NCT02841540

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

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

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 127 (actual)
• Sponsor: Hemavant Sciences GmbH · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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

Detailed description

This study is a Phase 1, open-label, first-in-human (FIH) study designed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary antitumor activity of H3B-8800 (RVT-2001) in subset of participants with Myelodysplastic Syndromes (MDS), Acute Myeloid Leukemia (AML), or Chronic Myelomonocytic Leukemia (CMML). The study consists of three parts, the dose escalation part (Part 1) exploring multiple once daily (QD) schedules and MDS Expansion part (Part 2) and Dose Optimization part (Part 3) exploring dosing schedules in lower-risk MDS.

Conditions

• Leukemia, Myeloid, Acute
• Myelodysplastic Syndromes
• Leukemia, Myelomonocytic, Chronic

Interventions

Type	Name	Description
DRUG	H3B-8800 (RVT-2001)	H3B-8800 (RVT-2001) orally at specified doses and schedules.

Timeline

• Start date: 2016-10-06
• Primary completion: 2024-02-13
• Completion: 2024-02-13
• First posted: 2016-07-22
• Last updated: 2024-02-14

Locations

49 sites across 7 countries: United States, Belgium, France, Italy, South Korea, Spain, Taiwan

Regulatory

• FDA-regulated drug study