Niclosamide in Pediatric Patients With Relapsed and Refractory AML

NCT ID: NCT05188170

Last Updated: 2026-01-12

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE1
• Total Enrollment: 16 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-11-21
• Study Completion Date: 2026-12-31

Brief Summary

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

Acute Myeloid Leukemia (AML)

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Niclosamide 250 mg/m2 /day divided BID

Group Type: EXPERIMENTAL

Niclosamide

Intervention Type: DRUG

Niclosamide will be administered orally for 14 days. Each dose will be followed by backbone chemotherapy

Niclosamide 500 mg/m2 /day divided BID

Group Type: EXPERIMENTAL

Niclosamide

Intervention Type: DRUG

Niclosamide will be administered orally for 14 days. Each dose will be followed by backbone chemotherapy

Niclosamide 800 mg/m2 /day divided BID

Group Type: EXPERIMENTAL

Niclosamide

Intervention Type: DRUG

Niclosamide will be administered orally for 14 days. Each dose will be followed by backbone chemotherapy

Niclosamide 1200 mg/m2 /day divided BID

Group Type: EXPERIMENTAL

Niclosamide

Intervention Type: DRUG

Niclosamide will be administered orally for 14 days. Each dose will be followed by backbone chemotherapy

Interventions

Niclosamide

Niclosamide will be administered orally for 14 days. Each dose will be followed by backbone chemotherapy

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. Prior morphologically-confirmed diagnosis of AML based on WHO Criteria 2. Has previously failed all available and suitable therapies for AML. Disease relapse or the presence of refractory disease after ≥ 2 cycles of intensive chemotherapy; or ≥ 4 cycles of non-intensive chemotherapy or hypomethylating agents (HMAs) must be documented by bone marrow (BM) examination demonstrating ≥ 5% blasts in the BM by morphology or ≥ 1% blasts by flow cytometry,

• 5% blasts in the peripheral blood (confirmed by flow cytometry, cytogenetics or FISH), ≥ 1% MRD

+ by flow cytometry, FISH, PCR or NGS, and not attributable to another cause (EXCEPTION: subjects with frank disease progression in the face of treatment with HMA with or without venetoclax will be considered eligible regardless of treatment cycles administered if they meet the other eligibility criteria). No prior treatment with niclosamide. 3. Age ≥ 2 and ≤ 30 years 4. Body surface area (BSA) ≤ 2.10 m2

, calculated per the Mostellar formula 5. Must be able to tolerate po or ng medications. 6. Performance status: Subject ≤ 16 years old: Lansky ≥ 50 Subject > 16 years old: Karnofsky ≥ 50% 7. Life expectancy of greater than 4 weeks 8. Platelets ≥ 10,000/mm3 (for subjects with platelets < 10,000/mm3 at baseline, platelet transfusion support is allowed) 9. Measured or calculated creatinine clearance

• 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

Exclusion Criteria

1. Received anticancer therapy (chemotherapy, immunotherapy, radiotherapy, or investigational therapy) within 2 weeks prior to starting study treatment. Administration of hydroxyurea 10 to 20 mg/kg/day PO (maximum 1000 mg PO BID) to control high WBC > 50 x 103

/mm3 is permitted at MD discretion (however, hydroxyurea should be stopped at least 24 hours prior to protocol therapy start).

2. Receiving any other investigational agents, including niclosamide.

4. Acute promyelocytic leukemia (French-American-British Class M3-AML)

5. Known active central nervous system (CNS) leukemia; subjects can enroll on study if CNS disease can be cleared with intrathecal chemotherapy, in the judgement of the treating physician

6. Known congenital bleeding disorders, including but not limited to hemophilia

7. Known active uncontrolled systemic infection

8. Malabsorption syndrome, disease significantly affecting gastrointestinal function, or resection of the stomach or small bowel, uncontrolled symptomatic inflammatory bowel disease or ulcerative colitis, or partial or complete bowel obstruction, at the time of study entry

9. Inability to receive administration of niclosamide in the available formulation(s)

10. Uncontrolled intercurrent illness including, but not limited to, uncontrolled active infection, or psychiatric illness/social situations that would limit compliance with study requirements

11. Lactating or pregnant female

12. Known active hepatitis C

• Minimum Eligible Age: 2 Years
• Maximum Eligible Age: 25 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

The Leukemia and Lymphoma Society

OTHER

Sponsor Role: collaborator

Pediatric Cancer Research Foundation (PCRF)

UNKNOWN

Sponsor Role: collaborator

CURE Childhood Cancer, Inc.

OTHER

Sponsor Role: collaborator

Stanford University

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Kathleen M Sakamoto, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Stanford University

Locations

Stanford University

Palo Alto, California, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

Nancy Sweeters

Role: CONTACT

nks2016@stanford.edu

650-724-4042

Facility Contacts

Nancy Sweeters, RN, PNP

Role: primary

nks2016@stanford.edu

650-721-4074

Other Identifiers

PEDSHEMAML0007

Identifier Type: OTHER

Identifier Source: secondary_id

641095

Identifier Type: OTHER_GRANT

Identifier Source: secondary_id

6587-20

Identifier Type: OTHER_GRANT

Identifier Source: secondary_id

NCI-2022-09974

Identifier Type: REGISTRY

Identifier Source: secondary_id

IRB-61916

Identifier Type: -

Identifier Source: org_study_id