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)
- Conditions
- Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
- Interventions
- Biological: Efgartigimod PH20 SC
- Registration Number
- NCT04280718
- Lead Sponsor
- argenx
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 229
-
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.
-
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.
-
Women of childbearing potential who have a negative urine pregnancy test at baseline before IMP administration.
-
Women of childbearing potential must use an acceptable method of contraception from signing the ICF until the date of the last administration of IMP.
- 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.
- Pregnant and lactating women and those intending to become pregnant during the trial.
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description efgartigimod PH20 SC Efgartigimod PH20 SC Patients treated with efgartigimod PH20 SC
- Primary Outcome Measures
Name Time Method 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 Outcome Measures
Name Time Method 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 Efgartigimod serum concentrations Up to 51 weeks 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 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 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 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 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 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 Percentage of patients with titers of binding antibodies towards efgartigimod and the presence of neutralizing antibodies against efgartigimod. Up to 51 weeks 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 Change from baseline over time in HADS Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study 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 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 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 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 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
Trial Locations
- Locations (147)
Investigator site 0070021
🇷🇺Saransk, Russian Federation
Investigator Site 3800010
🇺🇦Ivano-Frankivs'k, Ukraine
Investigator site 0860036
🇨🇳Chengdu, China
Investigator site 0860032
🇨🇳Guanzhou, China
Investigator site 0860040
🇨🇳Nanchang, China
Investigator site 860049
🇨🇳Chifeng, China
Investigator site 0320016
🇧🇪Edegem, Belgium
Investigator site 0480020
🇵🇱Łódź, Poland
Investigator site 320024
🇧🇪Liège, Belgium
Investigator site 040004
🇷🇴Constanţa, Romania
Investigator site 3590006
🇧🇬Sofia, Bulgaria
Investigator site 0070014
🇷🇺Saint Petersburg, Russian Federation
Investigator site 3590007
🇧🇬Pleven, Bulgaria
Investigator site 70021
🇷🇺Moscow, Russian Federation
Investigator site 0390024
🇮🇹Genova, Italy
Investigator site 390027
🇮🇹Messina, Italy
Investigator site 0010013
🇺🇸Phoenix, Arizona, United States
Investigator site 0010006
🇺🇸Tampa, Florida, United States
Investigator site 0010064
🇺🇸Columbus, Ohio, United States
Investigator site 0010065
🇺🇸Birmingham, Alabama, United States
Investigator site 0010160
🇺🇸Rancho Mirage, California, United States
Investigator site 0010071
🇺🇸San Francisco, California, United States
Investigator site 0010059
🇺🇸Miami, Florida, United States
Investigator site 0010072
🇺🇸Boca Raton, Florida, United States
Investigator site 0010007
🇺🇸Philadelphia, Pennsylvania, United States
Investigator site 0010047
🇺🇸Philadelphia, Pennsylvania, United States
Investigator site 31
🇱🇻Riga, Latvia
Investigator site 0010055
🇺🇸Scottsdale, Arizona, United States
Investigator site 0010032
🇺🇸Carlsbad, California, United States
Investigator site 10190
🇺🇸Pomona, California, United States
Investigator site 0010057
🇺🇸Centennial, Colorado, United States
Investigator site 0010144
🇺🇸Coral Springs, Florida, United States
Investigator site 0010068
🇺🇸Maitland, Florida, United States
Investigator site 0010023
🇺🇸Jacksonville, Florida, United States
Investigator site 0010011
🇺🇸Iowa City, Iowa, United States
Investigator site 0010015
🇺🇸Fairway, Kansas, United States
Investigator site 10147
🇺🇸Lexington, Kentucky, United States
Investigator site 10168
🇺🇸New York, New York, United States
Investigator site 0010003
🇺🇸Chapel Hill, North Carolina, United States
Investigator Site 0010066
🇺🇸Austin, Texas, United States
Investigator site 0010009
🇺🇸San Antonio, Texas, United States
Investigator site 0010061
🇺🇸Richmond, Virginia, United States
Investigator site 0430007
🇦🇹Innsbruck, Austria
Investigator site 0430008
🇦🇹Linz, Austria
Investigator site 0430005
🇦🇹Wien, Austria
Investigator site 0320009
🇧🇪Leuven, Belgium
Investigator site 320022
🇧🇪Woluwe-Saint-Lambert, Belgium
Investigator site 3590005
🇧🇬Sofia, Bulgaria
Investigator site 3590008
🇧🇬Sofia, Bulgaria
Investigator site 0860033
🇨🇳Beijing, China
Investigator site 860041
🇨🇳Changsha, China
Investigator site 0860038
🇨🇳Fuzhou, China
Investigator site 0860050
🇨🇳Guangzhou, China
Investigator site 0860045
🇨🇳Guiyang, China
Investigator site 0860031
🇨🇳Jinan, China
Investigator site 860047
🇨🇳Shanghai, China
Investigator site 0860035
🇨🇳Hangzhou, China
Investigator site 0860028
🇨🇳Shanghai, China
Investigator site 0860043
🇨🇳Nanjing, China
Investigator site 0860048
🇨🇳Xi'an, China
Investigator site 0860042
🇨🇳Tianjin, China
Investigator site 0860034
🇨🇳Wuhan, China
Investigator site 0860029
🇨🇳Wuhan, China
Investigator site 0860054
🇨🇳Xianyang, China
Investigator site 0450001
🇩🇰Copenhagen, Denmark
Investigator site 4200010
🇨🇿Hradec Králové, Czechia
Investigator site 0450002
🇩🇰Aarhus, Denmark
Investigator site 0450003
🇩🇰Odense, Denmark
Investigator site 00330034
🇫🇷Angers, France
Investigator site 0330013
🇫🇷Bordeaux, France
Investigator site 330033
🇫🇷Clermont-Ferrand, France
Investigator site 0330021
🇫🇷Nice, France
Investigator site 330022
🇫🇷Nantes, France
Investigator site 0330023
🇫🇷Le Kremlin-Bicêtre, France
Investigator site 0330035
🇫🇷Paris, France
Investigator site 0330024
🇫🇷Limoges, France
Investigator site 9950020
🇬🇪Kutaisi, Georgia
Investigator site 0330020
🇫🇷Strasbourg, France
Investigator site 9950004
🇬🇪Tbilisi, Georgia
Investigator site 9950005
🇬🇪Tbilisi, Georgia
Investigator site 490044
🇩🇪Bochum, Germany
Investigator Site 9950003
🇬🇪Tbilisi, Georgia
Investigator site 490021
🇩🇪Göttingen, Germany
Investigator site 490045
🇩🇪Essen, Germany
Investigator site 9950002
🇬🇪Tbilisi, Georgia
Investigator site 0490016
🇩🇪Kiel, Germany
Investigator site 0490019
🇩🇪Potsdam, Germany
Investigator site 9720004
🇮🇱Tel Aviv, Israel
Investigator site 0390022
🇮🇹Brescia, Italy
Investigator site 390029
🇮🇹Firenze, Italy
Investigator site 0490013
🇩🇪Köln, Germany
Investigator site 0390026
🇮🇹Milano, Italy
Investigator site 0390003
🇮🇹Milan, Italy
Investigator site 0390023
🇮🇹Pisa, Italy
Investigator site 0390042
🇮🇹Torino, Italy
Investigator site 0810035
🇯🇵Bunkyō-Ku, Japan
Investigator site 0390008
🇮🇹Roma, Italy
Investigator site 0810065
🇯🇵Ginowan, Japan
Investigator site 0810031
🇯🇵Fukuoka, Japan
Investigator site 0810003
🇯🇵Osaka, Japan
Investigator site 0810002
🇯🇵Chiba, Japan
Investigator site 0810066
🇯🇵Hakodate, Japan
Investigator site 0810030
🇯🇵Fuchū, Japan
Investigator site 0810058
🇯🇵Hiroshima, Japan
Investigator site 0810036
🇯🇵Itabashi, Japan
Investigator site 810061
🇯🇵Kyoto-shi, Japan
Investigator site 0810027
🇯🇵Mibu, Japan
Investigator site 0810029
🇯🇵Kawagoe, Japan
Investigator site 0810032
🇯🇵Nagoya, Japan
Investigator site 0810026
🇯🇵Kodaira, Japan
Investigator site 0810007
🇯🇵Osaka, Japan
Investigator site 0810063
🇯🇵Suita, Japan
Investigator site 0810064
🇯🇵Tokushima, Japan
Investigator site 0810060
🇯🇵Yokohama, Japan
Investigator site 0310010
🇳🇱Amsterdam, Netherlands
Investigator site 0310011
🇳🇱Rotterdam, Netherlands
Investigator Site 0480017
🇵🇱Lublin, Poland
Investigator site 0480024
🇵🇱Kraków, Poland
Investigator site 0480018
🇵🇱Kraków, Poland
Investigator site 0480022
🇵🇱Warszawa, Poland
Investigator site 040002
🇷🇴Braşov, Romania
Investigator site 040001
🇷🇴Bucharest, Romania
Investigator site 040003
🇷🇴Timişoara, Romania
Investigator site 0070019
🇷🇺Rostov-on-Don, Russian Federation
Investigator site 070017
🇷🇺Kazan, Russian Federation
Investigator site 0070020
🇷🇺Moscow, Russian Federation
Investigator site 0070023
🇷🇺Kazan, Russian Federation
Investigator site 3810003
🇷🇸Belgrade, Serbia
Investigator site 3810004
🇷🇸Kragujevac, Serbia
Investigator site 0340021
🇪🇸Badalona, Spain
Investigator site 3810001
🇷🇸Belgrade, Serbia
Investigator site 0340038
🇪🇸Barcelona, Spain
Investigator site 0340018
🇪🇸Madrid, Spain
Investigator site 8860013
🇨🇳Tainan, Taiwan
Investigator site 8860012
🇨🇳Taipei, Taiwan
Investigator site 8860017
🇨🇳Taoyuan, Taiwan
Investigator site 8860016
🇨🇳Taipei, Taiwan
Investigator site 900021
🇹🇷İzmir, Turkey
Investigator site 900025
🇹🇷Bursa, Turkey
Investigator site 900022
🇹🇷Samsun, Turkey
Investigator Site 3800012
🇺🇦Dnipro, Ukraine
Investigator site 3100013
🇺🇦Kyiv, Ukraine
Investigator site 3800008
🇺🇦Luts'k, Ukraine
Investigator site 0440016
🇬🇧Oxford, United Kingdom
Investigator site 3800011
🇺🇦Zaporizhzhya, Ukraine
Investigator site 440026
🇬🇧London, United Kingdom
Investigator site 0440018
🇬🇧Sheffield, United Kingdom