A Study of BH-30236 in Relapsed/ Refractory Acute Myelogenous Leukemia and Higher Risk Myelodysplastic Syndrome
A Phase 1/1b Open-Label, Dose Escalation, First-in- Human Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Preliminary Anti-leukemic Activity of the Orally Available CDC-Like Kinase (CLK) Inhibitor, BH-30236, in Adults With Relapsed or Refractory Acute Myelogenous Leukemia (R/R AML) or Higher-Risk Myelodysplastic Syndrome (HR-MDS)
1 other identifier
interventional
170
1 country
13
Brief Summary
Study BH-30236-01 is a first-in-human (FIH), Phase 1/1b, open-label, dose escalation and expansion study in participants with relapsed/refractory acute myelogenous leukemia (R/R AML) or higher-risk myelodysplastic syndrome (HR-MDS). Phase 1, Part 1 Dose Escalation - Monotherapy will evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary efficacy of BH-30236 administered orally. Approximately 50 participants may be enrolled in Phase 1, Part 1 Dose Escalation - Monotherapy. Phase 1, Part 2 Dose Escalation - Combination with Venetoclax will evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary efficacy of BH-30236 administered as a combination therapy with venetoclax. Approximately 48 participants may be enrolled in Phase 1, Part 2 Dose Escalation - Combination with Venetoclax. Phase 1b (Dose Expansion) will follow Phase 1 to further understand the relationships among dose, exposure, toxicity, tolerability, and clinical activity. Up to 72 participants may be enrolled in Phase 1b of the study as a monotherapy or in combination with venetoclax.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_1 leukemia
Started Jun 2024
13 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
June 17, 2024
CompletedStudy Start
First participant enrolled
June 19, 2024
CompletedFirst Posted
Study publicly available on registry
July 15, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 1, 2026
ExpectedStudy Completion
Last participant's last visit for all outcomes
June 1, 2027
September 24, 2025
September 1, 2025
2 years
June 17, 2024
September 19, 2025
Conditions
Outcome Measures
Primary Outcomes (3)
Dose Escalation: Frequency of dose limiting toxicities (DLTs)
DLTs are dose-limiting toxicities as defined in the study protocol.
Dose-limiting toxicities are collected during the first treatment cycle (28 days)
Dose Escalation and Expansion: Safety evaluation of BH-30236: Number of participants with treatment-related adverse events as assessed by National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) v5.0
Frequency, severity and relationship to study drug of AEs and SAEs
From first dose until 28 days after last dose of BH-30236
Dose Expansion: Composite Complete Remission (CR) Rate
Composite CR rate disease assessment in accordance with the following guidelines: European Leukemia Network (ELN) 2022 for acute myelogenous leukemia (AML) and International Working Group (IWG) 2023 for myelodysplastic syndrome (MDS).
From first dose of BH-30236 until disease progression (up to approximately 1 year)
Secondary Outcomes (10)
Dose Escalation and Expansion: Maximum observed blood concentration (Cmax) of BH-30236.
Evaluation performed in Cycle 1 (cycle duration is 28 days).
Dose Escalation: Area under the blood concentration time curve (AUC) of BH-30236.
Evaluation performed in Cycle 1 (cycle duration is 28 days).
Dose Escalation and Expansion: Concentration before dose at steady state (Ctrough).
Evaluation performed in all treatment cycles up to one year (cycle duration is 28 days).
Dose Escalation and Expansion: Objective Response Rate (ORR)
From first dose of BH-30236 until disease progression (up to approximately 1 year)
Dose Escalation and Expansion: Duration of Response (DoR)
Time from first documented response until disease progression or death (approximately 1 year).
- +5 more secondary outcomes
Study Arms (4)
Dose Escalation Cohort - Monotherapy
EXPERIMENTALBH-30236 Monotherapy for Dose Escalation
Dose Escalation Cohort - Combination with Venetoclax
EXPERIMENTALBH-30236 in Combination with Venetoclax for Dose Escalation.
Dose Expansion Cohort - Monotherapy
EXPERIMENTALBH-30236 based on Monotherapy dose escalation data.
Dose Expansion Cohort - Combination with Venetoclax
EXPERIMENTALBH-30236 in Combination with Venetoclax based on Dose Escalation data
Interventions
BH-30236 will be provided as either a 5 mg, 15 mg or 30 mg tablet. Participants will take BH-30236 tablets orally depending on their dose level assignment.
Venetoclax will be provided as 10 mg, 50 mg or 100 mg tablets. Participants will take venetoclax orally per label instructions.
Eligibility Criteria
You may qualify if:
- ≥18 years.
- Prior treatment history must include 1-5 prior lines of therapy.
- ECOG performance status ≤2.
- Adequate organ function evidenced by the following laboratory values:
- Hepatic: Transaminase levels aspartate aminotransferase \[AST\]/ alanine transaminase \[ALT\] ≤ 2.5 × upper limit of normal (ULN). In cases of liver involvement by AML or MDS, AST and ALT \< 5.0 × ULN is acceptable. Total bilirubin ≤ 1.5 × ULN in the absence of documented Gilbert's disease.
- Renal: Measured or calculated creatinine clearance ≥ 60 mL/min (Cockcroft-Gault formula)
You may not qualify if:
- Diagnosis of acute promyelocytic leukemia or chronic myeloid leukemia with blast crisis.
- Prior allogeneic HSCT within 3 months or donor lymphocyte infusion within 30 days of start of therapy;
- Active and uncontrolled infections.
- Unresolved AEs greater than Grade from prior therapies.
- History of other active malignancy (with certain exceptions)
- Prior treatment with a CLK inhibitor.
- Any acute or chronic graft versus host disease requiring systemic therapy within 4 weeks prior to study drug administration with the exception of topical steroids or the equivalent of 20 mg of prednisone or less.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (13)
City of Hope Medical Center
Duarte, California, 91010, United States
University of California Los Angeles
Los Angeles, California, 90095, United States
Stanford Cancer Center
Palo Alto, California, 94304, United States
Sylvester Comprehensive Cancer Center
Miami, Florida, 33136, United States
Moffitt Cancer Center
Tampa, Florida, 33612, United States
Northwestern Medicine - Northwestern Memorial Hospital Galter Pavilion
Chicago, Illinois, 60611, United States
Roswell Park Cancer Institute
Buffalo, New York, 14263, United States
Memorial Sloan Kettering Cancer Center
New York, New York, 10065, United States
The Ohio State University Wexner Medical Center - James Cancer Hosp
Columbus, Ohio, 43210, United States
Sarah Cannon Research Institute
Nashville, Tennessee, 37203, United States
The University of Texas M.D. Anderson Cancer Center
Houston, Texas, 77030, United States
Fred Hutchinson Cancer Center
Seattle, Washington, 98109, United States
University of Wisconsin Clinical Science Center
Madison, Wisconsin, 53792, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Sponsor Contact
BlossomHill Therapeutics, Inc.
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
June 17, 2024
First Posted
July 15, 2024
Study Start
June 19, 2024
Primary Completion (Estimated)
June 1, 2026
Study Completion (Estimated)
June 1, 2027
Last Updated
September 24, 2025
Record last verified: 2025-09
Data Sharing
- IPD Sharing
- Will not share