Clinical Trial of Pirfenidone in Adult Patients With Neurofibromatosis 1
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.
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Phase II Clinical Trial of Pirfenidone for the Treatment of Patients With Neurofibromatosis Type I|
- tumor volume [ Time Frame: 24 months ] [ Designated as safety issue: No ]
|Study Start Date:||September 2000|
|Primary Completion Date:||August 2004 (Final data collection date for primary outcome measure)|
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.