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Polyamine Synthesis Inhibitor

DFMO for Neuroblastoma

Phase 2
Waitlist Available
Research Sponsored by Giselle SaulnierSholler
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 2 years
Awards & highlights

Study Summary

The purpose of this research study is to evaluate a new investigational drug to prevent reoccurrence of neuroblastoma that is in remission. This study drug is called DFMO. The objectives of this study will be to monitor for safety and look at efficacy of DFMO. The safety of the proposed dosing regimen in this trial will be tested by an on-going risk/benefit assessment during the study. A patient benefiting from treatment, not progressing on therapy, and in the absence of any safety issues associated with DFMO may continue on treatment up to 27 cycles with the expectation that there will be an overall clinical benefit. The procedures involved in this study include Medical history, Physical exam, Vital signs (blood pressure, pulse, temperature), Blood tests, Urine tests, MRI or CT scan of the tumor(s), MIBG scans, and Bone marrow aspirations. All of these tests and procedures are considered standard of care for this population. Drug administration is also part of this protocol, including an investigational new drug called DFMO. The proposed dosing regimen is an oral dose of DFMO tablets two times a day for each day while on study. There will be 27 cycles. Each cycle will be 28 days in length.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~2 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and 2 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
To evaluate the preventative activity of DFMO as a single agent in patients that are in remission based on: Event free survival (EFS)
Secondary outcome measures
Biology studies
Number of Participants with Adverse Events as a Measure of Safety and Tolerability
To evaluate the preventative activity of DFMO as a single agent in patients with neuroblastoma who are in remission based on: Overall Survival (OS)

Side effects data

From 2023 Phase 2 trial • 140 Patients • NCT02395666
10%
ALT elevation
8%
Neutrophil count decrease
7%
AST elevation
6%
Infection
6%
Hearing Loss
6%
Diarrhea
4%
Anemia
4%
Otitis Media
2%
Vomiting
1%
Fever
1%
Pain
1%
Hyponatremia
1%
Benign vascular lesion
1%
Hypotension
1%
hearing loss
1%
Fracture
1%
Influenza
1%
Bacteremia
1%
Obstruction
1%
Swelling of Eye
1%
Hypoglycemia
1%
Urticaria
1%
Constipation
1%
Respiratory Disorder
100%
80%
60%
40%
20%
0%
Study treatment Arm
All Study Subjects

Trial Design

1Treatment groups
Experimental Treatment
Group I: DFMOExperimental Treatment1 Intervention
Subjects will receive twenty-seven (27) cycles of oral DFMO at a dose of 500 to 1000 mg/m2 BID on each day of a 28 day cycle.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Eflornithine
FDA approved

Find a Location

Who is running the clinical trial?

Giselle SaulnierShollerLead Sponsor
21 Previous Clinical Trials
2,129 Total Patients Enrolled
18 Trials studying Neuroblastoma
1,502 Patients Enrolled for Neuroblastoma
Cancer Prevention Pharmaceuticals, Inc.Industry Sponsor
7 Previous Clinical Trials
1,693 Total Patients Enrolled
1 Trials studying Neuroblastoma
21 Patients Enrolled for Neuroblastoma
University of ArizonaOTHER
514 Previous Clinical Trials
148,182 Total Patients Enrolled
1 Trials studying Neuroblastoma
21 Patients Enrolled for Neuroblastoma

Frequently Asked Questions

~7 spots leftby Apr 2025