An Open-label Single Arm Phase 2 Proof of Concept Study to Assess the Efficacy and Safety of ASCT01 in Patients With Critical Limb Ischemia
Phase: PHASE2
Conditions: Critical Limb Ischemia
Sponsor: Lifecells, LLC.

   
        
Final Study Summary Report An open-label single arm phase 2 proof of concept study to assess the efficacy 
and safety of ASCT01 in Patients with Critical Limb Ischemia 
 
1 
 
Final Study Summary Report 
 
 An open-label single arm phase 2 proof of concept study to assess the efficacy and safety of ASCT01 
in Patients with Critical Limb Ischemia 
IND 15069 
 
Summary for end of phase 2 meeting package 
August 4, 2017 

   
        
Final Study Summary Report An open-label single arm phase 2 proof of concept study to assess the efficacy 
and safety of ASCT01 in Patients with Critical Limb Ischemia 
 
2 
 
TABLE OF CONTENTS 
 
1. INTRODUCTION AND EXECUTIVE SUMMARY ………………………… 
 3  
2. SUMMARY OF PRIMARY EFFICACY RESULTS…………………………    .. … 4 
3. SUMMARY OF MAJOR SECONDARY EFFICACY RESULTS …………  ………5 
4. SUMMARY OF SAFETY VARIABLES ……………..………  ………………… ... 5 
5. SUMMARY OF STUDY ………  ………………………………………………… ...7 
6. STUDY SYNOPSIS …………………………………………………………….…    10 
 TABLES: 
Table 1: Clinical Trial Monitoring Plan …………………………………………….  …  15 
Table 2: Efficacy Outcome Based on Primary Endpoints at 90 days ……………….  … 18 
Table 3: Demographic Data of All Patients Treated ………………………………    …. 20 
Table 4, 4a, 4b and 4c: Adverse Events recorded during the Study ……… ………       . 22 
    Table 5: Major and Minor Amputations Entire Study Amputations after Dosing …    …31 
Table 6: ASCT01 Stem Cell Product Information – All patients …………………..   . .. 32 
 
 

   
        
Final Study Summary Report An open-label single arm phase 2 proof of concept study to assess the efficacy 
and safety of ASCT01 in Patients with Critical Limb Ischemia 
 
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INTRODUCTION 
Indication/Objectives 
Definition of indication 
Critical limb ischemia is a condition in which the blood circulation to the extremities (mostly the legs) is 
decreased to a degree that pain and non-healing wounds ensue. Mostly, this is a consequence of 
arteriosclerosis and/or diabetes. If surgical and other methods for improvement of revascularization have 
failed or are not possible, as a final option these patients need limb amputation. 
The Rutherford classification is a standard clinical staging system for peripheral artery disease. The 
classification has 7 stages; 
 
- 
Stage 0 – Asymptomatic  
- 
Stage 1 – Mild claudication  
- 
Stage 2 – Moderate claudication  
- 
Stage 3 – Severe claudication  
- 
Stage 4 – Continuous pain  
- 
Stage 5 – Ischemic ulceration not exceeding the digits of the foot  
- 
Stage 6 – Severe ischemic ulcers or frank gangrene 
An upward shift of 1 stage indicates improvement, except for stage 5 and 6, where a shift towards stage 
3 is required for improvement/ 
 The trial was originally intended to a randomized placebo control but was converted to be performed as 
an open label single arm clinical trial with ASCT01 administration with approval from FDA. All patients 
received the autologous ASCT01 treatment. A staggered product administration approach for enrolment 
was used -a minimum of one week between administrations of the product to the first five subjects was 
allowed to study any potential effects of the product and/or the administration process. 
 
The treatment- was a one-time, one dose application carried out by the treating physician - via 
angiographic application (80% of the dose). The remaining 20% ASCT01 was injected intramuscular 
distal of the obstruction and along the anatomic course of the artery or the neurovascular bundle in the 
ischemic limb. Selection and follow-up evaluation was performed by the investigators at the site.  
This Phase II (proof of concept) trial was conducted as a clinical study.  A total of 33 male and/or 
female patients were screened, and 24 evaluable patients, completed the study.  The patients were 
evaluated till one year (12 months) post-administration with last patient out on March 30, 2017. 
 
Other variables also effect outcomes and reported mortality in success of revascularization, which also 
correlates with the severity of the disease at presentation.  Rutherford stage 4 patients generally appear 

   
        
Final Study Summary Report An open-label single arm phase 2 proof of concept study to assess the efficacy 
and safety of ASCT01 in Patients with Critical Limb Ischemia 
 
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to do better than stage 5; specifically, those requiring early major amputation. The stage 5 group 
generally is expected to have aggressive atherosclerosis, and rapid progression happens.  This may occur 
with intra-plaque hemorrhage, or just proliferation. Patients may progressive because of proximal 
disease changes as seen on a few MRA's. Resumption of smoking also appears to have a clear negative 
effect. 
  
The primary efficacy outcome is considered a success if at 3 months after administration of the cell 
treatment there is no major amputation or has shown a 15% increase in ankle-brachial index (ABI) or 
transcutaneous oximetry measurement (TCOM) from the measured baseline data. Other secondary 
variables such as pain analysis, would healing, angiography and walking distance, were also evaluated 
in this study for investigative purpose. The following summary is a review of the outcomes. 
 
SUMMARY OF PRIMARY EFFICACY RESULTS: 
Major Amputations: There were 2 major amputations (both below knee) during the 3-month period 
(Table 5). Both major amputations occurred in patients who were Rutherford 5 at screening. Among 
patients who were Rutherford 5 at baseline, major below amputations occurred in 8% of the patients 
during the 3-month period. 2 more major amputation occurred during the 6-month period evaluation and 
an additional 2 amputations occurred between the 6-month and the 12-month monitoring period or a total 
of 6 major amputations, however one of these patients received an amputation at a different clinic within 
33 days of the treatment. There were minor toe amputations recorded for 2 patients after the 3-month 
period. Major amputation rates of 30 to 40% are not uncommon in such patients and has been seen in 
other studies. Rutherford 5s enrolled with insufficient time for the stem cell effect may also exhibit 
rapid progression of other disease potentially leading to amputation. The late amputation patients who 
underwent below knee amputations have shown improvement after the cell therapy and in some cases 
preventing an above knee amputation.  
ABI and TcPO2 
The ankle-brachial index is an efficient tool for objectively documenting the presence of lower-
extremity peripheral arterial disease (PAD) and critical limb ischemia (CLI). It is a simple, reproducible, 
and cost-effective assessment that can be used to detect lower-extremity arterial stenosis in the primary 
care setting and transcutaneous oxygen measurement (TCOM or TcPO2) is a non-invasive method of 
measuring the oxygen level of the tissue below the skin. Since oxygen is carried by the blood, TCOM 
can be used as an indirect measure of blood flow to the tissue. Since blood flow is important for wound 
healing, TCOM is often used to gauge the ability of tissue to effectively heal. These parameters are used 
as combined primary parameters in the study. 
 

   
        
Final Study Summary Report An open-label single arm phase 2 proof of concept study to assess the efficacy 
and safety of ASCT01 in Patients with Critical Limb Ischemia 
 
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There was more than 15% improvement in ABI and/TcPO2 measurements in 12 patients from that 
obtained at treatment date and in the open-label phase of the trial at 3 months with some showing 
additional improvements at 6 months. One patient showed a 14.89% more increase (Table 2). 
SUMMARY OF MAJOR SECONDARY EFFICACY RESULTS: 
Pain Analysis 
Pain was assessed using a 10-point VAS scale. No absolute change on the VAS scale from baseline was 
required to denote either improvement or worsening, the intent was to evaluate change only from 
baseline. 13 patients showed a significant reduction of pain in the compared to the pain scale at 
treatment and 3- month endpoint and 14 at 6-month visit. The pain analysis was however inconclusive 
since most of the study patients were also under standard analgesic treatment.  
Walking distance  
Walking distance by walking distance measurements at baseline and various visits during the study was 
evaluated. However, due to pain or associated comorbidities, many did not complete or even attempt the 
test. Some improvement was observed in a number of patients from baseline vs various visits.  
Quality of life 
Quality of life was measured using the EQ-5D questionnaire at baseline and 3 months and continued 
at all visits. Some patients demonstrated improvements compared baseline. 
Ulcer and Wound Healing 
Wound size, wound healing stages according to the Wagner classification were reviewed. At 3 months 
significant changes were not observed, longer period and repeat therapy maybe required for healing 
progression of ulcers. Rutherford scale changes in some patients is indicative of potential healing was 
observed however. consistency could not be observed in this patent group. 
 
SUMMARY OF SAFETY EVALUATION: 
Safety and adverse events 
There was one death 6 months after treatment however the coroner’s information and PIs statement have 
conclusively ruled out any causative link to the stem cell treatment. There were no severe unexpected 
adverse events during this 3-month reporting period and later (Table 4 (a, b and c)). Bone marrow 
aspiration was well tolerated with no complications in most and only minor complications in some related 
to injection pricks.  The hematology values have not shown any trends. Muscle injury due to the 
intramuscular injections was not observed clinically or any adverse effects or embolism with the infusion 
process were noted. Ophthalmologic examinations at screening demonstrated baseline proliferative 

   
        
Final Study Summary Report An open-label single arm phase 2 proof of concept study to assess the efficacy 
and safety of ASCT01 in Patients with Critical Limb Ischemia 
 
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retinopathy in some patients, but there were no cases of worsened or new retinopathy. There were no 
clinically evident cases of new or recurrent malignancy. No significant cardiovascular adverse events 
were observed. 
 
 

   
        
Final Study Summary Report An open-label single arm phase 2 proof of concept study to assess the efficacy 
and safety of ASCT01 in Patients with Critical Limb Ischemia 
 
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SUMMARY OF STUDY  
1. 
SUMMARY PAGE 
Study title 
An open-label single arm phase 2 study to assess the efficacy and 
safety of ASCT01 in Patients with Critical Limb Ischemia. 
Treatment  
About 200 mL (150 +/- 50 mL) of the patient’s bone marrow 
(BM) is aspirated from the ileac crest (s). The bone marrow was 
then transported to a processing site and processed to produce 
ASCT01 by a qualified institution operating under FDA cGMPs 
and cGTCPs.  
 
In the current study al patients, BM harvest, processing and 
treatment steps were completed on the same day, however the 
stability profile of ASCT01 allows for treatment within three 
days (72 hrs) after the bone marrow harvest. 
 
After angiographic visualization of the arterial stenosis in the 
affected limb, 80% of ASCT01 was infused intra-arterially 
proximal to the stenosis. The remaining 20% ASCT01 was 
injected intramuscular (IM) distal to the obstruction along the 
anatomic course of the artery and the neuro-vascular bundle in 
the ischemic limb (at least 6 injections). The infusion dose was 
approximately 10 million cells per mL infused at a rate of 5 ml 
per minute using suitable catheters qualified for the 
administration. The sequence of the IM and IA administration 
was at the investigators discretion. The intra-arterial infusion 
occurred via a metered infusion system (BBraun Syringe pump 
or similar) that accommodating a 50-60 ml syringe and infuse 
rates capability of 0-999ml/hr) with either a Quick Cross 
Catheter of 0.035” (catalog 518-038) or 0.018” (catalog 518-
035) ID.  
 
The ASCT01 (cellular composition) prepared by minimal 
manipulation is administered as entirely prepared from the bone 
marrow, cell count and viability varies with individual 
characteristics. The agency has reviewed the ASCT01 process 
and has confirmed that this process can be characterized as 
minimally manipulated -  Appendix 1. 

   
        
Final Study Summary Report An open-label single arm phase 2 proof of concept study to assess the efficacy 
and safety of ASCT01 in Patients with Critical Limb Ischemia 
 
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Indication studied 
Patients with established CLI (confirmed by Rutherford Stages 4 
to 5) with angiographic evidence of significant infra-inguinal 
arterial occlusive disease. 
Study design 
An open-label single arm phase 2 interventional single arm 
proof of concept study to assess the efficacy and safety of 
ASCT01 in Patients with Critical Limb Ischemia (CLI) who 
would otherwise need amputation. For this trial, patients with a 
severe grade of CLI and categorized in the group of failed 
traditional revascularization treatment patients (no option 
patients) were enrolled.  
Trial Objective 
At 3 months, a combined primary endpoint of major amputation 
(above the ankle) or persisting critical limb ischemia (no clinical 
or perfusion improvement) will be evaluated by comparison of 
treatment group with baseline data. 
Primary Outcome 
measures 
The primary outcome variable is "treatment failure" defined as 
major amputation (above the ankle) of the affected limb within 3 
months or an unchanged critical limb ischemia of the affected 
limb after 3 months defined as less than 15% change in tcPO2 or 
ABI or absolute ankle pressure. 
Sponsor 
Lifecells, LLC. 
68 Discovery 
Irvine, CA 92618 
USA  
Study Protocol No. 
IND 15609 (Approved on December 13, 2012) 
Development phase of 
study 
Phase 2a (Proof of Concept) 
Number of Patients 
● No. of subjects planned: 24 
● No. of subjects screened:  38 
● No. of subjects treated: 24 
● No. of subjects withdrawn: 01 (KCV2-016 voluntary after 6 
months) 
● No. of subjects dropped out: 02 (KCV2-004 non-complaint) 
● KCV2-006 deceased due to unrelated issue after completion 
of 6 months 
● No. of subjects completed the study: 24 
● No. of subjects analyzed for efficacy statistical analysis: 24 

   
        
Final Study Summary Report An open-label single arm phase 2 proof of concept study to assess the efficacy 
and safety of ASCT01 in Patients with Critical Limb Ischemia 
 
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Number of Study Sites 
One (Kansas City Vascular Foundation, now Midwest Aortic 
and Vein Institute ( MAVI)),  
First Patient in Date: 
2 February 2014 (Informed consent date) Dose Date: 
02/20/2014 
Last Patient Dosed  
30 March 2016  
Last Patient Primary 
end point date (3 mos) 
3 June 2016  
Last Patient Out 
(scheduled)  
30 March 2017 (safety parameter only- Visit 7) 
Principal Investigator  
Dr. Karl Stark, MD 
Principal Investigator 
Kansas City Vascular Foundation 
2750 Clay Edwards Drive Suite 310 
City, State, Zip:   North Kansas City, MO 64116 
Email:   kstark@mavi.com  
Phone   816-842-5555 
 
Sponsor’s 
Representative 
Paul T Sudhakar 
President and CEO 
Lifecells LLC. 
68 Discovery 
Irvine, CA 92618 
Telephone: (816) 507-8249 
Email:   pauls@ptspharma.com 
Date of study report 
July 29, 2017 
This study was performed per the current protocol at the time of dosing and IRB 
approval and in compliance with Good Clinical Practices (GCP) and Good Laboratory 
Practices (GLP) 
 
 
 
 

   
        
Final Study Summary Report An open-label single arm phase 2 proof of concept study to assess the efficacy 
and safety of ASCT01 in Patients with Critical Limb Ischemia 
 
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2. 
SYNOPSIS 
Name 
of 
Sponsor 
/ 
Company: 
Lifecells LLC., USA 
 
 
Name of Finished Product: 
ASCT01 (Autologous Stem 
Cell Transplantation)  
 
 
Treatment: 
ASCT01 (autologous stem 
cell transplantation) 
 
 
 
Title of Study 
An open-label single arm phase 2study to assess the efficacy 
and safety of ASCT01 of Lifecells LLC, in Patients with 
Critical Limb Ischemia. 
Investigators 
Dr. Karl Stark, MD 
Principal Investigator 
Kansas City Vascular Foundation 
2750 Clay Edwards Drive Suite 310 
City, State, Zip:   North Kansas City, MO 64116 
Email:   kstark@mavi.com  
Phone   816-842-5555 
Scott W. Kujath, MD 
Kansas City Vascular Foundation 
Email:   skujath@mavi.com  
Phone    816-842-5555 
Role:      Co-PI  
Clinical Research Coordinator: 
Rebecca Thomas, ANP-BC 
Kansas City Vascular Foundation 
Email:   rthomas@mavi.com  
Phone   816-842-5555 
Statistician: 
Alex Dmitrienko, PhD 
Principal biostatistician 
Mediana, Inc 

   
        
Final Study Summary Report An open-label single arm phase 2 proof of concept study to assess the efficacy 
and safety of ASCT01 in Patients with Critical Limb Ischemia 
 
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Study Center 
Kansas City Vascular Foundation 
2750 Clay Edwards Drive Suite 310 
City, State, Zip:   North Kansas City, MO 64116 
Email:   kstark@mavi.com  
Phone   816-842-5555 
 
Publication (reference) 
None at This - To come later 
Statistical Facility 
Mediana, Inc 
Study start date 
2 February 2014 
Study end date 
30 March 2017 
Phase of development 
Phase 2a (Proof of Concept) 
Objectives 
Primary Objective: 
At 3 months, a combined primary endpoint of major amputation 
(above the ankle) or persisting critical limb ischemia (no clinical 
or perfusion improvement) was evaluated by comparison of 
treatment group with baseline data. 
Secondary Objectives 
After 3 months: 
- 
Time to treatment failure 
- 
Time to amputation 
- 
Changes in transcutaneous oxygen pressure (TcPO2) 
- 
Changes in ankle-brachial pressure index (ABI)/toe 
brachial pressure index (TBI) 
- 
Rate of minor amputations in the affected limb (below 
the ankle) 
- 
Wound/Ulcer healing (wound size, wound stage 
according to the Wagner classification, wound healing stages 
according to the Reike classification picture) 
- 
Pain (visual analog scale) 
- 
Analgesics use 
- 
Quality of life (EQ-5D Questionnaire, Peripheral 
Vascular Questionnaire)  
- 
Rutherford grade and stage  
- 
Collateral arteries as judged by magnetic resonance 
angiography after 3 months 
- 
Measurement maximal walking ability in speed and 
distance without or with pain 
- 
Rate of major adverse cardiovascular events 
 

   
        
Final Study Summary Report An open-label single arm phase 2 proof of concept study to assess the efficacy 
and safety of ASCT01 in Patients with Critical Limb Ischemia 
 
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Design Methodology 
An open-label single arm phase 2 study to assess the efficacy 
and safety of ASCT01 in Patients with Critical Limb 
Ischemia (CLI) who would otherwise need amputation. For 
this trial, patients with a severe grade of CLI and categorized 
in the group of failed traditional revascularization treatment 
patients (no option patients) were enrolled.  
 
Selected patients received ASCT01 treatment. The patients in 
the treatment group underwent bone marrow collection for 
aspiration of about 200 mL of bone marrow from the Ileac 
crest(s). The bone marrow was processed to the required 
dilution of the ASCT01 at Kansas University Midwest Stem 
Cell Research Center and Biomedical Devices of Kansas 
operating under FDA cGMPs and cGTCPs. 
The bone marrow collection and the treatment occurred for 
all patients on the same day. Although there is a provision for 
treatment up to 72 hrs hold time if required, all the patients in 
this group were treated with the stem cell product on the 
same day of the bone marrow harvest. 
 
After angiographic visualization of the arterial stenosis in the 
affected limb, about 80% of diluted ASCT01 was infused 
intra-arterially (IA) proximal to the stenosis. The remaining 
20% ASCT01 was injected intramuscularly (IM) distal to the 
obstruction along the anatomic course of the artery and the 
neuro-vascular bundle in the ischemic limb (at least 6 
injections). The infusion dose is 10 million cells per mL 
infused at a rate of 5 ml per minute using suitable catheters 
qualified for the administration. The sequence of the IM and 
IA administration was at the investigators discretion.  
 
The intra-arterial infusion occurred via metered infusion 
system (Braun Syringe pump or similar) that accommodated 
a 50-60 ml syringe and can infuse at rates of 0-999ml/hr) 
with either a Quick Cross Catheter of 0.035” (catalog 518-
038) or 0.018” (catalog 518-035) ID.  
After ASCT01 administration, the patients were evaluated at 
3 months for the primary end points evaluation and 
monitoring continued for a total period of 12 months (the 
visit schedule table is at the end of the synopsis).  

   
        
Final Study Summary Report An open-label single arm phase 2 proof of concept study to assess the efficacy 
and safety of ASCT01 in Patients with Critical Limb Ischemia 
 
13 
 
 A staggered product administration approach for enrolment 
was used -a minimum of one week between administrations 
of the product to the first five subjects for safety check of the 
administration.   
 
Number of subjects (Table 3 
– Demographics) 
 
● No. of subjects planned: 24 
● No. of subjects screened:  38 
● 5 excluded after informed consent 
● 9 excluded before informed consent 
● No. of subjects treated: 24 
● No. of subjects withdrawn: 02 (KCV2-016 Voluntary 
KCV2-017 BKA) 
● No. of subjects dropped out: 01 (KCV2-004 non-
complaint) 
● One subject KCV2-006 deceased after 6 mos (determined 
unrelated to the treatment  
● No. of subjects completed the study: 24 
● No. of subjects analyzed for efficacy statistical analysis: 
24  
Diagnosis and criteria for 
inclusion 
1. 
Male and Female patients in the age group of 18-80yrs. 
2. 
Established CLI (confirmed by Rutherford 4 to 5) with 
angiographic evidence of significant infra-inguinal 
arterial occlusive disease  
3. 
Ankle Brachial Pressure Index (ABI) ≤ 0.6 or the absolute 
ankle blood pressure < 60 mm Hg or TcPO2<20 mmHg 
without tissue loss or TcPO2<40 mmHg if there is tissue 
loss or alternatively toe Brachial Pressure Index (TBI) 
less 0.5 or the absolute toe blood pressure less than 50 
mm Hg  
4. 
No surgical or interventional option for revascularization 
and no response to best standard care delivered as 
confirmed by a vascular surgeon and/or physician. 
5. 
No immediate life-threatening complication from CLI 
which would demand immediate amputation. 

   
        
Final Study Summary Report An open-label single arm phase 2 proof of concept study to assess the efficacy 
and safety of ASCT01 in Patients with Critical Limb Ischemia 
 
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6. 
Patients who are able to understand the requirements of 
the study, and willing to provide voluntary written 
informed consent, abide by the study requirements, and 
agree to return for required follow-up visits. 
7. 
On optimal medical therapy  
8. 
If diabetic, HgbA1c <10% 
Exclusion Criteria 
1. Acute life threatening complication of limb ischemia with 
the need for immediate limb amputation to avoid death or 
clinical deterioration 
2. Patients with confirmed Rutherford 6 condition with 
extensive tissue damage 
3. Patients with documented terminal illness or cancer or 
any concomitant disease process with a life expectancy 
of less than 6 months. 
4. Patients with a history of severe alcohol or drug abuse 
within 3 months of screening. 
5. Known 
bone 
marrow 
diseases 
which 
preclude 
transplantation. 
6. End-stage renal failure on regular dialysis treatment. 
Creatinine ≥2.0 mg/dl 
7. Patients already enrolled in another investigational drug 
trial or completed within 1month. 
8. Pregnancy. 
9. Patients tested positive for HIV screen1or2, Hepatitis C 
Antibody Hepatitis Bsurface-antigen, HepatitisBcore 
Antibody, Syphilis screen 
10. Myocardial infarction / CVA / TIA within the past three 
months prior to enrollment 
11. Revascularization procedure in target limb within 6 
weeks prior to enrollment 
12. Laboratory values as show below* 
13. Currently taking immunosuppressive agents 
14. If diabetic, diagnosis of proliferative retinopathy 
15. Patients with infected ulcers or systemic infections 

   
        
Final Study Summary Report An open-label single arm phase 2 proof of concept study to assess the efficacy 
and safety of ASCT01 in Patients with Critical Limb Ischemia 
 
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*Laboratory Values: 
Hemoglobin  <10 g/dL 
Platelet count  <100,000/microL 
ALT  
>60 U/L 
AST   
>60 U/L 
Bilirubin  
>1.0 mg/dL 
INR   
>1.3 unless on Coumadin and at Investigator 
discretion 
APTT  
 
>40 second unless on Lovenox or 
Heparin and at Investigator’s discretion 
 
 
 
 
Treatment  
Lifecells Stem Cell Product (ASCT01) diluted to 10 million 
CD45+ per mL for infusion and injections 
Lot No. 
Each patient is treated as a lot number for the ASCT01 
(Table 6 - product information). The certificates of release 
testing and analysis will be provided in module 3 of the IND 
file for the phase 3 study or as requested by the Agency. 
Treatment and mode of 
administration 
Single treatment only in the proof of concept study 
administered about 80% of diluted ASCT01 as infusion 
intra-arterially (IA) proximal to the stenosis. The remaining 
20% ASCT01 was injected intramuscularly (IM) distal to the 
obstruction along the anatomic course of the artery and the 
neuro-vascular bundle in the ischemic limb (at least 6 
injections). The infusion dose is 10 million cells per mL 
infused at a rate of 5 ml per minute using suitable catheters 
qualified for the administration. 
Duration of the treatment 
A single treatment was administered on same day. Total 
duration of the study from the check in of period one to the 
last evaluation was 365 days. 
Criteria for evaluation: 
Safety Evaluation 
- 
Adverse events 
- 
Vital signs 
- 
Standard biochemical safety variables 
Table 1: Clinical Trial Monitoring Chart. 
Visit 
1 
2 
3 
4 
5 
6 
7 

   
        
Final Study Summary Report An open-label single arm phase 2 proof of concept study to assess the efficacy 
and safety of ASCT01 in Patients with Critical Limb Ischemia 
 
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Written informed consent 
X 
 
 
 
 
 
 
Inclusion criteria 
X 
X 
 
 
 
 
 
Exclusion criteria 
X 
X 
 
 
 
 
 
Randomization 
 
X 
 
 
 
 
 
Demographic data 
X 
X 
X 
X 
X 
X 
X 
Medical History-Angiogram CD & Medical 
Records Required to be provided at this time 
X 
 
 
 
 
 
 
6 min Walking test  
X 
 
 
X 
X 
X 
X 
Quality 
of 
Life& 
Peripheral 
Vascular 
Questionnaires, 
VAS (Pain Score) 
X 
 
 
X 
X 
X 
X 
Ankle-Brachial Pressure Index (ABI) / Toe 
Brachial Pressure Index (TBI) 
X 
 
X 
X 
X 
X 
X 
Transcutaneous oxygen pressure (TcPO2) 
X 
 
 
X 
X 
X 
X 
Wound/ulcer 
examination(Wagner/Reike/Picture) 
X 
 
 
X 
X 
X 
X 
Physical Examination  
X 
 
X 
X 
X 
X 
X 
Rutherford assessment 
X 
 
 
X 
X 
X 
X 
Vital signs 
X 
X 
X 
X 
X 
X 
X 
ECG 
X 
X 
 
 
 
 
X 
Infectious Serology-Screening HIV1-2, 
Hepatitis C Antibody Hepatitis B surface-
antigen, Hepatitis B Core Antibody, Syphilis 
screen, 
X 
 
 
 
 
 
 
Pregnancy test / Use of contraception 
X 
 
 
 
 
 
 
Blood Labs- Hematology-CBC with Diff, 
CMP, lipid panel, C-Reactive Protein 
*At Day 7 only CBC with Diff 
X 
 
X* 
X 
X 
X 
X 
INR & APTT for patients on anticoagulation 
therapy 
X 
X 
 
 
 
 
 
HbA1c for DM subject’s only 
X 
 
 
 
X 
X 
X 

   
        
Final Study Summary Report An open-label single arm phase 2 proof of concept study to assess the efficacy 
and safety of ASCT01 in Patients with Critical Limb Ischemia 
 
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Retinal examination (diabetic subjects only) 
X 
 
 
 
 
X 
 
Bone Marrow Collection  
 
X 
 
 
 
 
 
Angiography with contrast  
 
X 
 
 
 
 
 
MR Angiography with contrast  
 
X 
 
 
X 
 
 
Stem cell transplantation: ASCT01 - 
 
X 
 
 
 
 
 
Record Adverse Events/Serious 
Adverse 
Events 
 
X 
X 
X 
X 
X 
X 
Concomitant medications 
X 
X 
X 
X 
X 
X 
X 
Record Analgesic(s) Use 
X  
X 
X 
X 
X 
X 
X 
 
Statistical Evaluation 
Statistical analysis was performed using SAS® package 
(SAS Institute Inc., USA, Version 9.3). for subject size 
requirement and power of the study based on the outcomes 
in the proof of concept. Comparative analysis for the 
number of treatment failures (i.e. major amputations or the 
number of unchanged Critical Limb Ischemia) for the 
treatment group with baseline data and the outcomes. 
Other Analyses of time to treatment failure and of time to 
amputation for the treatment group was evaluated. 
An external Data Safety Monitoring Board (DSMB) was 
used to oversee the conduct of the study as outlined in the 
DSMB Charter.  The DSMB had access to the data and 
performed the following functions: 
1. Review the conduct of the study and accruing safety 
data at interim analysis. 
2. Review safety data on an ad hoc basis as safety 
questions arise. 
The DSMB was allowed to stop the study at any point in 
time, if the safety of the patients is at stake. The decision to 
stop the study can also be made by the PI, for safety reasons. 
Members of DSMB: 
Chairman: Charles Ward Van Way III, M.D , Professor of 
Surgery 
Sosland / Missouri Endowed Chair of Trauma 

   
        
Final Study Summary Report An open-label single arm phase 2 proof of concept study to assess the efficacy 
and safety of ASCT01 in Patients with Critical Limb Ischemia 
 
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Services, Director, Shock Trauma Research Center, 
University of Missouri - Kansas City 
2411 Holmes Street, Kansas City, Missouri, 64108 TEL: 
(816) 404-5364, (816) 235-2553. 
EMAIL: vanwayc@umkc.edu 
Robert Rick Carter, M.D 
Vascular Surgeon 
Kansas City Vascular, PC 
2750 Clay Edwards Drive Suite 304 
Kansas City MO 64116-3256 
TEL: (816) 842-5555 
FAX: (816) 842-8888 
EMAIL: rcarter©kcvascular.com 
Statistical Consult:  Nilofer Qureshi, Ph.D Professor, 
Basic Medical Science, , University of Missouri - Kansas 
City 
2411 Holmes Street, Kansas City, Missouri, 64108 TEL: 
816-587-3642, office; 816-235-1056, Cell: 816-721-2281 
EMAIL: qureshin@umkc.edu 
Specific statistical measures and methods (per FDA 
guidance, regulations and scientifically accepted methods) 
Table 2: Efficacy Outcome Based on 
Primary Endpoints at 90 days (visit 5) 
Evaluation 
 
Primary 
Endpoi
nt 
Subject 
Identifier 
for the 
Study  
KCV2-00 
ABI 
at 
Visit 
1 
ABI 
at 
Visit 
5 
TcPO
2 at 
Visit 
1 
TcPO2 
at Visit 
5 
Amput
ation 
Percent 
Change 
in ABI 
at Visit 
5 
Percent 
Change 
in 
TcPO2 
at Visit 
5 
Comment 
Failure 
1 
0.74 
0.64 
29 
14   
-13.51 
-51.72 
Failure % change is less 
than 15% for both ABI 
and TcPO2 
Failure 
2 
0.01 
0.44 
5   
Major 
4300   
Failure due to major 
amputation 

   
        
Final Study Summary Report An open-label single arm phase 2 proof of concept study to assess the efficacy 
and safety of ASCT01 in Patients with Critical Limb Ischemia 
 
19 
 
Failure 
3 
0.01 
0.01 
7 
5   
0 
-28.57 
Failure % change is less 
than 15% for both ABI 
and TcPO2 
Failure 
4 
0.23   
10   
Major 
  
  
Failure due to major 
amputation 
Success 
5 
0.46 
0.73 
39 
28   
58.7 
-28.21 
  
Success 
6 
0.38 
0.52 
2 
27   
36.84 
1250 
  
Success 
7 
0.49 
0.58 
40 
35   
18.37 
-12.5 
  
Success 
8 
0.58 
0.83 
3 
39   
43.1 
1200 
  
Success 
9 
0.47 
0.58 
41 
60   
23.4 
46.34 
  
Failure 
10   
  
53 
51   
  
-3.77 
Failure % change is less 
than 15% for TcPO2 
Success 
11 
0.49 
0.57 
15 
47   
16.33 
213.33 
  
Failure 
12 
0.55 
0.57 
60 
28   
3.64 
-53.33 
Failure % change is less 
than 15% for both ABI 
and TcPO2 
Failure 
13 
0.5 
0.56 
19 
20   
12 
5.26 
Failure % is less than 
15% for both ABI and 
TcPO2 
Failure 
14 
0.54 
0.46 
66 
49   
-14.81 
-25.76 
Failure %is less than 15% 
for both ABI and TcPO2 
Success 
15 
0.21 
0.51 
2 
20   
142.86 
900 
  
Failure 
16 
0.24 
0.18 
19 
2   
-25 
-89.47 
Failure % change is less 
than 15% for both ABI and 
TcPO2 
Success 
17 
0.48 
0.3 
2 
3   
-37.5 
50 
  
Failure 
18 
0.46 
0.25 
44 
33   
-45.65 
-25 
Failure % is less than 15% 
for both ABI and TcPO2 
Failure 
19   
  
32 
13   
  
-59.38 
Failure % change is less 
than 15% for TcPO2 
Success 
20 
0.17 
0.31 
16 
11   
82.35 
-31.25 
  

   
        
Final Study Summary Report An open-label single arm phase 2 proof of concept study to assess the efficacy 
and safety of ASCT01 in Patients with Critical Limb Ischemia 
 
20 
 
Success 
21 
0.19 
0.34 
11 
26   
78.95 
136.36 
  
Success 
23 
0.4 
0.48 
41 
41   
20 
0 
  
Failure 
24 
0.43 
0.43 
47 
54   
0 
14.89 
Failure % change is less 
than 15% for both ABI 
and TcPO2 
(nearly a 15% TcP02 
increase) 
Success 
25 
0.3 
0.36 
17 
42   
20 
147.06   
*KCV2-002 – BKA 4/22/14 
 
*KCV2-004 - Had amputation from another doctor outside the study within the 30 days after  
the stem cell transplantation (non-complaint) 
 
*KCV2-010 - ABI could not be measured occluded. 
 
*KCV2-017- non-complaint withdrew from study  
 
*KCV2-019 - ABI is of Toe (not taken into consideration will pass with TCOM) 
Success Rate:  All 24 patients    = 50% 
Table 3: Demographic Data of All Patients Treated 
 
Patie
nt ID 
Sex 
Age 
(Year
s) 
Race 
Height 
(cm) 
Weig
ht 
(Kg) 
Treatm
ent 
Limb 
Smoking 
Status 
Remarks 
KCV2
-001 
Male 
81 
Caucasian 
180 
65.4 
Left 
Former 
Diabetic 
KCV2
-002 
Male 
54 
Caucasian 
170.2 
93 
Left 
Current 
Diabetic 
KCV2
-003 
Male 
50 
Caucasian 
172.7 
104.3 
Left 
Former 
Non-diabetic 
KCV2
-004 
Femal
e 
59 
Caucasian 
160 
49.9 
Left 
Current 
Non-diabetic 
KCV2
-005 
Femal
e 
62 
Caucasian 
162.56 
90.9 
Left 
Former 
Diabetic 
KCV2
-006 
Male 
50 
Caucasian 
180.3 
170 
Right 
Former 
Non-diabetic 

   
        
Final Study Summary Report An open-label single arm phase 2 proof of concept study to assess the efficacy 
and safety of ASCT01 in Patients with Critical Limb Ischemia 
 
21 
 
KCV2
-007 
Male 
60 
Caucasian 
175.3 
106.3 
Right 
Current 
Non-diabetic 
KCV2
-008 
Male 
75 
Caucasian 
172.7 
60.5 
Left 
Former 
Non-diabetic 
KCV2
-009 
Femal
e 
43 
Caucasian 
154.9 
103.6 
Left 
Former 
Non-diabetic 
KCV2
-010 
Male 
77 
Caucasian 
177.8 
79.5 
Right 
Former 
Diabetic 
KCV2
-011 
Femal
e 
80 
Caucasian 
152.4 
62.2 
Left 
Never 
Diabetic 
KCV2
-012 
Male 
62 
Caucasian 
175.3 
83.6 
Left 
Former 
Diabetic 
KCV2
-013 
Male 
74 
Hispanic 
167.6 
56.4 
Right 
Former 
Diabetic 
KCV2
-014 
Male 
59 
Caucasian 
172.7 
65.8 
Left 
Current 
Non-diabetic 
KCV2
-015 
Male 
78 
Caucasian 
175.3 
65.9 
Left 
Former 
Non-diabetic 
KCV2
-016 
Male 
67 
Caucasian 
187 
86 
Right 
Current 
Non-diabetic 
KCV2
-017 
Male 
72 
Caucasian 
165.1 
65.9 
Left 
Current 
Non-diabetic 
KCV2
-018 
Male 
82 
Caucasian 
190.5 
89.5 
Left 
Former 
Non-diabetic 
KCV2
-019 
Male 
82 
Caucasian 
172.7 
76.1 
Left 
Former 
Non-diabetic 
KCV2
-020 
Male 
68 
African 
American 
187.9 
118.3 
Left 
Former 
Diabetic 
KCV2
-021 
Femal
e 
66 
Caucasian 
167.6 
68.3 
Right 
Former 
Non-diabetic 
KCV2
-023 
Femal
e 
76 
Caucasian 
175 
72.9 
Left 
Never 
Diabetic 
KCV2
-024 
Male 
59 
Caucasian 
180 
66 
Right 
Former 
Diabetic 
KCV2
-025 
Male 
52 
Caucasian 
175.3 
81.7 
Left 
Current 
Non-diabetic 
 
 
Mean  
= 66.2 
 
 
 
 
 
 

   
        
Final Study Summary Report An open-label single arm phase 2 proof of concept study to assess the efficacy 
and safety of ASCT01 in Patients with Critical Limb Ischemia 
 
22 
 
Male 
– 18 
Femal
e - 6 
High = 
82 
Low = 
43 
Caucasian = 
22 
Hispanic = 1  
African-
American = 1 
High = 
190.5 
Low  = 
152.4 
High = 
170 
Low = 
49.9 
Left 
= 
17 
Right = 
7 
Current = 
7 
Former = 
15 
Never = 2 
Diabetic = 10 
Non-diabetic = 
14 
 
 
Safety Results 
 
 
 
Suspected Adverse events and adverse events were recorded 
during the study at various visits. None of the major the events 
reported below can be attributed directly to the product or the 
treatment procedure by the PI.  
The firm concludes that the product /treatment is safe to use 
as intended in future studies. 
Table 4: (AEs and SAEs, detailed presentation with start and end dates) 
SUBJECT 
START 
DATE 
DESRIPTION 
SAE 
OR 
AE 
END DATE 
KCV2-001 
5/21/2014 Left foot Debridement 
SAE 
5/21/2014 
5/23/2014 Incision & Drainage of Left foot/IN OR 
SAE 
5/24/2014 
8/19/2014 Left foot osteomyelitis/left & transmetatarsal Amputation 
SAE 
8/25/2014 
KCV2-002 
1/28/2015 Non-healing ulcer Right foot/great toe amputation 
SAE 
1/31/2015 
12/18/2014 
Right Femoral popliteal Bypass with saphenious vein graft 
Right common iliac vent placement -hospital 
SAE 
12/21/2014 
4/22/2014 severe ischemic gangrene with necrosis & infection Left BKA 
SAE 
4/26/2014 
4/23/2014 anemia  5.4 /TU PRBC OP 
AE 
4/23/2014 
7/11/2014 
Non-healing Left BKA site wound /Hospital revision of Left 
BKA 
SAE 
7/14/2014 
9/22/2014 Non-healing Left BKA/Admit skin graft 
SAE 
9/23/2014 
KCV2-003 
8/27/2014 cellulities, uncontrolled pain/Admit  IVAB, IV pain control 
SAE 
9/3/2014 
9/7/2014 recurrent cellulities/Hosp IVAB pain mgmt. 
SAE 
9/12/2014 
9/16/2014 recurrent dependent ruber R/T PAD/ Admit IVAB 
SAE 
9/18/2014 
2/16/2015 
1)Increase pain, edema/ In Patient referred to HB.2) New 
Left 3 RD toe with ischemic ulcer 
SAE 
2/18/2015 
3/11/2015 Intractable pain/admit LF BKA 
SAE 
3/26/2015 
KCV2-004 
5/20/2014 Anemia /In Patient TU PRBC & IV ferritin 
SAE 
5/23/2014 

   
        
Final Study Summary Report An open-label single arm phase 2 proof of concept study to assess the efficacy 
and safety of ASCT01 in Patients with Critical Limb Ischemia 
 
23 
 
6/6/2014 Anemia, dehydration/Admit IV fluids 
SAE 
6/6/2014 
6/10/2014 Cellulitis & Temp/ PO ABX IN PATIENT 
SAE 
6/13/2014 
6/19/2014 LF  wound Infection/Admit LBKA 
SAE 
6/23/2014 
9/9/2014 Fever non healing  surgical wound/PO ABX Out Patient 
AE 
9/10/2014 
9/10/2014 Non-healing BKA incision - Out Patient 
AE 
9/10/2014 
9/17/2014 Osteo UE/Admit LBKA revision 
SAE 
9/20/2014 
12/6/2014 stump infection/surgical revision. Left Knee disarticulation 
SAE 
12/12/2014 
KCV2-005 
12/15/2014 Rt hand trauma/Fell seen in ER released 
AE 
12/15/2015 
12/22/2014 
chest pain, SOB/Admit - placed on Beta -blocker echo-
cardiac catheter with no intervention 
SAE 
12/25/2014 
2/15/2015 Rt upper Ext DVT, restarted warfarin (in hospital) 
SAE 
2/23/2015 
2/6/2015 ABD pain/Admit - colon ostomy Resection 
SAE 
2/23/2015 
KCV2-006 
8/26/2014 
LL Critical Limb ischemia/Admit IV AB-IV pain med, IV 
Anticoagulant Removed Thrombus 
SAE 
8/29/2014 
9/17/2014 high Platelet - 1 unit Phlebectomy 
AE 
9/17/2014 
9/28/2014 
Increased pain of Non-index foot/ER-IV pain, Meds with pain 
Mgmt. 
AE 
9/28/2014 
10/2/2014 limb dry gangrene with infect/BKA 
SAE 
10/7/2014 
KCV2-007 
. 
none mentioned 
  
  
KCV2-008 
7/2/2014 
Hypotension secondary to sedation requiring 250 cc NS 
bolus 
AE 
7/2/2014 
KCV2-009 
10/3/2014 one visit with primary care 
AE 
10/3/2014 
12/8/2014 visit with PCP x1 
AE 
12/8/2014 
3/26/2015 
Eye infection placed on antibiotics X 10 days, Abnormal 
mammogram left breast 
N/A No record 
KCV2-010 
10/2/2014 Multiple iliac crest access  
N/A   
11/6/2014 PCP, Endo, ID, visit 
N/A   
KCV2-011 
11/25/2014 Respiratory infection/Admit pneumonia 
SAE 
11/26/2014 
KCV2-012 
11/13/2014 
shoulder pain/Admit Rotation cuff Repair NOT AN 
ADMISSION  
AE 
11/14/2014 
KCV2-013 
none mentioned 
  
  
KCV2-014 
7/29/2015 
Patient having left foot pain been seeing podiatry for 
neuroma's, currently getting injections to "kill nerve" 
AE 
9/1/2015, 
7/29/15-
9/1/2015 
KCV2-015 
8/2/2015 hypoxia/admitted Pneumonia CXP-IVAB 
SAE 
8/6/2015 

   
        
Final Study Summary Report An open-label single arm phase 2 proof of concept study to assess the efficacy 
and safety of ASCT01 in Patients with Critical Limb Ischemia 
 
24 
 
8/2/2015 Bursitis-cellulitis / aspiration of Bursitis Admitted IVAB 
SAE 
8/6/2015 
11/13/2015 Pneumonia 
SAE 
11/14/2015 
1/3/2016 C-Diff Colitis (abdominal pain / diarrhea) 
SAE 
2/27/2016 
1/3/2016 gross hematuria 
SAE 
ongoing @ 
study exit 
2/19/2016 Increased drainage LLE wound 
SAE 
ongoing @ 
study exit 
2/19/2016 UTI & hematuria 
SAE 
ongoing @ 
study exit 
2/19/2016 Sepsis 
SAE 
2/27/2016 
KCV2-016 
. 
none mentioned 
  
  
KCV2-017 
5/28/2015 Hgb 7.6 - low labs value - 
AE 
6/24/2015 
6/4/2015 Rt groin Hematoma Ultrasound 
AE 
6/16/2015 
7/14/2015 Intractable foot pain/admitted med adjust 
SAE 
8/27/2015 
8/14/2015 Intractable foot pain/Hosp pain control 
SAE 
8/27/2015 
8/26/2015 progresses demarcation of left foot with infection/Hosp BKA 
SAE 
8/27/2015 
9/1/2015 s/p LBKA painful Rt toenails/Rehab - toenail debrided 
AE 
9/2/2015 
KCV2-018 
8/6/2015 Rt Groin wound /Bactrim Out Patient 
AE 
8/16/2015 
8/21/2015 UTI - Bactrim 
AE 
10/21/2015 
1/3/2016 
New pressure ulcer - referred to podiatry- 0.4 cm wound X 
0.1 cm deep ⏀erudite, healthy wound bed foam applied & 
to follow up with Podiatry. 
AE 
ongoing @ 
study exit 
KCV2-019 
4/2/2016 respiratory failure 
SAE 
4/9/2016 
4/9/2016 L groin pain - possible inguinal hernia 
AE 
4/10/2016 
KCV2-020 
12/30/2015 New wound LF heal & toe - Referred to wound clinic 
AE 
4/8/2016 
12/30/2015 worsening wound - referred to HBO 
AE 
4/8/2016 
4/6/2016 patient in hospital below BKA on index limb 
SAE 
4/8/2016 
7/13/2016 ongoing L BKA wound dehiscence 
SAE 
ongoing @ 
study exit 
KCV2-021 
10/7/2015 post procedural bleeding / 2 units FFP 
SAE 
10/7/2015 
10/10/2015 uncontrolled pain / mod start on PCP Fentanyl patch 
AE 
10/11/2015 
10/16/2015 R leg cellulitis 
SAE 
11/12/2015 
10/19/2015 Supratheraputic INR / Admit 
SAE 
10/20/2015 
10/19/2015 Anemia  - Hospital PRBC picc placed  
SAE 
10/26/2015 
10/22/2015 Shortness of breath with anemia 
SAE 
10/23/2015 
10/30/2015 mental status changes 
SAE 
10/30/2015 

   
        
Final Study Summary Report An open-label single arm phase 2 proof of concept study to assess the efficacy 
and safety of ASCT01 in Patients with Critical Limb Ischemia 
 
25 
 
10/30/2015 increased pain & increased edema rle 
SAE 
10/31/2015 
11/2/2015 Altered mental status -admit med adjust 
SAE 
11/5/2015 
3/9/2016 
Open transmetatarsal amputation of index limb for ischemic 
ulcer with gangrene 
SAE 
ongoing @ 
study exit 
4/15/2016 worsening of R forefoot ulcer with revision of R forefoot  
SAE 
ongoing @ 
study exit 
5/20/2016 c-diff colitis 
SAE 
5/27/2016 
5/25/2016 
Hospitalized at NKCH for non-healing right foot 
transmetatarsal amputation site) 
SAE 
ongoing @ 
study exit 
8/19/2016 Split thickness skin graft to R foot 
SAE 
ongoing @ 
study exit 
11/7/2016 admitted for hip fracture after fall 
SAE 
ongoing @ 
study exit 
KCV2-023 
  
none mentioned 
  
  
KCV2-024 
4/7/2016 
Diagnostic Colonoscopy for continued GI disturbance & 
diarrhea 
AE 
4/7/2016 
KCV2-025 
4/28/2016 
Subject bit by a pit bull while fixing AC. States he went to a 
doctor where it happened & no antibiotic or dressing 
changes given 
AE 
4/28/2016 
 
Lifecells LLC, Protocol ASCT01 
          Table 4a: Overall Summary of Treatment Emergent Adverse Events (TEAE) 
                                                        Analysis Set: All Randomized 
Subjects 
 
_______________________________________________________________________________________ 
                                                                        Autologous Stem 
Cell Transplant 01 
                                                                                      
(N=24) 
_______________________________________________________________________________________ 
Subjects with TEAEs 
Yes                                                                      20 ( 83.3%) 
No                                                                        4 ( 16.7%) 
 
Subjects with TESAEs 
No                                                                       12 ( 50.0%) 
Yes                                                                      12 ( 50.0%) 
 
 
 
Lifecells LLC, Protocol ASCT01 

   
        
Final Study Summary Report An open-label single arm phase 2 proof of concept study to assess the efficacy 
and safety of ASCT01 in Patients with Critical Limb Ischemia 
 
26 
 
 Table 4 (a): Treatment Emergent Adverse Events (TEAE) by MedDRA Primary System Organ Class and 
Preferred Term 
                                      Analysis Set: All Subjects 
 
________________________________________________________________________________________________________ 
System Organ Class                                                      Autologous Stem Cell Transplant 
01 
   Preferred Term                                                                     (N=24) 
________________________________________________________________________________________________________ 
 
Subjects with TEAEs                                                       20 ( 83.3%) 
 
Total Number of TEAEs                                                     79 
 
Blood and lymphatic system disorders                                       3 ( 12.5%) 
   Anaemia                                                                 3 ( 12.5%) 
 
Cardiac disorders                                                          1 (  4.2%) 
   Angina pectoris                                                         1 (  4.2%) 
 
Gastrointestinal disorders                                                 3 ( 12.5%) 
   Abdominal pain                                                          1 (  4.2%) 
   Gastrointestinal disorder                                               1 (  4.2%) 
   Inguinal hernia                                                         1 (  4.2%) 
 
General disorders and administration site conditions                       2 (  8.3%) 
   Pain                                                                    2 (  8.3%) 
 
Infections and infestations                                                9 ( 37.5%) 
   Bursitis infective                                                      1 (  4.2%) 
   Cellulitis                                                              3 ( 12.5%) 
   Clostridium difficile colitis                                           2 (  8.3%) 
   Eye infection                                                           1 (  4.2%) 
   Localised infection                                                     1 (  4.2%) 
   Osteomyelitis                                                           1 (  4.2%) 
   Pneumonia                                                               2 (  8.3%) 
   Postoperative wound infection                                           1 (  4.2%) 
   Sepsis                                                                  1 (  4.2%) 
   Urinary tract infection                                                 2 (  8.3%) 
   Wound infection                                                         1 (  4.2%) 
 
Injury, poisoning and procedural complications                             9 ( 37.5%) 
   Animal bite                                                             1 (  4.2%) 
   Hip fracture                                                            1 (  4.2%) 
   Limb injury                                                             2 (  8.3%) 
   Post procedural complication                                            2 (  8.3%) 
   Post procedural haemorrhage                                             1 (  4.2%) 
   Postoperative wound complication                                        2 (  8.3%) 
   Procedural hypotension                                                  1 (  4.2%) 
   Wound                                                                   2 (  8.3%) 
   Wound dehiscence                                                        1 (  4.2%) 
 
Investigations                                                             3 ( 12.5%) 

   
        
Final Study Summary Report An open-label single arm phase 2 proof of concept study to assess the efficacy 
and safety of ASCT01 in Patients with Critical Limb Ischemia 
 
27 
 
   Haemoglobin decreased                                                   1 (  4.2%) 
   International normalised ratio increased                                1 (  4.2%) 
   Platelet count increased                                                1 (  4.2%) 
 
Musculoskeletal and connective tissue disorders                            4 ( 16.7%) 
   Musculoskeletal pain                                                    1 (  4.2%) 
   Pain in extremity                                                       3 ( 12.5%) 
 
Neoplasms benign, malignant and unspecified (incl                          1 (  4.2%) 
cysts and polyps) 
   Neuroma                                                                 1 (  4.2%) 
 
 
Psychiatric disorders                                                      1 (  4.2%) 
   Mental status changes                                                   1 (  4.2%) 
 
Renal and urinary disorders                                                1 (  4.2%) 
   Haematuria                                                              1 (  4.2%) 
 
Respiratory, thoracic and mediastinal disorders                            2 (  8.3%) 
   Dyspnoea                                                                1 (  4.2%) 
   Respiratory failure                                                     1 (  4.2%) 
 
Skin and subcutaneous tissue disorders                                     5 ( 20.8%) 
   Decubitus ulcer                                                         1 (  4.2%) 
   Onychalgia                                                              1 (  4.2%) 
   Skin ulcer                                                              3 ( 12.5%) 
 
Surgical and medical procedures                                            7 ( 29.2%) 
   Bone operation                                                          1 (  4.2%) 
   Debridement                                                             1 (  4.2%) 
   Incisional drainage                                                     2 (  8.3%) 
   Leg amputation                                                          2 (  8.3%) 
   Office visit                                                            1 (  4.2%) 
   Skin graft                                                              1 (  4.2%) 
   Specialist consultation                                                 1 (  4.2%) 
   Vascular operation                                                      1 (  4.2%) 
 
Vascular disorders                                                         4 ( 16.7%) 
   Deep vein thrombosis                                                    1 (  4.2%) 
   Dry gangrene                                                            1 (  4.2%) 
   Haematoma                                                               1 (  4.2%) 
   Peripheral arterial occlusive disease                                   1 (  4.2%) 
   Peripheral ischaemia                                                    1 (  4.2%) 
 
 
Lifecells LLC, Protocol ASCT01 
 Table 4b: Treatment Emergent Serious Adverse Events (TESAE) by MedDRA Primary System Organ Class and 
Preferred Term 
                                                        Analysis Set: All Subjects 
 
________________________________________________________________________________________________________ 
System Organ Class                                                      Autologous Stem Cell Transplant 

   
        
Final Study Summary Report An open-label single arm phase 2 proof of concept study to assess the efficacy 
and safety of ASCT01 in Patients with Critical Limb Ischemia 
 
28 
 
01 
   Preferred Term                                                               (N=24) 
________________________________________________________________________________________________________ 
 
Subjects with TESAEs                                                      12 ( 50.0%) 
 
Total Number of TESAEs                                                    53 
 
Blood and lymphatic system disorders                                       2 (  8.3%) 
   Anaemia                                                                 2 (  8.3%) 
 
Cardiac disorders                                                          1 (  4.2%) 
   Angina pectoris                                                         1 (  4.2%) 
 
Gastrointestinal disorders                                                 1 (  4.2%) 
   Abdominal pain                                                          1 (  4.2%) 
 
General disorders and administration site conditions                       1 (  4.2%) 
   Pain                                                                    1 (  4.2%) 
 
Infections and infestations                                                7 ( 29.2%) 
   Bursitis infective                                                      1 (  4.2%) 
   Cellulitis                                                              3 ( 12.5%) 
   Clostridium difficile colitis                                           2 (  8.3%) 
   Localised infection                                                     1 (  4.2%) 
   Osteomyelitis                                                           1 (  4.2%) 
   Pneumonia                                                               2 (  8.3%) 
   Postoperative wound infection                                           1 (  4.2%) 
   Sepsis                                                                  1 (  4.2%) 
   Urinary tract infection                                                 1 (  4.2%) 
   Wound infection                                                         1 (  4.2%) 
 
 
Injury, poisoning and procedural complications                             4 ( 16.7%) 
   Hip fracture                                                            1 (  4.2%) 
   Limb injury                                                             1 (  4.2%) 
   Post procedural complication                                            2 (  8.3%) 
   Post procedural haemorrhage                                             1 (  4.2%) 
   Postoperative wound complication                                        1 (  4.2%) 
   Wound dehiscence                                                        1 (  4.2%) 
 
Investigations                                                             1 (  4.2%) 
   International normalised ratio increased                                1 (  4.2%) 
 
Musculoskeletal and connective tissue disorders                            2 (  8.3%) 
   Pain in extremity                                                       2 (  8.3%) 
 
Psychiatric disorders                                                      1 (  4.2%) 
   Mental status changes                                                   1 (  4.2%) 
 
Renal and urinary disorders                                                1 (  4.2%) 
   Haematuria                                                              1 (  4.2%) 

   
        
Final Study Summary Report An open-label single arm phase 2 proof of concept study to assess the efficacy 
and safety of ASCT01 in Patients with Critical Limb Ischemia 
 
29 
 
 
Respiratory, thoracic and mediastinal disorders                            2 (  8.3%) 
   Dyspnoea                                                                1 (  4.2%) 
   Respiratory failure                                                     1 (  4.2%) 
 
Skin and subcutaneous tissue disorders                                     3 ( 12.5%) 
   Skin ulcer                                                              3 ( 12.5%) 
 
Surgical and medical procedures                                            5 ( 20.8%) 
 
 
Surgical and medical procedures (Continued) 
   Debridement                                                             1 (  4.2%) 
   Incisional drainage                                                     1 (  4.2%) 
   Leg amputation                                                          2 (  8.3%) 
   Skin graft                                                              1 (  4.2%) 
   Vascular operation                                                      1 (  4.2%) 
 
Vascular disorders                                                         3 ( 12.5%) 
   Deep vein thrombosis                                                    1 (  4.2%) 
   Dry gangrene                                                            1 (  4.2%) 
   Peripheral arterial occlusive disease                                   1 (  4.2%) 
   Peripheral ischaemia                                                    1 (  4.2%) 
 
Lifecells LLC, Protocol ASCT01 
 Table 4c: Treatment Emergent Serious Adverse Events (TESAE) by MedDRA Primary System Organ Class and 
Preferred Term 
Analysis Set: All Subjects 
 
________________________________________________________________________________________________________ 
System Organ Class                                                      Autologous Stem Cell Transplant 
01 
   Preferred Term                                                                     (N=24) 
________________________________________________________________________________________________________ 
Subjects with TESAEs                                                      12 ( 50.0%) 
 
Total Number of TESAEs                                                    53 
 
Blood and lymphatic system disorders                                       2 (  8.3%) 
   Anaemia                                                                 2 (  8.3%) 
 
Cardiac disorders                                                          1 (  4.2%) 
   Angina pectoris                                                         1 (  4.2%) 
 
Gastrointestinal disorders                                                 1 (  4.2%) 
   Abdominal pain                                                          1 (  4.2%) 
 
General disorders and administration site conditions                       1 (  4.2%) 
   Pain                                                                    1 (  4.2%) 
 
Infections and infestations                                                7 ( 29.2%) 
   Bursitis infective                                                      1 (  4.2%) 
   Cellulitis                                                              3 ( 12.5%) 

   
        
Final Study Summary Report An open-label single arm phase 2 proof of concept study to assess the efficacy 
and safety of ASCT01 in Patients with Critical Limb Ischemia 
 
30 
 
   Clostridium difficile colitis                                           2 (  8.3%) 
   Localised infection                                                     1 (  4.2%) 
   Osteomyelitis                                                           1 (  4.2%) 
   Pneumonia                                                               2 (  8.3%) 
   Postoperative wound infection                                           1 (  4.2%) 
   Sepsis                                                                  1 (  4.2%) 
   Urinary tract infection                                                 1 (  4.2%) 
   Wound infection                                                         1 (  4.2%) 
 
 
Injury, poisoning and procedural complications                             4 ( 16.7%) 
   Hip fracture                                                            1 (  4.2%) 
   Limb injury                                                             1 (  4.2%) 
   Post procedural complication                                            2 (  8.3%) 
   Post procedural haemorrhage                                             1 (  4.2%) 
   Postoperative wound complication                                        1 (  4.2%) 
   Wound dehiscence                                                        1 (  4.2%) 
 
Investigations                                                             1 (  4.2%) 
   International normalised ratio increased                                1 (  4.2%) 
 
Musculoskeletal and connective tissue disorders                            2 (  8.3%) 
   Pain in extremity                                                       2 (  8.3%) 
 
Psychiatric disorders                                                      1 (  4.2%) 
   Mental status changes                                                   1 (  4.2%) 
 
Renal and urinary disorders                                                1 (  4.2%) 
   Haematuria                                                              1 (  4.2%) 
 
Respiratory, thoracic and mediastinal disorders                            2 (  8.3%) 
   Dyspnoea                                                                1 (  4.2%) 
   Respiratory failure                                                     1 (  4.2%) 
 
Skin and subcutaneous tissue disorders                                     3 ( 12.5%) 
   Skin ulcer                                                              3 ( 12.5%) 
 
Surgical and medical procedures                                            5 ( 20.8%) 
 
Surgical and medical procedures (Continued) 
   Debridement                                                             1 (  4.2%) 
   Incisional drainage                                                     1 (  4.2%) 
   Leg amputation                                                          2 (  8.3%) 
   Skin graft                                                              1 (  4.2%) 
   Vascular operation                                                      1 (  4.2%) 
 
Vascular disorders                                                         3 ( 12.5%) 
   Deep vein thrombosis                                                    1 (  4.2%) 
   Dry gangrene                                                            1 (  4.2%) 
   Peripheral arterial occlusive disease                                   1 (  4.2%) 
   Peripheral ischaemia                                                    1 (  4.2%) 
 

   
        
Final Study Summary Report An open-label single arm phase 2 proof of concept study to assess the efficacy 
and safety of ASCT01 in Patients with Critical Limb Ischemia 
 
31 
 
 
Table 5:  Major and Minor Amputations Entire Study Amputations after Dosing 
 
Subject ID 
Age 
(yrs) 
Rutherford 
Scale on 
Enrolled Day 
Date of Dosing 
Date of 
Amputation 
Type of 
Amputation 
(Major/Min
or) 
Remarks 
Time 
from 
dosing 
(Days)  
Primary 
objective 
effect 
KCV2-001 
81 
5 
02/20/14 
8/19/2014 
Minor 
Left trans-metatarsal 
amputation (TMA) 
180 
None 
KCV2-002 
54 
5 
03/20/14 
1/31/2015 
Minor 
Right great toe 
amputation 
317 
None 
KCV2-002 
 
 
 
4/22/2014 
Major 
LBKA 
33 
Fail 
KCV2-003 
50 
4 
04/03/14 
3/1/2015 
Major 
LBKA* 
332 
None 
KCV2-004 
59 
5 
04/24/14 
6/19/2014 
Major 
LBKA 
56 
Fail 
KCV2-006 
50 
5 
06/05/14 
10/30/2014 
Major 
LBKA 
147 
None 
KCV2-017 
72 
4 
05/20/15 
8/26/2015 
Major 
LBKA 
98 
None 
KCV2-020 
68 
4 
09/30/15 
4/6/2016 
Major 
LBKA 
189 
None 
KCV2-021 
66 
5 
10/07/15 
3/9/2016 
Minor 
Right trans-
metatarsal 
amputation (TMA) 
154 
None 
 
PIs Note: Per Dr. Stark the PI, in some AKA (Above Knee Amputation) was prevented –due to increased vascularity in the limb area 
 
 * LBKA: Left Below Knee Amputation 
Table 6: ASCT01 Stem Cell Product Information - patients KCV2-001 to KCV2-025 
Patie
nt ID 
Bone 
Marrow 
Aspirated 
Volume mL 
(iliac 
Crest both 
sides) 
Conc. 
ASCT01 
Stem cell 
Processed 
by 
Lifecells 
ASCT01 
Final 
Volume at 
10 million 
cells/mL 
Prepared 
by 
Lifecells 
Administered 
by IA 
catheter 
infusion at 
5 ml/minute 
with Braun 
metered 
infusion 
pump-80%  
Administered by 
IM injections 
along the 
neurovascular 
bundle - 20% -6 
to 40 injections 
divided into 
equal volumes in 
OR 
Total 
CD45+
Cells 
Billi
ons 
CD45+ 
Cells/
mL 
Millio
ns 
Total 
CD34+Ce
llsMill
ion 
CD45+Ce
ll 
Viabili
ty 
KCV2-
001 
220 
3.5 
137 
110 
27 
2.5 
18.4 
42.7 
76 
KCV2-
002 
223 
6.0 
175 
143 
32 
2.0 
11.2 
24.2 
90.2 

   
        
Final Study Summary Report An open-label single arm phase 2 proof of concept study to assess the efficacy 
and safety of ASCT01 in Patients with Critical Limb Ischemia 
 
32 
 
KCV2-
003 
207 
5.0 
121 
96 
25 
1.2 
9.8 
13.1 
88.3 
KCV2-
004 
203 
3.0 
264 
209 
55 
1.7 
6.3 
12 
89.4 
KCV2-
005 
211 
6.3 
562 
452 
110 
4.3 
7.7 
156.7 
76.1 
KCV2-
006 
231 
4.0 
81 
65 
16 
0.8 
9.3 
23.4 
78.4 
KCV2-
007 
200 
6.0 
384 
306 
78 
2.4 
6.4 
33.3 
74.6 
KCV2-
008 
200 
4.8 
165 
130 
35 
1.4 
8.7 
50.2 
74.9 
KCV2-
009 
217 
5.0 
480 
383 
97 
3.7 
7.6 
31.9 
78.4 
KCV2-
010 
161 
2.8 
68 
54 
14 
0.4 
6.6 
9.4 
69.3 
KCV2-
011 
202 
2.3 
69 
55 
14 
0.7 
10.6 
6.7 
88.5 
KCV2-
012 
239 
4.5 
164 
131 
33 
1.6 
9.7 
22 
95 
KCV2-
013 
209 
2.5 
88 
70 
18 
0.9 
10.4 
13.8 
97.4 
KCV2-
014 
203 
3.4 
137 
108.5 
28.5 
1.4 
10.4 
19.5 
93.3 
KCV2-
015 
218 
2.5 
154 
123 
31 
1.3 
8.7 
21.9 
92.5 
KCV2-
016 
244 
4.0 
237 
189 
48 
2.2 
9.4 
31.8 
97.2 
KCV2-
017 
235 
3.0 
126 
100 
26 
1.2 
9.5 
7.7 
94.8 
KCV2-
018 
264 
2.0 
84 
68 
16 
0.5 
5.4 
6.3 
96 
KCV2-
019 
247 
1.0 
47.6 
38 
9.5 
0.3 
7.1 
4.4 
96 

   
        
Final Study Summary Report An open-label single arm phase 2 proof of concept study to assess the efficacy 
and safety of ASCT01 in Patients with Critical Limb Ischemia 
 
33 
 
KCV2-
020 
210 
2.5 
117.5 
94 
23.5 
0.4 
3.8 
5 
89.4 
KCV2-
021 
223 
3.3 
160 
128 
32 
1.8 
11.1 
15.9 
98.3 
KCV2-
023 
227 
2.8 
183.5 
146 
37.5 
1.9 
10.5 
29.6 
97.3 
KCV2-
024 
224 
2.0 
88 
70 
18 
1.0 
11.6 
8.9 
96.7 
KCV2-
025 
195 
3.0 
156.5 
125 
31.5 
1.6 
10.4 
14.3 
95 
 Rang
e 
161 to 
264 
1 to 
6.25 
47.6 to 
562 
38 to 452 
9.5 to 110 
0.3 
to 
4.3 
3.8 
to 
18.4 
4.4 
to 
156.7 
69.3 
to 
98.3 
 
 
 
Conclusion 
The 12-month follow-up has shown that the test treatment was 
safe and well tolerated by all the study patients administered 
as single treatment. Efficacy outcome of over 50% was 
observed in this CLI study. On this basis, the sponsor intends 
to complete a pivotal phase 3 study after review and discussion 
with the Agency 
 
DATE OF THE REPORT August 4, 2017