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Clinical Trial of Pirfenidone in Adult Patients With Neurofibromatosis 1

NCT00754780 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 24

Last updated 2012-03-19

No results posted yet for this study

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

Interventions

DRUG

Pirfenidone

Capsule 800 mg TID, oral

Sponsors & Collaborators

Principal Investigators

  • Dusica Babovic-Vuksanovic, M.D. · Mayo Clinic

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Max Age
70 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2000-09-30
Primary Completion
2004-08-31

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00754780 on ClinicalTrials.gov