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Home > "P" Clinical Trials Conditions > Pirfenidone in Treating Young Patients With Neurofibromatosis Type 1 and Plexiform Neurofibromas  Pirfenidone in Treating Young Patients With Neurofibromatosis Type 1 and Plexiform Neurofibromas
Pirfenidone in Treating Young Patients With Neurofibromatosis Type 1 and Plexiform Neurofibromas
For Condition: childhood neurofibrosarcoma,childhood soft tissue sarcoma
Status: Recruiting
Sponsor(s): National Cancer Institute (NCI) ,
Synopsis: 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.
Details: OBJECTIVES: - Determine the maximum tolerated dose or "comparable dose" of pirfenidone in pediatric patients with neurofibromatosis type 1 and inoperable, symptomatic plexiform neurofibromas. - Determine the toxic effects of this drug in these patients. - Determine the plasma pharmacokinetics of this drug in these patients. - Determine, preliminarily, if this drug could be beneficial for pediatric patients with refractory solid tumors. - Assess the quality of life of patients treated with this drug. 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.
Eligibility:
Study Type: Interventional, Prevention
Minimum Age/Maximum Age: 3 Years/21 Years
Genders: Both
Protocol Entry Criteria: DISEASE CHARACTERISTICS: - Diagnosis of neurofibromatosis type 1 (NF1) AND - Plexiform neurofibromas - Neurofibromas that have grown along the length of a nerve and may involve multiple fascicles and branches (spinal neurofibromas involve 2 or more levels with connection between the levels or extending laterally along the nerve) - Potential to cause significant morbidity such as: - Head and neck lesions that could compromise airway or great vessels - Brachial or lumbar plexus lesions that could cause nerve compression and loss of function - Lesions that could result in major deformity (e.g., orbital lesions) or significant cosmetic problems - Lesions of the extremity that cause limb hypertrophy or loss of function - Painful lesions - Meets at least 1 other diagnostic criteria for NF1 - 6 or more cafe-au-lait spots (at least 0.5 cm in prepubertal patients or at least 1.5 cm in postpubertal patients) - Freckling in the axilla or groin - Optic glioma - 2 or more Lisch nodules - Distinctive bony lesion (dysplasia of the sphenoid bone or dysplasia or thinning of long bone cortex) - First-degree relative with NF1 - Measurable plexiform neurofibromas - At least 3 cm in 1 dimension - Tumor resection not feasible - No history of malignant peripheral nerve sheath tumor or other cancer - No evidence of an active optic glioma requiring chemotherapy or radiotherapy - No malignant glioma PATIENT CHARACTERISTICS: Age - 3 to 21 Performance status - Karnofsky 50-100% (over 10 years of age) - Lansky 50-100% (10 years and under) Life expectancy - Not specified Hematopoietic - Absolute granulocyte count at least 1,500/mm^3 - Hemoglobin at least 9.0 g/dL - Platelet count at least 150,000/mm^3 Hepatic - Bilirubin normal - SGPT no greater than 2 times upper limit of normal - No clinically significant hepatic dysfunction that would preclude study participation Renal - Creatinine normal for age OR - Creatinine clearance at least 70 mL/min Cardiovascular - No clinically significant cardiac dysfunction that would preclude study participation Pulmonary - No clinically significant pulmonary dysfunction that would preclude study participation Other - Not pregnant or nursing - Negative pregnancy test - Fertile patients must use effective contraception during and for 2 months after study - Must be able to take pirfenidone orally - No serious infections - No clinically significant unrelated systemic illness or organ dysfunction that would preclude study participation PRIOR CONCURRENT THERAPY: Biologic therapy - At least 30 days since prior immunotherapy - No concurrent immunotherapy - No concurrent hematopoietic growth factors Chemotherapy - At least 30 days since prior chemotherapy - No concurrent chemotherapy directed at the tumor Endocrine therapy - At least 30 days since prior hormonal therapy directed at the tumor - No concurrent hormonal therapy directed at the tumor Radiotherapy - At least 90 days since prior radiotherapy to the site of the plexiform neurofibroma - No concurrent radiotherapy directed at the tumor Surgery - Not specified Other - Recovered from prior therapy - More than 30 days since prior investigational agents - No prior pirfenidone - No other concurrent investigational agents
Total Enrollment:
Location and Contact Information:
Overall Study Official:
BrigitteWidemann, Study Chair, Pediatric Oncology Branch
University Hospital at State University of New York - Upstate Medical University *Recruiting*
Syracuse, New York, 13210
United States
Recruiting Ronald Dubowy 315-464-5294
Cleveland Clinic Taussig Cancer Center *Recruiting*
Cleveland, Ohio, 44195
United States
Recruiting Bruce Cohen 216-444-9182
Texas Children's Cancer Center *Recruiting*
Houston, Texas, 77030-2399
United States
Recruiting Susan Blaney 832-822-4215
Beth Israel Medical Center - Singer Division *Recruiting*
New York City, New York, 10128
United States
Recruiting Jeffrey Allen 212-870-9407
Warren Grant Magnuson Clinical Center - NCI Clinical Studies Support *Recruiting*
Bethesda, Maryland, 20892
United States
Recruiting Patient Recruitment 888-NCI-1937
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins *Recruiting*
Baltimore, Maryland, 21231
United States
Recruiting Robert Arceci 410-502-7518
Washington University School of Medicine *Recruiting*
St. Louis, Missouri, 63110
United States
Recruiting Allison King 314-454-4291
Children's Hospital of Philadelphia *Recruiting*
Philadelphia, Pennsylvania, 19104
United States
Recruiting Jean Belasco 215-590-2848
Children's Memorial Hospital - Chicago *Recruiting*
Chicago, Illinois, 60614
United States
Recruiting Stewart Goldman 773-880-4598 ext. 3270
Children's National Medical Center *Recruiting*
Washington D.C., District of Columbia, 20010-2970
United States
Recruiting Roger Packer 202-884-2120
Mayo Clinic Cancer Center *Recruiting*
Rochester, Minnesota, 55905
United States
Recruiting Dusica Babovic-Vuksanovic 507-284-3215
Cancer Institute at Oregon Health and Science University *Recruiting*
Portland, Oregon, 97239-3098
United States
Recruiting H. Nicholson 503-494-1543
University of Alabama at Birmingham Comprehensive Cancer Center *Recruiting*
Birmingham, Alabama, 35294-3300
United States
Recruiting Alyssa Reddy 205-939-9285
Dana-Farber/Harvard Cancer Center at Dana Farber Cancer Institute *Recruiting*
Boston, Massachusetts, 02115
United States
Recruiting Mark Kieran 617-632-4907
Children's Hospital of Pittsburgh *Recruiting*
Pittsburgh, Pennsylvania, 15213
United States
Recruiting Regina Jakacki 412-692-5055
Additional Information:
Study ID Numbers: CDR0000269598; NCI-03-C-0058A
Study Start Date:
Record last reviewed: January 2003
Additional information available at: clinicaltrials.gov
Clinicaltrials.gov Reference link: NCT00053937
Other Childhood Soft Tissue Sarcoma Studies:
1. Imatinib Mesylate in Treating Patients With Relapsed or Refractory Solid Tumors of Childhood
2. Surgery Followed by Chemotherapy in Treating Young Patients With Soft Tissue Sarcoma
3. Combination Chemotherapy in Treating Children With Metastatic Rhabdomyosarcoma or Other Malignant Mesenchymal Tumors
4. Combination Chemotherapy in Treating Patients With Advanced Cancer
5. Genetic Study of Children With Soft Tissue Sarcoma or Rhabdomyosarcoma
Related Studies:
Other childhood soft tissue sarcoma Clinical Trials
Other Pennsylvania Clinical Trials
Other Pittsburgh Clinical Trials
Pirfenidone in Treating Young Patients With Neurofibromatosis Type 1 and Plexiform Neurofibromas
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