Clinical Studies Search

Protocol Summary


Protocol No.
PSHCI-16-023
Prinicipal Investigator
Brown, Valerie
Phase
Phase II
Age Group
Both
Scope
National
Secondary Protocol No.
NMTRC 014
Title
NMTT- Neuroblastoma Maintenance Therapy Trial Using Difluoromethylornithine (DFMO)
Objective
Primary:
1. To evaluate the efficacy of difluoromethylornithine (DFMO) as a single agent in preventing relapse in patients with high-risk neuroblastoma who are in remission, based upon:
a. Event free survival (EFS) from time of enrollment.

Secondary:
1. To evaluate the efficacy of difluoromethylornithine (DFMO) as a single agent in preventing relapse in patients with high-risk neuroblastoma who are in remission, based upon:
a. Overall Survival (OS) from time of enrollment.
b. EFS of the subset of patients with pre-immunotherapy Curie Scores >0.

2. To develop a complete safety and tolerability profile of difluoromethylornithine (DFMO) as a single agent in pediatric and young adult patients with high risk neuroblastoma that is in remission.

3. To further define the pharmacokinetics (PK) of DFMO in pediatric patients.

4. Biological Correlates to minimally include:
a. Blood: ODC SNP analysis in DNA isolated from nucleated cells, circulating tumor cell analysis, and explorative biomarker analysis.
Applicable Disease Sites
Brain and Nervous System
Status
Open
Participating Institutions
Hershey Medical Center