PHASE I STUDY OF PERFENIDIONE IN NF1 AND PLEXIFORM NEUROFIBROMAS

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This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Neurofibromatosis type 1 is an autosomal dominant progressive genetic disorder characterized by diverse clinical manifestations. Patients with NF1 have an increased risk of developing tumors of the central and peripheral nervous system including plexiform neurofibromas. Such growth may cause severe morbidity adn possible mortality. This study is a standard Phase 1 study evaluating the toxicity of Pirfenidone and determining, using pharacokinetic data, a dose of Pirfenidone equivalent to the dose being used in adult studies ofa the agent. It si planned that immediatly following the Phase I study, a Phase II study will be undertaken evaluating the efficacy of Pirfenidone in children 3 and 21 years of age with progressive plexiform neurofibromas and NF1.