EPIK-P2
Research type
Research Study
Full title
A Phase II double-blind study with an upfront, 16-week randomized, placebo-controlled period, to assess the efficacy, safety and pharmacokinetics of alpelisib (BYL719) in pediatric and adult patients with PIK3CA-related overgrowth spectrum (PROS)
IRAS ID
291786
Contact name
Robert Semple
Contact email
Sponsor organisation
Novartis Pharma AG
Eudract number
2020-000561-16
Clinicaltrials.gov Identifier
Duration of Study in the UK
8 years, 9 months, 26 days
Research summary
This Phase II study will assess the efficacy, safety/tolerability and pharmacokinetics (PK) of alpelisib in participants of different ages with a confirmed diagnosis of PROS.
PROS is a group of conditions where one or more parts of the body grow too much. It is caused by changes in a gene called the PIK3CA gene. The extra growth often affects fatty tissue, blood vessels, nerves, muscle, skin, and bone, but only in the parts of the body where the gene change is found. Complications from PROS vary, but can include problems directly arising from increased growth (e.g. impaired walking, use of hands, breathing or swallowing), or problems with bleeding, blot clots, infections, and/or pain.
Current medical care in PROS is limited and addresses symptoms and complications rather than treating the underlying problem. There is no approved medicine for PROS targeting the cause of the disease.
This study will be the first prospective study of alpelisib in participants with PROS.
The study will enrol:
• adult participants (Group 1),
• 6-17 years old paediatric participants (Group 2),
• an exploratory set of 2-5 years old paediatric participants (Group 3).
It will be a randomised study including an upfront 16-week placebo-controlled period for Group 1 and Group 2.
Group 3 will be exploratory and will be based on a small number of participants; in Group 3, participants will not be randomised and instead will be treated with alpelisib in an open label fashion after the primary analysis of patients in Group 1 and Group 2 are available. Following the primary analysis of patients in Group 1 and Group 2 at week 24 and prior to group 3 starting the study Novartis will amend the study protocol as required to provide instruction on dosing and procedures required for this group.REC name
East of Scotland Research Ethics Service REC 2
REC reference
21/ES/0003
Date of REC Opinion
23 Mar 2021
REC opinion
Further Information Favourable Opinion