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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00053937
Recruitment Status : Completed
First Posted : February 6, 2003
Last Update Posted : April 30, 2015
Information provided by:
National Cancer Institute (NCI)

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.

Condition or disease Intervention/treatment Phase
Neurofibromatosis Type 1 Precancerous Condition Drug: pirfenidone Phase 1

Detailed Description:


  • 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.

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Study Type : Interventional  (Clinical Trial)
Masking: None (Open Label)
Primary Purpose: Prevention
Official Title: Phase I Trial Of Pirfenidone In Children With Neurofibromatosis Type 1 And Plexiform Neurofibromas
Study Start Date : December 2002

Information from the National Library of Medicine

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Ages Eligible for Study:   3 Years to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • 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



  • 3 to 21

Performance status

  • Karnofsky 50-100% (over 10 years of age)
  • Lansky 50-100% (10 years and under)

Life expectancy

  • Not specified


  • Absolute granulocyte count at least 1,500/mm^3
  • Hemoglobin at least 9.0 g/dL
  • Platelet count at least 150,000/mm^3


  • Bilirubin normal
  • SGPT no greater than 2 times upper limit of normal
  • No clinically significant hepatic dysfunction that would preclude study participation


  • Creatinine normal for age OR
  • Creatinine clearance at least 70 mL/min


  • No clinically significant cardiac dysfunction that would preclude study participation


  • No clinically significant pulmonary dysfunction that would preclude study participation


  • 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


Biologic therapy

  • At least 30 days since prior immunotherapy
  • No concurrent immunotherapy
  • No concurrent hematopoietic growth factors


  • 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


  • At least 90 days since prior radiotherapy to the site of the plexiform neurofibroma
  • No concurrent radiotherapy directed at the tumor


  • Not specified


  • Recovered from prior therapy
  • More than 30 days since prior investigational agents
  • No prior pirfenidone
  • No other concurrent investigational agents

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00053937

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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
Sponsors and Collaborators
National Cancer Institute (NCI)
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Study Chair: Brigitte C. Widemann, MD National Cancer Institute (NCI)
Layout table for additonal information Identifier: NCT00053937    
Obsolete Identifiers: NCT00050453
Other Study ID Numbers: CDR0000269598
First Posted: February 6, 2003    Key Record Dates
Last Update Posted: April 30, 2015
Last Verified: April 2004
Keywords provided by National Cancer Institute (NCI):
plexiform neurofibroma
neurofibromatosis type 1
Additional relevant MeSH terms:
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Neurofibromatosis 1
Precancerous Conditions
Neurofibroma, Plexiform
Nerve Sheath Neoplasms
Neoplasms, Nerve Tissue
Neoplasms by Histologic Type
Neoplastic Syndromes, Hereditary
Neurocutaneous Syndromes
Nervous System Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Peripheral Nervous System Diseases
Neuromuscular Diseases
Peripheral Nervous System Neoplasms
Nervous System Neoplasms
Sensory System Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Anti-Inflammatory Agents, Non-Steroidal
Analgesics, Non-Narcotic
Anti-Inflammatory Agents
Antirheumatic Agents
Antineoplastic Agents