Testing BH-30236 for relapsed or 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)

This study is testing a new drug called BH-30236 to see if it can help adults with relapsed or hard-to-treat acute myelogenous leukemia or higher-risk myelodysplastic syndrome.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 170 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: BlossomHill Therapeutics (Industry-sponsored)
• Drugs / interventions: prednisone
• Locations: 13 sites (Duarte, California and 12 other locations)
• Trial ID: NCT06501196 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the safety and effectiveness of BH-30236, a CLK inhibitor, in adults with relapsed or refractory acute myelogenous leukemia (R/R AML) or higher-risk myelodysplastic syndrome (HR-MDS). The study is divided into two phases: Phase 1 focuses on dose escalation to determine the maximum tolerated dose and preliminary efficacy, while Phase 1b involves dose expansion to further assess safety and anti-leukemic activity at the recommended doses. Approximately 74 participants will be enrolled across both phases, with careful monitoring of pharmacokinetics and pharmacodynamics.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 and older with relapsed or refractory acute myelogenous leukemia or higher-risk myelodysplastic syndrome.

Not a fit: Patients with acute promyelocytic leukemia or chronic myeloid leukemia with blast crisis may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with difficult-to-treat forms of leukemia.

How similar studies have performed: While this approach is novel, similar studies targeting other forms of leukemia have shown promising results.

Eligibility criteria

Inclusion criteria:

* ≥18 years.
* Diagnosis of relapsed/refractory acute myelogenous leukemia (R/R) AML or higher-risk myelodysplastic syndrome (HR-MDS) with ≥5% bone marrow blast at time of inclusion.
* 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)

The above are a summary, other inclusion criteria details may apply.

Exclusion Criteria:

* 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.

The above is a summary, other exclusion criteria details may apply.

Where this trial is running

Duarte, California and 12 other locations

• City of Hope Medical Center — Duarte, California, United States (Recruiting)
• University of California Los Angeles — Los Angeles, California, United States (Recruiting)
• Stanford Cancer Center — Palo Alto, California, United States (Recruiting)
• Sylvester Comprehensive Cancer Center — Miami, Florida, United States (Recruiting)
• Moffitt Cancer Center — Tampa, Florida, United States (Recruiting)
• Northwestern Medicine - Northwestern Memorial Hospital Galter Pavilion — Chicago, Illinois, United States (Recruiting)
• Roswell Park Cancer Institute — Buffalo, New York, United States (Recruiting)
• Memorial Sloan Kettering Cancer Center — New York, New York, United States (Recruiting)
• The Ohio State University Wexner Medical Center - James Cancer Hosp — Columbus, Ohio, United States (Recruiting)
• Sarah Cannon Research Institute — Nashville, Tennessee, United States (Recruiting)
• The University of Texas M.D. Anderson Cancer Center — Houston, Texas, United States (Recruiting)
• Fred Hutchinson Cancer Center — Seattle, Washington, United States (Recruiting)
• University of Wisconsin Clinical Science Center — Madison, Wisconsin, United States (Recruiting)

Study contacts

• Study coordinator: Sponsor Contact

Conditions Leukemia; Leukemia, Myeloid; Leukemia, Myeloid, Acute; Preleukemia; Myelodysplastic Syndromes; Refractory Acute Myeloid Leukemia