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

NCT ID: NCT00969722

Last Updated: 2013-03-08

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2
• Total Enrollment: 1 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2009-08-31

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.

Conditions

Primary Refractory Neuroblastoma

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

ARM I

Intravenous MAb-3F8 plus Subcutaneous Granulocyte Macrophage Colony Stimulating Factor (GM-CSF)

Group Type: EXPERIMENTAL

MAb-3F8

Intervention Type: BIOLOGICAL

Subcutaneous Granulocyte Macrophage Colony Stimulating Factor (GM-CSF)

Intervention Type: BIOLOGICAL

ARM II

Oral 13-cis-Retinoic Acid (RA) plus Subcutaneous Granulocyte Macrophage Colony Stimulating Factor (GM-CSF)

Group Type: ACTIVE_COMPARATOR

Subcutaneous Granulocyte Macrophage Colony Stimulating Factor (GM-CSF)

Intervention Type: BIOLOGICAL

13-cis-Retinoic Acid

Intervention Type: BIOLOGICAL

Interventions

MAb-3F8

Intervention Type: BIOLOGICAL

Subcutaneous Granulocyte Macrophage Colony Stimulating Factor (GM-CSF)

Intervention Type: BIOLOGICAL

13-cis-Retinoic Acid

Intervention Type: BIOLOGICAL

Eligibility 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.

• Minimum Eligible Age: 18 Months
• Maximum Eligible Age: 13 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

United Therapeutics

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Peter E. Zage, M.D.

Role: PRINCIPAL_INVESTIGATOR

M.D. Anderson Cancer Center

Locations

Phoenix Children's Hospital

Phoenix, Arizona, United States

Rady Children's Hospital of San Diego

San Diego, California, United States

Georgetown Medical Center

Washington D.C., District of Columbia, United States

All Children's Hospital in Florida

St. Petersburg, Florida, United States

LSU Health Sciences Center; Children's Hospital

New Orleans, Louisiana, United States

Children's Hospitals and Clinics of Minnesota

Minneapolis, Minnesota, United States

Children's Hospital at Montefiore

The Bronx, New York, United States

Duke University Medical Center

Durham, North Carolina, United States

Nationwide Childrens Hospital

Columbus, Ohio, United States

University of Oklahoma Cancer Center

Oklahoma City, Oklahoma, United States

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, United States

US Oncology

Dallas, Texas, United States

The University of Texas M.D. Anderson Cancer Center

Houston, Texas, United States

University of Utah Medical Center

Salt Lake City, Utah, United States

Vermont Cancer Center

Burlington, Vermont, United States

Countries

United States

Other Identifiers

3F8-NB-201

Identifier Type: -

Identifier Source: org_study_id