Home
Search
Study Topics
Glossary
|
|
|
|
|
|
Sponsored by: |
National Cancer Institute (NCI) |
---|---|
Information provided by: | National Cancer Institute (NCI) |
ClinicalTrials.gov Identifier: | NCT00029354 |
RATIONALE: Chemoprevention therapy is the use of certain drugs to try to prevent the development or recurrence of cancer. Tipifarnib may be effective in preventing the development of cancer in patients who have neurofibromatosis type 1 and plexiform neurofibromas.
PURPOSE: This randomized phase II trial is studying tipifarnib to see how well it works in preventing cancer in young patients who have neurofibromatosis type 1 and progressive plexiform neurofibromas.
Condition | Intervention | Phase |
---|---|---|
Neurofibromatosis Type 1 (nf1) Precancerous/Nonmalignant Condition Sarcoma |
Drug: placebo Drug: tipifarnib |
Phase II |
Study Type: | Interventional |
Study Design: | Prevention, Randomized, Double-Blind, Placebo Control |
Official Title: | A Phase II Randomized, Cross-Over, Double-Blinded, Placebo-Controlled Trial Of The Farnesyltransferase Inhibitor R115777 In Pediatric Patients With Neurofibromatosis Type I And Progressive Plexiform Neurofibromas |
Estimated Enrollment: | 63 |
Study Start Date: | July 2001 |
Estimated Primary Completion Date: | February 2004 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
---|---|
Arm I: Experimental
Patients receive oral tipifarnib every 12 hours on days 1-21. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
|
Drug: tipifarnib
Given orally
|
Arm II: Placebo Comparator
Patients receive oral placebo every 12 hours on days 1-21. Courses repeat as in arm I.
|
Drug: placebo
Given orally
|
OBJECTIVES:
OUTLINE: This is a randomized, cross-over, double-blind, placebo-controlled, multicenter study. Patients are randomized to 1 of 2 arms.
After documentation of disease progression, patients on both arms cross over to the other arm and (after a 2-week washout period) receive treatment as above in the absence of further disease progression or unacceptable toxicity.
Quality of life is assessed at baseline, prior to courses 4, 7, and 10, and then after every 6 courses thereafter.
PROJECTED ACCRUAL: A total of 63 patients will be accrued for this study.
Ages Eligible for Study: | 3 Years to 25 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
DISEASE CHARACTERISTICS:
Diagnosis of one of the following:
Progressive plexiform neurofibromas
Potential to cause significant morbidity such as:
Meets at least 1 other diagnostic criteria for NF1
Measurable plexiform neurofibromas
PATIENT CHARACTERISTICS:
Age:
Performance status:
Life expectancy:
Hematopoietic:
Hepatic:
Renal:
Cardiovascular:
Pulmonary:
Other:
PRIOR CONCURRENT THERAPY:
Biologic therapy:
Chemotherapy:
Endocrine therapy:
Radiotherapy:
Surgery:
Other:
United States, Alabama | |
Lurleen Wallace Comprehensive Cancer at University of Alabama - Birmingham | |
Birmingham, Alabama, United States, 35294 | |
United States, California | |
Hospital for Sick Children | |
Los Angeles, California, United States, 90027-0700 | |
United States, Maryland | |
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins | |
Baltimore, Maryland, United States, 21231-2410 | |
Warren Grant Magnuson Clinical Center - NCI Clinical Trials Referral Office | |
Bethesda, Maryland, United States, 20892-1182 | |
United States, Missouri | |
Siteman Cancer Center at Barnes-Jewish Hospital - Saint Louis | |
Saint Louis, Missouri, United States, 63110 | |
United States, Pennsylvania | |
Children's Hospital of Philadelphia | |
Philadelphia, Pennsylvania, United States, 19104 |
Study Chair: | Brigitte C. Widemann, MD | NCI - Pediatric Oncology Branch |
Study ID Numbers: | CDR0000068922, NCI-01-C-0222, NCI-T99-0090 |
Study First Received: | January 10, 2002 |
Last Updated: | January 14, 2009 |
ClinicalTrials.gov Identifier: | NCT00029354 |
Health Authority: | United States: Food and Drug Administration |
childhood neurofibrosarcoma childhood soft tissue sarcoma neurofibromatosis type 1 (NF1) plexiform neurofibroma |
Precancerous Conditions Malignant mesenchymal tumor Neurodegenerative Diseases Neurofibromatosis type 1 Soft tissue sarcomas Neurofibromatosis 1 Neoplasms, Connective and Soft Tissue Heredodegenerative Disorders, Nervous System Neoplastic Syndromes, Hereditary Genetic Diseases, Inborn Neurofibroma |
Neuromuscular Diseases Peripheral Nervous System Diseases Sarcoma Neurofibromatoses Peripheral Nervous System Neoplasms Neurofibroma, Plexiform Nerve Sheath Neoplasms Nervous System Neoplasms Tipifarnib Neurocutaneous Syndromes |
Neoplasms Neoplasms by Histologic Type Antineoplastic Agents Therapeutic Uses |
Nervous System Diseases Neoplasms, Nerve Tissue Pharmacologic Actions |