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A Study of H3B-8800 (RVT-2001) in Participants With Lower Risk Myelodysplastic Syndromes | Clinical Trials | Clareo Health

TERMINATEDPHASE1

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

NCT02841540•Hemavant Sciences GmbH

View on ClinicalTrials.gov

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.

Eligibility

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•1. Confirmed diagnosis of MDS, CMML, or AML.
•For the MDS Expansion cohort, participants must be lower-risk MDS, defined as 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 IPSS-R criteria that carries a missense SF3B1 mutation.
•2. Participants must meet the following criteria relevant to their specific diagnosis:
•A. 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.
•B. 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 (\>) 500 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 \[HMA\]).
+5 more criteria

Exclusion Criteria

•1. Diagnosis of a core binding factor leukemia (t(8;21), t(16;16) or inv(16)). Diagnosis of acute promyelocytic leukemia (t(15;17))
•2. Participants are deemed candidate for hematopoietic stem cell transplants at the time of enrollment (for AML participants only).
•3. Known prior or current retinal or optic nerve disease (example, Retinitis Pigmentosa, diabetic retinopathy, optic neuritis) not stable for at least 6 months.
•4. History of clinically significant, uncorrected vitamin B12 or folate deficiency.

Locations (49)

Arizona Oncology Associates

Tucson, Arizona, United States

City of Hope

Duarte, California, United States

City of Hope

Irvine, California, United States

Rocky Mountain Cancer Center

Aurora, Colorado, United States

Mayo Clinic Jacksonville

Jacksonville, Florida, United States

University of Miami

Miami, Florida, United States

Massachusetts General Hospital

Boston, Massachusetts, United States

Beth Israel Deaconess Medical Center

Boston, Massachusetts, United States

Dana Farber Cancer Institute

Boston, Massachusetts, United States

Karmanos Cancer Institute

Detroit, Michigan, United States

Key Dates

Start Date

October 6, 2016

Primary Completion Date

February 13, 2024

Completion Date

February 13, 2024

Last Updated

February 14, 2024

Enrollment

127

ACTUAL participants

Conditions

Leukemia, Myeloid, AcuteMyelodysplastic SyndromesLeukemia, Myelomonocytic, Chronic

Interventions

H3B-8800 (RVT-2001)

DRUG

A Study of Bleximenib, Venetoclax and Azacitidine For Treatment of Participants With Newly Diagnosed Acute Myeloid Leukemia (AML)

NCT06852222

Leukemia, Myeloid, Acute

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RECRUITINGPHASE1

Phase Ib Study of CD33 FPBMC in Patients With MRD+ AML or MDS

NCT07270978

Acute Myeloid LeukemiaMyelodysplastic Syndromes+1 more

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: February 14, 2024Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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

Inclusion Criteria

Exclusion Criteria

A Study of Bleximenib, Venetoclax and Azacitidine For Treatment of Participants With Newly Diagnosed Acute Myeloid Leukemia (AML)

Phase Ib Study of CD33 FPBMC in Patients With MRD+ AML or MDS

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