A Phase 2 Study to Assess the Efficacy, Safety, and Pharmacodynamic Activity of PTC299 in Patients With Neurofibromatosis Type 2
The study will be conducted in 2 stages. In Stage 1 of the study, 11 patients will receive
daily treatment with PTC299 administered at 100 mg/dose twice per day for up to 1 year or
until tumor progression. If no subject responds with tumor shrinkage or an improvement in
hearing, then the study will be stopped. If ≥1 out of 11 subjects respond, then the study
will proceed to Stage 2 to enroll an additional 14 subjects for a total of 25 subjects.
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
To assess the effects of PTC299 on tumor volume and/or word recognition in patients with NF2.
Jay Barth, MD
United States: Food and Drug Administration
|Massachusetts General Hospital||Boston, Massachusetts 02114-2617|