Acute Myeloid Leukemia Clinical Trial

A Study of H3B-8800 (RVT-2001) in Participants With Myelodysplastic Syndromes, Acute Myeloid Leukemia, and Chronic 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

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Full 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.

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Eligibility Criteria

Inclusion Criteria:

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.

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]).

Eastern Cooperative Oncology Group (ECOG) performance score of 0-2.
For MDS expansion and Dose optimization cohorts - absolute neutrophil count (ANC) greater than or equal to (>=) 500/ microliter (mcL) (0.5*10^9/L).
For expansion and Dose optimization cohorts- platelet count >50,000/mcL (50*10^9/L).
For Dose-optimization cohort: No prior HMA or lenalidomide in participants with lower-risk MDS.
Adequate baseline organ function.

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.

Study is for people with:

Acute Myeloid Leukemia

Phase:

Phase 1

Estimated Enrollment:

200

Study ID:

NCT02841540

Recruitment Status:

Recruiting

Sponsor:

Hemavant Sciences GmbH

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There are 27 Locations for this study

See Locations Near You

Stanford Cancer Center
Stanford California, 94305, United States
SCRI - Colorado Blood Cancer Institute
Denver Colorado, 80218, United States
Mayo Clinic Jacksonville
Jacksonville Florida, 32224, United States
University of Miami
Miami Florida, 33136, United States
H Lee Moffitt Cancer Center and Research Institute
Tampa Florida, 33612, United States
University of Chicago
Chicago Illinois, 60637, United States
Massachusetts General Hospital
Boston Massachusetts, 02114, United States
Beth Israel Deaconess Medical Center
Boston Massachusetts, 02115, United States
Dana Farber Cancer Institute
Boston Massachusetts, 02215, United States
Karmanos Cancer Institute
Detroit Michigan, 48201, United States
Mayo Clinic
Rochester Minnesota, 55905, United States
Rosewell Park Cancer Center
Buffalo New York, 14263, United States
Memorial Sloan Kettering Cancer Center
New York New York, 10065, United States
University of North Carolina Chapel Hill
Chapel Hill North Carolina, 27514, United States
Cleveland Clinic
Cleveland Ohio, 21287, United States
SCRI - Tennessee Oncology
Nashville Tennessee, 37203, United States
MD Anderson Cancer Center
Houston Texas, 77030, United States
Fred Hutchinson Cancer Research Center
Seattle Washington, 98109, United States
Institut Gustave Roussy
Villejuif Val-de-Marne, 94805, France
Hôpital Saint Louis
Paris , 75475, France
Eisai Trial Site 1
Hannover Lower Saxony, 30625, Germany
Eisai Trial Site 2
Dresden Sachsen, 01307, Germany
Eisai Trial Site 3
Leipzig Sachsen, 04103, Germany
Clinica Universidad Navarra
Pamplona Navarra, 31008, Spain
Hospital General Universitario Gregorio Marañon
Madrid , 28007, Spain
Hospital Universitario Fundacion Jimenez Diaz
Madrid , 28040, Spain
Hospital Universitario HM Sanchinarro - CIOCC
Madrid , 28050, Spain

How clear is this clinincal trial information?

Study is for people with:

Acute Myeloid Leukemia

Phase:

Phase 1

Estimated Enrollment:

200

Study ID:

NCT02841540

Recruitment Status:

Recruiting

Sponsor:


Hemavant Sciences GmbH

How clear is this clinincal trial information?

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