EPIK-P4: Alpelisib for PIK3CA-related Overgrowth Spectrum (PROS)
This study is testing alpelisib (BYL719) in children and adults with PIK3CA-related Overgrowth Spectrum (PROS), a condition causing overgrowth in certain body parts. Alpelisib is a drug that targets the PI3K mechanism, which is linked to PROS. The study aims to see how well alpelisib works to shrink or improve the overgrowth and to check its safety. You might be eligible if you are at least 2 years old, have a diagnosis of PROS with symptoms and progressive overgrowth, and have the PIK3CA biomarker. Success in this study means a certain percentage of participants show a confirmed improvement in their overgrowth within 48 weeks. The study plans to enroll 104 participants, but its current recruitment status is unclear.
- Study design
- This is a single-arm study, meaning all participants will receive alpelisib. It is open-label, so both you and the study team will know you are receiving the drug. The study plans to enroll 104 participants.
- What's involved
- You would undergo a screening period of up to 42 days, followed by a core treatment period of 48 weeks. There is also an extension period of up to 2 years where you would continue treatment.
- Compensation
- Not stated in the trial record.
- Follow-up
- Participants will be followed for efficacy and safety up to Week 48 during the core period, and then for an extension period of up to 2 years.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
EPIK-P4: A Phase II Single-arm Study to Assess the Efficacy, Safety and Pharmacokinetics of Alpelisib (BYL719) in Pediatric and Adult Patients With PIK3CA-related Overgrowth Spectrum (PROS)
At a glance
Conditions
Where it's being run
37 sites across 24 statesStudy leadership
- Novartis Pharmaceuticals · STUDY_DIRECTOR · Novartis Pharmaceuticals
Who to contact
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What this trial measures
- Proportion of participants with a confirmed objective response by BIRCUp to Week 48
Confirmed objective response is defined as achieving radiological response, confirmed by a subsequent assessment performed at least after 4 weeks. The achievement of radiological response requires ≥20% reduction from baseline in the sum of target lesion volumes (1 to 3 target lesions, assessed by Magnetic Resonance Imaging (MRI) by a blinded independent review committee (BIRC)), provided that none of the individual target lesions has ≥20% increase from nadir, and in absence of progression of non-target lesions and without new lesions.