New hope for tough blood cancers: first human trial of GLB-001 begins

NCT ID NCT06146257

First seen Oct 01, 2025

Summary

This early-stage trial tests an experimental oral drug called GLB-001 in adults whose acute myeloid leukemia (AML) or high-risk myelodysplastic syndromes (MDS) have come back or not responded to treatment. The study aims to find a safe dose and see if the drug shows any signs of working. About 48 participants will be enrolled in two parts: dose escalation and dose expansion.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Alliance for Multispecialty Research, LLC

    TERMINATED

    Merriam, Kansas, 66204, United States

  • City of Hope Medical Center

    RECRUITING

    Duarte, California, 91010, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Icahn School of Medicine at Mount Sinai

    RECRUITING

    New York, New York, 10029, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Memorial Sloan Kettering Cancer Center-David H. Koch Center

    TERMINATED

    New York, New York, 10021, United States

  • Roswell Park Comprehensive Cancer Center

    TERMINATED

    Buffalo, New York, 14263, United States

  • University of California Irvine

    TERMINATED

    Irvine, California, 92697, United States

  • University of Kansas Medical Center Research Institute, Inc.

    RECRUITING

    Kansas City, Kansas, 66160, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • University of Texas M. D. Anderson Cancer Center

    TERMINATED

    Houston, Texas, 77030, United States

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance

GLB-001 (an oral drug)

What this could lead to

If successful, this could lead to a new oral treatment option for people with hard-to-treat AML or MDS.

What could go wrong

This is a very early Phase 1 trial with only 48 participants, so safety and effectiveness are not yet known. Many drugs fail at this stage.

Conditions

The condition(s) this trial relates to.

acute myeloid leukemia myelodysplastic syndrome Myelodysplastic Syndromes

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.