NCT04516967

Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects With Immune Thrombocytopenia for ≥6 Months

Randomized, Double-blind, Placebo-controlled Study With Open-label Extension Phase to Evaluate the Efficacy and Safety of Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects With Immune Thrombocytopenia for ≥6 Months

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 75 (actual)
• Sponsor: Sobi, Inc. · Industry
• Sex: All
• Age: 1 Year – 17 Years
• Healthy volunteers: Not accepted

Summary

A Phase 3b Study to Evaluate the Efficacy and Safety of Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects with Immune Thrombocytopenia for ≥6 Months

Detailed description

Subjects will be randomized in to blinded therapy of avatrombopag or placebo in a 3:1 ratio for a period of 12 weeks. Subjects who complete the 12 week treatment period and are eligible may continue to the open label extension phase which will last 2 years.

Conditions

• Immune Thrombocytopenia

Interventions

Type	Name	Description
DRUG	Avatrombopag	Oral avatrombopag tablet or sprinkle capsule
DRUG	Placebo	Placebo comparator tablet or sprinkle capsule

Timeline

• Start date: 2021-03-05
• Primary completion: 2023-11-08
• Completion: 2025-10-28
• First posted: 2020-08-18
• Last updated: 2026-01-15
• Results posted: 2025-01-17

Locations

62 sites across 9 countries: United States, France, Germany, Hungary, Poland, Russia, Turkey (Türkiye), Ukraine, United Kingdom

Regulatory

• FDA-regulated drug study