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

NCT04562766 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 232

Last updated 2026-02-23

Study results available
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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

Interventions

DRUG

Rilzabrutinib

400mg Caplet

DRUG

Placebo

400mg Caplet

Sponsors & Collaborators

  • Principia Biopharma, a Sanofi Company

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
10 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-12-14
Primary Completion
2025-01-15
Completion
2026-08-26
FDA Drug
Yes

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

Study Locations

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Entities

Drugs
Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04562766 on ClinicalTrials.gov