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Neuroblastoma Maintenance Therapy Trial (NMTT)

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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02679144
Recruitment Status : Recruiting
First Posted : February 10, 2016
Last Update Posted : April 17, 2020
Sponsor:
Collaborators:
KC Pharma
Beat NB Cancer Foundation
Information provided by (Responsible Party):
Giselle Sholler, Spectrum Health Hospitals

Tracking Information
First Submitted Date  ICMJE February 4, 2016
First Posted Date  ICMJE February 10, 2016
Last Update Posted Date April 17, 2020
Actual Study Start Date  ICMJE February 2016
Estimated Primary Completion Date February 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: February 5, 2016)
Number of participants with event free survival (EFS) during study. [ Time Frame: 2 years ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: February 5, 2016)
  • Length of time that participants experience Overall Survival (OS) [ Time Frame: 7 years ]
  • Number of Participants with Adverse Events as a Measure of Safety and Tolerability [ Time Frame: 2 years ]
  • Peak Plasma Concentration (Cmax) [ Time Frame: 1 year ]
    Pharmacokinetic assay
  • Area under the plasma concentration versus time curve (AUC) [ Time Frame: 1 year ]
    Pharmacokinetic assay
  • Time to reach Peak Plasma Concentration (Tmax) [ Time Frame: 1 year ]
    Pharmacokinetic assay
  • Number of participants with ODC (Ornithine decarboxylase) single nucleotide polymorphisms. [ Time Frame: 1 year ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Neuroblastoma Maintenance Therapy Trial
Official Title  ICMJE NMTT- Neuroblastoma Maintenance Therapy Trial Using Difluoromethylornithine (DFMO)
Brief Summary Difluoromethylornithine (DFMO) will be used in an open label, single agent, multicenter, study for patients with neuroblastoma in remission. In this study subjects will receive 730 Days of oral difluoromethylornithine (DFMO) at a dose of 500 to 1000 mg/m2 BID on each day of study. This study will focus on the use of DFMO in high risk neuroblastoma patients that are in remission as a strategy to prevent recurrence.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Prevention
Condition  ICMJE Neuroblastoma
Intervention  ICMJE Drug: Difluoromethylornithine (DFMO)
Subjects will receive 730 Days of oral difluoromethylornithine (DFMO) at a dose of 500 to 1000 mg/m2 BID on each day of study.
Other Name: eflornithine
Study Arms  ICMJE Experimental: Difluoromethylornithine (DFMO)
Subjects will receive 730 Days of oral difluoromethylornithine (DFMO) at a dose of 500 to 1000 mg/m2 twice daily on each day of study.
Intervention: Drug: Difluoromethylornithine (DFMO)
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: February 5, 2016)
258
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE February 2025
Estimated Primary Completion Date February 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • All patients must have a pathologically confirmed diagnosis of neuroblastoma, < 30.99 years of age and classified as high risk at the time of diagnosis. Exception: patients who are initially diagnosed as non-high-risk neuroblastoma, but later converted (and/or relapsed) to high risk neuroblastoma are also eligible.
  • All patients must be in complete remission (CR):

    1. No evidence of residual disease on scan
    2. No evidence of disease metastatic to bone marrow.
  • Specific Criteria by Stratum:

Stratum 1: All patients must have completed standard upfront therapy that replicates treatment which patients who were enrolled on ANBL0032 received, including:

intensive induction chemotherapy and (if feasible) resection of primary tumor, followed by: consolidation with high-dose chemotherapy with stem cell transplant and radiotherapy, followed by: immunotherapy with Ch14.18/IL-2/GM-CSF (dinutuximab) and retinoic acid;.

All subjects on Stratum 1 must have also met the following criteria:

• A pre-transplant disease status evaluation that met International Neuroblastoma Response Criteria (INRC) for CR (complete response), VGPR (very good partial response), or PR (partial response) for primary site, soft tissue metastases and bone metastases. Patients who meet those criteria must also meet the protocol-specified criteria for bone marrow response prior to transplant as outlined below: No more than 10% tumor involvement (based on total nucleated cellular content) seen on any specimen from a bilateral bone marrow aspirate/biopsy.

Stratum 2: Neuroblastoma that is in first complete remission following standard upfront therapy different from that described for Stratum 1.

Stratum 3: Neuroblastoma that failed to have a response of at least PR following induction chemotherapy and surgical resection of the primary tumor, but that has achieved CR following additional therapy.

Stratum 4: Patients who have achieved a second or subsequent CR following relapse(s).

  • Pre-enrollment tumor survey: Prior to enrollment on this study, a determination of mandatory disease staging must be performed:

    • Tumor imaging studies including
    • Bilateral bone marrow aspirates and biopsy
    • This disease assessment is required for eligibility and preferably should be done within 2 weeks prior to enrollment, but must be done within a maximum of 4 weeks before enrollment.
  • Timing from prior therapy:

Stratum 1: Enrollment no later than 60 days after completion of upfront therapy, (last dose of cis-retinoic acid) with a maximum of 6 cycles of cis-retinoic acid maintenance therapy.

Stratum 2, 3 and 4: Enrollment no later than 60 days from last dose of the most recent therapy.

  • Patients must have a Lansky or Karnofsky Performance Scale score of > 50% and patients must have a life expectancy of ≥ 2 months.
  • All clinical and laboratory studies for organ functions to determine eligibility must be performed within 7 days prior to enrollment unless otherwise indicated below.
  • Patients must have adequate organ functions at the time of registration:

    • Hematological: Total absolute phagocyte count ≥1000/μL
    • Liver: Subjects must have adequate liver function
    • Renal: Adequate renal function
  • Females of childbearing potential must have a negative pregnancy test. Patients of childbearing potential must agree to use an effective birth control method. Female patients who are lactating must agree to stop breast-feeding.
  • Written informed consent in accordance with institutional and FDA (food and drug administration) guidelines must be obtained from all subjects (or patients' legal representative).

Exclusion Criteria:

  • BSA (Body Surface Area) of <0.25 m2.
  • 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 hematological and bone marrow suppression effects of prior chemotherapy.
  • 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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 1 Year to 30 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Genevieve Bergendahl, MSN 6162670335 genevieve.bergendahl@helendevoschildrens.org
Contact: Alyssa VanderWerff, MS 6162670327 alyssa.vanderwerff@helendevoschildrens.org
Listed Location Countries  ICMJE Canada,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02679144
Other Study ID Numbers  ICMJE NMTRC014
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Undecided
Responsible Party Giselle Sholler, Spectrum Health Hospitals
Study Sponsor  ICMJE Giselle Sholler
Collaborators  ICMJE
  • KC Pharma
  • Beat NB Cancer Foundation
Investigators  ICMJE
Study Chair: Giselle Sholler, MD Spectrum Health Hospitals
PRS Account Spectrum Health Hospitals
Verification Date April 2020

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