Primary Refractory Neuroblastoma Clinical Trial
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
| Verified date | February 2013 |
| Source | United Therapeutics |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | United States: Food and Drug Administration |
| Study type | Interventional |
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.
| Status | Terminated |
| Enrollment | 1 |
| Est. completion date | |
| Est. primary completion date | August 2010 |
| Accepts healthy volunteers | No |
| Gender | Both |
| Age group | 18 Months to 13 Years |
| Eligibility |
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. |
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
| Country | Name | City | State |
|---|---|---|---|
| United States | Children's Hospital at Montefiore | Bronx | New York |
| United States | Vermont Cancer Center | Burlington | Vermont |
| United States | Nationwide Childrens Hospital | Columbus | Ohio |
| United States | US Oncology | Dallas | Texas |
| United States | Duke University Medical Center | Durham | North Carolina |
| United States | The University of Texas M.D. Anderson Cancer Center | Houston | Texas |
| United States | Children's Hospitals and Clinics of Minnesota | Minneapolis | Minnesota |
| United States | LSU Health Sciences Center; Children's Hospital | New Orleans | Louisiana |
| United States | University of Oklahoma Cancer Center | Oklahoma City | Oklahoma |
| United States | Phoenix Children's Hospital | Phoenix | Arizona |
| United States | Children's Hospital of Pittsburgh of UPMC | Pittsburgh | Pennsylvania |
| United States | University of Utah Medical Center | Salt Lake City | Utah |
| United States | Rady Children's Hospital of San Diego | San Diego | California |
| United States | All Children's Hospital in Florida | St. Petersburg | Florida |
| United States | Georgetown Medical Center | Washington | District of Columbia |
| Lead Sponsor | Collaborator |
|---|---|
| United Therapeutics |
United States,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | 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 | No | |
| Secondary | 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 | No |