Clinical Trial of Pirfenidone in Adult Patients With Neurofibromatosis 1 - Full Text View

Sponsored by:	Mayo Clinic
Information provided by:	Mayo Clinic
ClinicalTrials.gov Identifier:	NCT00754780

Purpose

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	Intervention	Phase
Neurofibromatosis	Drug: Pirfenidone	Phase II

Genetics Home Reference related topics: familial encephalopathy with neuroserpin inclusion bodies neurofibromatosis type 1 neurofibromatosis type 2

MedlinePlus related topics: Cancer Neurofibromatosis

Drug Information available for: Pirfenidone

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety Study
Official Title:	Phase II Clinical Trial of Pirfenidone for the Treatment of Patients With Neurofibromatosis Type I

Further study details as provided by Mayo Clinic:

Primary Outcome Measures:

tumor volume [ Time Frame: 24 months ] [ Designated as safety issue: No ]

Enrollment:	24
Study Start Date:	September 2000
Estimated Study Completion Date:	August 2010
Primary Completion Date:	August 2004 (Final data collection date for primary outcome measure)

Intervention Details:

Capsule 800 mg TID, oral

Detailed Description:

Specific aims of this study are:

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

Eligibility

Ages Eligible for Study:	18 Years to 70 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

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)

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00754780

Sponsors and Collaborators

Mayo Clinic

Investigators

Principal Investigator:

Dusica Babovic-Vuksanovic, M.D.

Mayo Clinic

More Information

Mayo Clinic Clinical Trials This link exits the ClinicalTrials.gov site

Responsible Party:	Mayo Clinic ( Dusica Babovic-Vuksanovic, M.D. )
Study ID Numbers:	1835-99, MC0077, Mayo Pirfenidone Study
Study First Received:	September 16, 2008
Last Updated:	September 17, 2008
ClinicalTrials.gov Identifier:	NCT00754780
Health Authority:	United States: Institutional Review Board

Keywords provided by Mayo Clinic:

NF1
plexiform neurofibroma

Study placed in the following topic categories:

Neurodegenerative Diseases
Neurofibromatosis 1
Neurofibromatosis type 1
Pirfenidone
Neoplastic Syndromes, Hereditary
Heredodegenerative Disorders, Nervous System
Neurofibroma

Genetic Diseases, Inborn
Neuromuscular Diseases
Peripheral Nervous System Diseases
Neurofibromatoses
Neurofibroma, Plexiform
Nerve Sheath Neoplasms
Neurocutaneous Syndromes

Additional relevant MeSH terms:

Anti-Inflammatory Agents
Neoplasms by Histologic Type
Antineoplastic Agents
Neoplasms, Nerve Tissue
Nervous System Diseases
Physiological Effects of Drugs
Pharmacologic Actions
Neoplasms

Sensory System Agents
Analgesics, Non-Narcotic
Therapeutic Uses
Anti-Inflammatory Agents, Non-Steroidal
Analgesics
Peripheral Nervous System Agents
Antirheumatic Agents
Central Nervous System Agents

ClinicalTrials.gov processed this record on January 13, 2009