Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE2
441 participants
INTERVENTIONAL
2016-02-29
2033-02-28
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
PREVENTION
NONE
Study Groups
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Difluoromethylornithine (DFMO)
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.
Difluoromethylornithine (DFMO)
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.
Interventions
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Difluoromethylornithine (DFMO)
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.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
* 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.
1 Year
30 Years
ALL
No
Sponsors
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Beat NB Cancer Foundation
OTHER
Team Parker for Life
UNKNOWN
Giselle Sholler
OTHER
Responsible Party
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Giselle Sholler
Beat Childhood Cancer Chair
Principal Investigators
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Giselle Sholler, MD
Role: STUDY_CHAIR
Beat Childhood Cancer at Atrium Health
Locations
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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
University of Massachusetts Medical School Worcester
Worcester, 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
Levine Children's Hospital
Charlotte, North Carolina, United States
Duke University
Durham, North Carolina, United States
Cleveland Clinic Children's
Cleveland, Ohio, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Randall Children's Hospital
Portland, Oregon, United States
Penn State Milton S. Hershey Medical Center and Children's Hospital
Hershey, Pennsylvania, United States
Hasbro Children's Hospital
Providence, Rhode Island, United States
Medical University of South Carolina
Charleston, South Carolina, United States
Monroe Carrell Jr. Children's Hospital at Vanderbilt
Nashville, Tennessee, United States
Dell Children's Blood and Cancer Center
Austin, Texas, United States
Children's Medical Center
Dallas, Texas, United States
Texas Children's Cancer and Hematology Centers
Houston, Texas, United States
Primary Children's Hospital
Salt Lake City, Utah, United States
Children's Hospital of The King's Daughters
Norfolk, Virginia, United States
Virginia Commonwealth University
Richmond, Virginia, United States
Medical College of Wisconsin
Milwaukee, Wisconsin, United States
Alberta Children's Hospital
Calgary, Alberta, Canada
CancerCare Manitoba
Winnipeg, Manitoba, Canada
Janesway Children's Health and Rehabilitation Centre
St. John's, Newfoundland and Labrador, Canada
UHC Sainte-Justine
Montreal, Quebec, Canada
Montreal Children's Hospital
Montreal, Quebec, Canada
CHUQ
Québec, Quebec, Canada
CIUSSS de l'Estrie-CHUS
Sherbrooke, Quebec, Canada
Countries
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Central Contacts
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Facility Contacts
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References
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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.
Related Links
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Beat Childhood Cancer Consortium Website
Other Identifiers
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NMTRC014
Identifier Type: -
Identifier Source: org_study_id
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