Actively Recruiting
A First-in-human, Phase 1, Dose Escalation and Expansion Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of GLB-001 in Patients With Relapsed or Refractory Acute Myeloid Leukemia or Relapsed or Refractory Higher-risk Myelodysplastic Syndromes
Led by GluBio Therapeutics Inc. · Updated on 2026-04-13
48
Participants Needed
8
Research Sites
14 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating GLB-001, an oral drug, in a first-in-human Phase 1 clinical trial for adults with relapsed or refractory acute myeloid leukemia RR AML or relapsed or refractory higher-risk myelodysplastic syndromes RR HR-MDS. The study aims to assess the safety, tolerability, how the drug moves and acts in the body, and initial effectiveness. The trial includes a dose escalation phase to find the highest safe dose and a dose expansion phase to confirm tolerability and explore clinical activity. The trial uses a standard dose-escalation design in Phase 1a to evaluate several dose levels of GLB-001, adjusting dose amounts or frequency based on safety and pharmacokinetic data. After determining the maximum tolerated or administered dose, Phase 1b will enroll participants at one or two selected dose levels to confirm tolerability and assess whether the drug shows enough activity to continue development. GLB-001 is given orally based on the assigned treatment schedule. Participants will be closely monitored for dose-limiting toxicities during the first 28 days, as well as adverse events over up to two years. Researchers will measure drug levels in the body, response rates such as remission, progression-free survival, and overall survival. Safety reviews will guide dose selection for future studies. The total study duration includes long-term follow-up to track outcomes and safety for up to two years after treatment begins.
CONDITIONS
Brief Title
A Study of GLB-001 in Patients With Relapsed or Refractory Acute Myeloid Leukemia or Relapsed or Refractory Higher Risk Myelodysplastic Syndromes
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants are 18 years of age or older at the time of signing the consent form.
- Participants must understand and voluntarily sign the informed consent form before any study procedures.
- Participants are willing and able to follow the study visit schedule and protocol requirements.
- Participants have histologically or cytologically confirmed acute myeloid leukemia (de novo or secondary) or higher-risk myelodysplastic syndromes.
- Participants have relapsed or refractory disease and have failed or are ineligible for all available beneficial therapies.
- Participants must have total white blood cell count less than 25 x 10^9/L before first dose.
- Liver enzymes AST and ALT must be ≤3 times upper limit of normal unless due to leukemic liver involvement (then ≤5 times).
- Serum total bilirubin must be ≤1.5 times upper limit of normal unless due to Gilbert's syndrome (then <3 times).
- Estimated creatinine clearance must be ≥60 mL/min.
- INR and aPTT must be ≤1.5 times upper limit of normal.
- Life expectancy of at least 12 weeks.
- Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2.
- Female participants of child-bearing potential must have a negative pregnancy test at screening and before first dose.
You will not qualify if you...
- Participants with acute promyelocytic leukemia.
- Participants with known leukemic involvement in the central nervous system.
- Recent anticancer treatments within 5 half-lives or 28 days before first dose.
- Unresolved significant non-hematologic toxicities ≥ Grade 2 from prior therapies, except residual alopecia.
- Chronic graft versus host disease requiring systemic immunosuppressive therapy.
- Active malignancies other than acute myeloid leukemia or myelodysplastic syndromes.
- Major surgery within 4 weeks prior to first dose.
- Life-threatening severe complications such as uncontrolled infection or bleeding.
- Known chronic active infections like hepatitis B, hepatitis C, or HIV.
- Unable to swallow oral medications or with significant gastrointestinal issues limiting absorption.
- Other significant medical, laboratory, or psychiatric conditions that increase risk or prevent study compliance.
- Use of medications known to strongly affect CYP3A4, P-glycoprotein, or CYP2C8 within 14 days or 5 half-lives before first dose.
- Pregnant or lactating women.
Research Team
K
Kimberly Glen
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here