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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 belowSelect...
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:Select...
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 weeks3 visits
Treatment ~ Varies
Follow Up ~ 30 days
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~30 days
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 • NCT0439935621%
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.
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
Eligibility Criteria:
This trial includes the following eligibility criteria:- My kidney function is within the required range for the study.I am between 2 and 25 years old.I have side effects from cancer treatment that haven't fully gone away, except for hair loss.I am a woman who can have children and have a recent negative pregnancy test.I have been diagnosed with AML according to WHO standards.My leukemia has spread to my brain but may be treatable with specific chemotherapy.I have an ongoing, untreated infection.I do not have any uncontrolled illnesses or social situations that would stop me from following the study's requirements.I can take medications by mouth or through a tube.My platelet count is at least 10,000/mm3, or I can receive a transfusion if it's lower.My bilirubin levels are within the normal range, or high due to Gilbert's syndrome.I cannot take niclosamide in its current forms.My AML has not responded to at least 2 chemotherapy treatments, confirmed by a bone marrow test.I do not have major issues with my digestive system that are uncontrolled.I am currently pregnant or breastfeeding.I have active hepatitis C.I agree to use effective birth control or practice abstinence during and for 30 days after the study.I have been diagnosed with a specific type of leukemia (APL).I had a bone marrow transplant and am currently experiencing severe GvHD symptoms.My body surface area is 2.10 m2 or less.I haven't had cancer treatment in the last 2 weeks, except for hydroxyurea for high WBC.I have a bleeding disorder like hemophilia.I am under 16 with a Lansky score over 50, or over 16 with a Karnofsky score over 50%.
Research Study Groups:
This trial has the following groups:- Group 1: Niclosamide 250 mg/m2 /day divided BID
- Group 2: Niclosamide 1200 mg/m2 /day divided BID
- Group 3: Niclosamide 500 mg/m2 /day divided BID
- Group 4: Niclosamide 800 mg/m2 /day divided BID
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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