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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01586260
Recruitment Status : Active, not recruiting
First Posted : April 26, 2012
Last Update Posted : March 18, 2020
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

Brief Summary:

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.


Condition or disease Intervention/treatment Phase
Neuroblastoma Drug: DFMO Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 89 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Prevention
Official Title: A Phase II Preventative Trial of DFMO as a Single Agent in Patients With High Risk Neuroblastoma in Remission
Actual Study Start Date : June 2012
Estimated Primary Completion Date : April 26, 2021
Estimated Study Completion Date : April 26, 2021

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Neuroblastoma

Arm Intervention/treatment
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.
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




Primary Outcome Measures :
  1. 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 ]
    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)


Secondary Outcome Measures :
  1. 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 ]
    To evaluate the preventative activity of DFMO as a single agent in patients with neuroblastoma who are in remission based on: Overall Survival (OS)

  2. Number of Participants with Adverse Events as a Measure of Safety and Tolerability [ Time Frame: 2 years ]
    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.

  3. Biology studies [ Time Frame: 2 years ]
    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



Information from the National Library of Medicine

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Ages Eligible for Study:   up to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01586260


Locations
Show Show 18 study locations
Sponsors and Collaborators
Giselle Sholler
Cancer Prevention Pharmaceuticals, Inc.
University of Arizona
University of Hawaii
University of Vermont
Beat NB Cancer Foundation
Investigators
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Study Chair: Giselle Sholler, MD The Spectrum Health Group
Additional Information:
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Responsible Party: Giselle Sholler, Study Chair, Spectrum Health Hospitals
ClinicalTrials.gov Identifier: NCT01586260    
Other Study ID Numbers: NMTRC 003
First Posted: April 26, 2012    Key Record Dates
Last Update Posted: March 18, 2020
Last Verified: March 2020
Keywords provided by Giselle Sholler, Spectrum Health Hospitals:
Neuroblastoma in remission
Relapsed Neuroblastoma
Refractory Neuroblastoma
Additional relevant MeSH terms:
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Neuroblastoma
Neuroectodermal Tumors, Primitive, Peripheral
Neuroectodermal Tumors, Primitive
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Eflornithine
Antineoplastic Agents
Trypanocidal Agents
Antiprotozoal Agents
Antiparasitic Agents
Anti-Infective Agents
Ornithine Decarboxylase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action