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Report for Clinical Trial NCT02395666

Developed by Shray Alag, 2020.
SNP Clinical Trial Gene

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

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.

NCT02395666 Neuroblastoma
MeSH:Neuroblastoma
HPO:Neuroblastoma

1 Interventions

Name: DFMO

Description: 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.
Type: Drug
Group Labels: 1

DFMO twice daily


Primary Outcomes

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

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

Time: 2 Years

Secondary Outcomes

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

Measure: Percentage of Participants With Overall Survival (OS)

Time: 2 Years

Description: 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.

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

Time: 2 years

Description: 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

Measure: Test the Association of Survival With ODC1 Genotype

Time: 2 years

Description: circulating tumor cell analysis

Measure: Circulating Tumor Cell Analysis

Time: 5 years

Description: 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.

Measure: Peak Plasma Concentration (Cmax)

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

Description: 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

Measure: Area Under the Plasma Concentration Versus Time Curve (AUC)

Time: 0 (pre dose), 30min, 1 hour, 3 hours, and 6 hours post-dose on two different days

Description: 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

Measure: Time to Reach Peak Plasma Concentration (Tmax)

Time: 0 (pre dose), 30min, 1 hour, 3 hours, and 6 hours post-dose on two different days

Purpose: Prevention

Allocation: N/A

Single Group Assignment


There is one SNP

SNPs


1 rs2302616

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



HPO Nodes


HPO: