Trial Search Results
A Study of H3B-8800 in Participants With Myelodysplastic Syndromes, Acute Myeloid Leukemia, and Chronic Myelomonocytic Leukemia
The main purpose of this study is to determine the safety of the study drug H3B-8800 in humans and to understand better how the study drug works in your type of cancer. The study will also collect information on how long you live and the side effects that you may experience during the study.
Stanford is currently accepting patients for this trial.
H3 Biomedicine Inc.
Collaborator: Eisai Inc.
- Drug: H3B-8800
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 at a variant allele frequency of 5 percent (%) or higher.
2. The participant 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 dose escalation portion, participants with lower-risk MDS/CMML must be
transfusion-dependent for red blood cells or platelets (as determined by instructional
practices or local standard of care). For enrollment MDS expansion cohort, lower risk
MDS participants must be RBC transfusion dependent according to IWG 2006 criteria
(having received at least 4 units (U) of RBCs within 8 weeks for hemoglobin (Hb) of
less than (<) 9 gram per deciliter (g/dL) prior to first dose of H3B-8800).
Participants who are RBC transfusion-dependent must also have failed erythropoiesis
stimulating agents (ESA) (primary resistance or relapse after a response) or have
serum erythropoietin (EPO) levels greater than (>) 500 units per liter (U/L).
C. Participants with AML must either refuse or not be considered candidates for
intensive induction chemotherapy using consensus criteria for defining such
participants. Previously treated participants should have evidence of persistent or
recurrent AML in the peripheral blood and/or bone marrow that is refractory to, or has
relapsed from, their most recent prior line of treatment. For AML, participants must
have white blood cells (WBC) < 15*10^9/liter (L).
D. 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 expansion cohort- absolute neutrophil count (ANC) greater than or equal to (>=)
500/ microliter (mcL) (0.5*10^9/L).
5. For expansion cohort- platelet count >50,000/mcL (50*10^9/L).
6. Adequate baseline organ function.
1. Diagnosis of a core binding factor leukemia (t(8;21), t(16;16) or inv(16)).
2. Participant is deemed candidate for hematopoietic stem cell transplants at the time of
enrollment (for AML participants only).
3. Family history of Leber Hereditary Optic Neuropathy, Autosomal Dominant Optic Atrophy,
Late-Onset Retinal Degeneration, Familial Dysautonomia or other hereditary
mitochondrial disease, unless the causative mutation(s) in the family have been
determined and the participant has tested negative for the mutation(s).
4. Known prior or current retinal or optic nerve disease (example, Retinitis Pigmentosa,
diabetic retinopathy, optic neuritis) based on screening ophthalmology assessment for
5. Corrected vision is worse than 20/40 unless due to cataracts.
6. Vitamin B12, folate or vitamin A deficiency. Rescreening following repletion therapy
Ages Eligible for Study
18 Years - N/A
Genders Eligible for Study