Study Assessing Long-term Safety and Efficacy of Alpelisib in Patients With PIK3CA-Related Overgrowth Spectrum (PROS) Who Previously Participated in Study CBYL719F12002 (EPIK-P1)

Summary

This is a prospective interventional Phase II multi center study, open label, preceded by a retrospective non-interventional period, to assess the long-term safety and efficacy of alpelisib, in pediatric and adult participants with PROS.

Timeline

Start

2022-01-27

Primary completion

2027-08-20

Completion

2027-08-20

Publications

• Cheng YL, Bustamante D, Yates DM, Menendez S, Chi AC. TEK-related venous malformation of the orofacial region: report of a case with multidisciplinary treatment guided by molecular diagnosis. Oral Surg Oral Med Oral Pathol Oral Radiol. 2025 Nov;140(5):587-593. doi: 10.1016/j.oooo.2025.05.009. Epub 2025 Jun 6.

Drugs

Indications