Alpelisib for Lymphatic Malformations with PIK3CA Mutation
This study is testing a drug called alpelisib for people with lymphatic malformations (abnormal growths of the lymphatic system) that have a specific genetic change called a PIK3CA mutation. The study aims to see if alpelisib can shrink these malformations and improve symptoms compared to a placebo (an inactive substance). Adults and children aged 0 to 100 years can participate if they have a PIK3CA mutation. The main goal is to measure how much the malformations respond to treatment after 24 weeks. The study is currently recruiting 232 participants.
- Study design
- This is a multi-center study with two stages. Stage 1 is open-label (everyone knows what treatment they are getting) to find the right dose, and Stage 2 is randomized, double-blind (neither you nor your doctor will know if you are receiving alpelisib or placebo), and placebo-controlled.
- What's involved
- You will need to remain at the clinical site as required by the study and follow all study restrictions and examination schedules. The core treatment phase in Stage 1 and Stage 2 is 24 weeks.
- Compensation
- Not stated in the trial record.
- Follow-up
- Radiological response will be measured at Baseline and Week 24 of Stage 2.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA Mutation.
At a glance
Conditions
Where it's being run
61 sites across 34 statesStudy leadership
- Novartis Pharmaceuticals · STUDY_DIRECTOR · Novartis Pharmaceuticals
Who to contact
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What this trial measures
- Stage 2:Radiological response rate at Week 24 of Stage 2 (adult and pediatric (6 - 17 years of age) participants)Baseline, Week 24
Radiological response defined by achieving at least 20% reduction in the sum of target lesion volumes (1 to 3 lesions), assessed by MRI by a BIRC at Week 24, provided that none of the individual target lesions has at least 20% increase from baseline and in absence of progression of non-target lesions and without new lesions. The percentage of participants with a radiological response at Week 24 of Stage 2 in adult and pediatric (6-17 years of age) groups will be assessed