Phase II Clinical Trial of Pirfenidone for the Treatment of Patients With Neurofibromatosis Type I
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
Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Dusica Babovic-Vuksanovic, M.D.
United States: Institutional Review Board