A Phase 1 Study to Evaluate H3B-8800 in Participants With Myelodysplastic Syndromes, Acute Myeloid Leukemia, and Chronic Myelomonocytic Leukemia



Status:Recruiting
Conditions:Blood Cancer, Blood Cancer, Blood Cancer, Blood Cancer, Hematology
Therapuetic Areas:Hematology, Oncology
Healthy:No
Age Range:18 - Any
Updated:2/24/2019
Start Date:October 6, 2016
End Date:December 1, 2019
Contact:Eisai Medical Information
Email:esi_oncmedinfo@eisai.com
Phone:1-888-274-2378

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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

This is a Phase 1, open-label, first-in-human (FIH) study designed to evaluate the safety,
tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary activity of
H3B-8800 in participants with Myelodysplastic Syndromes (MDS), Acute Myeloid Leukemia (AML),
or Chronic Myelomonocytic Leukemia (CMML). The study consists of two parts, a dose escalation
part (Part 1) and an expansion part (Part 2) exploring a multiple once daily (QD) schedules
at the recommended phase 2 dose (RP2D).


Inclusion Criteria:

1. Confirmed diagnosis of MDS, CMML, or AML.

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. 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). Participants who are red blood cell transfusion-dependent must also have failed
erythropoiesis stimulating agents (ESA) (primary resistance or relapse after a
response) or have serum EPO levels > 500 U/L. These lower-risk participants must have
platelet counts above 50,000 mm^3 in the absence of transfusion for 8 weeks.

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 WBC < 15 × 10^9/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. Adequate baseline organ function

Exclusion Criteria:

1. Diagnosis of a core binding factor leukemia (t(8;21), t(16;16) or inv(16)).

2. Participant is candidate for hematopoietic stem cell transplants at the time of
enrollment.

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
eligibility.

5. Corrected vision is worse than 20/40 unless due to cataracts.

6. Vitamin B12, folate or vitamin A deficiency. Rescreening following repletion therapy
is acceptable.
We found this trial at
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Denver, Colorado 80218
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185 Cambridge Street
Boston, Massachusetts 02114
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330 Brookline Ave
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Chapel Hill, North Carolina 27599
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5801 South Ellis Avenue
Chicago, Illinois 60637
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9500 Euclid Avenue
Cleveland, Ohio 44106
216.444.2200
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Miami, Florida 33124
(305) 284-2211
University of Miami A private research university with more than 15,000 students from around the...
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1100 Fairview Avenue North
Seattle, Washington 98109
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Fred Hutchinson Cancer Research Center At Fred Hutchinson Cancer Research Center, our interdisciplinary teams of...
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12902 USF Magnolia Dr
Tampa, Florida 33612
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H. Lee Moffitt Cancer Center & Research Institute Moffitt Cancer Center in Tampa, Florida, has...
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450 Brookline Ave
Boston, Massachusetts 2215
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Buffalo, New York 14263
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1275 York Ave
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Rochester, Minnesota 55905
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Villejuif, Val-de-Marne 94805
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