A Phase 3, Multicenter, Randomized, Double-Blinded, Placebo-Controlled Trial to Evaluate the Efficacy and the Safety of Efgartigimod (ARGX-113) PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia

argenx200 sites in 8 countries207 target enrollmentDecember 16, 2020

ConditionsPrimary Immune Thrombocytopenia

InterventionsPlacebo PH20 SC Efgartigimod PH20 SC

DrugsEfgartigimod PH20 SC

Overview

Phase: Phase 3
Intervention: Placebo PH20 SC
Conditions: Primary Immune Thrombocytopenia
Sponsor: argenx
Enrollment: 207
Locations: 200
Primary Endpoint: Percentage of Participants With Chronic Immune Thrombocytopenia (ITP) With a Sustained Platelet Count Response Between Weeks 19 and 24
Status: Completed
Last Updated: last year

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

This is a phase 3, multicenter, randomized, double-blinded, placebo-controlled, parallel-group trial to evaluate the efficacy, safety, and effect on QoL/PRO of efgartigimod PH20 SC treatment in adult patients with primary ITP.

Registry

clinicaltrials.gov

Start Date

December 16, 2020

End Date

October 9, 2023

Last Updated

last year

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

argenx

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Arms & Interventions

Placebo PH20 SC

Patients receiving placebo PH20 SC treatment

Intervention: Placebo PH20 SC

Efgartigimod PH20 SC

Patients receiving efgartigimod PH20 SC treatment

Intervention: Efgartigimod PH20 SC

Outcomes

Primary Outcomes

Percentage of Participants With Chronic Immune Thrombocytopenia (ITP) With a Sustained Platelet Count Response Between Weeks 19 and 24

Time Frame: Up to 6 weeks (between Weeks 19 and 24)

A participant was considered a responder for this endpoint (i.e., had a sustained platelet count response) if the participant had platelet counts of ≥50 × 10\^9/L for ≥4 of the 6 analysis visits between Weeks 19 and 24.

Secondary Outcomes

Extent of Disease Control Over the 24-Week Treatment Period in the Chronic ITP Population(Up to 24 weeks)
Percentage of Participants in the Overall Population (Chronic and Persistent ITP) With a Sustained Platelet Count Response Between Weeks 19 and 24(Up to 6 weeks (between Weeks 19 and 24))
Percentage of Participants in the Overall Population With Sustained Platelet Count Response Between Weeks 17 and 24(Up to 8 weeks (between Weeks 17 and 24))
Percentage of Participants in the Overall Population Achieving Overall Platelet Count Response at Any Time During the 24-week Treatment Period(Up to 24 weeks)
Extent of Disease Control Until Week 12 in the Overall Population(Up to 12 weeks)
Percentage of Participants in the Overall Population Achieving Overall Platelet Count Response at Any Time Until Week 12(Up to 12 weeks)
Mean Change From Baseline in Platelet Count at Each Visit in the Overall Population(Baseline and Weeks 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23, 24, Safety and Efficacy Follow-up Visit 1 (SEFU1) (up to Week 29), and SEFU2 (up to Week 33))
Time to Platelet Count Response in the Overall Population(Up to 24 weeks)
Extent of Disease Control Over the 24-Week Treatment Period in the Overall Population(Up to 24 weeks)
Extent of Disease Control Over the 24-Week Treatment Period in the Overall Population for Participants With Baseline Platelet Count of <15×10^9/L(Up to 24 weeks)
Number of the World Health Organization (WHO)-Classified Bleeding Events (Grade ≥1) in the Overall Population(Up to 24 weeks)
Percentage of Participants With a Platelet Count International Working Group (IWG) Response(Up to 24 weeks)
Rate of Receipt of Rescue Therapy (Rescue Per Participant Per Month) in the Overall Population(Up to 24 weeks)
Percentage of Participants for Whom Dose and/or Frequency of Concurrent ITP Therapies Have Increased at Week 12 or Later in the Overall Population(Up to 13 weeks (between Weeks 12 and 24))
Change From Baseline in Functional Assessment of Chronic Illness Therapy Fatigue Scale (FACIT-Fatigue) at Week 24 in the Overall Population(Baseline and Week 24)
Change From Baseline in Functional Assessment of Cancer Therapy Questionnaire-Th6 (Fact-Th6) at Week 24 in the Overall Population(Baseline and Weeks 4, 8, 12, 16, 20, and 24)
Change From Baseline in Short Form-36 (SF-36) at Week 24 in the Overall Population(Baseline and Week 24)
Incidence and Prevalence of Antibodies to Efgartigimod and/or rHuPH20 in the Overall Population(Up to 35 weeks)
Titers of Antibodies to Efgartigimod and/or rHuPH20 in the Overall Population(Weeks 3, 7, 11, 15, 19, 23, 24, SEFU1 (up to Week 29), and SEFU2 (up to Week 33))
Incidence and Prevalence of Neutralizing Antibodies (NAb) to Efgartigimod and/or rHuPH20 in the Overall Population(Up to 35 weeks)
Serum Efgartigimod Trough Concentration (Ctrough) in the Overall Population(Predose on Weeks 1, 2, 3, 17, 19, 21, 23, and 24)
Percentage Change From Baseline in Total IgG in the Overall Population(Baseline and Weeks 1, 2, 3, 17, 19, 21, 23, and 24)
Number of Participants With Antiplatelet Antibodies in the Overall Population(Weeks 7, 15, 23, and 24)