NMTT- Neuroblastoma Maintenance Therapy Trial Using Difluoromethylornithine (DFMO)

Who is this study for? Patients with Neuroblastoma
What treatments are being studied? Difluoromethylornithine (DFMO)
Status: Recruiting
Location: See all (47) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 1
Maximum Age: 30
Healthy Volunteers: f
View:

• 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

• 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).

Locations
United States
Alabama
University of Alabama, Children's of Alabama
RECRUITING
Birmingham
Arkansas
Arkansas Children's Hospital
RECRUITING
Little Rock
California
UCSF Benioff Children's Hospital Oakland-
RECRUITING
Oakland
Rady Children's Hospital
RECRUITING
San Diego
Colorado
Rocky Mountain Pediatric Hematology
RECRUITING
Denver
Connecticut
Connecticut Children's Hospital
RECRUITING
Hartford
Florida
University of Florida
RECRUITING
Gainesville
Arnold Palmer Hospital for Children
RECRUITING
Orlando
All Children's Hospital Johns Hopkins Medicine
RECRUITING
St. Petersburg
St. Joseph's Children's Hospital
RECRUITING
Tampa
Georgia
Augusta University Health
RECRUITING
Augusta
Hawaii
Kapiolani Medical Center for Women and Children
RECRUITING
Honolulu
Idaho
St. Lukes
RECRUITING
Boise
Illinois
Rebecca McFall
RECRUITING
Chicago
Kentucky
Kentucky Children's Hospital
RECRUITING
Lexington
University of Louisville
RECRUITING
Louisville
Massachusetts
Tufts Medical Center
COMPLETED
Boston
University of Massachusetts Medical School Worcester
WITHDRAWN
Worcester
Michigan
Helen DeVos Children's Hospital
RECRUITING
Grand Rapids
Minnesota
Children's Hospital and Clinics of Minnesota
RECRUITING
Minneapolis
Missouri
Children's Mercy Hospitals and Clinics
RECRUITING
Kansas City
Gina Martin
RECRUITING
St Louis
North Carolina
Levine Children's Hospital
RECRUITING
Charlotte
Duke University
RECRUITING
Durham
New Jersey
Hackensack University Medical Center
RECRUITING
Hackensack
New York
New York University
RECRUITING
New York
Ohio
Cleveland Clinic Children's
RECRUITING
Cleveland
Nationwide Children's Hospital
ACTIVE_NOT_RECRUITING
Columbus
Oregon
Randall Children's Hospital
RECRUITING
Portland
Pennsylvania
Penn State Milton S. Hershey Medical Center and Children's Hospital
RECRUITING
Hershey
Rhode Island
Hasbro Children's Hospital
RECRUITING
Providence
South Carolina
Medical University of South Carolina
RECRUITING
Charleston
Tennessee
Monroe Carrell Jr. Children's Hospital at Vanderbilt
RECRUITING
Nashville
Texas
Dell Children's Blood and Cancer Center
RECRUITING
Austin
Children's Medical Center
RECRUITING
Dallas
Texas Children's Cancer and Hematology Centers
RECRUITING
Houston
Utah
Primary Children's Hospital
RECRUITING
Salt Lake City
Virginia
Children's Hospital of The King's Daughters
RECRUITING
Norfolk
Virginia Commonwealth University
RECRUITING
Richmond
Wisconsin
Medical College of Wisconsin
RECRUITING
Milwaukee
Other Locations
Canada
Alberta Children's Hospital
RECRUITING
Calgary
Montreal Children's Hospital
RECRUITING
Montreal
UHC Sainte-Justine
RECRUITING
Montreal
CHUQ
RECRUITING
Québec
CIUSSS de l'Estrie-CHUS
RECRUITING
Sherbrooke
Janesway Children's Health and Rehabilitation Centre
RECRUITING
St. John's
CancerCare Manitoba
RECRUITING
Winnipeg
Contact Information
Primary
BCC Enroll
BCCEnroll@pennstatehealth.psu.edu
7175310003
Time Frame
Start Date: 2016-02
Estimated Completion Date: 2033-02
Participants
Target number of participants: 441
Treatments
Experimental: 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.
Sponsors
Collaborators: Beat NB Cancer Foundation, Team Parker for Life
Leads: Giselle Sholler

This content was sourced from clinicaltrials.gov

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