Clinical trial NCT04280718: A Study to Assess the Long-term Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod in Adults With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP, an Autoimmune Disorder That Affects the Peripheral Nerves)
Official title: Open-label Extension of the ARGX-113-1802 Trial to Investigate the Long-term Safety, Tolerability, and Efficacy of Efgartigimod PH20 SC in Patients With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
Conditions: Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
Lead sponsor: argenx
Sponsor class: INDUSTRY
First posted: 2020-02-21
Enrollment: 229
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This is the open-label extension study of phase II ARGX-113-1802 to evaluate the long-term safety and efficacy of the subcutaneous formulation of efgartigimod in adults with CIDP.

Patients already stabilized on efgartigimod PH20 SC will also have the opportunity to participate in a sub study to explore less frequent dosing of efgartigimod PH20 SC.


Eligibility criteria: Inclusion Criteria:

1. Ability to understand the requirements of the trial, provide written informed consent (including consent for the use and disclosure of research-related health information), willingness and ability to comply with the trial protocol procedures (including required trial visits) of this trial.
2. Male or female patient with one of the following options:

   * Have completed the Week-48 visit of Stage B of the ARGX-113-1802 trial and are considered to be eligible for treatment with efgartigimod PH20 SC; or
   * Have deteriorated during Stage B of the ARGX-113-1802 trial and are considered to be eligible for treatment with efgartigimod PH20 SC, or
   * Have been offered the participation in the OLE trial due to early termination of the ARGX-113-1802 trial (because sufficient events for the primary endpoint analysis of the that trial have been reached and it is stopped) and are considered to be eligible for treatment with efgartigimod PH20 SC treatment; or
   * Have completed the Week-48 visit of the previous cycle of the OLE trial and are considered to be eligible to continue with efgartigimod PH20 SC treatment.
3. Women of childbearing potential who have a negative urine pregnancy test at baseline before IMP administration.
4. Women of childbearing potential must use an acceptable method of contraception from signing the ICF until the date of the last administration of IMP.

Exclusion Criteria:

1. Week-48/ED visit in the ARGX-113-1802 trial or the Week-48 visit of the previous OLE participation occurred more than 14 days prior to SD1 of the OLE trial or the start of a new treatment cycle in the OLE trial and more than 21 days since the last dose of IMP.
2. Pregnant and lactating women and those intending to become pregnant during the trial.
3. Patients with clinical evidence of other significant serious disease or patients who underwent a recent or have a planned major surgery, or patients who (intend to) use prohibited medications (see protocol) and therapies during the trial, or any other reason which could confound the results of the trial or put the patient at undue risk.

Primary outcomes:
measure: Incidence of treatment-emergent adverse events and serious adverse events
timeFrame: Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study
Secondary outcomes:
measure: Change from baseline over time of the adjusted INCAT score
timeFrame: Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study
measure: Change from baseline over time of the MRC Sum score
timeFrame: Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study
measure: Change from baseline over time of I-RODS disability scores
timeFrame: Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study
measure: Change from baseline over time of mean grip strength
timeFrame: Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study
measure: Change from baseline over time of TUG score
timeFrame: Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study
measure: Percentage of patients without clinical deterioration over time, defined by adjusted INCAT deterioration ≥1 point compared to baseline.
timeFrame: Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study
measure: Percentage of patients with titers of binding antibodies towards efgartigimod and the presence of neutralizing antibodies against efgartigimod.
timeFrame: Up to 51 weeks
measure: Efgartigimod serum concentrations
timeFrame: Up to 51 weeks
measure: Changes from baseline over time of serum IgG levels (total)
timeFrame: Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study
measure: Change from baseline over time in EQ-5D-5L
timeFrame: Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study
measure: Change from baseline over time in BPI SF
timeFrame: Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study
measure: Change from baseline over time in TSQM-9
timeFrame: Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study
measure: Change from baseline over time in RT-FSS
timeFrame: Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study
measure: Change from baseline over time in HADS
timeFrame: Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study
measure: Percentage of patients performing self-administration over time
timeFrame: Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study
measure: Percentage of patients with treatment administered by caregiver over time.
timeFrame: Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Investigator site 0010065
state: Alabama
city: Phoenix
country: United States
facility: Investigator site 0010013
state: Arizona
city: Scottsdale
country: United States
facility: Investigator site 0010055
state: Arizona
city: Carlsbad
country: United States
facility: Investigator site 0010032
state: California
city: Pomona
country: United States
facility: Investigator site 10190
state: California
city: Rancho Mirage
country: United States
facility: Investigator site 0010160
state: California
city: San Francisco
country: United States
facility: Investigator site 0010071
state: California
city: Centennial
country: United States
facility: Investigator site 0010057
state: Colorado
city: Boca Raton
country: United States
facility: Investigator site 0010072
state: Florida
city: Coral Springs
country: United States
facility: Investigator site 0010144
state: Florida
city: Jacksonville
country: United States
facility: Investigator site 0010023
state: Florida
city: Maitland
country: United States
facility: Investigator site 0010068
state: Florida
city: Miami
country: United States
facility: Investigator site 0010059
state: Florida
city: Tampa
country: United States
facility: Investigator site 0010006
state: Florida
city: Iowa City
country: United States
facility: Investigator site 0010011
state: Iowa
city: Fairway
country: United States
facility: Investigator site 0010015
state: Kansas
city: Lexington
country: United States
facility: Investigator site 10147
state: Kentucky
city: New York
country: United States
facility: Investigator site 10168
state: New York
city: Chapel Hill
country: United States
facility: Investigator site 0010003
state: North Carolina
city: Columbus
country: United States
facility: Investigator site 0010064
state: Ohio
city: Philadelphia
country: United States
facility: Investigator site 0010007
state: Pennsylvania
city: Philadelphia
country: United States
facility: Investigator site 0010047
state: Pennsylvania
city: Austin
country: United States
facility: Investigator Site 0010066
state: Texas
city: San Antonio
country: United States
facility: Investigator site 0010009
state: Texas
city: Richmond
country: United States
facility: Investigator site 0010061
state: Virginia
city: Innsbruck
country: Austria
facility: Investigator site 0430007
city: Linz
country: Austria
facility: Investigator site 0430008
city: Wien
country: Austria
facility: Investigator site 0430005
city: Edegem
country: Belgium
facility: Investigator site 0320016
city: Leuven
country: Belgium
facility: Investigator site 0320009
city: Liège
country: Belgium
facility: Investigator site 320024
city: Woluwe-Saint-Lambert
country: Belgium
facility: Investigator site 320022
city: Pleven
country: Bulgaria
facility: Investigator site 3590007
city: Sofia
country: Bulgaria
facility: Investigator site 3590005
city: Sofia
country: Bulgaria
facility: Investigator site 3590008
city: Sofia
country: Bulgaria
facility: Investigator site 3590006
city: Beijing
country: China
facility: Investigator site 0860033
city: Changsha
country: China
facility: Investigator site 860041
city: Chengdu
country: China
facility: Investigator site 0860036
city: Chifeng
country: China
facility: Investigator site 860049
city: Fuzhou
country: China
facility: Investigator site 0860038
city: Guangzhou
country: China
facility: Investigator site 0860050
city: Guanzhou
country: China
facility: Investigator site 0860032
city: Guiyang
country: China
facility: Investigator site 0860045
city: Hangzhou
country: China
facility: Investigator site 0860035
city: Jinan
country: China
facility: Investigator site 0860031
city: Nanchang
country: China
facility: Investigator site 0860040
city: Nanjing
country: China
facility: Investigator site 0860043
city: Shanghai
country: China
facility: Investigator site 0860028
city: Shanghai
country: China
facility: Investigator site 860047
city: Tianjin
country: China
facility: Investigator site 0860042
city: Wuhan
country: China
facility: Investigator site 0860034
city: Wuhan
country: China
facility: Investigator site 0860029
city: Xi'an
country: China
facility: Investigator site 0860048
city: Xianyang
country: China
facility: Investigator site 0860054
city: Hradec Králové
country: Czechia
facility: Investigator site 4200010
city: Aarhus
country: Denmark
facility: Investigator site 0450002
city: Copenhagen
country: Denmark
facility: Investigator site 0450001
city: Odense
country: Denmark
facility: Investigator site 0450003
city: Angers
country: France
facility: Investigator site 00330034
city: Bordeaux
country: France
facility: Investigator site 0330013
city: Clermont-Ferrand
country: France
facility: Investigator site 330033
city: Le Kremlin-Bicêtre
country: France
facility: Investigator site 0330023
city: Limoges
country: France
facility: Investigator site 0330024
city: Nantes
country: France
facility: Investigator site 330022
city: Nice
country: France
facility: Investigator site 0330021
city: Paris
country: France
facility: Investigator site 0330035
city: Strasbourg
country: France
facility: Investigator site 0330020
city: Kutaisi
country: Georgia
facility: Investigator site 9950020
city: Tbilisi
country: Georgia
facility: Investigator site 9950004
city: Tbilisi
country: Georgia
facility: Investigator site 9950005
city: Tbilisi
country: Georgia
facility: Investigator site 9950002
city: Tbilisi
country: Georgia
facility: Investigator Site 9950003
city: Bochum
country: Germany
facility: Investigator site 490044
city: Essen
country: Germany
facility: Investigator site 490045
city: Göttingen
country: Germany
facility: Investigator site 490021
city: Kiel
country: Germany
facility: Investigator site 0490016
city: Köln
country: Germany
facility: Investigator site 0490013
city: Potsdam
country: Germany
facility: Investigator site 0490019
city: Tel Aviv
country: Israel
facility: Investigator site 9720004
city: Brescia
country: Italy
facility: Investigator site 0390022
city: Firenze
country: Italy
facility: Investigator site 390029
city: Genova
country: Italy
facility: Investigator site 0390024
city: Messina
country: Italy
facility: Investigator site 390027
city: Milano
country: Italy
facility: Investigator site 0390026
city: Milan
country: Italy
facility: Investigator site 0390003
city: Pisa
country: Italy
facility: Investigator site 0390023
city: Roma
country: Italy
facility: Investigator site 0390008
city: Torino
country: Italy
facility: Investigator site 0390042
city: Bunkyō-Ku
country: Japan
facility: Investigator site 0810035
city: Chiba
country: Japan
facility: Investigator site 0810002
city: Fuchū
country: Japan
facility: Investigator site 0810030
city: Fukuoka
country: Japan
facility: Investigator site 0810031
city: Ginowan
country: Japan
facility: Investigator site 0810065
city: Hakodate
country: Japan
facility: Investigator site 0810066
city: Hiroshima
country: Japan
facility: Investigator site 0810058
city: Itabashi
country: Japan
facility: Investigator site 0810036
city: Kawagoe
country: Japan
facility: Investigator site 0810029
city: Kodaira
country: Japan
facility: Investigator site 0810026
city: Kyoto-shi
country: Japan
facility: Investigator site 810061
city: Mibu
country: Japan
facility: Investigator site 0810027
city: Nagoya
country: Japan
facility: Investigator site 0810032
city: Osaka
country: Japan
facility: Investigator site 0810003
city: Osaka
country: Japan
facility: Investigator site 0810007
city: Suita
country: Japan
facility: Investigator site 0810063
city: Tokushima
country: Japan
facility: Investigator site 0810064
city: Yokohama
country: Japan
facility: Investigator site 0810060
city: Riga
country: Latvia
facility: Investigator site 31
city: Amsterdam
country: Netherlands
facility: Investigator site 0310010
city: Rotterdam
country: Netherlands
facility: Investigator site 0310011
city: Kraków
country: Poland
facility: Investigator site 0480024
city: Kraków
country: Poland
facility: Investigator site 0480018
city: Lublin
country: Poland
facility: Investigator Site 0480017
city: Warszawa
country: Poland
facility: Investigator site 0480022
city: Łódź
country: Poland
facility: Investigator site 0480020
city: Braşov
country: Romania
facility: Investigator site 040002
city: Bucharest
country: Romania
facility: Investigator site 040001
city: Constanţa
country: Romania
facility: Investigator site 040004
city: Timişoara
country: Romania
facility: Investigator site 040003
city: Kazan
country: Russian Federation
facility: Investigator site 070017
city: Kazan
country: Russian Federation
facility: Investigator site 0070023
city: Moscow
country: Russian Federation
facility: Investigator site 0070020
city: Moscow
country: Russian Federation
facility: Investigator site 70021
city: Rostov-on-Don
country: Russian Federation
facility: Investigator site 0070019
city: Saint Petersburg
country: Russian Federation
facility: Investigator site 0070014
city: Saransk
country: Russian Federation
facility: Investigator site 0070021
city: Belgrade
country: Serbia
facility: Investigator site 3810001
city: Belgrade
country: Serbia
facility: Investigator site 3810003
city: Kragujevac
country: Serbia
facility: Investigator site 3810004
city: Badalona
country: Spain
facility: Investigator site 0340021
city: Barcelona
country: Spain
facility: Investigator site 0340038
city: Madrid
country: Spain
facility: Investigator site 0340018
city: Tainan
country: Taiwan
facility: Investigator site 8860013
city: Taipei
country: Taiwan
facility: Investigator site 8860012
city: Taipei
country: Taiwan
facility: Investigator site 8860016
city: Taoyuan
country: Taiwan
facility: Investigator site 8860017
city: Bursa
country: Turkey
facility: Investigator site 900025
city: İzmir
country: Turkey
facility: Investigator site 900021
city: Samsun
country: Turkey
facility: Investigator site 900022
city: Dnipro
country: Ukraine
facility: Investigator Site 3800012
city: Ivano-Frankivs'k
country: Ukraine
facility: Investigator Site 3800010
city: Kyiv
country: Ukraine
facility: Investigator site 3100013
city: Luts'k
country: Ukraine
facility: Investigator site 3800008
city: Zaporizhzhya
country: Ukraine
facility: Investigator site 3800011
city: London
country: United Kingdom
facility: Investigator site 440026
city: Oxford
country: United Kingdom
facility: Investigator site 0440016
city: Sheffield
country: United Kingdom
facility: Investigator site 0440018
Study officials:
References: