Neuroblastoma Maintenance Therapy Trial

Running, not enrolling · Phase 2

Conditions studied: Neuroblastoma

In brief

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.

Key facts

Study ID

NCT02679144

Run by

Giselle Sholler

People needed

441

Starts

2016-02-01

Expected to finish

2033-02-01

Last updated by the study team

2026-06-12

Who can join

Age: 1 and older, up to 30. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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):
• No evidence of residual disease on scan
• 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

You may not qualify if…

• 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.

Where it is running

• University of Alabama, Children's of Alabama — Birmingham, Alabama, United States
• Arkansas Children's Hospital — Little Rock, Arkansas, United States
• UCSF Benioff Children's Hospital Oakland- — Oakland, California, United States
• Rady Children's Hospital — San Diego, California, United States
• Rocky Mountain Pediatric Hematology — Denver, Colorado, United States
• Connecticut Children's Hospital — Hartford, Connecticut, United States
• University of Florida — Gainesville, Florida, United States
• Arnold Palmer Hospital for Children — Orlando, Florida, United States
• All Children's Hospital Johns Hopkins Medicine — St. Petersburg, Florida, United States
• St. Joseph's Children's Hospital — Tampa, Florida, United States
• Augusta University Health — Augusta, Georgia, United States
• Kapiolani Medical Center for Women and Children — Honolulu, Hawaii, United States
• St. Lukes — Boise, Idaho, United States
• Advocate Aurora Research Institute — Chicago, Illinois, United States
• University of Iowa — Iowa City, Iowa, United States
• Kentucky Children's Hospital — Lexington, Kentucky, United States
• Norton Children's Research Institute/Affiliated with University of Louisville School of Medicine — Louisville, Kentucky, United States
• Tufts Medical Center — Boston, Massachusetts, United States
• Helen DeVos Children's Hospital — Grand Rapids, Michigan, United States
• Children's Hospital and Clinics of Minnesota — Minneapolis, Minnesota, United States
• Children's Mercy Hospitals and Clinics — Kansas City, Missouri, United States
• Cardinal Glennon Children's Medical Center — St Louis, Missouri, United States
• Hackensack University Medical Center — Hackensack, New Jersey, United States
• NYU Langone Health Hassenfeld Children's Hospital — New York, New York, United States
• The Children's Hospital at Montefiore — The Bronx, New York, United States

Full record on ClinicalTrials.gov

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