A Long-term Study to Assess the Safety and Efficacy of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ITP).
- Conditions
- Primary Immune Thrombocytopenia
- Interventions
- Biological: efgartigimod
- Registration Number
- NCT04225156
- Lead Sponsor
- argenx
- Brief Summary
This is an open-label long-term multicenter phase 3 trial to evaluate the efficacy and safety of ARGX-113 in adult patients with primary ITP.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 101
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description efgartigimod efgartigimod patients receiving efgartigimod
- Primary Outcome Measures
Name Time Method Frequency and severity of laboratory assessments Up to 60 weeks Frequency and severity of vital signs Up to 60 weeks Frequency and severity of Adverse Events Up to 60 weeks
- Secondary Outcome Measures
Name Time Method Extent of disease control defined as the percentage of weeks in the trial with platelet counts of ≥50×10E9/L. Over the 52 weeks of treatment Mean change from baseline in platelet count at each visit. Up to 60 weeks, at each visit In patients with first exposure to efgartigimod: proportion of patients in the overall population achieving platelet counts of at least 50x10^9/L for at least 6 of the 8 visits between week 17 and 24 of the trial. Up to 7 weeks, between visit 17 and 24 of the trial Rate of receipt of rescue therapy (rescue per patient per month). Up to 60 weeks, at each visit Incidence of anti-drug antibodies (ADA) to efgartigimod. Up to 216 weeks For patients rolling-over from the ARGX-113-1801 trial with a platelet count of <30×10^9/L: time to response is defined as the time to achieve 2 consecutive platelet counts of ≥50×10^9/L Up to 60 weeks, at each visit The percentage of weeks in the trial with platelet counts of ≥30×109/L and at least 20×10E9/L above baseline. Over the 52 weeks of treatment Percentage of patients with overall platelet count response defined as achieving a platelet count of ≥50×10^9/L on at least 4 occasions at any time during the 52-week treatment period. Over the 52 weeks of treatment In patients with baseline platelet count of <15×10E9/L in the current trial (ARGX-113-1803), the percentage of weeks in the trial with platelet counts of ≥30×10E9/L and at least 20×10E9/L above baseline. Over the 52 weeks of treatment Incidence and severity of the WHO-classified bleeding events. Up to 60 weeks, at each visit Change from baseline in Patient reported Outcomes (Fact-Th6) at planned visits. Up to 52 weeks In patients with first exposure to efgartigimod: proportion of patients who achieve a sustained platelet 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. Up to 5 weeks, between visit 19 and 24 of the trial Change from baseline in Patient reported Outcomes (FACIT-Fatigue) at planned visits. Up to 52 weeks Reduction in concurrent ITP therapy. Up to 60 weeks, at each visit Change from baseline in Quality of Life (SF-36) at planned visits. Up to 52 weeks Pharmacokinetic parameter of efgartigimod: serum concentration observed predose (Ctrough). Up to 60 weeks Pharmacodynamics markers: total IgG. Up to 60 weeks
Trial Locations
- Locations (87)
Investigator Site 0360015
🇭🇺Győr, Hungary
Investigator Site 0390009
🇮🇹Siena, Italy
Investigator Site 0390016
🇮🇹Trieste, Italy
Investigator Site 0360004
🇭🇺Budapest, Hungary
Investigator Site 0810015
🇯🇵Hirakata, Japan
Investigator Site 0810010
🇯🇵Hiroshima, Japan
Investigator Site 0070013
🇷🇺Rostov-on-Don, Russian Federation
Investigator Site 0900006
🇹🇷Ankara, Turkey
Investigator Site 0810018
🇯🇵Maebashi, Japan
Investigator Site 0810021
🇯🇵Niigata, Japan
Investigator Site 0070007
🇷🇺Petrozavodsk, Russian Federation
Investigator Site 0900019
🇹🇷Trabzon, Turkey
Investigator Site 0070012
🇷🇺Tula, Russian Federation
Investigator Site 0900004
🇹🇷İzmir, Turkey
Investigator Site 0070010
🇷🇺Ufa, Russian Federation
Investigator Site 0900010
🇹🇷Mersin, Turkey
Investigator Site 3800006
🇺🇦Mykolaiv, Ukraine
Investigator Site 0900009
🇹🇷Samsun, Turkey
Investigator Site 0430002
🇦🇹Vienna, Austria
Investigator Site 0010040
🇺🇸Columbus, Ohio, United States
Investigator Site 4200001
🇨🇿Brno, Czechia
Investigator Site 0430003
🇦🇹Vienna, Austria
Investigator Site 4200008
🇨🇿Olomouc, Czechia
Investigator Site 0320012
🇧🇪Brasschaat, Belgium
Investigator Site 0320011
🇧🇪Brugge, Belgium
Investigator Site 0320014
🇧🇪Turnhout, Belgium
Investigator Site 0320002
🇧🇪Yvoir, Belgium
Investigator Site 4200007
🇨🇿Praha, Czechia
Investigator Site 0330018
🇫🇷Montpellier, France
Investigator site 9950009
🇬🇪Tbilisi, Georgia
Investigator site 9950011
🇬🇪Tbilisi, Georgia
Investigator Site 0490010
🇩🇪Düsseldorf, Germany
Investigator Site 0490008
🇩🇪Essen, Germany
Investigator Site 0360010
🇭🇺Nyíregyháza, Hungary
Investigator Site 0340012
🇪🇸Palma De Mallorca, Spain
Investigator Site 0480026
🇵🇱Nowy Sącz, Poland
Investigator Site 0070015
🇷🇺Syktyvkar, Russian Federation
Investigator Site 0810016
🇯🇵Shibukawa, Japan
Investigator Site 0440012
🇬🇧Southampton, United Kingdom
Investigator Site 0440008
🇬🇧London, United Kingdom
Investigator Site 0310006
🇳🇱Den Haag, Netherlands
Investigator Site 0900016
🇹🇷Edirne, Turkey
Investigator Site 0900007
🇹🇷Sakarya, Turkey
Investigator Site 0070008
🇷🇺Moscow, Russian Federation
Investigator Site 0480014
🇵🇱Lublin, Poland
Investigator Site 0340006
🇪🇸Barcelona, Spain
Investigator Site 0480012
🇵🇱Gdańsk, Poland
Investigator Site 0340007
🇪🇸Barcelona, Spain
Investigator Site 0070006
🇷🇺Kaluga, Russian Federation
Investigator Site 0340013
🇪🇸Sevilla, Spain
Investigator Site 0340015
🇪🇸Pozuelo De Alarcón, Spain
Investigator Site 4200006
🇨🇿Ostrava, Czechia
Investigator Site 0010042
🇺🇸Iowa City, Iowa, United States
Investigator Site 0010037
🇺🇸Ocala, Florida, United States
Investigator site 9950007
🇬🇪Tbilisi, Georgia
Investigator Site 0330016
🇫🇷Périgueux, France
Investigator Site 0900015
🇹🇷Ankara, Turkey
Investigator site 9950008
🇬🇪Tbilisi, Georgia
Investigator Site 0330008
🇫🇷Pessac, France
Investigator Site 0810022
🇯🇵Kashiwa, Japan
Investigator site 9950012
🇬🇪Tbilisi, Georgia
Investigator Site 0390018
🇮🇹Reggio Emilia, Italy
Investigator Site 0390019
🇮🇹Rimini, Italy
Investigator Site 0810023
🇯🇵Shimotsuke, Japan
Investigator Site 0480013
🇵🇱Katowice, Poland
Investigator Site 0310005
🇳🇱Rotterdam, Netherlands
Investigator Site 0480011
🇵🇱Łódź, Poland
Investigator Site 0340004
🇪🇸Valencia, Spain
Investigator Site 0340014
🇪🇸Madrid, Spain
Investigator Site 0900003
🇹🇷Ankara, Turkey
Investigator Site 0900013
🇹🇷Istanbul, Turkey
Investigator Site 0340011
🇪🇸Valencia, Spain
Investigator Site 0340009
🇪🇸Madrid, Spain
Investigator Site 0900017
🇹🇷Tekirdağ, Turkey
Investigator Site 0810017
🇯🇵Iruma, Japan
Investigator Site 0390020
🇮🇹Monza, Italy
Investigator Site 0390015
🇮🇹Novara, Italy
Investigator Site 0390011
🇮🇹Reggio Calabria, Italy
Investigator Site 0010045
🇺🇸Washington, District of Columbia, United States
Investigator Site 3590001
🇧🇬Pleven, Bulgaria
Investigator Site 3590002
🇧🇬Sofia, Bulgaria
Investigator Site 0330009
🇫🇷Créteil, France
Investigator Site 0360006
🇭🇺Debrecen, Hungary
Investigator Site 0360014
🇭🇺Szombathely, Hungary
Investigator Site 0390014
🇮🇹Milano, Italy
Investigator Site 0390010
🇮🇹Ravenna, Italy
Investigator Site 0810014
🇯🇵Sapporo, Japan