RECRUITING

Niclosamide in Pediatric Patients With Relapsed and Refractory AML

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

Phase 1 Study of Niclosamide (ANA001) in Pediatric Patients With Relapsed and Refractory AML

Quick Facts

Study Start:2022-11-21

Study Completion:2026-12

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT05188170

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:2 Years to 25 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT

Inclusion Criteria	Exclusion Criteria
* Prior morphologically confirmed diagnosis of AML based on WHO Criteria * Has previously failed all available and suitable therapies for AML Disease relapse or the presence of refractory disease after ≥ 2 cycles of chemotherapy must be documented by bone marrow (BM) examination demonstrating \> 5% blasts in the BM not attributable to another cause. Administration of hydroxyurea 10 to 20 mg/kg/day PO (maximum 1000 mg PO BID) to control high WBC count is permitted. * Age ≥ 2 and ≤ 25 years * Body surface area (BSA) ≤ 2.10 m2, calculated per the Mostellar formula * Must be able to tolerate po or ng medications. * Performance status: * Subject age * Life expectancy of greater than 4 weeks * Platelets ≥ 10,000/mm3 (for subjects with platelets \< 10,000/mm3 at baseline, platelet transfusion support is allowed) * Serum creatinine ≤ 2.0 mg/dL or estimated creatinine clearance ≥ 30 mL/min (Cockcroft Gault) within 14 days prior to treatment initiation * Total bilirubin ≤ 2.0 x Institutional upper limit of normal (ULN) within 14 days prior to treatment initiation, unless the elevation is due primarily to elevated unconjugated hyperbilirubinemia secondary to Gilbert's syndrome or hemolysis or non hepatic origin, and not to liver dysfunction * SGOT (AST) ≤ 3.0 x ULN and SGPT (ALT) ≤ 3.0 x ULN within 14 days prior to treatment initiation * 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) for the duration of study participation * Men only: Men must agree to use adequate contraception (eg, hormonal or barrier methods of birth control; abstinence; sterilized partner) prior to the study treatment (from date of consent), for the duration of study participation, and 30 days after completion of niclosamide administration * 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	* 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 is permitted. * Receiving any other investigational agents. * Unresolved toxicities due to prior anticancer therapy, defined as not having resolved to Grade 0 or 1 (by CTCAE version 5 criteria), unless otherwise defined in the inclusion/exclusion criteria with the exception of alopecia * Acute promyelocytic leukemia (French American British Class M3 AML) * 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 * Prior bone marrow transplant presenting with active uncontrolled graft versus host disease (GvHD) * Known congenital bleeding disorders, including but not limited to hemophilia * Known active uncontrolled systemic infection * 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 * Inability to receive administration of niclosamide in the available formulation(s) * Uncontrolled intercurrent illness including, but not limited to, uncontrolled active infection, or psychiatric illness/social situations that would limit compliance with study requirements * Lactating or pregnant female * Known active hepatitis C

Inclusion Criteria

Exclusion Criteria

* Prior morphologically confirmed diagnosis of AML based on WHO Criteria
* Has previously failed all available and suitable therapies for AML Disease relapse or the presence of refractory disease after ≥ 2 cycles of chemotherapy must be documented by bone marrow (BM) examination demonstrating \> 5% blasts in the BM not attributable to another cause. Administration of hydroxyurea 10 to 20 mg/kg/day PO (maximum 1000 mg PO BID) to control high WBC count is permitted.
* Age ≥ 2 and ≤ 25 years
* Body surface area (BSA) ≤ 2.10 m2, calculated per the Mostellar formula
* Must be able to tolerate po or ng medications.
* Performance status:
* Subject age
* Life expectancy of greater than 4 weeks
* Platelets ≥ 10,000/mm3 (for subjects with platelets \< 10,000/mm3 at baseline, platelet transfusion support is allowed)
* Serum creatinine ≤ 2.0 mg/dL or estimated creatinine clearance ≥ 30 mL/min (Cockcroft Gault) within 14 days prior to treatment initiation
* Total bilirubin ≤ 2.0 x Institutional upper limit of normal (ULN) within 14 days prior to treatment initiation, unless the elevation is due primarily to elevated unconjugated hyperbilirubinemia secondary to Gilbert's syndrome or hemolysis or non hepatic origin, and not to liver dysfunction
* SGOT (AST) ≤ 3.0 x ULN and SGPT (ALT) ≤ 3.0 x ULN within 14 days prior to treatment initiation
* 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) for the duration of study participation
* Men only: Men must agree to use adequate contraception (eg, hormonal or barrier methods of birth control; abstinence; sterilized partner) prior to the study treatment (from date of consent), for the duration of study participation, and 30 days after completion of niclosamide administration
* 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

* 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 is permitted.
* Receiving any other investigational agents.
* Unresolved toxicities due to prior anticancer therapy, defined as not having resolved to Grade 0 or 1 (by CTCAE version 5 criteria), unless otherwise defined in the inclusion/exclusion criteria with the exception of alopecia
* Acute promyelocytic leukemia (French American British Class M3 AML)
* 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
* Prior bone marrow transplant presenting with active uncontrolled graft versus host disease (GvHD)
* Known congenital bleeding disorders, including but not limited to hemophilia
* Known active uncontrolled systemic infection
* 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
* Inability to receive administration of niclosamide in the available formulation(s)
* Uncontrolled intercurrent illness including, but not limited to, uncontrolled active infection, or psychiatric illness/social situations that would limit compliance with study requirements
* Lactating or pregnant female
* Known active hepatitis C

Contacts and Locations

Study Contact

Nancy Sweeters

CONTACT

650-724-4042

nks2016@stanford.edu

Principal Investigator

Kathleen M Sakamoto, MD, PhD

PRINCIPAL_INVESTIGATOR

Stanford University

Study Locations (Sites)

Stanford University

Palo Alto, California, 94305

United States

Collaborators and Investigators

Sponsor: Stanford University

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

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2022-11-21

Study Completion Date2026-12

Study Record Updates

Study Start Date2022-11-21

Study Completion Date2026-12

Terms related to this study

Additional Relevant MeSH Terms

Acute Myeloid Leukemia (AML)