Study Stopped
Lack of enrollment.
A Study of MAb-3F8 Plus Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF) Versus 13-cis-Retinoic Acid (RA) Plus GM-CSF in Primary Refractory Neuroblastoma Patients
A Randomized Study of Monoclonal Antibody 3F8 Plus Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF) Compared to 13-cis-Retinoic Acid Plus GM-CSF in High Risk Stage 4, Primary Refractory Neuroblastoma Patients
1 other identifier
interventional
1
1 country
15
Brief Summary
This is a multicenter, randomized, controlled, open-label study. Patients meeting inclusion/exclusion criteria will be randomized (1:1) to receive two cycles of MAb-3F8 plus GM-CSF or RA plus GM-CSF. Patients who do not respond to their assigned treatment after two cycles may cross-over to receive the alternate treatment. Disease response and safety will be assessed in all patients after cycle 2 and after cycle 4.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
15 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
August 1, 2009
CompletedFirst Submitted
Initial submission to the registry
August 31, 2009
CompletedFirst Posted
Study publicly available on registry
September 1, 2009
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 1, 2010
CompletedMarch 8, 2013
February 1, 2013
1 year
August 31, 2009
February 28, 2013
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
To compare the proportion of patients achieving a complete bone marrow response measured by an absence of histological evidence of bone marrow disease and by MIBG scan after two cycles of treatment.
two years
Secondary Outcomes (1)
A comparison in treatment arms for disease response as measured by CT/MRI scan and urine catecholamines, MIBG extent of disease scores, disease response in cross-over patients.
two years
Study Arms (2)
ARM I
EXPERIMENTALIntravenous MAb-3F8 plus Subcutaneous Granulocyte Macrophage Colony Stimulating Factor (GM-CSF)
ARM II
ACTIVE COMPARATOROral 13-cis-Retinoic Acid (RA) plus Subcutaneous Granulocyte Macrophage Colony Stimulating Factor (GM-CSF)
Interventions
Eligibility Criteria
You may qualify if:
- Have a diagnosis of stage 4 neuroblastoma diagnosed in accordance with the International Neuroblastoma Staging System: either (a) histologic confirmation which may involve immunohistochemical, ultrastructural, and/or cytogenetic studies, or (b) elevated urinary catecholamines plus tumor cells/clumps in the bone marrow.
- Have evaluable disease or biopsy-proven stable disease in BM by histology or MIBG scan with MIBG-positive disease confined to the bone or bone marrow, plus urine catecholamine results, documented \>3 weeks after conventional chemotherapy or \>6 weeks after stem-cell transplantation. CT, MRI, or bone scan (if necessary) can be done at 2-3 weeks after conventional chemotherapy confirming that the chemotherapy, radiotherapy, and ABMT are not realistic curative options.
- Be between 18 months to 13 years old at diagnosis.
- Have recovered to grade 2 or better toxicities since their prior therapy.
- Must, if female of childbearing potential, be willing to use two forms of medically acceptable contraception (at least one barrier method) and have a negative pregnancy test at screening and monthly thereafter through the first four cycles of treatment.
- Have a performance score of at least 60 from Lansky Play Performance Scale if aged up to 16 years or at least 60 from Karnofsky Scale if aged more than 16 years.
- Have voluntarily agreed to participate.
You may not qualify if:
- Have measurable disease ≥ 1 cm assessed by CT or MRI.
- Have progressive disease (any new lesion; increase of any measurable lesion by \>25%; or previous negative marrow positive for tumor).
- Have disease detectable in CNS (confirmed by CT or MRI of the brain at screening or within 8 weeks of randomization).
- Be receiving alternative therapy for the treatment of neuroblastoma, e.g. radiotherapy or chemotherapy within 3 weeks of randomization.
- Require additional therapy (such as radiotherapy) during the first two treatment cycles.
- Have detectable human anti-mouse antibody titers at screening.
- Have received prior anti-GD2 investigational therapies.
- Have a history of allergies to mouse proteins.
- Have an active infection requiring IV infusion of antibiotics.
- Be currently receiving long-term chronic treatment with immunosuppressive drugs such as cyclosporine, adrenocorticotropic hormone (ACTH), or systemic corticosteroids.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (15)
Phoenix Children's Hospital
Phoenix, Arizona, 85016, United States
Rady Children's Hospital of San Diego
San Diego, California, 92123, United States
Georgetown Medical Center
Washington D.C., District of Columbia, 20057, United States
All Children's Hospital in Florida
St. Petersburg, Florida, 33701, United States
LSU Health Sciences Center; Children's Hospital
New Orleans, Louisiana, 70118, United States
Children's Hospitals and Clinics of Minnesota
Minneapolis, Minnesota, 55404, United States
Children's Hospital at Montefiore
The Bronx, New York, 10467, United States
Duke University Medical Center
Durham, North Carolina, 27705, United States
Nationwide Childrens Hospital
Columbus, Ohio, 43205, United States
University of Oklahoma Cancer Center
Oklahoma City, Oklahoma, 73104, United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224, United States
US Oncology
Dallas, Texas, 75230, United States
The University of Texas M.D. Anderson Cancer Center
Houston, Texas, 77030, United States
University of Utah Medical Center
Salt Lake City, Utah, 84113, United States
Vermont Cancer Center
Burlington, Vermont, 05405, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Peter E. Zage, M.D.
M.D. Anderson Cancer Center
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
August 31, 2009
First Posted
September 1, 2009
Study Start
August 1, 2009
Primary Completion
August 1, 2010
Last Updated
March 8, 2013
Record last verified: 2013-02