Could a cancer drug help patients with rare gene changes?

NCT ID NCT03953898

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This phase 2 trial tests the drug olaparib in 14 adults with acute myeloid leukemia or myelodysplastic syndrome that has returned or not responded to treatment, and whose cancer has a specific change in the IDH gene. Olaparib is a pill that may block enzymes cancer cells need to grow. The study aims to see if the drug can shrink or control the cancer.

What this could mean

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

Active substance

Olaparib (a PARP inhibitor drug taken by mouth)

What this could lead to

If it works, this could point toward a new treatment option for people with hard-to-treat AML or MDS that has an IDH mutation.

What could go wrong

This is a very early, small phase 2 trial with only 14 participants. The drug may not work well enough, and side effects are possible. Results may not apply to all patients.

Disclaimer Read more

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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Conditions

The condition(s) this trial relates to.

acute myeloid leukemia acute myeloid leukemia with multilineage dysplasia myelodysplastic syndrome Myelodysplastic Syndromes therapy related acute myeloid leukemia and myelodysplastic syndrome

As listed by the trial registrant

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

Contacts and locations

Locations

  • Dartmouth Hitchcock Medical Center/Dartmouth Cancer Center

    Lebanon, New Hampshire, 03756, United States

  • UC Irvine Health/Chao Family Comprehensive Cancer Center

    Orange, California, 92868, United States

  • UM Sylvester Comprehensive Cancer Center at Coral Gables

    Coral Gables, Florida, 33146, United States

  • UM Sylvester Comprehensive Cancer Center at Deerfield Beach

    Deerfield Beach, Florida, 33442, United States

  • UM Sylvester Comprehensive Cancer Center at Plantation

    Plantation, Florida, 33324, United States

  • University of Maryland/Greenebaum Cancer Center

    Baltimore, Maryland, 21201, United States

  • University of Miami Miller School of Medicine-Sylvester Cancer Center

    Miami, Florida, 33136, United States

  • Wake Forest Baptist Health - Wilkes Medical Center

    Wilkesboro, North Carolina, 28659, United States

  • Wake Forest University Health Sciences

    Winston-Salem, North Carolina, 27157, United States

  • Wake Forest University at Clemmons

    Clemmons, North Carolina, 27012, United States

  • Yale University

    New Haven, Connecticut, 06520, United States