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Preventative Trial of DFMO in Patients With High Risk Neuroblastoma in Remission

This study is currently recruiting participants. (see Contacts and Locations)
Verified November 2014 by Spectrum Health Hospitals
Sponsor:
Collaborators:
Cancer Prevention Pharmaceuticals, Inc.
University of Arizona
University of Hawaii
University of Vermont
Beat NB Cancer Foundation
Information provided by (Responsible Party):
Giselle Sholler, Spectrum Health Hospitals
ClinicalTrials.gov Identifier:
NCT01586260
First received: April 24, 2012
Last updated: November 19, 2014
Last verified: November 2014

April 24, 2012
November 19, 2014
June 2012
June 2016   (final data collection date for primary outcome measure)
To evaluate the preventative activity of DFMO as a single agent in patients that are in remission based on: Event free survival (EFS) [ Time Frame: 2 years ] [ Designated as safety issue: No ]
3.1.1 To evaluate the preventative activity of DFMO as a single agent in patients that are in remission based on: Event free survival (EFS)
Same as current
Complete list of historical versions of study NCT01586260 on ClinicalTrials.gov Archive Site
  • To evaluate the preventative activity of DFMO as a single agent in patients with neuroblastoma who are in remission based on: Overall Survival (OS) [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    To evaluate the preventative activity of DFMO as a single agent in patients with neuroblastoma who are in remission based on: Overall Survival (OS)
  • Number of Participants with Adverse Events as a Measure of Safety and Tolerability [ Time Frame: 2 years ] [ Designated as safety issue: Yes ]
    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.
  • Biology studies [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    Biological Correlates to minimally include: 1) Urine: polyamine levels and blood inflammatory markers, 2) Blood: microRNA analysis as predictor of DFMO effect, ODC SNP analysis in DNA isolated from nucleated cells, explorative biomarker analysis 3) Bone Marrow: flow cytometry of minimal residual disease of tumor; explorative biomarker analysis
Same as current
Not Provided
Not Provided
 
Preventative Trial of DFMO in Patients With High Risk Neuroblastoma in Remission
A Phase II Preventative Trial of DFMO 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), 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.

Not Provided
Interventional
Phase 2
Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Prevention
Neuroblastoma in Remission
Drug: 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.
Other Names:
  • eflornithine HCl
  • Difluoromethylornithine
Experimental: 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.
Intervention: Drug: DFMO
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
160
June 2020
June 2016   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • Age: 0-21 years at the time of diagnosis.
  • Diagnosis: histologic verification at either the time of original diagnosis or a previous relapse of high risk neuroblastoma.
  • Disease Status: Neuroblastoma that is in remission
  • Greater than 30 days from completion of cytotoxic and biologic therapy and less than 120 days from previous therapy.
  • A negative urine pregnancy test is required for female subjects of child bearing potential (onset of menses or ≥13 years of age).
  • Both male and female post-pubertal study subjects need to agree to use one of the more effective birth control methods during treatment and for six months after treatment is stopped. These methods include total abstinence (no sex), oral contraceptives ("the pill"), an intrauterine device (IUD), levonorgestrol implants (Norplant), or medroxyprogesterone acetate injections (Depo-provera shots). If one of these cannot be used, contraceptive foam with a condom is recommended.
  • ANC > 500/μl and platelet count >50,000/μl
  • Organ Function Requirements: Subjects must have adequate liver function as defined by:

    • AST and ALT <10x upper limit of normal
    • Serum bilirubin must be ≤ 2.0 mg/dl
    • Serum creatinine based on age/gender
  • Informed Consent: All subjects and/or legal guardians must sign informed written consent. Assent, when appropriate, will be obtained according to institutional guidelines

Exclusion Criteria:

  • Lansky score < 60%
  • BSA (m2) of <0.25
  • Investigational Drugs: Subjects who are currently receiving another investigational drug are excluded from participation.
  • Anti-cancer Agents: Subjects who are currently receiving other anticancer agents are not eligible. Subjects must have fully recovered from the effects of prior chemotherapy (hematological and bone marrow suppression effects).
  • Infection: Subjects who have an uncontrolled infection are not eligible until the infection is judged to be well controlled in the opinion of the investigator.
  • Subjects who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study, or in whom compliance is likely to be suboptimal, should be excluded.
Both
up to 21 Years
No
Contact: Genevieve Bergendahl, RN 616-267-0335 genevieve.bergendahl@helendevoschildrens.org
Contact: Alyssa VanderWerff (616) 267-0327 alyssa.vanderwerff@helendevoschildrens.org
United States
 
NCT01586260
NMTRC 003
Yes
Giselle Sholler, Spectrum Health Hospitals
Giselle Sholler
  • Cancer Prevention Pharmaceuticals, Inc.
  • University of Arizona
  • University of Hawaii
  • University of Vermont
  • Beat NB Cancer Foundation
Study Chair: Giselle Sholler, MD The Spectrum Health Group
Spectrum Health Hospitals
November 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP