A Study to Assess the Efficacy and Safety of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ITP).
- Conditions
- Primary Immune Thrombocytopenia
- Interventions
- Other: Placebo
- Registration Number
- NCT04188379
- Lead Sponsor
- argenx
- Brief Summary
This is a randomized, double-blind placebo-controlled multicenter phase 3 trial to evaluate the efficacy and safety of ARGX-113 in participants with primary ITP.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 131
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description efgartigimod efgartigimod Patient receiving efgartigimod Placebo Placebo Patients receiving placebo
- Primary Outcome Measures
Name Time Method Percentage of Participants With Chronic ITP With a Sustained Platelet Count Response Defined as Achieving Platelet Counts of at Least 50×10^9/L for at Least 4 of the 6 Visits Between Week 19 and 24 of the Trial. From Week 19 up to Week 24 Percentage of participants with chronic ITP with a sustained platelet count response was defined as achieving platelet counts of at least 50 × 10\^9/L for at least 4 of the 6 visits between Week 19 and 24 of the study.
- Secondary Outcome Measures
Name Time Method Extent of Disease Control Defined as the Number of Cumulative Weeks Over the Planned 24-week Treatment Period With Platelet Counts of ≥50×10^9/L in the Chronic ITP Population From Week 1 up to Week 24 Extent of disease control, defined as the cumulative number of weeks over the planned 24-week treatment period with platelet counts of ≥50 × 10\^9/L in the chronic ITP population.
Percentage of Participants With a Sustained Platelet Count Response for at Least 4 of the 6 Visits Between Week 19 and 24 of the Study From Week 19 up to Week 24 Percentage of participants in the overall population (chronic and persistent ITP) with a sustained platelet count response, defined as achieving platelet counts of at least 50 × 10\^9/L for at least 4 of the 6 visits between weeks 19 and 24 of the study.
Incidence and Severity of the WHO-classified Bleeding Events From Week 1 to Week 24 Incidence of the World Health Organization (WHO)-classified bleeding events in the overall population.
This secondary endpoint used the WHO-classified bleeding scale. Bleeding was the predominant clinical manifestation of ITP and was typically related to platelet count. Accordingly, measuring bleeding was important for monitoring this participant population.Percentage of Participants in the Overall Population Achieving Platelet Counts of at Least 50×10^9/L for at Least 6 of the 8 Visits Between Week 17 and Week 24 From Week 17 up to Week 24 Percentage of participants in the overall population achieving platelet counts of at least 50 × 10\^9/L for at least 6 of the 8 visits between Week 17 and 24 of the study.
Trial Locations
- Locations (121)
Investigator Site 0010040
🇺🇸Columbus, Ohio, United States
Investigator Site 9950008
🇬🇪Tbilisi, Georgia
Investigator Site 0480008
🇵🇱Katowice, Poland
Investigator Site 0320015
🇧🇪Leuven, Belgium
Investigator Site 0810024
🇯🇵Bunkyō-Ku, Japan
Investigator Site 0360004
🇭🇺Budapest, Hungary
Investigator Site 0320014
🇧🇪Turnhout, Belgium
Investigator site 0360010
🇭🇺Nyiregyhaza, Hungary
Investigator site 0810016
🇯🇵Shibukawa, Japan
Investigator Site 0320012
🇧🇪Brasschaat, Belgium
Investigator Site 9950006
🇬🇪Tbilisi, Georgia
Investigator Site 0810015
🇯🇵Hirakata, Japan
Investigator site 0810020
🇯🇵Minato-Ku, Japan
Investigator site 0810014
🇯🇵Sapporo, Japan
Investigator site 0480026
🇵🇱Nowy Sącz, Poland
Investigator Site 0070015
🇷🇺Syktyvkar, Russian Federation
Investigator Site 0810023
🇯🇵Shimotsuke, Japan
Investigator Site 0310007
🇳🇱Rotterdam, Netherlands
Investigator Site 0430004
🇦🇹Linz, Austria
Investigator Site 0480013
🇵🇱Chorzów, Poland
Investigator Site 0340004
🇪🇸Valencia, Spain
Investigator Site 0440014
🇬🇧Truro, United Kingdom
Investigator Site 0010038
🇺🇸Tucson, Arizona, United States
Investigator Site 0010045
🇺🇸Washington, District of Columbia, United States
Investigator Site 0010034
🇺🇸Jacksonville, Florida, United States
Investigator site 0010037
🇺🇸Ocala, Florida, United States
Investigator Site 0010042
🇺🇸Iowa City, Iowa, United States
Investigator Site 0010046
🇺🇸Greenville, North Carolina, United States
Investigator Site 0010049
🇺🇸Cleveland, Ohio, United States
Investigator Site 0010041
🇺🇸Philadelphia, Pennsylvania, United States
Investigator Site 0430003
🇦🇹Vienna, Austria
Investigator Site 0430002
🇦🇹Vienna, Austria
Investigator Site 0320011
🇧🇪Brugge, Belgium
Investigator Site 0320020
🇧🇪Verviers, Belgium
Investigator site 0320002
🇧🇪Yvoir, Belgium
Investigator Site 3590001
🇧🇬Pleven, Bulgaria
Investigator Site 3590002
🇧🇬Sofia, Bulgaria
Investigator Site 4200001
🇨🇿Brno, Czechia
Investigator Site 4200008
🇨🇿Olomouc, Czechia
Investigator Site 4200006
🇨🇿Ostrava, Czechia
Investigator Site 0330009
🇫🇷Créteil, France
Investigator Site 0330019
🇫🇷Clermont-Ferrand, France
Investigator Site 0330015
🇫🇷Le Mans, France
Investigator Site 0330018
🇫🇷Montpellier, France
Investigator Site 0330008
🇫🇷Pessac, France
Investigator Site 0330016
🇫🇷Périgueux, France
Investigator Site 0330017
🇫🇷Rouen, France
Investigator Site 9950007
🇬🇪Tbilisi, Georgia
Investigator Site 9950009
🇬🇪Tbilisi, Georgia
Investigator Site 9950011
🇬🇪Tbilisi, Georgia
Investigator Site 9950012
🇬🇪Tbilisi, Georgia
Investigator Site 0490008
🇩🇪Essen, Germany
Investigator Site 0490010
🇩🇪Düsseldorf, Germany
Investigator Site 0490012
🇩🇪Gießen, Germany
Investigator Site 0360006
🇭🇺Debrecen, Hungary
Investigator Site 0360015
🇭🇺Győr, Hungary
Investigator Site 0390012
🇮🇹Campobasso, Italy
Investigator Site 0360014
🇭🇺Szombathely, Hungary
Investigator Site 0390020
🇮🇹Monza, Italy
Investigator Site 0390014
🇮🇹Milan, Italy
Investigator Site 0390015
🇮🇹Novara, Italy
Investigator Site 0390010
🇮🇹Ravenna, Italy
Investigator Site 0390011
🇮🇹Reggio Calabria, Italy
Investigator Site 0390018
🇮🇹Reggio Emilia, Italy
Investigator Site 0390019
🇮🇹Rimini, Italy
Investigator Site 0390021
🇮🇹Roma, Italy
Investigator Site 0390009
🇮🇹Siena, Italy
Investigator Site 0390017
🇮🇹Torino, Italy
Investigator Site 0390016
🇮🇹Trieste, Italy
Investigator Site 0810010
🇯🇵Hiroshima, Japan
Investigator site 0810017
🇯🇵Iruma, Japan
Investigator site 0810011
🇯🇵Isehara, Japan
Investigator Site 0810022
🇯🇵Kashiwa, Japan
Investigator site 0810018
🇯🇵Maebashi, Japan
Investigator Site 0810021
🇯🇵Niigata, Japan
Investigator Site 0810025
🇯🇵Shinjuku-Ku, Japan
Investigator site 0310006
🇳🇱Den Haag, Netherlands
Investigator Site 0310005
🇳🇱Rotterdam, Netherlands
Investigator Site 0480030
🇵🇱Białystok, Poland
Investigator Site 0480015
🇵🇱Brzozów, Poland
Investigator Site 0480010
🇵🇱Bydgoszcz, Poland
Investigator Site 0480012
🇵🇱Gdańsk, Poland
Investigator Site 0480011
🇵🇱Lodz, Poland
Investigator Site 0480014
🇵🇱Lublin, Poland
Investigator Site 0480016
🇵🇱Wrocław, Poland
Investigator site 0070006
🇷🇺Kaluga, Russian Federation
Investigator Site 0070007
🇷🇺Petrozavodsk, Russian Federation
Investigator Site 0070013
🇷🇺Rostov-on-Don, Russian Federation
Investigator Site 0070012
🇷🇺Tula, Russian Federation
Investigator site 0070010
🇷🇺Ufa, Russian Federation
Investigator Site 0340006
🇪🇸Barcelona, Spain
Investigator Site 0340007
🇪🇸Barcelona, Spain
Investigator Site 0340030
🇪🇸Burgos, Spain
Investigator Site 0340009
🇪🇸Madrid, Spain
Investigator Site 0340014
🇪🇸Madrid, Spain
Investigator Site 0340015
🇪🇸Madrid, Spain
Investigator site 0340012
🇪🇸Palma De Mallorca, Spain
Investigator Site 0340013
🇪🇸Sevilla, Spain
Investigator Site 0340011
🇪🇸Valencia, Spain
Investigator Site 0900002
🇹🇷Adana, Turkey
Investigator Site 0900008
🇹🇷Ankara, Turkey
Investigator Site 0900007
🇹🇷Adapazarı, Turkey
Investigator Site 0900003
🇹🇷Ankara, Turkey
Investigator Site 0900006
🇹🇷Ankara, Turkey
Investigator Site 0900015
🇹🇷Ankara, Turkey
Investigator Site 0900016
🇹🇷Edirne, Turkey
Investigator Site 0900013
🇹🇷Istanbul, Turkey
Investigator Site 0900004
🇹🇷Izmir, Turkey
Investigator Site 0900014
🇹🇷Kocaeli, Turkey
Investigator Site 0900018
🇹🇷Malatya, Turkey
Investigator site 3800006
🇺🇦Mykolayiv, Ukraine
Investigator Site 0440008
🇬🇧London, United Kingdom
Investigator Site 0900005
🇹🇷Manisa, Turkey
Investigator Site 0900010
🇹🇷Mersin, Turkey
Investigator Site 0900009
🇹🇷Samsun, Turkey
Investigator Site 0440010
🇬🇧Rhyl, United Kingdom
Investigator Site 0900017
🇹🇷Tekirdağ, Turkey
Investigator Site 0900019
🇹🇷Trabzon, Turkey
Investigator Site 0440012
🇬🇧Southampton, United Kingdom
Investigator Site 3800022
🇺🇦Kharkiv, Ukraine
Investigator Site 4200007
🇨🇿Praha, Czechia