Alpelisib for Overgrowth Spectrum Disorders
(EPIK-P3 Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This is a prospective interventional Phase II multi center study, open label, preceded by a retrospective non-interventional period, to assess the long-term safety and efficacy of alpelisib, in pediatric and adult participants with PROS.
Will I have to stop taking my current medications?
The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.
Is Alpelisib safe for humans?
How is the drug Alpelisib unique for treating overgrowth spectrum disorders?
What data supports the effectiveness of the drug Alpelisib for treating overgrowth spectrum disorders?
Research shows that Alpelisib, a drug targeting the PIK3CA gene, was effective in reducing the size of overgrowth lesions in 27% of patients with PIK3CA-related overgrowth spectrum disorders. Additionally, 60% of those who responded to the treatment maintained their improvement for at least 12 months.123511
Who Is on the Research Team?
Novartis Pharmaceuticals
Principal Investigator
Novartis Pharmaceuticals
Are You a Good Fit for This Trial?
This trial is for pediatric and adult patients with PIK3CA-Related Overgrowth Spectrum (PROS) who were previously in the EPIK-P1 study. They must have taken at least one dose of alpelisib after March 9, 2020, and give informed consent. People can't join if they stopped taking alpelisib due to severe side effects or have certain medical conditions like uncontrolled diabetes.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Retrospective Period
Non-interventional period where key safety and efficacy information is collected from medical charts
Prospective Period
Interventional period where participants receive alpelisib and safety and efficacy data are collected
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Alpelisib
Alpelisib is already approved in United States, European Union for the following indications:
Find a Clinic Near You
Who Is Running the Clinical Trial?
Novartis Pharmaceuticals
Lead Sponsor
Dr. Vas Narasimhan
Novartis Pharmaceuticals
Chief Executive Officer since 2018
MD from Harvard Medical School
Dr. Shreeram Aradhye
Novartis Pharmaceuticals
Chief Medical Officer since 2021
MD