Difluoromethylornithine (DFMO) will be used in an open label, single agent, multicenter, study for patients with neuroblastoma in remission. In this study 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. This study will focus on the use of DFMO in high risk neuroblastoma patients that are in remission as a strategy to prevent recurrence.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
PREVENTION
Masking
NONE
Enrollment
441
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.
Number of participants with event free survival (EFS) during study.
Time frame: 2 years
Length of time that participants experience Overall Survival (OS)
Time frame: 7 years
Number of Participants with Adverse Events as a Measure of Safety and Tolerability
Time frame: 2 years
Peak Plasma Concentration (Cmax)
Pharmacokinetic assay
Time frame: 1 year
Area under the plasma concentration versus time curve (AUC)
Pharmacokinetic assay
Time frame: 1 year
Time to reach Peak Plasma Concentration (Tmax)
Pharmacokinetic assay
Time frame: 1 year
Number of participants with ODC (Ornithine decarboxylase) single nucleotide polymorphisms.
Time frame: 1 year
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
University of Alabama, Children's of Alabama
Birmingham, Alabama, United States
Arkansas Children's Hospital
Little Rock, Arkansas, United States
UCSF Benioff Children's Hospital Oakland-
Oakland, California, United States
Rady Children's Hospital
San Diego, California, United States
Rocky Mountain Pediatric Hematology
Denver, Colorado, United States
Connecticut Children's Hospital
Hartford, Connecticut, United States
University of Florida
Gainesville, Florida, United States
Arnold Palmer Hospital for Children
Orlando, Florida, United States
All Children's Hospital Johns Hopkins Medicine
St. Petersburg, Florida, United States
St. Joseph's Children's Hospital
Tampa, Florida, United States
...and 37 more locations