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Sponsored by: |
National Cancer Institute (NCI) |
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Information provided by: | National Cancer Institute (NCI) |
ClinicalTrials.gov Identifier: | NCT00053937 |
RATIONALE: Chemoprevention therapy is the use of certain drugs to try to prevent the development or recurrence of cancer. Pirfenidone may slow the growth or prevent further development of plexiform neurofibromas.
PURPOSE: Phase I trial to study the effectiveness of pirfenidone in treating young patients who have neurofibromatosis type 1 and plexiform neurofibroma.
Condition | Intervention | Phase |
---|---|---|
Neurofibromatosis Type 1 (nf1) Precancerous/Nonmalignant Condition |
Drug: pirfenidone |
Phase I |
Study Type: | Interventional |
Study Design: | Prevention, Open Label |
Official Title: | Phase I Trial Of Pirfenidone In Children With Neurofibromatosis Type 1 And Plexiform Neurofibromas |
Study Start Date: | December 2002 |
OBJECTIVES:
OUTLINE: This is an open-label, multicenter, dose-escalation study.
Patients receive oral pirfenidone three times daily on days 1-28. Courses repeat every 28 days for up to 2 years in the absence of disease progression or unacceptable toxicity.
Cohorts of 3-6 patients receive escalating doses of pirfenidone 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.
Quality of life is assessed at baseline, before course 4, and then after every 6 courses.
PROJECTED ACCRUAL: A total of 3-18 patients will be accrued for this study within 18 months.
Ages Eligible for Study: | 3 Years to 21 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
DISEASE CHARACTERISTICS:
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 | |
University of Alabama at Birmingham Comprehensive Cancer Center | |
Birmingham, Alabama, United States, 35294-3300 | |
United States, District of Columbia | |
Children's National Medical Center | |
Washington, District of Columbia, United States, 20010-2970 | |
United States, Illinois | |
Children's Memorial Hospital - Chicago | |
Chicago, Illinois, United States, 60614 | |
United States, Maryland | |
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins | |
Baltimore, Maryland, United States, 21231 | |
Warren Grant Magnuson Clinical Center - NCI Clinical Studies Support | |
Bethesda, Maryland, United States, 20892 | |
United States, Massachusetts | |
Dana-Farber/Harvard Cancer Center at Dana Farber Cancer Institute | |
Boston, Massachusetts, United States, 02115 | |
United States, Minnesota | |
Mayo Clinic Cancer Center | |
Rochester, Minnesota, United States, 55905 | |
United States, Missouri | |
Washington University School of Medicine | |
Saint Louis, Missouri, United States, 63110 | |
United States, New York | |
Beth Israel Medical Center - Singer Division | |
New York, New York, United States, 10128 | |
University Hospital at State University of New York - Upstate Medical University | |
Syracuse, New York, United States, 13210 | |
United States, Ohio | |
Cleveland Clinic Taussig Cancer Center | |
Cleveland, Ohio, United States, 44195 | |
United States, Oregon | |
Cancer Institute at Oregon Health and Science University | |
Portland, Oregon, United States, 97239-3098 | |
United States, Pennsylvania | |
Children's Hospital of Philadelphia | |
Philadelphia, Pennsylvania, United States, 19104 | |
Children's Hospital of Pittsburgh | |
Pittsburgh, Pennsylvania, United States, 15213 | |
United States, Texas | |
Texas Children's Cancer Center | |
Houston, Texas, United States, 77030-2399 |
Study Chair: | Brigitte C. Widemann, MD | NCI - Pediatric Oncology Branch |
Study ID Numbers: | CDR0000269598, NCI-03-C-0058A |
Study First Received: | February 5, 2003 |
Last Updated: | December 13, 2008 |
ClinicalTrials.gov Identifier: | NCT00053937 |
Health Authority: | United States: Federal Government |
plexiform neurofibroma neurofibromatosis type 1 (NF1) |
Precancerous Conditions Neurodegenerative Diseases Neurofibromatosis type 1 Neurofibromatosis 1 Pirfenidone Neoplastic Syndromes, Hereditary Heredodegenerative Disorders, Nervous System Genetic Diseases, Inborn Neurofibroma |
Neuromuscular Diseases Peripheral Nervous System Diseases Neurofibromatoses Peripheral Nervous System Neoplasms Neurofibroma, Plexiform Nerve Sheath Neoplasms Nervous System Neoplasms Neurocutaneous Syndromes |
Anti-Inflammatory Agents Neoplasms by Histologic Type Antineoplastic Agents Neoplasms, Nerve Tissue Nervous System Diseases Physiological Effects of Drugs Pharmacologic Actions Neoplasms |
Sensory System Agents Analgesics, Non-Narcotic Therapeutic Uses Anti-Inflammatory Agents, Non-Steroidal Analgesics Peripheral Nervous System Agents Antirheumatic Agents Central Nervous System Agents |