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Acute Myeloid LeukemiaPhase 1NCT03760523

Dose Escalation Study of Minnelide in Relapsed or Refractory Acute Myeloid Leukemia

A Phase 1 study testing Minnelide in people with Acute Myeloid Leukemia, run by H. Lee Moffitt Cancer Center and Research Institute at 1 site in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

This study is to determine the safety and recommended dosing of Minnelide in Acute Myeloid Leukemia (AML) Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Acute Myeloid Leukemia
What is being tested?
Minnelide
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
27 participants
Where
1 siteUnited States
Study dates
18 Apr 2019to 9 Oct 2023
Phase
Phase 1
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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.
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  • 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)

Who cannot join

  • 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.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • H. Lee Moffitt Cancer Center and Research Institute

    Tampa, Florida, 33612

Other trials in United States

About this study

Official title: Phase 1/1b Dose Escalation Study of Minnelide in Relapsed or Refractory Acute Myeloid Leukemia

This study is to determine the safety and recommended dosing of Minnelide in Acute Myeloid Leukemia (AML)

Read the detailed description

This phase 1 dose escalation clinical trial will establish the maximum tolerated dose (MTD) and recommended phase 2 dose (RP2D) of Minnelide as single-agent in relapsed/refractory (R/R) acute myeloid leukemia (AML) patients who are ineligible to receive intensive chemotherapy. The oral formulation of Minnelide will be used. Minnelide is a prodrug of triptolide (a potent heat shock protein (HSP) 70 inhibitor) with promising preclinical activity in AML.

Being tested
Minnelide
Keywords
leukemia, myeloid leukemia
Medical subject headings
Leukemia, Myeloid, Acute, Leukemia, Leukemia, Myeloid

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • MinnelideDrug

    Patients will take Minnelide orally once daily on Days 1-21 of 28 day Cycle. Dose Escalation Schedule: Dose Level -1: .5 mg, Dose Level 1: .75 mg, Dose Level 2: 1 mg, Dose Level 3: 1.25 mg.

Study groups in NCT03760523
Study groupRoleWhat they receive
Minnelide Dose EscalationExperimentalA 3+3 design will be used. The first 3 patients will be treated at dose level 1. If none experience a Dose Limiting Toxicity (DLT), the next 3 patients will be treated at dose level 2. If a DLT is observed in 1 out of 3 patients at dose level 1, up to an 3 more patients will be enrolled and treated at that dose level. If 2 patients at dose level have DLTs, dosing will be lowered to dose level -1 (.5 mg daily, taken orally). If 2 or more of the up to 6 patients at any dose level have DLTs, the preceding dose will be declared the Maximum Tolerated Dose (MTD). If more than 1 DLT occurs at Dose Level -1, the investigators will consider stopping the study. Once the MTD has been established, an additional 10 patients will be enrolled at this level to better characterize safety and tolerability.
Receives: Drug: Minnelide

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Maximum Tolerated Dose (MTD) of Minnelide

    MTD will be determined by testing increasing doses up to 1.25 mg daily. MTD reflects highest dose of drug that did not cause a Dose Limiting Toxicity (DLT).

    Measured: Up to 28 days for each dosing cohort

  2. Number of Participants Who Experience Dose Limiting Toxicities (DLTs)

    A DLT is any Grade 3 or 4 drug-related non-hematologic toxicity, with some exceptions per protocol.

    Measured: Up to 28 days for each dosing cohort

Secondary outcomes (4)

  1. Complete Response (CR)

    Participants who experience Complete Response (CR) and Complete Response with Incomplete Blood Count Recovery (CRi) rate as defined by 2003 International Working Group (IWG) for AML.

    Measured: Up to 12 months

  2. Overall Response Rate (ORR)

    Overall Response Rate is defined as CR + CRi + partial response (PR) as defined by 2003 IWG criteria for AML.

    Measured: Up to 12 months

  3. Relapse Free Survival (RFS)

    RFS is defined as time interval between achievement of CR to time of relapse.

    Measured: Up to 18 months

  4. Overall Survival (OS)

    OS defined as time interval from time of enrollment onto the clinical trial to death from any cause.

    Measured: Up to 12 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted30 Nov 2018
  2. Study start18 Apr 2019
  3. Primary completion9 Oct 2023
  4. Study completion9 Oct 2023

Why it stopped: Toxicity -Two DLT events occurred

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
27 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03760523
Sponsor's protocol ID
MCC-19742
Status last verified
Jan 2026
First submitted
27 Nov 2018
Last updated
7 Jan 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Minnelide in people with Acute Myeloid Leukemia, run by H. Lee Moffitt Cancer Center and Research Institute at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 13 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 7 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was stopped early (reason given: Toxicity -Two DLT events occurred). Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Maximum Tolerated Dose (MTD) of Minnelide (time frame: Up to 28 days for each dosing cohort). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03760523.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Jan 2026.

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