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Sponsors and Collaborators: |
NCI - Center for Cancer Research-Medical Oncology National Cancer Institute (NCI) |
Information provided by: | National Cancer Institute (NCI) |
ClinicalTrials.gov Identifier: | NCT00078936 |
RATIONALE: Some tumors need growth factors produced by the body's white blood cells to keep growing. Pirfenidone may interfere with growth factors and stop the tumor from growing.
PURPOSE: Phase II trial to study the effectiveness of pirfenidone in treating young patients who have neurofibromatosis type 1 and recurrent or progressive plexiform neurofibroma.
Condition | Intervention | Phase |
Neurofibromatosis Type 1 (nf1) Precancerous/Nonmalignant Condition |
Drug: pirfenidone |
Phase II |
Genetics Home Reference related topics: | familial encephalopathy with neuroserpin inclusion bodies neurofibromatosis type 1 neurofibromatosis type 2 |
MedlinePlus related topics: | Cancer Neurofibromatosis |
Drug Information available for: | Pirfenidone |
Study Type: | Interventional |
Study Design: | Treatment, Open Label |
Official Title: | Phase II Trial Of Pirfenidone In Children, Adolescents, And Young Adults With Neurofibromatosis Type I And Progressive Plexiform Neurofibromas |
Estimated Enrollment: | 36 |
Study Start Date: | October 2005 |
OBJECTIVES:
Primary
Secondary
OUTLINE: This is an open-label, multicenter study.
Patients receive oral pirfenidone three times daily continuously for a course of 28 days. Courses repeat in the absence of disease progression or unacceptable toxicity.
For patients 6 to 18 years of age, quality of life is assessed at baseline, before courses 4 and 7, and then after every 6 courses.
PROJECTED ACCRUAL: A total of 36 patients will be accrued for this study within 12-14 months.
Ages Eligible for Study: | 3 Years to 21 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
DISEASE CHARACTERISTICS:
All of the following:
Histologically confirmed OR consistent clinical and radiographic findings of plexiform neurofibroma (defined as neurofibroma that has grown along the length of a nerve and may involve multiple fascicles and branches)
Recurrent disease (presence of new lesion) or progressive disease as documented on last 2 consecutive MRI or CT scans or within the past year by 1 of the following:
Meets 1 or more of the following other diagnostic criteria for NF1:
At least 6 cafe-au-lait spots
One of the following distinctive bony lesions:
Ineligible for or refused complete resection of plexiform neurofibroma
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, 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, 21231-2410 | |||||
Warren Grant Magnuson Clinical Center - NCI Clinical Trials Referral Office | |||||
Bethesda, Maryland, United States, 20892-1182 | |||||
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 | |||||
St. Louis Children's Hospital | |||||
Saint Louis, Missouri, United States, 63110 | |||||
United States, New York | |||||
Beth Israel Medical Center - Singer Division | |||||
New York, New York, United States, 10028 | |||||
SUNY Upstate Medical University Hospital | |||||
Syracuse, New York, United States, 13210 | |||||
United States, Ohio | |||||
Cleveland Clinic Taussig Cancer Center | |||||
Cleveland, Ohio, United States, 44195 | |||||
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 and Hematology Service at Texas Children's Hospital | |||||
Houston, Texas, United States, 77030-2399 |
NCI - Center for Cancer Research-Medical Oncology |
National Cancer Institute (NCI) |
Principal Investigator: | Brigitte C. Widemann, MD | NCI - Pediatric Oncology Branch |
Clinical trial summary from the National Cancer Institute's PDQ® database 
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Study ID Numbers: | CDR0000353200, NCI-04-C-0080 |
First Received: | March 8, 2004 |
Last Updated: | October 18, 2008 |
ClinicalTrials.gov Identifier: | NCT00078936 |
Health Authority: | United States: Food and Drug Administration |
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