Experimental AML drug minnelide tested in early trial
NCT ID NCT03760523
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-phase study tested the safety and best dose of a drug called Minnelide in 27 adults with acute myeloid leukemia (AML) that had returned or not responded to prior therapy. The goal was to find the highest safe dose and check for side effects. The study was terminated early, so results are limited.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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27 people
The number who actually took part.
- Started
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Apr 2019
- Finished
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Oct 2023
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
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Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Adults ages 18 years or older. * Participant must have relapsed or refractory acute myeloid leukemia (AML) (excluding acute promyelocytic leukemia). * Relapsed patients must have received at least 1 induction chemotherapy regimen or two cycles of a hypomethylating agent and achieved a Complete Response (CR), followed by relapse of disease. * Refractory patients must have received at least 1 induction chemotherapy regimen or two cycles of hypomethylating agent without achieving a CR. * Eastern Cooperative Oncology Group (ECOG) performance status \<2. * Participants must have acceptable organ function. * Be able and willing to adhere to the study visit schedule and other protocol requirements. * Must be able to swallow capsules and have no evidence of GI tract abnormality that would alter the absorption of oral medications. * The effects of Minnelide on the developing human fetus are unknown. For this reason, women of child-bearing potential must have a negative serum or urine pregnancy test within 24 hours prior to beginning study treatment. * Participants of childbearing potential must practice contraception. Females of childbearing potential: Recommendation is for 2 effective contraceptive methods during the study. Male participants with female partners who are of childbearing potential: Recommendation is for male and partner to use at least 2 effective contraceptive methods, as described above, during the study. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately. * Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with Minnelide, breastfeeding mothers must agree to discontinue nursing if the mother is treated with Minnelide. * Provision of signed and dated informed consent document * Patients with prior allogeneic stem cell transplant who experience relapse of AML are eligible if they are off of immunosuppressive therapy and without any evidence of graft-versus-host disease (GVHD) Exclusion Criteria: * Participants may not have received any therapy with any investigational products, systemic anti-neoplastic therapy, or radiotherapy within 14 days prior to Cycle 1 Day 1. Patients actively receiving hydroxyurea are eligible and may continue to receive hydroxyurea during protocol treatment. * Candidates for standard and/or potentially curative treatments. * Major surgery within 28 days prior to Cycle 1 Day 1. * New York Heart Association Class III or IV heart failure, myocardial infarction within the past 6 months, unstable arrhythmia, or evidence of ischemia on an electrocardiogram (EKG) * Active, uncontrolled bacterial, viral, or fungal infections, requiring systemic therapy * Known, active HIV, Hepatitis A, B or C infection (prior Hepatitis C infection that has been treated and determined to be cured is allowed) * Symptomatic central nervous system (CNS) involvement with leukemia * A concurrent second active and non-stable malignancy with the exception of non-melanoma skin cancer or carcinoma in-situ.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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H. Lee Moffitt Cancer Center and Research Institute
Tampa, Florida, 33612, United States
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