A Study to Assess the Efficacy and Safety of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ITP).

Phase 3

Completed

Conditions: Primary Immune Thrombocytopenia

Interventions: Biological: efgartigimod
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

Inclusion Criteria

Not provided

Exclusion Criteria

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 0010038
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Tucson, Arizona, United States
Investigator Site 0010045
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Washington, District of Columbia, United States
Investigator Site 0010034
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Jacksonville, Florida, United States
Investigator site 0010037
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Ocala, Florida, United States
Investigator Site 0010042
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Iowa City, Iowa, United States
Investigator Site 0010046
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Greenville, North Carolina, United States
Investigator Site 0010049
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Cleveland, Ohio, United States
Investigator Site 0010040
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Columbus, Ohio, United States
Investigator Site 0010041
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Philadelphia, Pennsylvania, United States
Investigator Site 0430004
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Linz, Austria
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Investigator Site 0010038
🇺🇸Tucson, Arizona, United States