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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
This study is currently recruiting participants.
Verified by United Therapeutics, December 2009
First Received: August 31, 2009   Last Updated: December 3, 2009   History of Changes
Sponsor: United Therapeutics
Information provided by: United Therapeutics
ClinicalTrials.gov Identifier: NCT00969722
  Purpose

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.


Condition Intervention Phase
Primary Refractory Neuroblastoma
Biological: MAb-3F8
Biological: Subcutaneous Granulocyte Macrophage Colony Stimulating Factor (GM-CSF)
Biological: 13-cis-Retinoic Acid
Phase II

Study Type: Interventional
Study Design: Treatment, Randomized, Open Label, Safety/Efficacy Study
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

Resource links provided by NLM:


Further study details as provided by United Therapeutics:

Primary Outcome Measures:
  • 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. [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • 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.

Estimated Enrollment: 40
Study Start Date: August 2009
Arms Assigned Interventions
ARM I: Experimental
Intravenous MAb-3F8 plus Subcutaneous Granulocyte Macrophage Colony Stimulating Factor (GM-CSF)
Biological: MAb-3F8 Biological: Subcutaneous Granulocyte Macrophage Colony Stimulating Factor (GM-CSF)
ARM II: Active Comparator
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

  Eligibility

Ages Eligible for Study:   18 Months to 13 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00969722

Contacts
Contact: Mary Smith, Ph.D. 919-425-8174 msmith@unither.com
Contact: Richard E. Stark, B.Sc. 507-536-0308 rstark@unither.com

Locations
United States, Arizona
Phoenix Children's Hospital Recruiting
Phoenix, Arizona, United States, 85016
Contact: Jessica Boklan, M.D.     602-546-0920     jboklan@phoenixchildrens.com    
Contact: Amanda Janitz, R.N.     602-546-0188     ajanitz@phoenixchildrens.com    
Principal Investigator: Jessica Boklan, M.D.            
United States, California
Rady Children's Hospital of San Diego Recruiting
San Diego, California, United States, 92123
Contact: Jennifer Willert, M.D.     858-966-5811     jwillert@chsd.org    
Contact: Sara Hingtgen, R.N.     858-966-8381     shingtgen@chsd.org    
Principal Investigator: Jennifer Willert, M.D.            
United States, District of Columbia
Georgetown Medical Center Recruiting
Washington, District of Columbia, United States, 20057
Contact: Corina Gonzales, M.D.     202-444-7599     gonzalec@georgetown.edu    
Contact: Anjum Zaki     202-687-1351     aaz2@georgetown.edu    
Principal Investigator: Corina Gonzales, M.D.            
United States, Florida
All Children's Hospital in Florida Recruiting
St. Petersburg, Florida, United States, 33701
Contact: Aleksandra Petrovic, M.D.     727-767-4665     petrovia@allkids.org    
Contact: Amber Herschberger, R.N.     727-767-4178     herschbergera@allkids.org    
Principal Investigator: Aleksandra Petrovic, M.D.            
United States, Louisiana
LSU Health Sciences Center; Children's Hospital Recruiting
New Orleans, Louisiana, United States, 70118
Contact: Lolie Yu, M.D.     504-896-9848     LYu@lsuhsc.edu    
Contact: Mimi Hermann, R.N.     504-894-5377     mhermann@clinicaltrialscenter.org    
Principal Investigator: Lolie Yu, M.D.            
United States, Minnesota
Children's Hospitals and Clinics of Minnesota Recruiting
Minneapolis, Minnesota, United States, 55404
Contact: Jawhar Rawwas, M.D.     612-813-5940     Jawhar.Rawwas@childrensmn.org    
Contact: Lezlie Rabine, R.N.     612-813-6751     Lezlie.Rabine@childrensmn.org    
Principal Investigator: Jawhar Rawwas, M.D.            
United States, New York
Children's Hospital at Montefiore Recruiting
Bronx, New York, United States, 10467
Contact: Rosanna Ricafort, M.D.     718-741-2342     rricafor@montefiore.org    
Contact: Catherine Stanford     718-741-2356     cstanfor@montefiore.org    
Principal Investigator: Rosanna Ricafort, M.D.            
United States, North Carolina
Duke University Medical Center Recruiting
Durham, North Carolina, United States, 27705
Contact: Timothy Driscoll, M.D.     919-668-1100     drisc002@mc.duke.edu    
Contact: Caroline Peiffer, R.N.     919-668-1280     caroline.peifer@duke.edu    
Principal Investigator: Timothy Driscoll, M.D.            
United States, Ohio
Nationwide Childrens Hospital Recruiting
Columbus, Ohio, United States, 43205
Contact: Mark Ranalli, M.D.     614-722-3563     Mark.ranalli@nationwidechildrens.org    
Contact: Barb Pugh, R.N.     614-722-3568     Barbara.Pugh@nationwidechildrens.org    
Principal Investigator: Mark Ranalli, M.D.            
United States, Oklahoma
University of Oklahoma Cancer Center Recruiting
Oklahoma City, Oklahoma, United States, 73104
Contact: Rene McNall-Knapp, M.D.     405-271-4412     Rene-McNall@ouhsc.edu    
Contact: Elaine Reeves, R.N.     405-271-5849     elaine-reeves@ouhsc.edu    
Principal Investigator: Rene McNall-Knapp, M.D.            
United States, Pennsylvania
Children's Hospital of Pittsburgh of UPMC Recruiting
Pittsburgh, Pennsylvania, United States, 15224
Contact: Jean Tersak, M.D.     412-692-5055     Jean.Tersak@chp.edu    
Contact: Michele Paris, R.N.     412-692-6815     michele.paris@chp.edu    
Principal Investigator: Jean Tersak, M.D.            
United States, Texas
The University of Texas M.D. Anderson Cancer Center Recruiting
Houston, Texas, United States, 77030
Contact: Peter E. Zage, M.D.     713-792-6624     pezage@mdanderson.org    
Contact: MaryAnn Giannan, R.N.     713-745-0774     mgianan@mdanderson.org    
Principal Investigator: Peter E. Zage, M.D.            
US Oncology Recruiting
Dallas, Texas, United States, 75230
Contact: Stan Goldman, M.D.     972-566-6647     stan.goldman@usoncology.com    
Contact: Tessa Bolt, R.N.     972-566-4449     Tessa.Bolt@usoncology.com    
Principal Investigator: Stan Goldman, M.D.            
United States, Utah
University of Utah Medical Center Recruiting
Salt Lake City, Utah, United States, 84113
Contact: Mark Fluchel, M.D.     801-662-4740     mark.fluchel@imail.org    
Contact: Stan Pies, R.N.     801-662-4715     stan.pies@imail.org    
Principal Investigator: Mark Fluchel, M.D.            
United States, Vermont
Vermont Cancer Center Recruiting
Burlington, Vermont, United States, 05405
Contact: Giselle Sholler, M.D.     802-847-2850     Giselle.Sholler@uvm.edu    
Contact: Shannon Lenox, R.N.     802-656-9283     Shannon.Lenox@med.uvm.edu    
Principal Investigator: Giselle Sholler, M.D.            
Sponsors and Collaborators
United Therapeutics
Investigators
Principal Investigator: Peter E. Zage, M.D. M.D. Anderson Cancer Center
  More Information

No publications provided

Study ID Numbers: 3F8-NB-201
Study First Received: August 31, 2009
Last Updated: December 3, 2009
ClinicalTrials.gov Identifier: NCT00969722     History of Changes
Health Authority: United States: Food and Drug Administration

Keywords provided by United Therapeutics:
Neuroblastoma
3F8
Primary refractory

Additional relevant MeSH terms:
Neuroectodermal Tumors, Primitive
Neoplasms by Histologic Type
Antineoplastic Agents
Neoplasms, Nerve Tissue
Pharmacologic Actions
Neuroblastoma
Keratolytic Agents
Neuroectodermal Tumors
Neoplasms
Therapeutic Uses
Neoplasms, Germ Cell and Embryonal
Isotretinoin
Tretinoin
Neoplasms, Neuroepithelial
Dermatologic Agents
Neuroectodermal Tumors, Primitive, Peripheral
Neoplasms, Glandular and Epithelial

ClinicalTrials.gov processed this record on February 08, 2010