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)

Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) Clinical Trial

— ADHERE+  

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)

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04280718
• Other study ID #: ARGX-113-1902
• Secondary ID: 2019-003107-35
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: September 18, 2020
• Est. completion date: March 1, 2027

Study information

• Verified date: August 2023
• Source: argenx
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 226
• Est. completion date: March 1, 2027
• Est. primary completion date: March 1, 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
• Polyneuropathies
• Polyradiculoneuropathy, Chronic Inflammatory Demyelinating

Intervention

Biological:
• Efgartigimod PH20 SC
Subcutaneous administration of efgartigimod

Locations

Country	Name	City	State
Austria	Investigator site 0430007	Innsbruck
Austria	Investigator site 0430008	Linz
Austria	Investigator site 0430005	Wien
Belgium	Investigator site 0320016	Edegem
Belgium	Investigator site 0320009	Leuven
Belgium	Investigator site 320024	Liège
Belgium	Investigator site 320022	Woluwe-Saint-Lambert
Bulgaria	Investigator site 3590007	Pleven
Bulgaria	Investigator site 3590005	Sofia
Bulgaria	Investigator site 3590006	Sofia
Bulgaria	Investigator site 3590008	Sofia
China	Investigator site 0860033	Beijing
China	Investigator site 860041	Changsha
China	Investigator site 0860036	Chengdu
China	Investigator site 860049	Chifeng
China	Investigator site 0860038	Fuzhou
China	Investigator site 0860050	Guangzhou
China	Investigator site 0860032	Guanzhou
China	Investigator site 0860045	Guiyang
China	Investigator site 0860035	Hangzhou
China	Investigator site 860035	Hangzhou
China	Investigator site 0860031	Jinan
China	Investigator site 0860040	Nanchang
China	Investigator site 0860043	Nanjing
China	Investigator site 0860028	Shanghai
China	Investigator site 860047	Shanghai
China	Investigator site 0860042	Tianjin
China	Investigator site 0860029	Wuhan
China	Investigator site 0860034	Wuhan
China	Investigator site 0860048	Xi'an
China	Investigator site 0860054	Xianyang
Czechia	Investigator site 4200010	Hradec Králové
Denmark	Investigator site 0450002	Aarhus
Denmark	Investigator site 0450001	Copenhagen
Denmark	Investigator site 0450003	Odense
France	Investigator site 00330034	Angers
France	Investigator site 0330013	Bordeaux
France	Investigator site 330033	Clermont-Ferrand
France	Investigator site 0330023	Le Kremlin-Bicêtre
France	Investigator site 0330024	Limoges
France	Investigator site 330022	Nantes
France	Investigator site 0330021	Nice
France	Investigator site 0330035	Paris
France	Investigator site 0330020	Strasbourg
Georgia	Investigator site 9950020	Kutaisi
Georgia	Investigator site 9950002	Tbilisi
Georgia	Investigator Site 9950003	Tbilisi
Georgia	Investigator site 9950004	Tbilisi
Georgia	Investigator site 9950005	Tbilisi
Germany	Investigator site 490044	Bochum
Germany	Investigator site 490045	Essen
Germany	Investigator site 490021	Göttingen
Germany	Investigator site 0490016	Kiel
Germany	Investigator site 0490013	Köln
Germany	Investigator site 0490019	Potsdam
Israel	Investigator site 9720004	Tel Aviv
Italy	Investigator site 0390022	Brescia
Italy	Investigator site 390029	Firenze
Italy	Investigator site 0390024	Genova
Italy	Investigator site 390027	Messina
Italy	Investigator site 0390003	Milan
Italy	Investigator site 0390026	Milano
Italy	Investigator site 0390023	Pisa
Italy	Investigator site 0390008	Roma
Italy	Investigator site 0390042	Torino
Japan	Investigator site 0810035	Bunkyo-Ku
Japan	Investigator site 0810002	Chiba
Japan	Investigator site 0810030	Fuchu
Japan	Investigator site 0810031	Fukuoka
Japan	Investigator site 0810065	Ginowan
Japan	Investigator site 0810066	Hakodate
Japan	Investigator site 0810058	Hiroshima
Japan	Investigator site 0810036	Itabashi
Japan	Investigator site 0810029	Kawagoe
Japan	Investigator site 0810026	Kodaira
Japan	Investigator site 810061	Kyoto-shi
Japan	Investigator site 0810027	Mibu
Japan	Investigator site 0810032	Nagoya
Japan	Investigator site 0810003	Osaka
Japan	Investigator site 0810007	Osaka
Japan	Investigator site 0810063	Suita
Japan	Investigator site 0810064	Tokushima
Japan	Investigator site 0810060	Yokohama
Latvia	Investigator site 31	Riga
Netherlands	Investigator site 0310010	Amsterdam
Netherlands	Investigator site 0310011	Rotterdam
Poland	Investigator site 0480018	Kraków
Poland	Investigator site 0480024	Kraków
Poland	Investigator site 0480020	Lódz
Poland	Investigator Site 0480017	Lublin
Poland	Investigator site 0480022	Warszawa
Romania	Investigator site 040002	Brasov
Romania	Investigator site 040001	Bucharest
Romania	Investigator site 040004	Constanta
Romania	Investigator site 040003	Timisoara
Russian Federation	Investigator site 0070023	Kazan
Russian Federation	Investigator site 070017	Kazan
Russian Federation	Investigator site 0070020	Moscow
Russian Federation	Investigator site 70021	Moscow
Russian Federation	Investigator site 0070019	Rostov-on-Don
Russian Federation	Investigator site 0070014	Saint Petersburg
Russian Federation	Investigator site 0070021	Saransk
Serbia	Investigator site 3810001	Belgrade
Serbia	Investigator site 3810003	Belgrade
Serbia	Investigator site 3810004	Kragujevac
Spain	Investigator site 0340021	Badalona
Spain	Investigator site 0340038	Barcelona
Spain	Investigator site 0340018	Madrid
Taiwan	Investigator site 8860013	Tainan
Taiwan	Investigator site 8860012	Taipei
Taiwan	Investigator site 8860016	Taipei
Taiwan	Investigator site 8860017	Taoyuan
Turkey	Investigator site 900025	Bursa
Turkey	Investigator site 900021	Izmir
Turkey	Investigator site 900022	Samsun
Ukraine	Investigator Site 3800012	Dnipro
Ukraine	Investigator Site 3800010	Ivano-Frankivs'k
Ukraine	Investigator site 3100013	Kyiv
Ukraine	Investigator site 3800008	Luts'k
Ukraine	Investigator site 3800011	Zaporizhzhya
United Kingdom	Investigator site 440026	London
United Kingdom	Investigator site 0440016	Oxford
United Kingdom	Investigator site 0440018	Sheffield
United States	Investigator Site 0010066	Austin	Texas
United States	Investigator site 0010065	Birmingham	Alabama
United States	Investigator site 0010072	Boca Raton	Florida
United States	Investigator site 0010032	Carlsbad	California
United States	Investigator site 0010057	Centennial	Colorado
United States	Investigator site 0010003	Chapel Hill	North Carolina
United States	Investigator site 0010064	Columbus	Ohio
United States	Investigator site 0010144	Coral Springs	Florida
United States	Investigator site 0010015	Fairway	Kansas
United States	Investigator site 0010011	Iowa City	Iowa
United States	Investigator site 0010023	Jacksonville	Florida
United States	Investigator site 10147	Lexington	Kentucky
United States	Investigator site 0010068	Maitland	Florida
United States	Investigator site 0010059	Miami	Florida
United States	Investigator site 10168	New York	New York
United States	Investigator site 0010007	Philadelphia	Pennsylvania
United States	Investigator site 0010047	Philadelphia	Pennsylvania
United States	Investigator site 0010013	Phoenix	Arizona
United States	Investigator site 10190	Pomona	California
United States	Investigator site 0010160	Rancho Mirage	California
United States	Investigator site 0010061	Richmond	Virginia
United States	Investigator site 0010009	San Antonio	Texas
United States	Investigator site 0010071	San Francisco	California
United States	Investigator site 0010055	Scottsdale	Arizona
United States	Investigator site 0010006	Tampa	Florida

Sponsors (1)

Lead Sponsor	Collaborator
argenx

Countries where clinical trial is conducted

United States,  Austria,  Belgium,  Bulgaria,  China,  Czechia,  Denmark,  France,  Georgia,  Germany,  Israel,  Italy,  Japan,  Latvia,  Netherlands,  Poland,  Romania,  Russian Federation,  Serbia,  Spain,  Taiwan,  Turkey,  Ukraine,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of treatment-emergent adverse events and serious adverse events		Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study
Secondary	Change from baseline over time of the adjusted INCAT score		Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study
Secondary	Change from baseline over time of the MRC Sum score		Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study
Secondary	Change from baseline over time of I-RODS disability scores		Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study
Secondary	Change from baseline over time of mean grip strength		Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study
Secondary	Change from baseline over time of TUG score		Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study
Secondary	Percentage of patients without clinical deterioration over time, defined by adjusted INCAT deterioration =1 point compared to baseline.		Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study
Secondary	Percentage of patients with titers of binding antibodies towards efgartigimod and the presence of neutralizing antibodies against efgartigimod.		Up to 51 weeks
Secondary	Efgartigimod serum concentrations		Up to 51 weeks
Secondary	Changes from baseline over time of serum IgG levels (total)		Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study
Secondary	Change from baseline over time in EQ-5D-5L		Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study
Secondary	Change from baseline over time in BPI SF		Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study
Secondary	Change from baseline over time in TSQM-9		Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study
Secondary	Change from baseline over time in RT-FSS		Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study
Secondary	Change from baseline over time in HADS		Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study
Secondary	Percentage of patients performing self-administration over time		Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study
Secondary	Percentage of patients with treatment administered by caregiver over time.		Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study