EPIK-P4
Research type
Research Study
Full title
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)
IRAS ID
1011861
Contact name
Fiona Morris
Contact email
Sponsor organisation
Novartis Pharmaceuticals UK Ltd.
Research summary
The main purpose of the trial is to see whether or not alpelisib works (is effective) and if it can be used safely (what side effects occur) in people people with a condition called PIK3CA-related overgrowth spectrum (PROS). PROS occurs when there are changes to a process that occurs inside cells, called the PI3K pathway, which helps control how cells grow and work. This change causes the pathway to become overactive and leads to abnormal growth in various parts of the body like fat, muscle, skin, bones, blood vessels, or nerves. PROS can cause serious health problems, and there are currently no approved treatments for this disease outside the US. Alpelisib is a medicine that blocks a protein called PI3K, which is involved in this disease. By blocking PI3K, alpelisib may help stop the abnormal growth of tissues.
In this trial, participants are treated with alpelisib tablets or granules (depending on age and/or drug dose) for at least 3 years. Participants will be enrolled into Group 1 for adults or Group 2 for children aged 2 to 17 years.
The trial will last at least 3 years (it will continue until the last enrolled participant has completed the treatment period) and during that time 16-20 visits to the doctor are planned. Physical examinations, blood tests and electrocardiograms (test which records the electrical activity of the heart) will be performed to monitor the safety of alpelisib. Bone and teeth development, as well as sexual maturation will also be monitored in children under 18 years old. MRI of PROS lesions (affected body parts) and questionnaires to assess participants’ health condition will be performed to study the efficacy of alpelisib.
This trial is also designed to learn more about how the amount of alpelisib changes in blood over time (pharmacokinetics) and the participant’s response to treatment (both desired and undesired responses).REC name
Yorkshire & The Humber - Sheffield Research Ethics Committee
REC reference
25/YH/0164
Date of REC Opinion
9 Oct 2025
REC opinion
Further Information Favourable Opinion