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Neuroblastoma Maintenance Therapy Trial (NMTT)

  • Status
    Accepting Candidates
  • Age
    1 Year - 30 Years
  • Sexes
    All
  • Healthy Volunteers
    No

Objective

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 750 mg/m2 ± 250 mg/m2 BID (strata 1, 2, 3, and 4) OR 2500 mg/m2 BID (stratum 1B) 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.

Details

Full study title NMTT- Neuroblastoma Maintenance Therapy Trial Using Difluoromethylornithine (DFMO)
Protocol number OCR33522
ClinicalTrials.gov ID NCT02679144
Phase Phase 2

Eligibility

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 1/1B: 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/B 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 1/1B: 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

Lead researcher

  • Pediatric Hematologist/Oncologist (Child Cancer Specialist)
    Joanne Lagmay

Participate in a study

Here are some general steps to consider when participating in a research study:

  1. Step
    1

    Contact the research team

    Call or email the research team listed within the specific clinical trial or study to let them know that you're interested. A member of the research team, such as the researcher or study coordinator, will be available to tell you more about the study and to answer any questions or concerns you may have.

    Primary contact

  2. Step
    2

    Get screened to confirm eligibility

    You may be asked to take part in prescreening to make sure you are eligible for a study. The prescreening process ensures it is safe for you to participate. During the prescreening process, you will be asked some questions and you may also be asked to schedule tests or procedures to confirm your eligibility.

  3. Step
    3

    Provide your consent to participate

    If you are eligible and want to join the clinical trial or study, a member of the research team will ask for your consent to participate. To give consent, you will be asked to read and sign a consent form for the study. This consent form explains the study's purpose, procedures, risks, benefits and provides other important information, such as the study team's contact information.

  4. Step
    4

    Participate

    If you decide to participate in a clinical trial or study, the research team will keep you informed of the study requirements and what you will need to do to throughout the study. For some trials or studies, your health care provider may work with the research team to ensure there are no conflicts with other medications or treatments.