Actively Recruiting

Phase 1
Age: 2Years - 25Years
All Genders
NCT05188170

Niclosamide in Pediatric Patients With Relapsed and Refractory AML

Led by Stanford University · Updated on 2026-01-12

16

Participants Needed

1

Research Sites

210 weeks

Total Duration

On this page

Sponsors

S

Stanford University

Lead Sponsor

T

The Leukemia and Lymphoma Society

Collaborating Sponsor

AI-Summary

What this Trial Is About

Protocol is designed to evaluate a niclosamide dose escalation scale in combination with cytarabine as a therapeutic modality for pediatric subjects with relapsed/refractory acute myeloid leukemia.

CONDITIONS

Official Title

Niclosamide in Pediatric Patients With Relapsed and Refractory AML

Who Can Participate

Age: 2Years - 25Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Prior confirmed diagnosis of AML based on WHO criteria
  • Disease relapse or refractory AML after at least 2 cycles of intensive chemotherapy or 4 cycles of non-intensive chemotherapy or hypomethylating agents
  • No prior treatment with niclosamide
  • Age 2 to 30 years
  • Body surface area (BSA) less than or equal to 2.10 m2
  • Able to tolerate oral or nasogastric medications
  • Performance status: Lansky score 50 or higher (if age 16 or younger), Karnofsky score 50% or higher (if older than 16 years)
  • Life expectancy greater than 4 weeks
  • Platelet count of at least 10,000/mm3 (platelet transfusion allowed if below this)
  • Creatinine clearance at least 60 mL/min/1.73 m2 within 14 days before treatment
  • Total bilirubin less than or equal to 2.0 times institutional upper limit of normal (ULN) within 14 days before treatment (except Gilbert's syndrome up to 3.0 x ULN)
  • AST and ALT less than or equal to 3.0 times ULN within 14 days before treatment
  • Last dose of anti-cancer therapy at least 2 weeks or 3 half-lives before study treatment, whichever is longer
  • For prior stem cell transplant recipients, no graft-versus-host disease and more than 60 days since transplant
  • Females of reproductive potential must have a negative pregnancy test within 14 days before treatment and agree to use contraception during and 30 days after treatment
  • Men must agree to use contraception during and 30 days after treatment
  • Ability to understand study purpose and risks and provide informed consent
Not Eligible

You will not qualify if you...

  • Received any anticancer therapy within 2 weeks before starting study treatment (hydroxyurea allowed to control high white blood cell count but must stop 24 hours before treatment)
  • Receiving other investigational agents including niclosamide
  • Unresolved toxicities from prior cancer therapy above Grade 1 except alopecia
  • Diagnosis of acute promyelocytic leukemia (M3-AML)
  • Active central nervous system leukemia unless cleared with intrathecal chemotherapy
  • Known congenital bleeding disorders
  • Active uncontrolled systemic infection
  • Significant gastrointestinal disorders affecting absorption or with bowel obstruction
  • Unable to take niclosamide in available forms
  • Uncontrolled illness or psychiatric/social conditions limiting study compliance
  • Pregnant or breastfeeding females
  • Known active hepatitis C

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 1 location

1

Stanford University

Palo Alto, California, United States, 94305

Actively Recruiting

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Research Team

N

Nancy Sweeters

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NON_RANDOMIZED

Model

SEQUENTIAL

Primary Purpose

TREATMENT

Number of Arms

4

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