Phase 2/3Recruiting
Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA Mutation. (EPIK-L1)
A Two-stage Double-blind, Randomized, Placebo-controlled Study to Assess the Efficacy, Safety and Pharmacokinetics of Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA Mutation.
- Sponsor
- Novartis Pharmaceuticals (Industry)
- Enrollment
- 232 planned
- Conditions
- Lymphatic Malformations
- Interventions
- Alpelisib; Placebo
- Ages
- children, adults, older adults
- Registry
- NCT05948943, CTIS2023-504146-60-00
Full record on ClinicalTrials.gov
From the registry
The main purpose of this study in participants with PIK3CA-mutated LyM is to assess the change in radiological response and symptom severity upon treatment with alpelisib film-coated tablets (FCT) as compared to placebo.
Primary outcomes
- Stage 2:Radiological response rate at Week 24 of Stage 2 (adult and pediatric (6 - 17 years of age) participants)
History
Study completion expected
Primary completion expected
Last registry update
Started
Registered