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Experimental AML drug minnelide tested in early trial

NCT ID NCT03760523

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early This study
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

27 people

The number who actually took part.

Started

Apr 2019

Finished

Oct 2023

Lead sponsor

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

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

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

  • H. Lee Moffitt Cancer Center and Research Institute

    Tampa, Florida, 33612, United States

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