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Clinical Trial of Pirfenidone in Adult Patients With Neurofibromatosis 1

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ClinicalTrials.gov Identifier: NCT00754780
Recruitment Status : Completed
First Posted : September 18, 2008
Last Update Posted : March 19, 2012
Information provided by:
Mayo Clinic

Brief Summary:

The study is a phase II, open label trial of oral Pirfenidone in 24 adult patients with neurofibromatosis type 1.

Pirfenidone is a new, broad-spectrum anti-fibrotic drug, with proven in vitro and in vivo negative effects on fibroblast growth and collagen matrix synthesis. Human studies indicate promising therapeutic effects in arresting and reversing fibrosis in a variety of different conditions, where the excessive formation of fibrous tissue is a major pathogenic mechanism. Since the fibrous tissue is a significant component of neurofibroma, reduction of fibrosis could diminish tumor progression and lead to tumor shrinkage. Therefore, Pirfenidone is an excellent candidate for the treatment of plexiform neurofibromas and surgically unresectable tumors in patients with NF1.

Condition or disease Intervention/treatment Phase
Neurofibromatosis Drug: Pirfenidone Phase 2

Detailed Description:

Specific aims of this study are:

  1. To evaluate efficacy of Pirfenidone in NF1 patients with disfiguring or disabling plexiform neurofibroma (PN) and spinal neurofibromas (SN)
  2. To determine the acute, subacute and chronic toxicity of Pirfenidone in patients with NF1.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 24 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase II Clinical Trial of Pirfenidone for the Treatment of Patients With Neurofibromatosis Type I
Study Start Date : September 2000
Actual Primary Completion Date : August 2004

Intervention Details:
  • Drug: Pirfenidone
    Capsule 800 mg TID, oral

Primary Outcome Measures :
  1. tumor volume [ Time Frame: 24 months ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Diagnosis of neurofibromatosis type I, based on clinical criteria (NIH Consensus Development Conference, 1988). Tumors will not be confirmed histologically, since the biopsy could cause a change in tumor growth and such interfere with effect of Pirfenidone treatment.
  • Male or female patients
  • Age 18 years old
  • All patients should be mentally capable of signing the consent form or should have a legal guardian to provide consent
  • Patients who are experiencing symptoms from neurofibromatous lesions and who refuse surgery or are not good surgical candidates, such as those with plexiform neurofibroma who are experiencing significant discomfort, disfigurement or nerve compression or
  • Presence of multiple spinal neurofibromas in which the surgical removal would carry a major risk for spinal cord damage.

Exclusion Criteria:

  • Tumors for which surgical removal could lead to permanent (or long-term) relief of symptoms
  • Patients with open skin lesions and patients for whom surgery is being contemplated or who had surgery less than 4 weeks from starting treatment
  • Patients for whom biopsy is warranted for suspected malignancies
  • Individuals younger than 18 years
  • Pregnant and lactating women
  • Inability to have MR imaging (e.g. claustrophobia, pacemaker or allergy to contrast dye, if administration is needed for neurofibroma imaging)

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 ClinicalTrials.gov identifier (NCT number): NCT00754780

Sponsors and Collaborators
Mayo Clinic
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Principal Investigator: Dusica Babovic-Vuksanovic, M.D. Mayo Clinic
Additional Information:
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Responsible Party: Dusica Babovic-Vuksanovic, M.D., Mayo Clinic
ClinicalTrials.gov Identifier: NCT00754780    
Other Study ID Numbers: 1835-99
Mayo Pirfenidone Study
First Posted: September 18, 2008    Key Record Dates
Last Update Posted: March 19, 2012
Last Verified: March 2012
Keywords provided by Mayo Clinic:
plexiform neurofibroma
Additional relevant MeSH terms:
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Neurofibromatosis 1
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