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
Information source: United Therapeutics
Information obtained from ClinicalTrials.gov on October 19, 2009
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Primary Refractory Neuroblastoma
Intervention: MAb-3F8 (Biological); Subcutaneous Granulocyte Macrophage Colony Stimulating Factor (GM-CSF) (Biological); 13-cis-Retinoic Acid (Biological)
Phase: Phase 2
Status: Recruiting
Sponsored by: United Therapeutics Official(s) and/or principal investigator(s):
Peter E. Zage, M.D., Principal Investigator, Affiliation: M.D. Anderson Cancer Center
Overall contact:
Mary Smith, Ph.D., Phone: 919-425-8174, Email: msmith@unither.com
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.
Clinical Details
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
Study design: Treatment, Randomized, Open Label, Safety/Efficacy Study
Primary outcome: 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.
Secondary outcome: 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.
Eligibility
Minimum age: 18 Months.
Maximum age: 13 Years.
Gender(s): Both.
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.
Locations and Contacts
Mary Smith, Ph.D., Phone: 919-425-8174, Email: msmith@unither.com
Phoenix Children's Hospital, Phoenix, Arizona 85016, United States; Recruiting
Jessica Boklan, M.D., Phone: 602-546-0920, Email: jboklan@phoenixchildrens.com
Christian Lawson, R.N., Phone: 602-546-0171, Email: clawson@phoenixchildrens.com
Jessica Boklan, M.D., Principal Investigator
Rady Children's Hospital of San Diego, San Diego, California 92123, United States; Recruiting
Jennifer Willert, M.D., Phone: 858-966-5811, Email: jwillert@chsd.org
Sara Hingtgen, R.N., Phone: 858-966-8381, Email: shingtgen@chsd.org
Jennifer Willert, M.D., Principal Investigator
All Children's Hospital in Florida, St. Petersburg, Florida 33701, United States; Recruiting
Aleksandra Petrovic, M.D., Phone: 727-767-4665, Email: petrovia@allkids.org
Amber Herschberger, R.N., Phone: 727-767-4178, Email: herschbergera@allkids.org
Aleksandra Petrovic, M.D., Principal Investigator
LSU Health Sciences Center; Children's Hospital, New Orleans, Louisiana 70118, United States; Recruiting
Lolie Yu, M.D., Phone: 504-896-9848, Email: LYu@lsuhsc.edu
Mimi Hermann, R.N., Phone: 504-894-5377, Email: mhermann@clinicaltrialscenter.org
Lolie Yu, M.D., Principal Investigator
Children's Hospitals and Clinics of Minnesota, Minneapolis, Minnesota 55404, United States; Recruiting
Jawhar Rawwas, M.D., Phone: 612-813-5940, Email: Jawhar.Rawwas@childrensmn.org
Lezlie Rabine, R.N., Phone: 612-813-6751, Email: Lezlie.Rabine@childrensmn.org
Jawhar Rawwas, M.D., Principal Investigator
University of Oklahoma Cancer Center, Oklahoma City, Oklahoma 73104, United States; Recruiting
Rene McNall-Knapp, M.D., Phone: 405-271-4412, Email: Rene-McNall@ouhsc.edu
Elaine Reeves, R.N., Phone: 405-271-5849, Email: elaine-reeves@ouhsc.edu
Rene McNall-Knapp, M.D., Principal Investigator
The University of Texas M.D. Anderson Cancer Center, Houston, Texas 77030, United States; Recruiting
Peter E. Zage, M.D., Phone: 713-792-6624, Email: pezage@mdanderson.org
MaryAnn Giannan, R.N., Phone: 713-745-0774, Email: mgianan@mdanderson.org
Peter E. Zage, M.D., Principal Investigator
US Oncology, Dallas, Texas 75230, United States; Recruiting
Stan Goldman, M.D., Phone: 972-566-6647, Email: stan.goldman@usoncology.com
Tessa Bolt, R.N., Phone: 972-566-4449, Email: Tessa.Bolt@usoncology.com
Stan Goldman, M.D., Principal Investigator
University of Utah Medical Center, Salt Lake City, Utah 84113, United States; Recruiting
Mark Fluchel, M.D., Phone: 801-662-4740, Email: mark.fluchel@imail.org
Stan Pies, R.N., Phone: 801-662-4715, Email: stan.pies@imail.org
Mark Fluchel, M.D., Principal Investigator
Vermont Cancer Center, Burlington, Vermont 05405, United States; Recruiting
Giselle Sholler, M.D., Phone: 802-847-2850, Email: Giselle.Sholler@uvm.edu
Shannon Lenox, R.N., Phone: 802-656-9283, Email: Shannon.Lenox@med.uvm.edu
Giselle Sholler, M.D., Principal Investigator
Additional Information
Starting date: August 2009
Last updated: September 22, 2009
|
