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Nonsteroidal Anti-inflammatory Drug (NSAID)

DFMO + Celecoxib + Cyclophosphamide + Topotecan for Neuroblastoma (DFMO Trial)

Phase 1
Waitlist Available
Research Sponsored by New Approaches to Neuroblastoma Therapy Consortium
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
All patients must have at least ONE site of evaluable disease
Patients must have adequate heart, kidney, liver and bone marrow function
Must not have
Patients status post allogeneic stem cell transplant
Females of childbearing potential that do not have a negative pregnancy test
Timeline
Screening 3 weeks
Treatment Varies
Follow Up approximately 1 year
Awards & highlights

Summary

This trial uses a mix of two pills and two IV medicines to treat tumors. It focuses on finding the safest dose and understanding side effects. The treatment works by reducing growth signals, lowering inflammation, and killing cancer cells.

Who is the study for?
This trial is for children and young adults aged 2-29 with high-risk neuroblastoma that's recurrent, progressive, or hasn't fully responded to standard treatment. Participants need evaluable disease but good heart, kidney, liver, and bone marrow function. They can't join if they have severe allergies to NSAIDs/aspirin/sulfonamides, are pregnant/breastfeeding without contraception use, have major organ issues or active infections.
What is being tested?
The study tests different doses of DFMO (oral) combined with celecoxib (oral), cyclophosphamide (IV), and topotecan (IV) in patients with neuroblastoma. It aims to find the highest tolerable dose of DFMO without severe side effects and assess its impact on tumor size and specific gene changes related to treatment response.
What are the potential side effects?
Potential side effects include those common to chemotherapy like nausea, hair loss, low blood cell counts leading to increased infection risk; plus possible stomach irritation from celecoxib. The severity may vary based on the DFMO dosage.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have at least one tumor that can be measured for treatment response.
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My heart, kidneys, liver, and bone marrow are functioning well.
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I am between 2 and 30 years old.
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My neuroblastoma is high-risk and has not fully responded to standard treatments.
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My bone marrow works well enough for me to join the study.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have had a stem cell transplant from a donor.
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I am a woman who can have children and I do not have a negative pregnancy test.
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I have not had a seizure or been on seizure medication in the last year.
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I do not have any active or uncontrolled infections.
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I have active stomach bleeding or symptoms like gastritis.
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My major organs are healthy enough to handle treatment.
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I am currently receiving hemodialysis.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~approximately 1 year
This trial's timeline: 3 weeks for screening, Varies for treatment, and approximately 1 year for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Number of participants with adverse events as a measure of safety and tolerability.

Side effects data

From 2024 Phase 1 & 2 trial • 16 Patients • NCT02139397
33%
Anemia
33%
Neutropenia
33%
Alanine aminotransferase increased
33%
Anorexia
33%
Peripheral sensory neuropathy
100%
80%
60%
40%
20%
0%
Study treatment Arm
Phase I: DFMO 1500 mg/m^2
Phase I: DFMO 2000 mg/m^2
Phase I: DFMO 2500 mg/m^2

Trial Design

1Treatment groups
Experimental Treatment
Group I: DFMO, Celecoxib, Cyclophosphamide & TopotecanExperimental Treatment4 Interventions
Reconstituted DFMO powder by mouth for 14 days and celecoxib capsule by mouth daily in each cycle. Cyclophosphamide and Topotecan IV on days 8-12 in cycle 1 and days 1-5 of cycles 2-17. Patients may continue for up to 17 cycles as long as therapy is tolerated (no DLT) and disease progression does not occur (SD or better). \*Cycle 1 will include a 7 day lead-in with DFMO and celecoxib to deplete tumor polyamines.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
DFMO
2014
Completed Phase 2
~240
Celecoxib
FDA approved
Cyclophosphamide
FDA approved
Topotecan
FDA approved

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
Neuroblastoma treatments often target the rapid cell growth characteristic of this cancer. DFMO (difluoromethylornithine) inhibits ornithine decarboxylase, reducing polyamine synthesis, which is crucial for cell proliferation. This mechanism is significant as it directly impedes the growth of cancer cells. Additionally, chemotherapy agents like cyclophosphamide and topotecan work by damaging DNA and inhibiting topoisomerase I, respectively, leading to cell death. Targeted therapies, such as those inhibiting specific genetic mutations, also play a role. These treatments are vital for Neuroblastoma patients as they offer multiple avenues to halt tumor growth and improve survival rates.
Parkinsonism. Clinical and neuropharmacologic aspects.Dopamine: an old target in a new therapy.Presynaptic regulation of extracellular dopamine levels in the medial prefrontal cortex and striatum during tyrosine depletion.

Find a Location

Who is running the clinical trial?

New Approaches to Neuroblastoma Therapy ConsortiumLead Sponsor
17 Previous Clinical Trials
1,666 Total Patients Enrolled
17 Trials studying Neuroblastoma
1,666 Patients Enrolled for Neuroblastoma
National Cancer Institute (NCI)NIH
13,868 Previous Clinical Trials
41,010,807 Total Patients Enrolled
206 Trials studying Neuroblastoma
53,695 Patients Enrolled for Neuroblastoma
Michael Hogarty, MDStudy ChairChildren's Hospital of Philadelphia
2 Previous Clinical Trials
679 Total Patients Enrolled
2 Trials studying Neuroblastoma
679 Patients Enrolled for Neuroblastoma

Media Library

Celecoxib (Nonsteroidal Anti-inflammatory Drug (NSAID)) Clinical Trial Eligibility Overview. Trial Name: NCT02030964 — Phase 1
Neuroblastoma Research Study Groups: DFMO, Celecoxib, Cyclophosphamide & Topotecan
Neuroblastoma Clinical Trial 2023: Celecoxib Highlights & Side Effects. Trial Name: NCT02030964 — Phase 1
Celecoxib (Nonsteroidal Anti-inflammatory Drug (NSAID)) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02030964 — Phase 1
~3 spots leftby Oct 2025