Neoplasms

All Conditions

Rare Diseases

Neuroblastoma

Neuroectodermal Tumors, Primitive, Peripheral

Neuroectodermal Tumors

Neuroectodermal Tumors, Primitive

Neoplasms, Neuroepithelial

Neoplasms, Germ Cell and Embryonal

Neoplasms by Histologic Type

Neoplasms, Glandular and Epithelial

Neoplasms, Nerve Tissue

Neuroepithelioma

Anti-Infective Agents

Antineoplastic Agents

All Drugs and Chemicals

Amino Acids

Eflornithine

Antiprotozoal Agents

Antiparasitic Agents

Enzyme Inhibitors

Ornithine

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.

DFMO

Giselle Saulnier-Sholler, MD

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), meta-iodobenzylguanidine (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.

Preventative Trial of Difluoromethylornithine (DFMO) in High Risk Patients With Neuroblastoma That is in Remission

A Phase II Preventative Trial of DFMO (Eflornithine HCl) as a Single Agent in Patients With High Risk Neuroblastoma in Remission

To evaluate the preventative activity of DFMO as a single agent in patients that are in remission based on: Event free survival (EFS)

To evaluate the preventative activity of DFMO as a single agent in patients with neuroblastoma who are in remission based on: Overall Survival (OS)

To continue to determine the safety and tolerability of DFMO as a single agent and in pediatric and young adult patients with high risk neuroblastoma that is in remission.

Tests (p-value) of the association of survival with ODC1 single nucleotide polymorphism rs2302616 genotype.

Blood: microRNA analysis as predictor of DFMO effect, ornithine decarboxylase (ODC) single nucleotide polymorphism (SNP) analysis in DNA isolated from nucleated cells

Pharmacokinetic assay Cmax/D

Samples drawn at 5 timepoints: (0 (pre dose), 30min, 1 hour, 3 hours, and 6 hours post-dose) on two different days.

Pharmacokinetic assay AUC(0-6 hr)/D

Samples drawn at 5 timepoints: (0 (pre dose), 30min, 1 hour, 3 hours, and 6 hours post-dose) on two different days

Pharmacokinetic assay- tmax, hr

Samples drawn at 5 timepoints: (0 (pre dose), 30min, 1 hour, 3 hours, and 6 hours post-dose) on two different days

Beat NB Cancer Foundation

Because of Ezra

K C Pharmaceuticals Inc.

Giselle Sholler

Beat Childhood Cancer Chair

All subjects that received at least one dose of DFMO. Since both arms received the same exact treatment, they were combined into one reporting group.

All Study Subjects

Subjects that are in remission at the end of upfront therapy defined as chemotherapy (5-7 cycles), surgery as indicated, consolidation therapy as indicated, radiation therapy as indicated, anti-GD2 antibody therapy with retinoic acid up to 6 cycles.

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.

Stratum 1

Subjects that are in remission after any previous relapse or refractory therapy.

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.

Stratum 2

Total

Age, Continuous

Sex: Female, Male

Race (NIH/OMB)

Dr. Giselle Sholler

One subject removed from Stratum 1 due to not fitting study criteria upon review

Number of Participants With Event Free Survival (EFS) During Study.

Percentage of Participants With Overall Survival (OS)

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.

DFMO: 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.

DFMO Twice Daily

Stratum 1 and 2 were analyzed together as one safety group as per statistical analysis plan.

Number of Participants With Adverse Events as a Measure of Safety and Tolerability

Tests of the association of survival with ODC1 single nucleotide polymorphism rs2302616 genotype.

GG, GT, TT

GG or GT, TT

GG, GT or TT

Stratum 1 and 2 were analyzed together as one group as per statistical analysis plan.

Test the Association of Survival With ODC1 Genotype

Includes study subjects from either stratum that signed an optional consent to have PK's collected

Study Subjects Consented to PK Collection

12 subjects from both Stratum 1 and 2 were analyzed together as one group as per statistical analysis plan.

Spots Global Cancer Trial Database for neuroblastoma in remission