Neuroblastoma Maintenance Therapy Trial
NMTT
NMTT- Neuroblastoma Maintenance Therapy Trial Using Difluoromethylornithine (DFMO)
1 other identifier
interventional
441
2 countries
48
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 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_2
Started Feb 2016
Longer than P75 for phase_2
48 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
February 1, 2016
CompletedFirst Submitted
Initial submission to the registry
February 4, 2016
CompletedFirst Posted
Study publicly available on registry
February 10, 2016
CompletedPrimary Completion
Last participant's last visit for primary outcome
February 1, 2028
ExpectedStudy Completion
Last participant's last visit for all outcomes
February 1, 2033
June 12, 2026
June 1, 2026
12 years
February 4, 2016
June 10, 2026
Conditions
Outcome Measures
Primary Outcomes (1)
Number of participants with event free survival (EFS) during study.
2 years
Secondary Outcomes (6)
Length of time that participants experience Overall Survival (OS)
7 years
Number of Participants with Adverse Events as a Measure of Safety and Tolerability
2 years
Peak Plasma Concentration (Cmax)
1 year
Area under the plasma concentration versus time curve (AUC)
1 year
Time to reach Peak Plasma Concentration (Tmax)
1 year
- +1 more secondary outcomes
Study Arms (1)
Difluoromethylornithine (DFMO)
EXPERIMENTALSubjects 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.
Interventions
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.
Eligibility Criteria
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
- +12 more criteria
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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Giselle Shollerlead
- Beat NB Cancer Foundationcollaborator
- Team Parker for Lifecollaborator
Study Sites (48)
University of Alabama, Children's of Alabama
Birmingham, Alabama, 35233, United States
Arkansas Children's Hospital
Little Rock, Arkansas, 72202, United States
UCSF Benioff Children's Hospital Oakland-
Oakland, California, 94609, United States
Rady Children's Hospital
San Diego, California, 92123, United States
Rocky Mountain Pediatric Hematology
Denver, Colorado, 80205, United States
Connecticut Children's Hospital
Hartford, Connecticut, 06106, United States
University of Florida
Gainesville, Florida, 32611, United States
Arnold Palmer Hospital for Children
Orlando, Florida, 32806, United States
All Children's Hospital Johns Hopkins Medicine
St. Petersburg, Florida, 33701, United States
St. Joseph's Children's Hospital
Tampa, Florida, 33614, United States
Augusta University Health
Augusta, Georgia, 30912, United States
Kapiolani Medical Center for Women and Children
Honolulu, Hawaii, 96813, United States
St. Lukes
Boise, Idaho, 83712, United States
Advocate Aurora Research Institute
Chicago, Illinois, 60453, United States
University of Iowa
Iowa City, Iowa, 52242, United States
Kentucky Children's Hospital
Lexington, Kentucky, 40536, United States
Norton Children's Research Institute/Affiliated with University of Louisville School of Medicine
Louisville, Kentucky, 40202, United States
Tufts Medical Center
Boston, Massachusetts, 02111, United States
Helen DeVos Children's Hospital
Grand Rapids, Michigan, 49503, United States
Children's Hospital and Clinics of Minnesota
Minneapolis, Minnesota, 55404, United States
Children's Mercy Hospitals and Clinics
Kansas City, Missouri, 64108, United States
Cardinal Glennon Children's Medical Center
St Louis, Missouri, 63104, United States
Hackensack University Medical Center
Hackensack, New Jersey, 07601, United States
NYU Langone Health Hassenfeld Children's Hospital
New York, New York, 10016, United States
The Children's Hospital at Montefiore
The Bronx, New York, 10467, United States
Levine Children's Hospital
Charlotte, North Carolina, 28204, United States
Duke University
Durham, North Carolina, 27708, United States
Cleveland Clinic Children's
Cleveland, Ohio, 44106, United States
Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
Randall Children's Hospital
Portland, Oregon, 97227, United States
Penn State Milton S. Hershey Medical Center and Children's Hospital
Hershey, Pennsylvania, 17033, United States
Hasbro Children's Hospital
Providence, Rhode Island, 02903, United States
Medical University of South Carolina
Charleston, South Carolina, 29425, United States
Monroe Carrell Jr. Children's Hospital at Vanderbilt
Nashville, Tennessee, 37232, United States
Dell Children's Blood and Cancer Center
Austin, Texas, 78723, United States
Children's Medical Center
Dallas, Texas, 75235, United States
Texas Children's Cancer and Hematology Centers
Houston, Texas, 77030, United States
Primary Children's Hospital
Salt Lake City, Utah, 84113, United States
Children's Hospital of The King's Daughters
Norfolk, Virginia, 23507, United States
Virginia Commonwealth University
Richmond, Virginia, 23284, United States
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226, United States
Alberta Children's Hospital
Calgary, Alberta, AB T3B 6A8, Canada
CancerCare Manitoba
Winnipeg, Manitoba, MB R3E 0V9, Canada
Janesway Children's Health and Rehabilitation Centre
St. John's, Newfoundland and Labrador, NL A1B 3V6, Canada
UHC Sainte-Justine
Montreal, Quebec, QC H3S 2G4, Canada
Montreal Children's Hospital
Montreal, Quebec, QC H4A 3H9, Canada
CHUQ
Québec, Quebec, QC G1V 4W6, Canada
CIUSSS de l'Estrie-CHUS
Sherbrooke, Quebec, QC J1H 5H3, Canada
Related Publications (1)
Duke ES, Bradford D, Sinha AK, Mishra-Kalyani PS, Lerro CC, Rivera D, Wearne E, Miller CP, Leighton J, Sabit H, Zhao H, Lane A, Scepura B, Pazdur R, Singh H, Kluetz PG, Donoghue M, Drezner N. US Food and Drug Administration Approval Summary: Eflornithine for High-Risk Neuroblastoma After Prior Multiagent, Multimodality Therapy. J Clin Oncol. 2024 Sep 1;42(25):3047-3057. doi: 10.1200/JCO.24.00546. Epub 2024 Jun 25.
PMID: 38917371DERIVED
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- STUDY CHAIR
Giselle Sholler, MD
Beat Childhood Cancer at Atrium Health
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- PREVENTION
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR INVESTIGATOR
- PI Title
- Beat Childhood Cancer Chair
Study Record Dates
First Submitted
February 4, 2016
First Posted
February 10, 2016
Study Start
February 1, 2016
Primary Completion (Estimated)
February 1, 2028
Study Completion (Estimated)
February 1, 2033
Last Updated
June 12, 2026
Record last verified: 2026-06