Clinical Trial of Pirfenidone in Adult Patients With Neurofibromatosis 1

NCT00754780 | Completed Phase 2

• 3 other identifiers: 1835-99MC0077Mayo Pirfenidone Study
• Study Type: interventional
• Target: 24
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Neurofibromatosis

Keywords

NF1; plexiform neurofibroma

Outcome Measures

Primary Outcomes (1)

• tumor volume

  24 months

Interventions

Pirfenidone DRUG

Capsule 800 mg TID, oral

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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)

Sponsors & Collaborators

• Mayo Clinic: lead

MeSH Terms

Conditions

Neurofibromatoses; Neurofibroma, Plexiform

Interventions

pirfenidone

Condition Hierarchy (Ancestors)

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; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Peripheral Nervous System Neoplasms; Nervous System Neoplasms; Peripheral Nervous System Diseases; Neuromuscular Diseases

Study Officials

• Dusica Babovic-Vuksanovic, M.D.

  Mayo Clinic

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: September 16, 2008
• First Posted: September 18, 2008
• Study Start: September 1, 2000
• Primary Completion: August 1, 2004
• Last Updated: March 19, 2012

Record last verified: 2012-03