Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP)
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.
Timeline
- Start
- 2020-12-14
- Primary completion
- 2025-01-15
- Completion
- 2026-09-02
Outcome
Met primary endpoint
registry analysis (superiority test); DB Period: Adults: Placebo vs DB Period: Adults: Rilzabrutinib 400 mg BID; p <0.0001; Risk Difference (RD) 23.1 NCT04562766 ↗
paper was observed in 31 (23%) rilzabrutinib vs 0 placebo patients (P < .0001). PMID 40090011 ↗
release “LUNA 3 study met its primary endpoint demonstrating a significantly higher proportion of patients receiving rilzabrutinib achieved the primary endpoint of durable platelet response versus placebo.” sanofi.com ↗
release “Approval was based on the pivotal LUNA 3 phase 3 study, in which Wayrilz met the primary and secondary endpoints” sanofi.com ↗
Publications
- Kuter DJ, Ghanima W, Cooper N, Liebman HA, Zhang L, Hu Y, Miyakawa Y, Hutter-Kronke ML, Pascual-Izquierdo C, Lucchini E, Audia S, Cordoba M, Krenz H, Yao M, Daak A. Clinical activity and safety of rilzabrutinib in patients with previously treated immune thrombocytopenia (LUNA3): results from the open-label period of a phase 3 trial. Lancet Haematol. 2026 Sep 17:S2352-3026(26)00193-6. doi: 10.1016/S2352-3026(26)00193-6. Online ahead of print.
- Kuter DJ, Ghanima W, Cooper N, Liebman HA, Zhang L, Hu Y, Miyakawa Y, Homenda W, Galindo LEM, Basquiera AL, Tan CW, Saydam G, Hutter-Kronke ML, Chai-Adisaksopha C, Gomez-Almaguer D, Tran H, Shin HJ, Dantas da Cunha Junior A, Lazar Z, Izquierdo CP, Kirgner I, Lucchini E, Kuzmina G, Fillitz M, Audia S, Taparia M, Cordoba M, Diab R, Yao M, Gouia I, Lee M, Daak A. Safety and efficacy of rilzabrutinib vs placebo in adults with immune thrombocytopenia: the phase 3 LUNA3 study. Blood. 2025 Jun 12;145(24):2914-2926. doi: 10.1182/blood.2024027336.
- Lin J, Radhakrishnan J. What Are Baskets, Umbrellas, and Platforms Doing in Nephrology Clinical Trials? J Am Soc Nephrol. 2025 Feb 3;36(8):1652-1654. doi: 10.1681/ASN.0000000648. No abstract available.
- Kuter DJ, Bussel JB, Ghanima W, Cooper N, Gernsheimer T, Lambert MP, Liebman HA, Tarantino MD, Lee M, Guo H, Daak A. Rilzabrutinib versus placebo in adults and adolescents with persistent or chronic immune thrombocytopenia: LUNA 3 phase III study. Ther Adv Hematol. 2023 Oct 18;14:20406207231205431. doi: 10.1177/20406207231205431. eCollection 2023.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Rilzabrutinib | Unknown | 400 mg | Oral |