NCT00754780

Clinical Trial of Pirfenidone in Adult Patients With Neurofibromatosis 1

Phase II Clinical Trial of Pirfenidone for the Treatment of Patients With Neurofibromatosis Type I

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 24 (actual)
• Sponsor: Mayo Clinic · Academic / Other
• Sex: All
• Age: 18 Years – 70 Years
• Healthy volunteers: Not accepted

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.

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.

Conditions

• Neurofibromatosis

Interventions

Type	Name	Description
DRUG	Pirfenidone	Capsule 800 mg TID, oral

Timeline

• Start date: 2000-09-01
• Primary completion: 2004-08-01
• First posted: 2008-09-18
• Last updated: 2012-03-19