Clinical Trial of Pirfenidone in Adult Patients With Neurofibromatosis 1
Status:
Completed
Trial end date:
1969-12-31
Target enrollment:
Participant gender:
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.