A Study of H3B-8800 (RVT-2001) in Participants With Lower Risk Myelodysplastic Syndromes
NCT ID: NCT02841540
Last Updated: 2024-02-14
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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TERMINATED
PHASE1
127 participants
INTERVENTIONAL
2016-10-06
2024-02-13
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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H3B-8800 (RVT-2001) Dose Escalation
H3B-8800 Acute Myeloid Leukemia or High Risk Myelodysplastic Syndromes/ Low Risk Myelodysplastic Syndromes/ Chronic Myelomonocytic Leukemia.
H3B-8800 (RVT-2001)
H3B-8800 (RVT-2001) orally at specified doses and schedules.
H3B-8800 (RVT-2001) MDS Expansion
Transfusion-dependent, lower-risk MDS subjects (very-low to intermediate risk categorization per IPSS-R) with missense mutations in SF3B1.
H3B-8800 (RVT-2001)
H3B-8800 (RVT-2001) orally at specified doses and schedules.
H3B-8800 (RVT-2001) Dose Optimization
Transfusion-dependent, lower-risk MDS subjects (very-low to intermediate risk categorization per IPSS-R) with missense mutations in SF3B1, and who have not been exposed to HMA's or lenalidomide in a prior line of therapy.
H3B-8800 (RVT-2001)
H3B-8800 (RVT-2001) orally at specified doses and schedules.
Interventions
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H3B-8800 (RVT-2001)
H3B-8800 (RVT-2001) orally at specified doses and schedules.
Eligibility Criteria
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Inclusion Criteria
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 \[HMA\]).
3. Eastern Cooperative Oncology Group (ECOG) performance score of 0-2.
4. For MDS expansion and Dose optimization cohorts - absolute neutrophil count (ANC) greater than or equal to (\>=) 500/ microliter (mcL) (0.5\*10\^9/L).
5. For expansion and Dose optimization cohorts- platelet count \>50,000/mcL (50\*10\^9/L).
6. For Dose-optimization cohort: No prior HMA or lenalidomide in participants with lower-risk MDS.
7. Adequate baseline organ function.
Exclusion Criteria
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.
18 Years
ALL
No
Sponsors
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Hemavant Sciences GmbH
INDUSTRY
Responsible Party
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Principal Investigators
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Keisuke Kuida, MD, PhD
Role: STUDY_DIRECTOR
Hemavant Sciences
Locations
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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
Centre Hospitalier Lyon Sud
Pierre-Bénite, , France
Azienda Ospedaliera Universitaria di Bologna - Policlinico S. Orsola Malpighi
Bologna, , Italy
Fondazione IRCCS Cà Granda Ospedale Policlinico Maggiore
Milan, , Italy
Fondazione IRCCS Policlinico San Matteo
Pavia, , Italy
IRCCS Istituto Clinico Humanitas Cancer Center
Rozzano, , Italy
National Cancer Center
Gyeonggi-do, Goyang-si, South Korea
Daegu Catholic University Medical Center
Daegu, , South Korea
Gachon University Gil Medical Center
Incheon, , South Korea
Asan Medical Center
Seoul, , South Korea
Hanyang University Seoul Hospital
Seoul, , South Korea
Samsung Medical Center
Seoul, , South Korea
Seoul National University Hospital
Seoul, , South Korea
The Catholic University of Korea Seoul St. Mary's Hospital
Seoul, , South Korea
Clinica Universidad de Navarra
Pamplona, Navarre, Spain
Hospital Universitario Valle de Hebrón
Barcelona, , Spain
Hospital General Universitario Gregorio Marañon
Madrid, , Spain
Hospital Universitario Fundación Jiménez Díaz
Madrid, , Spain
Complejo Asistencial Universitario de Salamanca
Salamanca, , Spain
Hospital Universitario y Politécnico La Fe de Valencia
Valencia, , Spain
Changhua Christian Hospital
Changhua, , Taiwan
Chang-Gung Memorial Hospital, Chiayi
Chiayi City, , Taiwan
China Medical University Hospital
Taichung, , Taiwan
National Cheng Kung University Hospital
Tainan City, , Taiwan
National Taiwan University Hospital
Taipei, , Taiwan
Countries
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References
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Foran JM, Sanz GF, Watts JM, Brunner AM, Fossard G, Della Porta MG, Tsai XC, Garcia-Manero G, Dimicoli-Salazar S, Fletcher L, Kim YJ, Font P, Alfonso-Pierola A, Alonso-Dominguez JM, Benton C, Hong J, Malcovati L, Mazure D, Lee JH, Yeh SP, Goursaud L, Barcellini W, Wu E, Corzo D, Kuida K, Stone RM. Phase 1 first-in-human dose-expansion study of the oral SF3B1 modulator H3B-8800 in lower-risk myelodysplastic syndrome. Leuk Res. 2025 Sep;156:107735. doi: 10.1016/j.leukres.2025.107735. Epub 2025 Jun 9.
Other Identifiers
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2016-001792-70
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
H3B-8800-G000-101
Identifier Type: -
Identifier Source: org_study_id