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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)
Sponsor: Novartis Pharmaceuticals
Summary
This Phase II, multicenter, open-label study, preceded by a retrospective non-interventional period, is assessing the long-term safety and efficacy of alpelisib in pediatric and adult participants with PIK3CA-related overgrowth spectrum (PROS).
Official title: A Phase II Study to Evaluate the Long-term Safety and Efficacy of Alpelisib in Patients With PIK3CA-Related Overgrowth Spectrum (PROS) Who Previously Participated in Study CBYL719F12002 (EPIK-P1)
Key Details
Gender
All
Age Range
2 Years - 100 Years
Study Type
INTERVENTIONAL
Enrollment
41
Start Date
2022-01-27
Completion Date
2027-06-22
Last Updated
2026-10-14
Healthy Volunteers
No
Interventions
Alpelisib
Retrospective period: Participants receive alpelisib under a Temporary Authorization for Use (ATU) or Managed Access Program (MAP) according to the treatment plan established by the treating physician. The recommended starting dose is 50 mg orally once daily for pediatric participants and 250 mg orally once daily for adult participants. Pediatric treatment interruptions and adult dose adjustments are permitted for toxicity. Prospective period: Participants receive alpelisib orally once daily with food. The starting dose is generally based on the last dose received during the retrospective period. Participants transitioning from fasted to fed administration may start at an adjusted dose. Dose escalation for response optimization and dose reduction or interruption for safety or tolerability are permitted. Dose escalation above 250 mg is not allowed. Treatment is planned for at least 5 years or until earlier discontinuation.
Locations (6)
Boston Childrens Hospital
Boston, Massachusetts, United States
Novartis Investigative Site
Dijon, France
Novartis Investigative Site
Montpellier, France
Novartis Investigative Site
Paris, France
Novartis Investigative Site
Dublin, Ireland
Novartis Investigative Site
Madrid, Spain