi ve n t he seri o us ness of t h e 
dis ease state, h o we ver, s o me s u bjects 
ma y feel c o m pelle d t o see k ot her 
i n vesti gati o nal treat me nts. I n s uc h 
cases, it w o ul d be m ore usef ul t o 
c o nti n ue t o f oll o w t hese s u bj ects t ha n 
t o recei ve n o f urt her data fr o m t h e m.  
S uc h c o nti n ue d f oll o w- u p s h o ul d n ot 
e ntail a n y si g nifica nt ris k t o st u d y 
s u bj ects.
7. 1 0 Re m o val of 
Patie nts fr o m t he Trial 
or St u d y Dr u g
St atisti c al A n al yses
Efficac y a nal ysis p o p ulati o ns 
re defi ne d as I T T, mI T T, a n d
Per- Pr ot oc ol. 
T he ne w d efi niti o ns are a better fit wit h 
I C H E 9.
Ne w te xt als o s p ecifies t hat 
s u bj ects will be a nal yze d f or efficac y as 
ra n d o mize d, re gar dless of act ual 
treat me nt recei ve d.
9. 3 A nal ysis 
P o p ulati o ns
Safet y a nal ysis p o p ulati o n 
clarifie d (starti n g p oi nts f or 
b ot h st u d y ar ms).
Ne w t e xt is better bala nce d b et wee n 
st u d y gr o u ps (i nitiati o n of vasc ular 
access f or s u bjects ra n d o mize d t o 
C A P- 1 0 0 2 i nf usi o n, vers us 9: 0 0 a m o n 
Da y 7 f or us ual care s u bjects). Ne w t e xt 
als o s pecifies t hat s u bj ects will be 
a nal yze d f or s afet y as act u all y treate d, 
re gar dless of ra n d o mizati o n.
9. 3 A nal ysis 
P o p ulati o ns
Efficac y a nal ysis pla n o utli ne d. 
Pri mar y a nal ysis of efficac y 
will utilize t he mI T T 
p o p ulati o n.
T his cl arificati o n is c o nsiste nt wit h t he 
i nte nti o n as s u g geste d i n t he pri or 
versi o ns of t h e pr ot oc ol.
9. 4 Effi cac y A n al ysis

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y, 2 0 1 6
C o nfi d e ntial
P a ge 1 0 2 of 1 1 1
Descri pti o n of C h a n ge
R ati o n ale/ J ustific ati o n
Secti o ns Affecte d
St atisti c al A n al yses (c o nti n ue d)
Safet y a nal ysis pla n o utli ne d. 
Pri mar y a nal ysis of efficac y 
will utilize t he Pr o d uct Safet y 
p o p ulati o n.
Safet y a nal ysis pla n was n ot pre vi o usl y 
o utli ne d i n t he pr ot oc ol.
9. 5 Safet y A nal ysis
H a n dli n g of missi n g d at a will 
be det aile d i n t he St atistic al 
A n alysis Pl a n ( S A P). N o 
i m p utati o ns will be ma de f or 
missi n g data.
I m p utati o ns f or missi n g data ma y be 
necessar y.
9. 6
Missi n g Data
T his s a m ple size is a de q u ate t o 
s u p p ort a n assess me nt of s af ety 
a n d t ol er a bility of 
i ntr ac or o n ary i nf usi o n of C A P-
1 0 0 2 i n t his dise ase 
p o p ul ati o n, as well as a n 
ex pl or at ory assess me nt of 
effi c acy. T we nt y-f o ur patie nts 
(ra n d o mize d 1: 1) will be 
e val uate d as e x pl orat or y 
efficac y e n d p oi nts at si x- a n d 
t wel ve- m o nt h f oll o w- u p visits.
T he f or mer te xt di d n ot disc uss t h e 
sa m ple si ze rati o nale i n t er ms of t he 
pri mar y st u d y o bj ecti ve (safet y).
9. 2 Deter mi n ati o n of 
Sa m pl e Size
Re d u n da nt te xt re m o ve d fr o m 
defi niti o ns of safet y 
p o p ulati o ns.
Pre vi o usl y, i de ntical te xt was s u p plie d 
i n b ot h secti o ns 9. 3. 2 a n d 9. 4. 1.
9. 3. 2 Safet y 
P o p ulati o n
T E A E defi niti o n i niti ates wit h 
pr o d uct e x p os ure ( pre vi o usl y 
i nitiate d wit h vasc ular access)
Cl arifi cati o n t o matc h T E A E defi niti o n 
as prese nt i n secti o n 9. 5. 2 ( A d verse 
E ve nts)
7. 1 3. 2 Ti mi n g
St u d y assess me nts
Hist orical M RI data ( 6 t o 1 5 
m o nt hs pri or t o scree ni n g), if 
a vail a ble, will be c ollecte d a n d 
se nt t o t he M RI c ore la b f or 
assess me nt.
If a vail a ble, t his data will be use d t o 
p ote ntiall y deter mi ne t he rate of car diac 
dis ease pr o gressi o n at t he ti me of 
scree ni n g.
6. 8. 4 Hist orical 
Car diac
M RI
7. 1 Scree ni n g Visit
A d diti o n of Da y 3 p h o ne 
assess me nt
T o assess A Es f or b ot h st u d y ar ms
bet wee n D 0 a n d t he Wee k 2 visit.
3. 1 O verall St u d y 
Desi g n a n d Pla n
7. 3 Da y 3 P h o ne Visit
A p pe n di x 1 Sc he d ule 
of E ve nts

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y, 2 0 1 6
C o nfi d e ntial
P a ge 1 0 3 of 1 1 1
Descri pti o n of C h a n ge
R ati o n ale/ J ustific ati o n
Secti o ns Affecte d
St u d y assess me nts (c o nti n ue d)
All st u d y assess me nts will be 
perf or me d b y ce ntral 
la b orat ori es, e xce pt f or l ocal 
la b ass ess me nts as nee de d f or 
s u bj ect safet y o n t h e da y of 
a n d da y after i nf usi o n wit h 
C A P- 1 0 0 2.
Ce ntral la b orat or y ser vices are a d de d t o 
i m pr o ve c o nsiste nc y of assess me nts 
bet wee n st u d y sites. L ocal la b orat or y 
assess me nts o n Da y 0 a n d Da y 1 f or 
s u bj ects recei vi n g C A P- 1 0 0 2 are 
i n dicate d t o ma na ge s u bj ect safet y.
6. 5. 2 Sa m ple 
C ollecti o n, St ora ge, 
a n d S hi p pi n g
A p pe n di x 2 Cli nical 
La b orat or y T ests
Assess me nt of hei g ht a d de d 
(scree ni n g, 6 wee ks, 3 m o nt hs, 
6 m o nt hs, a n d 1 2 m o nt hs). If 
st a n di n g hei g ht ca n n ot be 
meas ure d, hei g ht will be 
calc ul at e d usi n g a 
meas ure me nt of ul na le n gt h 
( Ga ul d et al 2 0 0 4)
S u bj ects as y o u n g as 1 2 years ol d ma y 
be eli gi ble f or t his st u d y. At t his a ge, 
trac ki n g of hei g ht is a sta n dar d 
c o m p o ne nt of r o uti n g p h ysical 
e xa mi n ati o ns a n d ma y be i nf or mati ve 
re gar di n g s u bj ect healt h. Hei g ht is 
a d de d at e ver y st u d y visit w here 
s pir o metr y is meas ure d. 
6. 3 Car diac a n d 
Li mite d P h ysical 
E xa mi nati o ns
7. 1 Scree ni n g Visit
7. 5
Wee k 6 Visit ( Da y 
4 0 ± 7 da ys)
7. 6
M o nt h 3 Visit 
( Da y 9 0 ± 7 da ys)
7. 7
M o nt h 6 Visit 
( Da y 1 8 0 ± 1 4 da ys)
7. 8
M o nt h 1 2 Visit 
( Da y 3 6 0 ± 3 0 da ys)
A p pe n di x 1 Sc he d ule 
of E ve nts
Tr o p o ni n a n d C K- M B will be 
o btai ne d e ver y 8 h o urs ( ± 3 0 
mi n utes) at mi ni m u m f or 2 0- 2 4
h o urs aft er C A P- 1 0 0 2 i nf usi o n.
T his 3 0- mi n ute wi n d o w f or eac h 
assess me nt is n o w s pecifie d i n t he 
pr ot oc ol t o all o w s o me fle xi bilit y i n t he 
ti mi n g of s peci me n c oll ecti o ns, w hile 
still pr o vi di n g a p pr o pri ate f oll o w- u p f or 
e vi de nce of m y ocar dial i nfarcti o n 
i nj ur y p ost-i nf usi o n.
3. 3 Safet y M o nit ori n g
6. 1 0. 1 C A P- 1 0 0 2
I nf usi o n
7. 2 Ra n d o mizati o n,
Da y 0
A n n ual f oll o w- u p p h o ne call 
ti e d t o a n ni versar y of Da y 0 f or 
b ot h st u d y ar ms. 
C h a n ge i m ple me nte d f or parit y 
bet wee n ar ms. Pre vi o usl y, f oll o w- u p
call was tie d t o i nf usi o n date f or C A P-
1 0 0 2 s u bj ects, b ut t o ra n d o mizati o n f or 
us ual care s u bjects.
7. 9 A n n ual F oll o w- u p
P h o ne Call
Ur o bili n o ge n re m o ve d fr o m 
uri nal ysis assess me nts
T his para meter n ot a c o m p o ne nt of t he 
ce ntral la b orat or y’s uri nal ysis 
assess me nt. Ur o bili n o ge n is u nli kel y t o 
be affect e d b y treat me nt wit h 
C A P- 1 0 0 2.
A p pe n di x 2 Cli nical 
La b orat or y T ests

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y, 2 0 1 6
C o nfi d e ntial
P a ge 1 0 4 of 1 1 1
Descri pti o n of C h a n ge
R ati o n ale/ J ustific ati o n
Secti o ns Affecte d
St u d y assess me nts (c o nti n ue d)
A d diti o n of clarif yi n g te xt 
re gar di n g w hic h assess me nts 
are f or b ot h st u d y ar ms vers us 
assess me nts f or C A P- 1 0 0 2
o nl y, a n d w hic h assess me nts 
are perf or me d b y ce ntral vs. 
l o cal la b
Cl arifi cati o n t o e ns ure t hat a p pr o priate 
la b orat or y assess me nts are perf or me d 
f or us ual care s u bjects, a n d t hat 
assess me nts are perf or me d b y t he 
ce ntral la b orat or y f or c o nsist e nc y.
A p pe n di x 2 Cli nical 
La b orat or y T ests
M o difie d te xt re gar di n g ke y 
safet y assess me nts t o be 
perf or me d t hr o u g h t he st u d y 
f or all s u bjects.
Cl arifi cati o n t hat ke y safet y 
assess me nts will be perf or me d f or all 
s u bj ects, wit h t he e xce pti o n of t h ose 
p ost-i nf usi o n assess me nts rele va nt o nl y 
t o s u bjects recei vi n g C A P- 1 0 0 2.
3. 1 O verall St u d y 
Desi g n a n d Pla n
S u bjects will be ev al u at e d 
over all, o nce all s u bjects 
e nr olle d i n t he st u dy h ave 
re ac he d t he Week 6 visit. 
is re place d wit h te xt defi ni n g 
D S M B re vie ws f or Gr o u p 1 
( 7 2- h o ur data) a n d f or all 
s u bj ects ( 6- m o nt h data).
T his te xt is c ha n ge d t o be c o nsist e nt 
wit h t he safet y re vie w pla n esta blis he d 
b y t h e D S M B, as defi ne d i n t he c urre nt 
D S M B c hart er v 1. 0.
3. 1 O verall St u d y 
Desi g n a n d Pla n
Cl arifi cati o n: A 2 4 h o ur 
a m b ulat or y E C G m o nit or … 
will be perf or me d at t he 
scree ni n g visit ( all s u bjects),
d uri n g t he 2 4- h o urs f oll o wi n g 
release ( disc har ge) fr o m t he 
h os pit al (f or s u bjects receivi n g 
C A P- 1 0 0 2 i nf usi o n o nl y) a n d 
als o f or all s u bjects at t h e 
Wee k 6, M o nt h 6, a n d M o nt h 
1 2 st u d y visits.
Cl arifi cati o n t hat all s u bj ects will 
recei ve t his E C G m o nit ori n g at 
ti me p oi nts as pre vi o usl y i n dicat e d i n 
A p pe n di x 1 Sc he d ule of E ve nts – wit h 
t h e e xce pti o n of p ost-i nf usi o n 
m o nit ori n g w hic h is o nl y f or s u bj ects 
recei vi n g C A P- 1 0 0 2.
6. 7 C o nti n u o us 
Car diac R h yt h m 
M o nit or
Cl arifi cati o n t hat b ot h t he 
pare nt/ g uar dia n a n d t he s u bj ect 
( n ot t he s u bject al o ne) 
c o m plete t h e Q o L assess me nts
T he Pe ds Q L a n d t he P O D CI 
assess me nts are desi g n e d t o be use d f or 
b ot h t he s u bject a n d t h e pare nt/ g uar dia n
6. 9 Cli nical F u ncti o n 
a n d Q ualit y of Life 
Meas ures
Cl arifi cati o n t hat t he PI will 
se paratel y assess rel ati o ns hi p 
of t he A E t o t he C A P- 1 0 0 2 
pr o d uct, a n d relati o ns hi p t o t h e 
i nf usi o n pr oce d ure.
Pre vi o usl y, A E re p orti n g f or ms 
re q uire d assess me nt of A E relati o ns hi p 
t o t he i nf usi o n pr oce d ure; h o we ver, t his 
was n ot s pecifie d i n t he pr ot o c ol.
7. 1 3. 4 Rel ati o ns hi p
Cl arif yi n g i nf or mati o n a d de d 
re gar di n g pr o d uct descri pti o n, 
rec o nstit uti o n, a n d st ora ge.
T e xt a d de d t o clarif y ha n dli n g of t h e 
pr o d uct.
5. 1 I n vesti gati o nal 
Pr o d uct
S u b hea di n gs a d de d t o t his 
secti o n
S u b hea di n gs a d de d t o e xisti n g t e xt i n 
t his secti o n t o se parate disti nct t o pics
5. 1 I n vesti gati o nal 
Pr o d uct

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y, 2 0 1 6
C o nfi d e ntial
P a ge 1 0 5 of 1 1 1
Descri pti o n of C h a n ge
R ati o n ale/ J ustific ati o n
Secti o ns Affecte d
Ot her Cl arific ati o ns a n d Mi n or C h a n ges
Cl arifi cati o n i n cite d secti o ns 
t h at 9 a m o n Da y 0 is defi ne d, 
f or us ual care s u bjects, as t he 
e q ui vale nt ti me p oi nt t o t he 
st art of i nf usi o n f or s u bjects 
recei vi n g C A P- 1 0 0 2 
A us ual-care e q ui vale nt t o t he start of 
i nf usi o n ti me was esta blis he d i n 
A me n d me nt 1 f or parit y bet wee n st u d y 
ar ms, t o t he e xte nt p ossi ble.  T hese 
c ha n ges are c orrecti o ns t o clarif y t he 
i m ple me ntati o n of t h e pri or c ha n ge. 
3. 1 O verall St u d y 
Desi g n a n d Pla n
7. 1 3. 2 Ti mi n g
7. 2 Ra n d o mizati o n, 
Da y 0
9. 5. 2 A d verse E ve nts
Cl arifi cati o n t hat f or L T F 
s u bj ects, E n d of St u d y date is 
date/ti me of last c o ntact or 
k n o w n e ve nt. Ne w s u b hea di n g 
“ L ost t o F oll o w- U p” hi g hli g hts 
t his secti o n. 
Cl arifi cati o n as pri or te xt di d n ot 
s pecif y assess me nt of “e n d of st u d y” 
date f or L T F s u bjects.
Ne w s u b hea di n g is a d de d t o hi g hli g ht 
pri or te xt re gar di n g ma na ge me nt of 
s u bj ects w h o ma y be l ost t o f oll o w- u p. 
7. 1 2 L ost t o F oll o w-
U p
N o s u bject has e x perie nce d a 
T E A E cl assifi e d as  c o nsi dere d 
relate d t o C A P- 1 0 0 2 b ut t h at 
w as n ot als o cl assifie d as 
rel at e d t o t he cat heteri zati o n 
a n d i nf usi o n  pr oce d ure. 
1. 2. 3. 2 Treat me nt 
E mer ge nt A d verse 
E ve nts ( T E A Es)
E dit orial clarificati o n
1. 2. 5. 1 Efficac y 
C o ncl usi o ns
p- val u e a d de d t o le ge n d of 
Fi g ure 7
T he p- val ue was n ot pre vi o usl y liste d 
Fi g ure 7
D S A = d o n or s pecifi c 
a nti b o dy , n ot d o n or-s pecific 
a nti ge n
C orrecti o n i n t his secti o n o nl y (t his 
a b bre viati o n a n d ter m pre vi o usl y were 
c orrect i n ot her pr ot oc ol secti o ns, 
i n cl u di n g t h e list of a b bre viati o ns) 
…tec h ni q ues will be 
pr ot oc olle d are s pecifie d i n t h e 
M RI I m a gi n g M a n u al .
E dit orial clarificati o n
6. 8
M a g netic 
Res o na nce I ma gi n g 
“e nr olle d” c ha n ge d t o 
“r a n d o miz e d” i n m ost 
i nsta nces pertai ni n g t o t h e 
H O P E trial 
“ra n d o mize d” is a m ore acc urate a n d 
precis e ter m t ha n “e nr olle d” i n t h e 
i nsta nces re place d
M ulti pl e secti o ns
Refere nce a d diti o ns: Ga ul d et 
al 2 0 0 4, I n ker et al 2 0 1 2 
Ga ul d refere nce: hei g ht esti mati o n per 
ul n a; I n ker refere nce: e G F R esti mati o n
1 2 Refere nce List

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y, 2 0 1 6
C o nfi d e ntial
P a ge 1 0 6 of 1 1 1 
A p p e n di x 6  
A m e n d m e nt 1 C h a n g e s 
( Pr ot o c ol V er si o n 2 7 J a n u ar y 2 0 1 6) 
T he f oll o wi n g t a ble pres e nts a c o m plete list of c o nte nt c ha n ges i n t his pr ot oc ol a me n d me nt. 
T o a v oi d re d u n da n c y, t he “secti o ns affect e d” c ol u m n d oes n ot list t he pr ot oc ol s y n o psis as a n 
affecte d secti o n. F or c ha n ges s h o w n v er bati m, delete d te x t is s h o w n i n stri ket hr o u g h f o nt, 
a n d ne w text is s h o w n i n re d it alicize d f o nt.
Descri pti o n of C h a n ge
R ati o n ale/ J ustific ati o n
Secti o ns Affecte d
Cli nic al D at a U p d ates: Ot her Tri als wit h C A P -1 0 0 2
  
Si nce t he ori gi nal pr ot o c ol was iss ue d, 
cli ni cal e x perie nce wit h t he pr o d uct f or 
treat me nt use has e x pa n de d. A d diti o nal 
safet y a n d efficac y data fr o m t he 
o n g oi n g trials s u p p ort a n i m pr o ve d 
ris k/ be nefit pr ofile f or t h e pr o d uct.
1. 2 S u m mar y of 
Cli ni cal Data
Fi g ure 7
M o dific ati o ns t o Eli gi bilit y Criteri a
A ge ra n ge eli gi ble f or 
ra n d o mizati o n i nt o Gr o u p 1 is 
e x pa n de d t o i ncl u de males 
1 2 + years ol d.
I ncl usi o n Criteri o n 5:
3. 1 O verall St u d y 
Desi g n a n d Pla n
4. 2 I ncl usi o n Criteria 
M RI assess me nt at 6- 1 5
m o nt hs pri or t o scree ni n g n o 
l o n ger re q uire d; i nstea d, 
eli gi bilit y assess me nt will 
utili ze c o ntrast-e n ha nce d M RI 
at Scree ni n g
Hist orical M RI sca n was pre vi o usl y 
re q uire d s olel y t o assist i n st u d y 
stratificati o n, b ut has bee n a barrier t o 
st u d y e ntr y f or s u bjects ot her wise 
eli gi ble. Gi ve n t h e s mall n u m ber of 
a vail a ble s u bjects a n d t he s mall st u d y 
si ze, t he utilit y of stratif yi n g t he 
p o p ulati o n di d n ot j ustif y e xcl u di n g 
s u bj ects s ol el y f or a bse nce of M RI 
hist or y.  
I ncl usi o n Criteri o n 3:
4. 2 I ncl usi o n Criteria 
T hera p y wit h c hr o nic 
i n otr o pes, or c urre ntl y 
recei vi n g i ntra ve n o us i n otr o pic 
or vas oacti ve me dicati o ns at 
t h e ti me of scree ni n g. 
E dit orial clarificati o n
E x cl usi o n criteri o n 1:
4. 3 E xcl usi o n Criteri a 
5. 4 Pri or T hera p y 
E xcl usi o ns 
… s u bj ects m ust be ta ki n g 
s yst e mic gl uc oc ortic oi ds f or at 
least si x m o nt hs pri or t o 
si g ni n g t h e c o nse nt a n d/ or 
asse nt scree ni n g. 
Re visi n g t he defi niti o n t o 6 m o nt hs of 
gl u c oc ortic oi d t hera p y pri or t o scree ni n g 
is me dicall y rel e va nt; t h e pri or 
re q uire me nt was n ot.
I ncl usi o n crit eri o n 4:
4. 2 I ncl usi o n Criteria 

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y, 2 0 1 6
C o nfi d e ntial
P a ge 1 0 7 of 1 1 1
Descri pti o n of C h a n ge
R ati o n ale/ J ustific ati o n
Secti o ns Affecte d
Scree ni n g a n d R a n d o miz ati o n
E nr oll me nt will c o nti n ue u ntil 
1 2 s u bj ects are i nf use d or 3 0 
s u bj ects are e nr oll e d. N o 
s u bj ects will be re place d. 
Pre vi o usl y, e nr oll me nt tar get 
was 1 2 i nf use d a n d 1 2 c o ntr ol 
s u bj ects, wit h re place me nt of 
s u bj ects ra n d o mize d t o C A P-
1 0 0 2 b ut w h o di d n ot c o m plete 
i nf usi o n.
Pri or e nr oll me nt pla n all o we d f or 
re place me nt of s u bj ects o n o nl y o ne 
st u d y ar m.  Ne w pl a n is m ore bal a nce d, 
as n o ra n d o mize d s u bjects will be 
re place d.
3. 1 O verall St u d y 
Desi g n a n d Pla n
9. 2 Deter mi n ati o n of 
Sa m pl e Si ze
Ra n d o mi zati o n will be 
si m plifi e d a n d will n o l o n ger 
atte m pt t o stratif y assi g n me nts 
base d o n hist orical M RI 
rea di n gs or c hr o nic use of 
st er oi ds.
T he st u d y size ( 2 4 s u bjects) is t o o s mall
t o s u p p ort stratifi cati o n, a n d t h e 
re q uire me nt f or hist orical M RI is bei n g 
re m o ve d (as e x plai ne d a b o ve).
3. 1 O verall St u d y 
Desi g n a n d Pla n
5. 2 Ra n d o mizati o n
A d diti o nal la b orat or y tests at 
scree ni n g: ve n o us bl o o d 
assess me nts f or Tr o p o ni n, C K-
M B ( pre vi o usl y t hese 
para meters were assesse d p ost-
scree ni n g o nl y)
Scree ni n g val ues f or t hese para meters 
will pr o vi de a bas eli ne f or t he 
assess me nts c ollecte d later i n t he st u d y.
7. 1 Scree ni n g Visit
A p pe n di x 1 Sc he d ule 
of E ve nts
A p pe n di x 2 Cli nical 
La b orat or y T ests
Ne w t esti n g f or C yst ati n C at 
scree ni n g, M 6, a n d M 1 2.
C ystati n C is a d de d as a t o ol f or 
m o nit ori n g car di o vasc ular disease.
Ve n o us bl o o d s p eci me n f or 
E LI S P O T i m m u n oassa y 
c ollecte d at Scree ni n g i nstea d 
of pre-i nf usi o n
Ti mi n g f or c ollecti o n of t his sa m ple is
a dj uste d t o i m pr o ve bala nce bet wee n 
st u d y ar ms
7. 1 Scree ni n g Visit
7. 2 Ra n d o mizati o n 
a n d Da y 0
A p pe n di x 1 Sc he d ule 
of E ve nts
S af et y a n d Effic ac y A n al ys es
Ne w t e xt dis c ussi n g 
I n de pe n de nt O versi g ht 
C o m mittees ( D S M B a n d C E C)
Ori gi nal pr ot oc ol di d n ot e x plicitl y state 
t h at st u d y w o ul d be o versee n b y a 
D S M B, a n d di d n ot disc uss t he C E C.
3. 1 O verall St u d y 
Desi g n a n d Pl a n
1 0 I n d e pe n de nt 
O versi g ht 
C o m mittees
St u d y st o p pi n g r ules are n o w 
defi ne d b y refere nce t o t he 
D S M B C harter.
Ori gi nal Pr ot oc ol di d n ot defi ne st u d y 
st o p pi n g r ules. T he st u d y st o p pi n g r ules 
ha ve n o w b ee n esta blis he d b y 
u na ni m o us v ote of t he D S M B, a n d are 
detaile d i n t he D S M B C harter.
9. 8 St u d y St o p pi n g 
R ules
Bi o mar kers f or t he st u d y are 
n o w defi ne d ( Galecti n- 3, I L-
1 0, Oste o p o nti n, S T 2)
Bi o mar kers t o be assesse d i n t h e 
pr ot oc ol were n ot liste d pre vi o usl y. 
A p pe n di x 2 Cli nical 
La b orat or y T ests

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y, 2 0 1 6
C o nfi d e ntial
P a ge 1 0 8 of 1 1 1
Descri pti o n of C h a n ge
R ati o n ale/ J ustific ati o n
Secti o ns Affecte d
T he st u d y will m o nit or t h e 
pr o p orti o n of s u bj ects 
e x perie nci n g a n y of t he 
f oll o wi n g e ve nts d uri n g or p ost 
i ntrac or o nar y i nf usi o n 
deli ver y. F or c o m p aris o n, 
t h ese s a me eve nts will be 
m o nit or e d i n us u al c are 
s u bj ects usi n g 9: 0 0 a m o n D ay 
0 as t h e refere nce ti me p oi nt :
Ori gi nal pr ot oc ol di d n ot e x plicitl y state 
a met h o d f or c o m paris o n of safet y 
bet wee n i nf use d s u bj ects a n d c o ntr ol 
s u bj ects, f or t he peri o d d uri n g a n d 
i m me diatel y f oll o wi n g i ntrac or o nar y 
i nf usi o n of t he s u bjects recei vi n g C A P-
1 0 0 2.
3. 3 Safet y M o nit ori n g
Cl arific ati o ns, A d mi nistr ati ve C h a n ges a n d U p d ates
C h a n ge of S p o ns or C o nt act 
a n d Me dical M o nit or
Pers o n nel c ha n ge
7. 1 1. 8. 2 Re p orti n g 
Seri o us A d verse 
E ve nts
1 1 . 1 I n vesti gat ors a n d 
St u d y A d mi nistrati ve 
Str uct ure
St u d y Da y 0 re defi ne d as date 
of pr o d uct i nf usi o n f or C A P-
1 0 0 2 s u bj ects, a n d as 7 da ys 
after ra n d o mizati o n f or c o ntr ol 
s u bj ects. Pre vi o usl y, da y 0 was 
t h e date of ra n d o mizati o n.
Re defi ni n g Da y 0 as t he date of i nitial 
pr o d uct e x p os ure is m ore mea ni n gf ul 
t h a n ra n d o mizati o n date f or statisti cal 
assess me nts of safet y a n d pr o d uct 
efficac y. “ Da y 0” f or c o ntr ol s u bjects is 
defi ne d as “ra n d o mizati o n + 7 da ys” t o 
be as c o nsist e nt as p ossi ble wit h t he 
ot h er st u d y ar m (i nf usi o n date is 
a ntici pate d t o occ ur 7 da ys aft er 
ra n d o mizati o n, b ut s o me sc he d ule 
variati o ns ma y occ ur).
3. 1 O verall St u d y 
Desi g n a n d Pla n
Fi g ure 1 0
7. 2 Ra n d o mizati o n 
a n d Da y 0
7. 1 1. 2 Ti mi n g
9. 6. 1 Safet y E n d p oi nts
9. 6. 2 A d verse E ve nts
T ar get i nf usi o n ti mes f or was h 
s ol uti o n, I P ( C A P- 1 0 0 2) a n d 
2 n d was h s ol uti o n n o w defi ne 
wi n d o ws ( ± 5- 3 0 sec o n ds) 
per mitte d f or eac h ste p.  
Ori gi nal pr ot o c ol di d n ot defi n e 
per mitte d ra n ge of de viati o n fr o m tar get 
i nf usi o n ti mes.
6. 1 0. 1 C A P- 1 0 0 2
I nf usi o n
Efficac y Ass ess me nt 2 
( F u ncti o nal): Seri al c ha n ges i n 
m o bilit y meas ure me nts a n d 
Perf or ma nce of U p per Li m b 
( P U L) scale, s pir o metr y, a n d 
6- mi n ute wal k t est ( 6 M W T) 
w he n dee me d a p pr o priat e b y 
t h e I n vesti gat or.
Cl arifi cati o n of f u ncti o nal ass ess me nts 
t o be perf or me d
3. 2 E x pl orat or y 
Efficac y E n d p oi nts
A d diti o n of ter ms t o t he “ List 
of A b bre viati o ns a n d 
Defi niti o ns” ( C E C, D S M B, 
L G E, T E A E, T M P G)
T hese a b bre viati o ns i n t he pr ot oc ol were 
n ot pre vi o usl y defi ne d i n t his secti o n
List of A b bre viati o ns 
a n d Defi niti o ns

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y, 2 0 1 6
C o nfi d e ntial
P a ge 1 0 9 of 1 1 1
Descri pti o n of C h a n ge
R ati o n ale/ J ustific ati o n
Secti o ns Affecte d
Visit wi n d o ws pre vi o usl y 
defi ne d i n A p pe n di x 1 a n d 
Secti o n 7 are n o w refere nce d 
i n Secti o n 3. 1 als o 
Visit wi n d o ws pre vi o usl y were defi ne d 
i n t he pr ot oc ol b ut were o mitte d i n 
secti o n 3. 1
3. 1 O verall St u d y 
Desi g n a n d Pla n
Fi g ure 4 – defi niti o n of X-a xis
X-a xis para meters f or t his fi g ure were 
n ot la bel e d pre vi o usl y.
Fi g ure 4
A Es will b e re gar de d as 
‘ pretreat me nt’ if t h e y occ ur 
pri or t o t he first e x p os ure t o 
treat me nt. 
T e xt re m o ve d was re d u n da nt wit h t h e 
para gra p h i m me diatel y f oll o wi n g, w hi c h 
defi ne d T E A Es. 
9. 6. 2 A d verse E ve nts
Refere nce a d diti o n: 
1 2 Refere nce List

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y, 2 0 1 6
C o nfi d e ntial
P a ge 1 1 0 of 1 1 1
A p p e n di x 7 
S p o n s or Si g n at ur e
St u d y Title:
A Ra n d o mize d, O pe n-la bel St u d y of t he Safet y a n d Efficac y of 
M ulti- Vessel I ntrac or o nar y Deli ver y of All o ge neic Car di os p here-
Deri ve d Cells i n Patie nts wit h C ar di o m y o pat h y Sec o n dar y t o 
D uc he n ne M us c ular D ystr o p h y [ H O P E - D uc he n n e (H alt 
car di o m yO P at h y pr o grE ssi o n i n D uc he n ne)]  
St u d y N u m ber:
C A P- 1 0 0 2- D M D- 0 1
Ori gi n al Pr ot oc ol:
0 1 Ma y 2 0 1 5
A me n d me nt 3. 0:
1 8 J ul y 2 0 1 6
T his cli nical st u d y pr ot oc ol was s u bject t o critical re vie w a n d h as bee n a p pr o ve d b y t h e 
s p o ns or.  
Si g ne d:
Date:
1 8 J ul y 2 0 1 6
, M D
 & Me dical M o nit or 
Ca pric or, I n c.

C A P- 1 0 0 2 All o ge neic Car di os p here- D eri ve d Cells
Cli nical Trial Pr ot oc ol: C A P- 1 0 0 2- D M D- 0 1, A me n d me nt 3. 0
Ca pric or, I nc.
1 8 J ul y, 2 0 1 6
C o nfi d e ntial
P a ge 1 1 1 of 1 1 1
A p p e n di x 8 
I n v e sti g at or’ s Si g n at ur e
St u d y Title:
A Ra n d o mize d, O pe n-la bel St u d y of t he Safet y a n d Efficac y of 
M ulti- Vessel I ntrac or o nar y Deli ver y of All o ge neic Car di os p here-
Deri ve d Cells i n Patie nts wit h C ar di o m y o pat h y Sec o n dar y t o 
D uc he n ne M us c ular D ystr o p h y [ H O P E - D uc he n n e (H alt 
car di o m yO P at h y pr o grE ssi o n i n D uc he n ne)]
St u d y N u m ber:
C A P- 1 0 0 2- D M D- 0 1
Ori gi n al Pr ot oc ol:
0 1 Ma y 2 0 1 5
A me n d me nt 3. 0:
1 8 J ul y 2 0 1 6
I h a ve r ea d t h e pr ot oc ol descri be d a b o ve. I a gree t o c o m pl y wit h all a p plica ble re g ulati o ns 
a n d t o c o n d uct t he st u d y as descri be d i n t he pr ot o c ol.
Si g ne d:
Date:
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Na me, Cre de ntials