NCT04562766

Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP)

A Phase 3, Multicenter, Randomized, Double-Blind, Placebo Controlled, Parallel-Group Study With an Open-Label Extension to Evaluate the Efficacy and Safety of Oral Rilzabrutinib (PRN1008) in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP)

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 232 (actual)
• Sponsor: Principia Biopharma, a Sanofi Company · Industry
• Sex: All
• Age: 10 Years
• Healthy volunteers: Not accepted

Summary

This was a randomized, double-blind study of rilzabrutinib in patients with persistent or chronic ITP, with an average platelet count of \<30,000/μL (and no single platelet count \>35,000/μL) on two counts at least 5 days apart in the 14 days before treatment begins. Patients received rilzabrutinib or placebo 400mg twice daily. For each patient, the study lasted up to 60 weeks from the start of the Screening Period to the End of Study (EOS) visit. This included Screening (up to 4 weeks) through a 12 to 24-week Blinded Treatment Period followed by a 28-week Open-Label Period. Followed by a 4-week post dose follow-up. For adult participants, the maximum duration of the long-term extension (LTE) period was 12 months from the date of the last adult participant to enter the LTE. For pediatric participants, the maximum duration of the LTE period was 12 months from the date of the last pediatric participant to enter the LTE.

Conditions

• Immune Thrombocytopenia

Interventions

Type	Name	Description
DRUG	Rilzabrutinib	400mg Caplet
DRUG	Placebo	400mg Caplet

Timeline

• Start date: 2020-12-14
• Primary completion: 2025-01-15
• Completion: 2026-08-26
• First posted: 2020-09-24
• Last updated: 2026-02-23
• Results posted: 2026-02-23

Locations

155 sites across 26 countries: United States, Argentina, Australia, Austria, Brazil, Canada, Chile, China, France, Germany, Hungary, Israel, Italy, Japan, Mexico, Netherlands, Norway, Poland, Russia, Singapore, South Korea, Spain, Thailand, Turkey (Türkiye), Ukraine, United Kingdom

Regulatory

• FDA-regulated drug study