A Study of H3B-8800 (RVT-2001) in Participants With Lower Risk Myelodysplastic Syndromes
Stopped early · Phase 1
Conditions studied: Leukemia, Myeloid, Acute, Myelodysplastic Syndromes, Leukemia, Myelomonocytic, Chronic
In brief
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
Key facts
- Study ID
- NCT02841540
- Run by
- Hemavant Sciences GmbH
- People needed
- 127
- Starts
- 2016-10-06
- Expected to finish
- 2024-02-13
- Last updated by the study team
- 2024-02-14
Who can join
Age: 18 and older. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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.
You may not qualify if…
- 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.
Where it is running
- 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
- Mayo Clinic — Rochester, Minnesota, United States
- Oncology Associates of Oregon — Eugene, Oregon, United States
- Texas Oncology — Austin, Texas, United States
- MD Anderson Cancer Center — Houston, Texas, United States
- Virginia Cancer Specialist — Fairfax, Virginia, United States
- Algemeen Ziekenhuis Klina — Brasschaat, Belgium
- AZ Sint-Jan Brugge Oostende AV — Bruges, Belgium
- Universiteit Gent — Ghent, Belgium
- University Hospitals Leuven — Leuven, Belgium
- Institut Gustave Roussy — Villejuif, Val-de-Marne, France
- CHU Amiens-Picardie — Amiens, France
- Centre Hospitalier Universitaire d'Angers (CHU d'Angers) — Angers, France
- Centre Hospitalier Universitaire (CHU) de Bordeaux — Bordeaux, France
- Centre Hospitalier - Le Mans — Le Mans, France
- Hôpital Claude Huriez — Lille, France
Full record on ClinicalTrials.gov
Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.