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

  • STATUS
    Recruiting
  • End date
    Dec 1, 2024
  • participants needed
    200
  • sponsor
    Hemavant Sciences GmbH
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Updated on 4 April 2023
See if I qualify
myeloid leukemia
myelodysplastic syndromes
hydroxyurea
chronic myelomonocytic leukemia
decitabine
leukemia
bone marrow procedure
induction chemotherapy
azacitidine
lenalidomide
blood transfusion
myelomonocytic leukemia

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

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.

Details
Condition Leukemia, Myeloid, Acute, Myelodysplastic Syndromes, Leukemia, Myelomonocytic, Chronic
Treatment H3B-8800, H3B-8800 (RVT-2001)
Clinical Study IdentifierNCT02841540
SponsorHemavant Sciences GmbH
Last Modified on4 April 2023

Eligibility

 Yes No Not Sure

Inclusion Criteria

Confirmed diagnosis of MDS, CMML, or AML
For the MDS Expansion cohort, participants must be lower-risk MDS, defined as
Participants with higher-risk MDS/CMML must be intolerant of hypomethylating agents (HMAs) or not have responded to 4 treatment cycles of decitabine or 6 treatment cycles of azacitidine, or must have progressed at any point after initiation of an HMA
low or intermediate-1 risk categorization per International Prognostic Scoring
System (IPSS) criteria that carries a missense SF3B1 mutation
For the Dose Optimization cohort, participants must be transfusion-dependent
lower-risk MDS, defined as very-low to intermediate risk categorization per
Eastern Cooperative Oncology Group (ECOG) performance score of 0-2
IPSS-R criteria that carries a missense SF3B1 mutation
Participants must meet the following criteria relevant to their specific diagnosis
Adequate baseline organ function
For the Dose Escalation portion, participants with lower-risk MDS/CMML must be transfusion-dependent for red blood cells or platelets
For the MDS expansion cohort, lower risk MDS participants must be RBC transfusion
dependent according to IWG 2006 criteria and must also have failed erythropoiesis
stimulating agents (ESA) or have serum erythropoietin (EPO) levels greater than (>)
units per liter (U/L)
C. For the Dose Optimization cohort, lower-risk MDS participants must be RBC
transfusion-dependent at baseline defined as ≥3 RBC units (concentrates) in 16-weeks
in at least 2 transfusion episodes prior to the first dose of H3B-8800 (RVT-2001) and
must also have failed ESA or have serum EPO levels > 500 U/L. Any ESA use should be
discontinued ≥6 weeks prior to enrollment
D. Participants with AML must either refuse or not be considered candidates for
intensive induction chemotherapy using consensus criteria for defining such
participants
E. Participants with CMML must have been treated with at least one prior therapy
(hydroxyurea or a hypomethylating agent [HMA])
For MDS expansion and Dose optimization cohorts - absolute neutrophil count (ANC)
greater than or equal to (>=) 500/ microliter (mcL) (0.510^9/L)
For expansion and Dose optimization cohorts- platelet count >50,000/mcL (5010^9/L)
For Dose-optimization cohort: No prior HMA or lenalidomide in participants with
lower-risk MDS

Exclusion Criteria

Diagnosis of a core binding factor leukemia (t(8;21), t(16;16) or inv(16)). Diagnosis
of acute promyelocytic leukemia (t(15;17))
Participants are deemed candidate for hematopoietic stem cell transplants at the time
of enrollment (for AML participants only)
Known prior or current retinal or optic nerve disease (example, Retinitis Pigmentosa
diabetic retinopathy, optic neuritis) not stable for at least 6 months
History of clinically significant, uncorrected vitamin B12 or folate deficiency
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myeloid leukemia
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hydroxyurea
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decitabine
leukemia
bone marrow procedure
induction chemotherapy
azacitidine
lenalidomide
blood transfusion
myelomonocytic leukemia

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