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| Sponsor: | New Approaches to Neuroblastoma Therapy Consortium |
|---|---|
| Collaborator: |
National Cancer Institute (NCI) |
| Information provided by: | New Approaches to Neuroblastoma Therapy Consortium |
| ClinicalTrials.gov Identifier: | NCT00005835 |
Purpose
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining chemotherapy with bone marrow or peripheral stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more tumor cells.
PURPOSE: Phase I trial to study the effectiveness of melphalan and buthionine sulfoximine followed by bone marrow or peripheral stem cell transplantation in treating children who have resistant or recurrent neuroblastoma.
| Condition | Intervention | Phase |
|---|---|---|
|
Neuroblastoma |
Drug: buthionine sulfoximine Drug: melphalan Procedure: Peripheral blood stem cell infusion Other: Filgrastim |
Phase I |
| Study Type: | Interventional |
| Study Design: | Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | Modulation of Intensive Melphalan (L-PAM) by Buthionine Sulfoximine (BSO) Autologous Stem Cell Support for Resistant or Recurrent High-Risk Neuroblastoma (IND 69-112) |
| Estimated Enrollment: | 30 |
| Study Start Date: | August 2001 |
| Estimated Primary Completion Date: | December 2009 (Final data collection date for primary outcome measure) |
OBJECTIVES:
OUTLINE: This is a multicenter, dose-escalation study of melphalan.
Patients receive buthionine sulfoximine IV as a bolus over 30 minutes followed by a 72-hour continuous infusion beginning on day -4; melphalan IV over 15 minutes on days -3 and -2; autologous peripheral blood stem cells or bone marrow IV over 15-30 minutes on day 0; and filgrastim (G-CSF) subcutaneously or IV once daily beginning on day 0 and continuing until blood counts recover.
Cohorts of 3-6 patients receive escalating doses of melphalan until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that at which 2 of 3 or 2 of 6 patients experience dose-limiting toxicity.
Patients are followed at 84 days and then 2 months later if there is a complete and/or partial response. Patients who continue therapy on other protocols are followed before starting the new therapy. All patients are followed for life for any delayed toxic effects to protocol therapy and secondary malignancies.
PROJECTED ACCRUAL: A total of 30 patients will be accrued for this study within 2-3 years.
Eligibility| Ages Eligible for Study: | up to 30 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
DISEASE CHARACTERISTICS:
Meets 1 of the following response status criteria:
Meets 1 of the following criteria:
Bone marrow disease documented by standard morphology of bilateral bone marrow aspirate and biopsy specimens
Meets 1 of the following criteria for harvested autologous stem cells:
Availability of at least 1.0 x 10^6 viable CD34-positive purged autologous peripheral blood stem cells per kg of body weight*
PATIENT CHARACTERISTICS:
Age:
Performance status:
Life expectancy:
Hematopoietic:
Hepatic:
Renal:
Cardiovascular:
Pulmonary:
Neurologic:
Other:
PRIOR CONCURRENT THERAPY:
Biologic therapy:
Chemotherapy:
Endocrine therapy:
Radiotherapy:
More than 6 months since prior radiotherapy to kidneys, liver, heart, skull, or face
Surgery:
Other:
Contacts and Locations| United States, California | |
| Childrens Hospital Los Angeles | Recruiting |
| Los Angeles, California, United States, 90027-0700 | |
| Contact: Judith G. Villablanca, MD 323-361-5654 jvillablanca@chla.usc.edu | |
| Lucile Salter Packer Children's Hospital at Stanford University Medical Center | Recruiting |
| Palo Alto, California, United States, 94304 | |
| Contact: Clare Twist, MD 650-723-5535 clare.twist@stanford.edu | |
| UCSF Helen Diller Family Comprehensive Cancer Center | Recruiting |
| San Francisco, California, United States, 94143 | |
| Contact: Katherine K. Matthay, MD 415-476-3831 matthayK@peds.ucsf.edu | |
| United States, Illinois | |
| University of Chicago Comer Children's Hospital | Recruiting |
| Chicago, Illinois, United States, 60637 | |
| Contact: Susan L. Cohn, MD 773-702-2571 scohn@peds.bsd.uchicago.edu | |
| United States, Massachusetts | |
| Childrens Hospital Boston, Dana-Farber Cancer Institute. | Recruiting |
| Boston, Massachusetts, United States, 02115 | |
| Contact: Suzanne - Shusterman, MD 617-632-3725 suzanne_shusterman@dfci.harvard.edu | |
| United States, Michigan | |
| C.S Mott Children's Hospital | Recruiting |
| Ann Arbor, Michigan, United States, 48109 | |
| Contact: Gregory Yanik, MD 734-936-8785 gyanik@umich.edu | |
| United States, New York | |
| Morgan Stanley Childrens Hospital of New York-Presbyterian, Herbert Irving Division of Child & Adolescent Oncology | Recruiting |
| New York, New York, United States, 10032 | |
| Contact: Julia - Glade-Bender, MD 212-305-3379 Jg589@columbia.edu | |
| United States, Ohio | |
| Cincinnati Children's Hospital Medical Center | Recruiting |
| Cincinnati, Ohio, United States, 45229-3039 | |
| Contact: John P. Perentesis, MD 513-636-6090 john.perentesis@chmcc.org | |
| United States, Pennsylvania | |
| Children's Hospital of Philadelphia | Recruiting |
| Philadelphia, Pennsylvania, United States, 19104-4318 | |
| Contact: John M. Maris, MD 215-590-5242 maris@chop.edu | |
| United States, Texas | |
| Cook Children's Medical Center - Fort Worth | Recruiting |
| Fort Worth, Texas, United States, 76104 | |
| Contact: Clinical Trials Office - Cook's Children's Medical Center 682-885-2103 | |
| Texas Childrens Hospital. Baylor College of Medicine | Recruiting |
| Houston, Texas, United States, 77030 | |
| Contact: Susan - Blaney, MD 832-822-4586 Sblaney@txccc.org | |
| United States, Washington | |
| Children's Hospital and Regional Medical Center - Seattle | Recruiting |
| Seattle, Washington, United States, 98105 | |
| Contact: Julie R. Park, MD 206-987-1947 Julie.park@seattlechildrens.org | |
| Canada, Ontario | |
| Hospital for Sick Children | Recruiting |
| Toronto, Ontario, Canada, M5G 1X8 | |
| Contact: Sylvain Baruchel, MD 416-813-7795 sylvain.baruchel@sickkids.ca | |
| Study Chair: | Judith G. Villablanca, MD | Children's Hospital Los Angeles |
More Information
| Responsible Party: | Judith G. Villablanca , M. D. Professor , Division of Pediatric Hematology/Oncology., Children's Hospital Los Angeles |
| ClinicalTrials.gov Identifier: | NCT00005835 History of Changes |
| Other Study ID Numbers: | CDR0000067849, P01CA081403, NANT-99-02 |
| Study First Received: | June 2, 2000 |
| Last Updated: | November 2, 2010 |
| Health Authority: | United States: Food and Drug Administration |
|
regional neuroblastoma disseminated neuroblastoma recurrent neuroblastoma localized unresectable neuroblastoma |
|
Neuroblastoma Neuroectodermal Tumors, Primitive, Peripheral Neuroectodermal Tumors, Primitive Neoplasms, Neuroepithelial Neuroectodermal Tumors Neoplasms, Germ Cell and Embryonal Neoplasms by Histologic Type Neoplasms Neoplasms, Glandular and Epithelial Neoplasms, Nerve Tissue Melphalan Buthionine Sulfoximine Lenograstim Myeloablative Agonists Immunosuppressive Agents |
Immunologic Factors Physiological Effects of Drugs Pharmacologic Actions Antineoplastic Agents Therapeutic Uses Antineoplastic Agents, Alkylating Alkylating Agents Molecular Mechanisms of Pharmacological Action Antimetabolites, Antineoplastic Antimetabolites Enzyme Inhibitors Radiation-Protective Agents Protective Agents Radiation-Sensitizing Agents Adjuvants, Immunologic |