Status:

TERMINATED

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

Lead Sponsor:

United Therapeutics

Conditions:

Primary Refractory Neuroblastoma

Eligibility:

All Genders

18-13 years

Phase:

PHASE2

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. ...

Eligibility Criteria

Inclusion

  • 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

  • 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.

Key Trial Info

Start Date :

August 1 2009

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

Estimated Enrollment :

1 Patients enrolled

Trial Details

Trial ID

NCT00969722

Start Date

August 1 2009

Last Update

March 8 2013

Active Locations (15)

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Page 1 of 4 (15 locations)

1

Phoenix Children's Hospital

Phoenix, Arizona, United States, 85016

2

Rady Children's Hospital of San Diego

San Diego, California, United States, 92123

3

Georgetown Medical Center

Washington D.C., District of Columbia, United States, 20057

4

All Children's Hospital in Florida

St. Petersburg, Florida, United States, 33701