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

February 28, 2013 updated by: United Therapeutics

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

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.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

1

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85016
        • Phoenix Children's Hospital
    • California
      • San Diego, California, United States, 92123
        • Rady Children's Hospital of San Diego
    • District of Columbia
      • Washington, District of Columbia, United States, 20057
        • Georgetown Medical Center
    • Florida
      • St. Petersburg, Florida, United States, 33701
        • All Children's Hospital in Florida
    • Louisiana
      • New Orleans, Louisiana, United States, 70118
        • LSU Health Sciences Center; Children's Hospital
    • Minnesota
      • Minneapolis, Minnesota, United States, 55404
        • Children's Hospitals and Clinics of Minnesota
    • New York
      • Bronx, New York, United States, 10467
        • Children's Hospital at Montefiore
    • North Carolina
      • Durham, North Carolina, United States, 27705
        • Duke University Medical Center
    • Ohio
      • Columbus, Ohio, United States, 43205
        • Nationwide Childrens Hospital
    • Oklahoma
      • Oklahoma City, Oklahoma, United States, 73104
        • University of Oklahoma Cancer Center
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15224
        • Children's Hospital of Pittsburgh of UPMC
    • Texas
      • Dallas, Texas, United States, 75230
        • US Oncology
      • Houston, Texas, United States, 77030
        • The University of Texas M.D. Anderson Cancer Center
    • Utah
      • Salt Lake City, Utah, United States, 84113
        • University of Utah Medical Center
    • Vermont
      • Burlington, Vermont, United States, 05405
        • Vermont Cancer Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year to 13 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: ARM I
Intravenous MAb-3F8 plus Subcutaneous Granulocyte Macrophage Colony Stimulating Factor (GM-CSF)
Biological: MAb-3F8
Biological: Subcutaneous Granulocyte Macrophage Colony Stimulating Factor (GM-CSF)
Active Comparator: ARM II
Oral 13-cis-Retinoic Acid (RA) plus Subcutaneous Granulocyte Macrophage Colony Stimulating Factor (GM-CSF)
Biological: Subcutaneous Granulocyte Macrophage Colony Stimulating Factor (GM-CSF)
Biological: 13-cis-Retinoic Acid

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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.
Time Frame: two years
two years

Secondary Outcome Measures

Outcome Measure
Time Frame
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.
Time Frame: two years
two years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

United Therapeutics

Investigators

  • Principal Investigator: Peter E. Zage, M.D., M.D. Anderson Cancer Center

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

August 1, 2009

Primary Completion (Actual)

August 1, 2010

Study Registration Dates

First Submitted

August 31, 2009

First Submitted That Met QC Criteria

August 31, 2009

First Posted (Estimate)

September 1, 2009

Study Record Updates

Last Update Posted (Estimate)

March 8, 2013

Last Update Submitted That Met QC Criteria

February 28, 2013

Last Verified

February 1, 2013

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 3F8-NB-201

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Primary Refractory Neuroblastoma

Clinical Trials on MAb-3F8

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Dominican Republic

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe