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Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP)
NCT04562766 · View on ClinicalTrials.gov ↗
Study 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 Studied
Interventions
- DRUG Placebo
- DRUG Rilzabrutinib
Study Locations (20)
California
- University of Southern California_Investigational Site Number 84024 — Los Angeles
- UCSF Benioff Children's Hospital San Francisco_Investigational Site Number 84020 — San Francisco
- Lundquist Institute for Biomedical Innovation at Harbor UCLA Medical Center_Investigational Site Number 84037 — Torrance
- The Oncology Institute of Hope and Innovation_Investigational Site Number 84031 — Whittier
Colorado
- Children's Hospital Colorado_Investigational Site Number 84025 — Aurora
- IMMUNOe International Research Centers_Investigational Site Number 84028 — Centennial
Ohio
- University Hospitals Cleveland Medical Center Site Number : 84036 — Cleveland
- Cleveland Clinic_Investigational Site Number 84026 — Cleveland
Buenos Aires
- Investigational Site Number : 3206 — Capital Federal
- Investigational Site Number : 3211 — La Plata
Florida
- ASCLEPES Research Centers_Investigational Site Number 84023 — Weeki Wachee
Georgia
- Children's Healthcare of Atlanta_Investigational Site Number 84034 — Atlanta
Illinois
- Rush University Medical Center_Investigational Site Number 84029 — Chicago
Kentucky
- University of Louisville - James Graham Brown Cancer Center_Investigational Site Number 84033 — Louisville
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 232 participants |
| Start Date | 2020-12-14 |
| Est. Completion | 2026-08-26 |
| Phase | Phase 3 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04562766
The ClinicalTrials.gov registry entry for NCT04562766 describes a study currently listed as active not recruiting. It is categorized as Phase 3, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 232 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Principia Biopharma, a Sanofi Company, which has 1 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Immune Thrombocytopenia appearing as the primary indexed condition, and to 2 interventions — of which Placebo is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT04562766 reports 20 study locations spanning 14 distinct geographic areas — top geographies include California, Colorado, Ohio. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT04562766 about?
NCT04562766 is a clinical study titled "Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP)". 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 ...
What is the current status of trial NCT04562766?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 232 participants. The study started on 2020-12-14. Estimated completion is 2026-08-26.
What conditions does trial NCT04562766 study?
This clinical trial studies the following conditions: Immune Thrombocytopenia. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT04562766?
The interventions under investigation include: Placebo (DRUG), Rilzabrutinib (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT04562766?
This trial is sponsored by Principia Biopharma, a Sanofi Company, which has 1 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT04562766 being conducted?
This trial has 20 study locations across California, Colorado, Florida, Georgia, Illinois. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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