441 Participants Needed

DFMO Maintenance Therapy for Neuroblastoma

(NMTT Trial)

Recruiting at 49 trial locations
GB
BE
Overseen ByBCC Enroll
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial
Approved in 1 JurisdictionThis treatment is already approved in other countries

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a medication called DFMO in patients with high-risk neuroblastoma who are in remission. The goal is to prevent the cancer from returning by stopping an enzyme that cancer cells need to grow. DFMO was initially developed as a cancer therapeutic agent but gained renewed interest as a preventive agent after showing effectiveness in inhibiting cancer development in rodent models.

Will I have to stop taking my current medications?

The trial requires that participants stop taking other anticancer agents before joining. The protocol does not specify about other medications, so it's best to discuss your current medications with the study team.

Is DFMO safe for use in humans?

DFMO (also known as Eflornithine) has been used safely in clinical trials for neuroblastoma, showing it is well tolerated in children. It is also FDA-approved for treating other conditions like African sleeping sickness, indicating its general safety in humans.12345

How does DFMO maintenance therapy for neuroblastoma differ from other treatments?

DFMO (2-difluoromethylornithine) is unique because it targets polyamine levels, which are compounds involved in cell growth, and has shown effectiveness in reducing tumor polyamine levels without severe toxicities. This approach is different from traditional chemotherapy, which often targets DNA synthesis or cell division directly.678910

What data supports the effectiveness of the drug DFMO for treating neuroblastoma?

Research shows that DFMO can reduce tumor growth by depleting polyamines, which are compounds that help cancer cells grow. This effect was observed in studies on head and neck cancers and feline oral cancers, suggesting potential benefits for other cancers like neuroblastoma.69111213

Who Is on the Research Team?

GS

Giselle Sholler, MD

Principal Investigator

Beat Childhood Cancer at Atrium Health

Are You a Good Fit for This Trial?

This trial is for high-risk neuroblastoma patients under 30.99 years old in remission after intensive treatment, including chemotherapy, surgery, stem cell transplant, radiotherapy, immunotherapy with dinutuximab and retinoic acid. They must have no residual disease or bone marrow involvement over 10%, be within a specific time frame post-treatment, agree to birth control if applicable, and not be on other investigational drugs.

Inclusion Criteria

I can do most activities and am expected to live for at least 2 more months.
I am under 31 and have high-risk neuroblastoma, or it became high-risk after diagnosis.
My scans show no signs of cancer, and my bone marrow is clear.
See 11 more

Exclusion Criteria

My body surface area is less than 0.25 square meters.
I am not currently on any cancer treatments and have recovered from previous ones.
My infections are under control.
See 2 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive 730 days of oral difluoromethylornithine (DFMO) to prevent recurrence of neuroblastoma

104 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

52 weeks

What Are the Treatments Tested in This Trial?

Interventions

  • Difluoromethylornithine (DFMO)
Trial Overview The trial tests DFMO as an oral maintenance therapy for preventing neuroblastoma recurrence. Participants will take DFMO daily for two years at doses adjusted by body surface area. The study includes multiple patient groups based on their previous response to standard therapies.
How Is the Trial Designed?
1Treatment groups
Experimental Treatment
Group I: Difluoromethylornithine (DFMO)Experimental Treatment1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

Giselle Sholler

Lead Sponsor

Trials
23
Recruited
2,500+

Giselle SaulnierSholler

Lead Sponsor

Trials
22
Recruited
2,400+

Wake Forest University Health Sciences

Lead Sponsor

Trials
1,432
Recruited
2,506,000+

Beat NB Cancer Foundation

Collaborator

Trials
6
Recruited
1,300+

Team Parker for Life

Collaborator

Trials
3
Recruited
1,100+

Published Research Related to This Trial

In a study involving 14 client-owned cats with oral squamous cell carcinoma (SCC), the polyamine inhibitor DFMO effectively reduced total polyamine levels in tumor tissue and normal mucosa, indicating its potential as a treatment option.
While DFMO treatment led to some side effects, such as ototoxicity in some cats and mild thrombocytopenia, it did not cause life-threatening toxicities, suggesting a favorable safety profile for further investigation.
Polyamine inhibitors for treatment of feline oral squamous cell carcinoma: a proof-of-concept study.Lewis, JR., O'Brien, TG., Skorupski, KA., et al.[2017]
The combination of 5-fluorouracil (5-FU) and leucovorin (FL) has improved response rates for colorectal cancer treatment to about 20-30%, with a median survival time of 10 to 12 months.
The FOLFOX 4 regimen, which combines the de Gramont method of 5-FU and leucovorin with oxaliplatin, has shown improved median survival times over a year and is now considered the standard treatment for advanced colorectal cancer due to its effectiveness and manageable toxicity.
[Chemotherapy].Aiba, K.[2018]
Combination therapy of 5-fluorouracil (5-FU) with leucovorin (LV) shows improved response rates in colorectal cancer patients compared to single-agent 5-FU (25% vs 14%), but it does not significantly extend median survival (12.2 months vs 11.4 months).
Different dosing schedules of 5-FU/LV are therapeutically equivalent, with higher doses of LV on a weekly schedule leading to slightly better efficacy but increased toxicity, such as severe diarrhea (26% vs 14%).
Systemic treatment options in advanced colorectal cancer: perspectives on combination 5-fluorouracil plus leucovorin.Grem, JL.[2013]

Citations

Polyamine inhibitors for treatment of feline oral squamous cell carcinoma: a proof-of-concept study. [2017]
[Chemotherapy]. [2018]
Systemic treatment options in advanced colorectal cancer: perspectives on combination 5-fluorouracil plus leucovorin. [2013]
Phase II clinical trial of DDMP (2,4-diamino-5-(3',4'-dichlorophenyl)-6-methylpyrimidine) and folinic acid (CF) in solid tumors. [2019]
Modulation of tumor cell proliferation and apoptosis by polyamine depletion in cells of head and neck squamous cell carcinomas. [2013]
Maintenance DFMO Increases Survival in High Risk Neuroblastoma. [2019]
Eflornithine as Postimmunotherapy Maintenance in High-Risk Neuroblastoma: Externally Controlled, Propensity Score-Matched Survival Outcome Comparisons. [2023]
Translational development of difluoromethylornithine (DFMO) for the treatment of neuroblastoma. [2020]
Probenecid increases renal retention and antitumor activity of DFMO in neuroblastoma. [2021]
10.United Statespubmed.ncbi.nlm.nih.gov
A subset analysis of a phase II trial evaluating the use of DFMO as maintenance therapy for high-risk neuroblastoma. [2021]
A pharmacokinetic and pharmacodynamic investigation of Modufolin® compared to Isovorin® after single dose intravenous administration to patients with colon cancer: a randomized study. [2021]
Hyperammonemia-induced impaired consciousness following mFOLFOX6 therapy in a patient with recurrent rectal cancer. [2021]
13.United Statespubmed.ncbi.nlm.nih.gov
Pathway analysis of genetic variants in folate-mediated one-carbon metabolism-related genes and survival in a prospectively followed cohort of colorectal cancer patients. [2023]
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