A study to test how well an oral medication called rilzabrutinib works and how safe it is for adults with immune thrombocytopenia (ITP) who did not respond to first-line treatments such as corticosteroids, intravenous immunoglobulin, and/or anti-D immunoglobulin.

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Sanofi-Aventis Recherche & Developpement

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

Trial duration

15 Dec 2025 → ongoing

Decision date (initial)

2025-10-13

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

External identifiers

EU CT number

2025-522070-36-00

WHO UTN

U1111-1320-4669

ClinicalTrials.gov

NCT07007962

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety, Others

The main goal of this study is to investigate if rilzabrutinib can provide a long-lasting increase in platelet count in adults with primary ITP who fail first-line treatment.

Secondary objectives 1

1. The study will also check how well rilzabrutinib works in these adults during the study and assess its safety and tolerability, as well as quality of life.

Conditions and MedDRA coding

immune thrombocytopenia (ITP)

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 10

1. I 01. Participant must be 18 or above years of age inclusive, at the time of signing the informed consent.
2. I 02. Male and female participants with a documented diagnosis of primary ITP in the medical history.
3. I 03. Participants with Eastern Cooperative Oncology Group (ECOG) performance status grade 2 or lower.
4. I 04. Participant received at least one course of first-line therapy per international guidelines or local standard of care (eg, oral or parenteral CS, IVIg, anti-D). Note: Participants may have received multiple courses of first-line therapy, either sequentially or in combination.
5. I 05. History of platelet response while on-treatment with first-line therapy.
6. I 06. Participant has loss of response, relapse, or steroid dependency.
7. I 07. Please see study protocol.
8. I 08. All contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
9. I 09. Adequate hematologic, hepatic, and renal function (hemoglobin >9 g/dL within 1 week prior to Study Day 1), absolute neutrophil count ≥1500/μL,, AST and ALT ≤1.5×upper limit of normal (ULN), albumin ≥3 g/dL, total bilirubin ≤1.5×ULN (unless the participant has documented Gilbert syndrome), estimated glomerular filtration rate (GFR) >50mL/min/1.73m² (CKD-EPI 2021 Creatinine Equation [Race-Free]), International normalized ratio (INR) and activated partial thromboplastin time (APTT) values are within 20% of the normal ranges.
10. I 10. Capable of giving signed informed consent as described in Appendix 1 Section 10.1 of the protocol which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol. In countries where legal age of majority is above 18 years, a specific ICF must also be signed by the participant’s legally authorized representative (Appendix 1 Section 10.1.3).

Exclusion criteria 30

1. E 01. Participants with diagnosis of secondary ITP.
2. E 02. Participants with a diagnosis of Evans syndrome.
3. E 03. Participants with history of myelodysplastic syndrome
4. E 04. Participant with history of or current life-threatening bleeding (eg., a bleed that results in hemodynamic instability or respiratory compromise) due to ITP.
5. E 05. Participants with medical history of lymphoma, leukemia, or any malignancy within the past 5 years except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for the past 3 years.
6. E 06. Participants with history of solid organ transplant.
7. E 07. Participants with history of coagulation or bleeding disorders, including genetic conditions, other than ITP.
8. E 08. Please see the study protocol.
9. E 09. Positive COVID-19 molecular test (if COVID-19 testing required per local guidelines to be determined for each site).
10. E 18. Participant received subsequent/advanced treatment for the treatment of ITP or was splenectomized before Study Day 1
11. E 19. Participant received drugs known to potentially improve thrombocytopenia to treat other conditions within 5 times the elimination half-life of the drug or within 14 days of Study Day 1, whichever is longer, or the participant is planned to receive treatment with drugs known to potentially improve thrombocytopenia for any indication during the course of the study.
12. E 10. HIV infection.
13. E 20. Please see study protocol.
14. E 21. Please see study protocol.
15. E 22. Please see study protocol.
16. E 23. Please see study protocol.
17. E 24. Please see study protocol.
18. E 25. Received any investigational drug within 6 months or 5 half-lives, whichever is longer, or is participating in another clinical trial.
19. E 26. Participant during pregnancy or nursing.
20. E 27. Individuals accommodated in an institution because of regulatory or legal order; prisoners or participants who are legally institutionalized.
21. E 28. Participant not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or participants potentially at risk of noncompliance to study procedures.
22. E 11. A history of active or latent tuberculosis (TB) (unless the participant has completed a full course of anti-tuberculosis therapy or it is documented by a specialist that the participant has been adequately treated and can begin treatment with an immunosuppressive agent). A positive test for TB (QuantiFERON-TB-Gold [QFT] or equivalent) performed at the screening visit or within the 3 months prior to screening.
23. E 29. Participants are employees of the clinical study site or other individuals directly involved in the conduct of the study, or immediate family members of such individuals (in conjunction with section 1.61 of the International Council for Harmonization Good Clinical Practice [GCP] Ordinance E6).
24. E 30. Sensitivity to any of the study intervention, or components thereof, or drug or other allergy that, in the opinion of the Investigator, contraindicates participation in the study.
25. E 12. Participants with uncontrolled or active HBV infection: Participants with positive HBsAg and/or HBV DNA.
26. E 13. Participants with active HCV infection: positive HCV-RNA and positive anti-HCV.
27. E 14. Current drug or alcohol abuse.
28. E 15. Refractory nausea and vomiting, malabsorption, external biliary shunt, significant bowel resection, or any other condition that would preclude adequate study drug absorption.
29. E 16. Participants who have undergone major surgery within 28 days prior to Study Day 1 or have planned surgery in the time frame of the study.
30. E 17. Please see study protocol.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. The main study endpoint is to check the durable platelet response ie, to see how many participants can keep their platelet count at a safe level.
2. The durable platelet response is the percentage of participants who can maintain a platelet count of at least 50,000 /μL (or between 30,000/μL and 50,000/μL and at least double their starting count) for at least half of their scheduled platelet checks every 2 weeks and for at least 4 valid visits in the last 12 weeks of the study, without needing rescue treatment.

Secondary endpoints 4

1. 1. Overall platelet response: The percentage of participants who can achieve 2 platelet counts, at least 5 days apart, of at least 50,000/μL (or between 30,000/μL and 50,000/μL but at least double their starting count) without needing rescue treatment in the 4 weeks before the first high platelet count.
2. 2. Duration of platelet response: The total number and proportion of weeks where participants maintain platelet counts of at least 50,000/μL (or between 30,000/μL and 50,000/μL but at least double their starting count) without needing rescue treatment in the 4 weeks before the high platelet count in participants who achieve a response.
3. 3. Bleeding: The change in immune thrombocytopenia bleeding scale score from the start of the study to the end of Week 28.
4. 4. Corticosteroid-sparing effect: The percentage of participants who can stop or reduce their corticosteroid dose by at least 50% or to less than 5 mg/day (prednisone equivalent) from the start of the study by the end of Week 28

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Sanofi-Aventis Recherche & Developpement

Sponsor organisation

Sanofi-Aventis Recherche & Developpement

Address

82 Avenue Raspail

City

Gentilly

Postcode

94250

Country

France

Scientific contact point

Organisation

Sanofi-Aventis Recherche & Developpement

Contact name

Clinical Sciences and Operations

Public contact point

Organisation

Sanofi-Aventis Recherche & Developpement

Contact name

Clinical Sciences and Operations

Locations

8 EU/EEA countries · 44 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 80 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

3 submissions — initial application plus substantial / non-substantial modifications