NCT07007962

Brief Summary

This is a multinational, open label, single arm study that will evaluate the impact of early multi-immune modulation with rilzabrutinib in adult ITP patients who failed first-line treatment. The study includes a screening period (up to 8 weeks), a primary analysis period (up to 28 weeks), a long-term extension period for selected participants (28 weeks) and a 24-week follow-up period only for eligible participants.

Trial Health

83
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
60

participants targeted

Target at below P25 for phase_3

Timeline
32mo left

Started Oct 2025

Typical duration for phase_3

Geographic Reach
7 countries

24 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress17%
Oct 2025Dec 2028

First Submitted

Initial submission to the registry

May 20, 2025

Completed
17 days until next milestone

First Posted

Study publicly available on registry

June 6, 2025

Completed
5 months until next milestone

Study Start

First participant enrolled

October 24, 2025

Completed
2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 5, 2027

Expected
1.2 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 30, 2028

Last Updated

May 1, 2026

Status Verified

April 1, 2026

Enrollment Period

2 years

First QC Date

May 20, 2025

Last Update Submit

April 30, 2026

Conditions

Immune Thrombocytopenia

Outcome Measures

Primary Outcomes (1)

  • Durable platelet response

    Defined as the percentage of participants able to achieve platelet counts ≥50 000/μL (or ≥30 000/μL and \<50 000/μL and at least double from baseline) for ≥50% of 6 biweekly scheduled platelet measurements and at least 4 non-missing, biweekly visits during the last 12 weeks of the primary analysis period (PAP) in absence of rescue therapy

    Until Week 28

Secondary Outcomes (6)

  • Overall platelet response

    Until Week 28

  • Duration of platelet response

    Until Week 28

  • Change from baseline in the immune thrombocytopenia bleeding scale (IBLS) score at the end of week 28

    Until Week 28

  • Percentage of participants able to discontinue or reduce CS dose by at least 50% or to <5 mg/day (prednisone equivalent) from baseline at the end of week 28

    Until Week 28

  • Frequency and severity of treatment emergent adverse events (TEAEs, including serious adverse events [SAEs], bleeding TEAEs, adverse event of special interest [AESI] and adverse events leading to discontinuation)

    Until Week 80

  • +1 more secondary outcomes

Study Arms (1)

rilzabrutinib

EXPERIMENTAL

400 mg BID

Drug: rilzabrutinib

Interventions

rilzabrutinibDRUG

Pharmaceutical form:Tablet-Route of administration:Oral

Also known as: SAR444671
rilzabrutinib

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (24)

Tulane University Health Sciences Center - Tulane Avenue-Investigational Site Number: 8400011

New Orleans, Louisiana, 70112, United States

RECRUITING

University of Michigan Health - Michigan Medicine - University Hospital-Investigational Site Number: 8400001

Ann Arbor, Michigan, 48109, United States

RECRUITING

Mayo Clinic_Investigational Site Number: 8400009

Rochester, Minnesota, 55905, United States

RECRUITING

New York Oncology Hematology-Investigational Site Number: 8400010

Albany, New York, 12208, United States

RECRUITING

Montefiore Medical Center-Investigational Site Number: 8400012

The Bronx, New York, 10467, United States

RECRUITING

Community Cancer Trials of Utah-Investigational Site Number: 8400002

Ogden, Utah, 84405, United States

RECRUITING

Investigational Site Number : 0400001

Vienna, 1140, Austria

RECRUITING

Investigational Site Number: 2500003

Créteil, 94000, France

RECRUITING

Investigational Site Number : 2500001

Dijon, 21079, France

RECRUITING

Investigational Site Number : 2500002

Pessac, 33604, France

RECRUITING

Investigational Site Number : 2500004

Toulouse, 31059, France

RECRUITING

Investigational Site Number : 3480002

Kaposvár, 7400, Hungary

RECRUITING

Investigational Site Number : 3800006

Naples, 80131, Italy

RECRUITING

Investigational Site Number : 3800007

Torino, 10126, Italy

RECRUITING

Investigational Site Number : 3800003

Vicenza, 36100, Italy

RECRUITING

Investigational Site Number : 6160002

Gdansk, 80-219, Poland

RECRUITING

Investigational Site Number : 6160003

Skorzewo, 60-185, Poland

RECRUITING

Investigational Site Number : 6160004

Słupsk, 76-200, Poland

RECRUITING

Investigational Site Number : 7240001

Barcelona, 8003, Spain

RECRUITING

Investigational Site Number : 7240002

Burgos, 9006, Spain

RECRUITING

Investigational Site Number : 7240007

Madrid, 28007, Spain

RECRUITING

Investigational Site Number : 7240008

Madrid, 28046, Spain

RECRUITING

Investigational Site Number : 7240005

Murcia, 30008, Spain

RECRUITING

Investigational Site Number : 7240004

Seville, 41013, Spain

RECRUITING

Related Links

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Condition Hierarchy (Ancestors)

Purpura, ThrombocytopenicPurpuraBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesThrombotic MicroangiopathiesThrombocytopeniaBlood Platelet DisordersCytopeniaHemorrhagic DisordersAutoimmune DiseasesImmune System DiseasesHemorrhagePathologic ProcessesPathological Conditions, Signs and SymptomsSkin ManifestationsSigns and Symptoms

Central Study Contacts

Trial Transparency email recommended (Toll free for US & Canada)

CONTACT

800-633-1610contact-us@sanofi.com

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 20, 2025

First Posted

June 6, 2025

Study Start

October 24, 2025

Primary Completion (Estimated)

November 5, 2027

Study Completion (Estimated)

December 30, 2028

Last Updated

May 1, 2026

Record last verified: 2026-04

Data Sharing

IPD Sharing
Will share

Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

Locations

HU(1)AU(1)ES(6)IT(3)FR(4)PL(3)US(6)