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Niclosamide for Relapsed/Refractory AML in Children and Young Adults

This study is testing a drug called niclosamide in children and young adults (ages 2 to 25) who have acute myeloid leukemia (AML) that has come back or hasn't responded to previous treatments. Niclosamide will be given by mouth for 14 days, along with standard chemotherapy. Researchers want to see if this combination causes any serious side effects (dose-limiting toxicity) within 30 days. This study is looking for about 16 participants. The study is focused on how niclosamide might work by affecting the immune system. To join, you must have AML that has been diagnosed before and has not responded to or has come back after other treatments.

Study design
This study plans to enroll about 16 participants. It is designed to test different doses of niclosamide in combination with chemotherapy.
What's involved
Participants will take niclosamide by mouth for 14 days, followed by backbone chemotherapy.
Compensation
Not stated in the trial record.
Follow-up
The main goal is to measure side effects within 30 days.

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NCT05188170

Niclosamide in Pediatric Patients With Relapsed and Refractory AML

Recruiting
PHASE1Ages 2–25InterventionalTreatment
Stanford University
~16 participants
Updated 2026-08-12 on ClinicalTrials.gov
What's tested:Niclosamide

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Dose-limiting toxicity
Measured over 30 days

Conditions

Acute Myeloid Leukemia (AML)

Explore related trials

Acute Myeloid Leukemia

Where it's being run

1 sites across 1 states
California1

Study leadership

  • Kathleen M Sakamoto, MD, PhD · PRINCIPAL_INVESTIGATOR · Stanford University

Who to contact

Nancy Sweeters
[email protected]
650-724-4042
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

5% blasts in the peripheral blood (confirmed by flow cytometry, cytogenetics or FISH), ≥ 1% MRD
60 mL/min/1.73 m2 (by the Cockcroft-Gault method) within 14 days prior to treatment initiation 10. Total bilirubin ≤ 2.0 x institutional upper limit of normal (ULN) within 14 days prior to treatment initiation (EXCEPTION: Subjects with Gilbert's syndrome may be included if the total bilirubin is
3.0 x ULN) 11. SGOT (AST) ≤ 3.0 x ULN and SGPT (ALT)
3.0 x ULN within 14 days prior to treatment initiation 12. Patients must have received their last dose of anti-cancer therapy (chemotherapy, immunotherapy, targeted agents, radiotherapy or investigational therapy) at least 2 weeks or 3 half-lives prior to the start of study treatment, whichever is longer. 13. For patients who have received prior hematopoietic stem cell transplants (HSCT), no evidence of GvHD and must be \> 60 days since the HSCT. HSCT recipients must have completed their last course of tacrolimus, cyclosporine, or mycophenolate \> 4 weeks before initiation of niclosamide 14. Females of reproductive potential (WOCBP) must have a negative pregnancy test within 14 days prior to study treatment. WOCBP must agree to use adequate contraception (eg, hormonal or barrier methods of birth control; abstinence; sterilized partner) from date of consent through the treatment period, and for 30 days after completion of niclosamide administration 15. Men only: Men must agree to use adequate contraception (eg, hormonal or barrier methods of birth control; abstinence; sterilized partner) from date of consent through the treatment period, and for 30 days after completion of niclosamide administration 16. Ability to understand the purpose and risks of the study and the willingness to sign a written informed consent document containing an authorization to use protected health information (in accordance with national and local subject privacy regulations

What this trial measures

  • Dose-limiting toxicity30 days

    Dose-limiting toxicities (DLTs) are defined as any events ≥ Grade 3 that are at least possibly, probably, or definitely related to niclosamide treatment