A Study of H3B-8800 (RVT-2001) in Participants With Lower Risk Myelodysplastic Syndromes

Phase 1 Terminated 127 enrolled Hemavant Sciences GmbH

Non-randomizedParallel-groupOpen-labelTreatment

Summary

A Phase 1, an Open-label, Multicenter Phase 1 Trial to Evaluate the Safety, Pharmacokinetics and Pharmacodynamics of Splicing Modulator H3B-8800 (RVT-2001) for Subjects With Myelodysplastic Syndromes, Acute Myeloid Leukemia, and Chronic Myelomonocytic Leukemia

Timeline

Start: 2016-10-06
Primary completion: 2024-02-13
Completion: 2024-02-13

Outcome

Missed primary endpoint

Stopped: “The study was terminated after conducting an interim analysis of the available data where it became evident that the observed efficacy results were not in alignment with the initially set expectations.”

paper However, the low RBC-TI rate suggests insufficient efficacy of H3B-8800 at the dose levels tested. PMID 40651101 ↗

Publications

Foran JM, Sanz GF, Watts JM, Brunner AM, Fossard G, Della Porta MG, Tsai XC, Garcia-Manero G, Dimicoli-Salazar S, Fletcher L, Kim YJ, Font P, Alfonso-Pierola A, Alonso-Dominguez JM, Benton C, Hong J, Malcovati L, Mazure D, Lee JH, Yeh SP, Goursaud L, Barcellini W, Wu E, Corzo D, Kuida K, Stone RM. Phase 1 first-in-human dose-expansion study of the oral SF3B1 modulator H3B-8800 in lower-risk myelodysplastic syndrome. Leuk Res. 2025 Sep;156:107735. doi: 10.1016/j.leukres.2025.107735. Epub 2025 Jun 9.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	H3B-8800	Unknown	5 mg	Oral
Subject	H3B-8800	Unknown	10 mg	Oral