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DFMO Maintenance Therapy for Neuroblastoma (NMTT Trial)
Phase 2
Recruiting
Research Sponsored by Giselle Sholler
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Patients must have adequate organ functions at the time of registration: Hematological: Total absolute phagocyte count ≥1000/μL, Liver: Subjects must have adequate liver function, Renal: Adequate renal function, Females of childbearing potential must have a negative pregnancy test. Patients of childbearing potential must agree to use an effective birth control method. Female patients who are lactating must agree to stop breast-feeding.
Patients must have a Lansky or Karnofsky Performance Scale score of > 50% and patients must have a life expectancy of ≥ 2 months.
Must not have
BSA (Body Surface Area) of <0.25 m2.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 year
Awards & highlights
No Placebo-Only Group
Summary
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.
Who is the study for?
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.
What is being tested?
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.
What are the potential side effects?
While the description doesn't specify side effects of DFMO directly, potential side effects may include hearing loss and mild blood disorders since it's being tested as a cancer drug; however individual experiences can vary.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I can do most activities and am expected to live for at least 2 more months.
Select...
My scans show no signs of cancer, and my bone marrow is clear.
Select...
I have had cancer come back but am now in remission again.
Select...
My neuroblastoma responded well to treatment before a transplant, with less than 10% cancer in my bone marrow.
Select...
My neuroblastoma is in its first complete remission after initial standard treatment.
Select...
I've completed the specific treatment steps for my cancer as outlined, including chemotherapy, surgery, stem cell transplant, radiotherapy, and immunotherapy.
Exclusion Criteria
You may be eligible for the trial if you check “No” for criteria below:Select...
My body surface area is less than 0.25 square meters.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 1 year
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1 year
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Secondary study objectives
Area under the plasma concentration versus time curve (AUC)
Peak Plasma Concentration (Cmax)
Time to reach Peak Plasma Concentration (Tmax)
Awards & Highlights
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Trial Design
1Treatment groups
Experimental Treatment
Group I: Difluoromethylornithine (DFMO)Experimental Treatment1 Intervention
Subjects will receive 730 Days of oral difluoromethylornithine (DFMO) at a dose of 750 mg/m2 ± 250 mg/m2 BID (strata 1, 2, 3, and 4) OR 2500 mg/m2 BID (stratum 1B) on each day of study.
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
Difluoromethylornithine (DFMO) treats Neuroblastoma by inhibiting ornithine decarboxylase, reducing polyamine synthesis necessary for cell proliferation, thereby limiting cancer cell growth. This targeted approach is crucial for Neuroblastoma patients as it may lead to fewer side effects and better outcomes.
Other treatments like chemotherapy and immunotherapy also play vital roles by targeting rapidly dividing cells and enhancing the immune response, respectively, offering multiple strategies to improve remission and survival rates.
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Who is running the clinical trial?
Giselle ShollerLead Sponsor
22 Previous Clinical Trials
2,049 Total Patients Enrolled
19 Trials studying Neuroblastoma
1,422 Patients Enrolled for Neuroblastoma
Giselle SaulnierShollerLead Sponsor
21 Previous Clinical Trials
1,964 Total Patients Enrolled
18 Trials studying Neuroblastoma
1,337 Patients Enrolled for Neuroblastoma
Wake Forest University Health SciencesLead Sponsor
1,398 Previous Clinical Trials
2,459,769 Total Patients Enrolled
19 Trials studying Neuroblastoma
1,394 Patients Enrolled for Neuroblastoma
Beat NB Cancer FoundationOTHER
5 Previous Clinical Trials
876 Total Patients Enrolled
5 Trials studying Neuroblastoma
876 Patients Enrolled for Neuroblastoma
Team Parker for LifeUNKNOWN
2 Previous Clinical Trials
631 Total Patients Enrolled
2 Trials studying Neuroblastoma
631 Patients Enrolled for Neuroblastoma
Giselle Sholler, MDStudy ChairBeat Childhood Cancer at Atrium Health
16 Previous Clinical Trials
1,691 Total Patients Enrolled
14 Trials studying Neuroblastoma
1,182 Patients Enrolled for Neuroblastoma
Media Library
Eligibility Criteria:
This trial includes the following eligibility 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.I have had cancer come back but am now in remission again.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.My neuroblastoma responded well to treatment before a transplant, with less than 10% cancer in my bone marrow.My neuroblastoma didn't improve with initial treatment but is now in complete remission after more therapy.My neuroblastoma is in its first complete remission after initial standard treatment.I have had a recent tumor check, including bone marrow tests, within the last 4 weeks.I finished my last cancer treatment less than 60 days ago.I've completed the specific treatment steps for my cancer as outlined, including chemotherapy, surgery, stem cell transplant, radiotherapy, and immunotherapy.
Research Study Groups:
This trial has the following groups:- Group 1: Difluoromethylornithine (DFMO)
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.