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Anti-helminthic

Niclosamide for Pediatric Acute Myeloid Leukemia

Phase 1
Recruiting
Led By Kathleen M Sakamoto, MD, PhD
Research Sponsored by Stanford University
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Serum creatinine ≤ 2.0 mg/dL or estimated creatinine clearance ≥ 30 mL/min (Cockcroft Gault) within 14 days prior to treatment initiation
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.
Must not have
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
Known active uncontrolled systemic infection
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 30 days
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing a combination of two drugs, niclosamide and cytarabine, to treat children with a type of leukemia that has come back or hasn't responded to other treatments. The goal is to see if niclosamide can help cytarabine work better by making cancer cells easier to destroy. Niclosamide is being tested to see if it can help destroy cancer cells more effectively.

Who is the study for?
This trial is for pediatric patients aged 2-25 with relapsed/refractory acute myeloid leukemia (AML) who have failed previous therapies. Participants must have adequate liver and kidney function, a minimum expected lifespan of 4 weeks, and be able to take oral or nasogastric medications. They should not be pregnant or breastfeeding and must agree to use contraception.
What is being tested?
The study tests the effects of increasing doses of Niclosamide combined with cytarabine in young patients with AML that has returned after treatment or hasn't responded to at least two chemotherapy cycles. The goal is to find an effective dose for this specific condition.
What are the potential side effects?
While the side effects specific to Niclosamide in this context are not detailed here, potential risks may include typical chemotherapy-related issues such as nausea, fatigue, increased risk of infection due to low blood cell counts, liver toxicity, and allergic reactions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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My kidney function is within the required range for the study.
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My AML has not responded to at least 2 chemotherapy treatments, confirmed by a bone marrow test.
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I am between 2 and 25 years old.
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I have been diagnosed with AML according to WHO standards.
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I can take medications by mouth or through a tube.
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My body surface area is 2.10 m2 or less.
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I am under 16 with a Lansky score over 50, or over 16 with a Karnofsky score over 50%.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I do not have major issues with my digestive system that are uncontrolled.
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I have an ongoing, untreated infection.
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I do not have any uncontrolled illnesses or social situations that would stop me from following the study's requirements.
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I cannot take niclosamide in its current forms.
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I am currently pregnant or breastfeeding.
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I have active hepatitis C.
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I have been diagnosed with a specific type of leukemia (APL).
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I had a bone marrow transplant and am currently experiencing severe GvHD symptoms.
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I have a bleeding disorder like hemophilia.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~30 days
This trial's timeline: 3 weeks for screening, Varies for treatment, and 30 days for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Dose-limiting toxicity
Secondary study objectives
Efficacy of niclosamide treatment clinical response

Side effects data

From 2021 Phase 2 trial • 73 Patients • NCT04399356
21%
Nausea
9%
Skin rash
6%
Vomiting
100%
80%
60%
40%
20%
0%
Study treatment Arm
Control Group
Niclosamide- Experimental Group

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

4Treatment groups
Experimental Treatment
Group I: Niclosamide 800 mg/m2 /day divided BIDExperimental Treatment1 Intervention
Group II: Niclosamide 500 mg/m2 /day divided BIDExperimental Treatment1 Intervention
Group III: Niclosamide 250 mg/m2 /day divided BIDExperimental Treatment1 Intervention
Group IV: Niclosamide 1200 mg/m2 /day divided BIDExperimental Treatment1 Intervention
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Niclosamide
2017
Completed Phase 3
~2280

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Common treatments for Acute Myeloid Leukemia (AML) include agents that inhibit key cellular pathways critical for cancer cell survival and proliferation. Niclosamide targets the Wnt/β-catenin signaling pathway, which is often dysregulated in AML, leading to uncontrolled cell growth and survival. By inhibiting this pathway, Niclosamide can reduce tumor cell proliferation and induce apoptosis. Cytarabine, on the other hand, inhibits DNA synthesis by incorporating into DNA during replication, causing chain termination and cell death. This is particularly effective in rapidly dividing cells, such as AML cells. These mechanisms are crucial for AML patients as they directly target the cellular processes that allow leukemia cells to grow and survive, thereby potentially improving treatment outcomes and survival rates.
γ-Catenin Overexpression in AML Patients May Promote Tumor Cell Survival via Activation of the Wnt/β-Catenin Axis.Knockdown of TRIM24 suppresses growth and induces apoptosis in acute myeloid leukemia through downregulation of Wnt/GSK-3β/β-catenin signaling.Molecular targeting in acute myeloid leukemia.

Find a Location

Who is running the clinical trial?

Stanford UniversityLead Sponsor
2,461 Previous Clinical Trials
17,494,858 Total Patients Enrolled
Kathleen M Sakamoto, MD, PhDPrincipal InvestigatorStanford University
1 Previous Clinical Trials
80 Total Patients Enrolled

Media Library

Niclosamide (Anti-helminthic) Clinical Trial Eligibility Overview. Trial Name: NCT05188170 — Phase 1
Acute Myeloid Leukemia Research Study Groups: Niclosamide 250 mg/m2 /day divided BID, Niclosamide 1200 mg/m2 /day divided BID, Niclosamide 500 mg/m2 /day divided BID, Niclosamide 800 mg/m2 /day divided BID
Acute Myeloid Leukemia Clinical Trial 2023: Niclosamide Highlights & Side Effects. Trial Name: NCT05188170 — Phase 1
Niclosamide (Anti-helminthic) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05188170 — Phase 1
~4 spots leftby Jul 2025