Pirfenidone in Treating Young Patients With Neurofibromatosis Type 1 and Plexiform Neurofibromas

NCT00053937 | Completed Phase 1

• 2 other identifiers: CDR0000269598NCI-03-C-0058A
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 15

Brief Summary

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.

Conditions

Neurofibromatosis Type 1; Precancerous Condition

Keywords

plexiform neurofibroma; neurofibromatosis type 1

Interventions

pirfenidone DRUG

Eligibility Criteria

• Age: 3 Years - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

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

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• National Cancer Institute (NCI): lead

Study Sites (15)

University of Alabama at Birmingham Comprehensive Cancer Center

Birmingham, Alabama, 35294-3300, United States

Location

Children's National Medical Center

Washington D.C., District of Columbia, 20010-2970, United States

Location

Children's Memorial Hospital - Chicago

Chicago, Illinois, 60614, United States

Location

Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Baltimore, Maryland, 21231, United States

Location

Warren Grant Magnuson Clinical Center - NCI Clinical Studies Support

Bethesda, Maryland, 20892, United States

Location

Dana-Farber/Harvard Cancer Center at Dana Farber Cancer Institute

Boston, Massachusetts, 02115, United States

Location

Mayo Clinic Cancer Center

Rochester, Minnesota, 55905, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Beth Israel Medical Center - Singer Division

New York, New York, 10128, United States

Location

University Hospital at State University of New York - Upstate Medical University

Syracuse, New York, 13210, United States

Location

Cleveland Clinic Taussig Cancer Center

Cleveland, Ohio, 44195, United States

Location

Cancer Institute at Oregon Health and Science University

Portland, Oregon, 97239-3098, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15213, United States

Location

Texas Children's Cancer Center

Houston, Texas, 77030-2399, United States

Location

MeSH Terms

Conditions

Neurofibromatosis 1; Precancerous Conditions; Neurofibroma, Plexiform

Interventions

pirfenidone

Condition Hierarchy (Ancestors)

Neurofibromatoses; Neurofibroma; Nerve Sheath Neoplasms; Neoplasms, Nerve Tissue; Neoplasms by Histologic Type; Neoplasms; Neoplastic Syndromes, Hereditary; Neurocutaneous Syndromes; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Peripheral Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Peripheral Nervous System Neoplasms; Nervous System Neoplasms

Study Officials

• Brigitte C. Widemann, MD

  National Cancer Institute (NCI)

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 1
• Masking: NONE
• Purpose: PREVENTION
• Sponsor Type: NIH

Study Record Dates

• First Submitted: February 5, 2003
• First Posted: February 6, 2003
• Study Start: December 1, 2002
• Last Updated: April 30, 2015

Record last verified: 2004-04

Locations

US (15)