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
Information source: United Therapeutics
ClinicalTrials.gov processed this data on August 23, 2015
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Primary Refractory Neuroblastoma
Intervention: MAb-3F8 (Biological); Subcutaneous Granulocyte Macrophage Colony Stimulating Factor (GM-CSF) (Biological); 13-cis-Retinoic Acid (Biological)
Phase: Phase 2
Status: Terminated
Sponsored by: United Therapeutics Official(s) and/or principal investigator(s):
Peter E. Zage, M.D., Principal Investigator, Affiliation: M.D. Anderson Cancer Center
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.
Clinical Details
Official title: 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
Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Primary outcome: 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.
Secondary outcome: 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.
Eligibility
Minimum age: 18 Months.
Maximum age: 13 Years.
Gender(s): Both.
Criteria:
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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.
Locations and Contacts
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, 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, 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
Additional Information
Starting date: August 2009
Last updated: February 28, 2013
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