A Long-term Study to Assess the Safety and Efficacy of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ITP).
Phase 3
Active, not recruiting
- Conditions
- Primary Immune Thrombocytopenia
- Interventions
- 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
Inclusion Criteria
Not provided
Exclusion Criteria
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 vital signs Up to 60 weeks Frequency and severity of laboratory assessments 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 0010045
🇺🇸Washington D.C., District of Columbia, United States
Investigator Site 0010037
🇺🇸Ocala, Florida, United States
Investigator Site 0010042
🇺🇸Iowa City, Iowa, United States
Investigator Site 0010040
🇺🇸Columbus, Ohio, United States
Investigator Site 0430002
🇦🇹Vienna, Austria
Investigator Site 0430003
🇦🇹Vienna, Austria
Investigator Site 0320012
🇧🇪Brasschaat, Belgium
Investigator Site 0320011
🇧🇪Bruges, Belgium
Investigator Site 0320014
🇧🇪Turnhout, Belgium
Investigator Site 0320002
🇧🇪Yvoir, Belgium
Scroll for more (77 remaining)Investigator Site 0010045🇺🇸Washington D.C., District of Columbia, United States