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.
Interventional
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.
48 weeks
No
Jay Barth, MD
Principal Investigator
PTC Therapeutics
United States: Food and Drug Administration
PTC299-ONC-007-NF2
NCT00911248
July 2009
May 2012
Name | Location |
---|---|
Massachusetts General Hospital | Boston, Massachusetts 02114-2617 |