Oral rilzabrutinib for adults with immune thrombocytopenia after first-line treatment fails.
A Multi-center, Open-label, Single-arm Study to Evaluate the Efficacy and Safety of Oral Rilzabrutinib in Adults With Immune Thrombocytopenia (ITP) Who Failed First-line Treatment
This study will test whether the oral drug rilzabrutinib can raise and keep platelet counts up in adults with ITP who did not have lasting benefit from first-line therapy.
Quick facts
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Enrollment | 60 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Sanofi Industry-sponsored |
| Drugs / interventions | rilzabrutinib |
| Locations | 24 sites (New Orleans, Louisiana and 23 other locations) |
| Trial ID | NCT07007962 on ClinicalTrials.gov |
What this trial studies
This multinational, open-label, single-arm Phase 3 study gives oral rilzabrutinib to adults with primary ITP who relapsed, lost response, or are steroid-dependent after first-line treatment. After a screening period of up to 8 weeks, participants enter a primary analysis period of up to 28 weeks to monitor platelet responses and safety, with a selected long-term extension of 28 weeks and a 24-week follow-up for eligible participants. Key exclusions include secondary ITP, Evans syndrome, prior splenectomy, prior advanced ITP therapy, certain recent malignancies, and pregnancy or nursing. The trial is sponsored by Sanofi and is being conducted at several academic medical centers in the United States.
Who should consider this trial
Good fit: Adults (18+) with primary ITP who responded to first-line treatment but later relapsed, lost response, or are steroid-dependent and who have not received advanced ITP therapies or undergone splenectomy are the intended candidates.
Not a fit: Patients with secondary ITP, Evans syndrome, recent malignancy, prior splenectomy or advanced ITP therapy, other clotting disorders, or who are pregnant or nursing are unlikely to be eligible and may not benefit from this treatment.
Why it matters
Potential benefit: If successful, rilzabrutinib could become an effective oral option to increase platelet counts and reduce bleeding risk for adults with ITP who fail first-line therapy.
How similar studies have performed: Earlier-phase studies of rilzabrutinib and other Bruton tyrosine kinase inhibitors have shown platelet count improvements in some patients with ITP, but confirmatory phase 3 evidence is limited.
Eligibility criteria
Show full inclusion / exclusion criteria
Key Inclusion Criteria: * Male or female participants aged 18 years and older with a documented diagnosis of primary ITP in the medical history * Participant received at least one course of first-line therapy and had a history of response while on treatment * Participant has loss of response, relapse, or steroid dependency Key Exclusion Criteria: * Participants with Secondary ITP * Participants with Evans syndrome or history of myelodysplastic syndrome * Participants with history of lymphoma, leukemia, or any malignancy within the past 5 years except for non-melanoma skin malignancy. * Participants with history of solid organ transplant * Participants with history of coagulation or bleeding disorders other than ITP, including genetic conditions, other than ITP * Participant received advanced therapy for ITP or was splenectomized * Pregnancy or nursing The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
Where this trial is running
New Orleans, Louisiana and 23 other locations
- Tulane University Health Sciences Center - Tulane Avenue-Investigational Site Number: 8400011 — New Orleans, Louisiana, United States (Recruiting)
- University of Michigan Health - Michigan Medicine - University Hospital-Investigational Site Number: 8400001 — Ann Arbor, Michigan, United States (Recruiting)
- Mayo Clinic_Investigational Site Number: 8400009 — Rochester, Minnesota, United States (Recruiting)
- New York Oncology Hematology-Investigational Site Number: 8400010 — Albany, New York, United States (Recruiting)
- Montefiore Medical Center-Investigational Site Number: 8400012 — The Bronx, New York, United States (Recruiting)
- Community Cancer Trials of Utah-Investigational Site Number: 8400002 — Ogden, Utah, United States (Recruiting)
- Investigational Site Number : 0400001 — Vienna, Austria (Recruiting)
- Investigational Site Number: 2500003 — Créteil, France (Recruiting)
- Investigational Site Number : 2500001 — Dijon, France (Recruiting)
- Investigational Site Number : 2500002 — Pessac, France (Recruiting)
- Investigational Site Number : 2500004 — Toulouse, France (Recruiting)
- Investigational Site Number : 3480002 — Kaposvár, Hungary (Recruiting)
- Investigational Site Number : 3800006 — Naples, Italy (Recruiting)
- Investigational Site Number : 3800007 — Torino, Italy (Recruiting)
- Investigational Site Number : 3800003 — Vicenza, Italy (Recruiting)
- Investigational Site Number : 6160002 — Gdansk, Poland (Recruiting)
- Investigational Site Number : 6160003 — Skorzewo, Poland (Recruiting)
- Investigational Site Number : 6160004 — Słupsk, Poland (Recruiting)
- Investigational Site Number : 7240001 — Barcelona, Spain (Recruiting)
- Investigational Site Number : 7240002 — Burgos, Spain (Recruiting)
- Investigational Site Number : 7240007 — Madrid, Spain (Recruiting)
- Investigational Site Number : 7240008 — Madrid, Spain (Recruiting)
- Investigational Site Number : 7240005 — Murcia, Spain (Recruiting)
- Investigational Site Number : 7240004 — Seville, Spain (Recruiting)
Study contacts
- Study coordinator: Trial Transparency email recommended (Toll free for US & Canada)
- Email: contact-us@sanofi.com
- Phone: 800-633-1610
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.