Study Assessing Long-term Safety and Efficacy of Alpelisib in Patients With PIK3CA-Related Overgrowth Spectrum (PROS) Who Previously Participated in Study CBYL719F12002 (EPIK-P1)
Alpelisib: Participant's treatment plan has been established by the treating physician, within the global compassionate use framework. Doses permitted are 50, 125, 200 and 250 mg.
Study summary
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.
Eligibility
Sex
ALL
Min age
2 Years
Max age
100 Years
Healthy volunteers
No
Inclusion Criteria:
* Participants who had previously participated in the study EPIK-P1.
* Signed informed consent form and assent (when applicable) from the participant, parent, or guardian must be obtained prior to any study related screening procedures being performed.
* Participant is treated with at least one dose of alpelisib after the EPIK-P1 study data cut- off date of 09-Mar-2020.
Exclusion Criteria:
For participants in the retrospective period
\- All EPIK-P1 participants who permanently discontinued the investigational drug on or prior to the cut-off date 09-Mar-2020.
For participants in the prospective period
* Previous alpelisib treatment discontinuation (after 09-Mar-2020) due to any of the following adverse events:
* Grade 4 skin and subcutaneous tissue disorders
* Stevens-Johnson-Syndrome (SJS)/ Toxic Epidermal Necrolysis (TEN) or other SJS/TEN-like severe skin reactions (any grade)
* Grade 4 hyperglycemia without confounding factors
* Pneumonitis (any grade)
* Grade 4 stomatitis
* Grade 4 pancreatitis
* Recurrent grade 4 thrombocytopenia
* Grade 3 or 4 serum creatinine increase
* Grade 4 isolated total bilirubin elevation
* Recurrent grade 3 or 4 QT interval corrected by Fridericia's formula prolongation (\>500 ms or \>60 ms change from baseline)
* Known impairment of GI function due to concomitant disease that may significantly alter the absorption of the study drug (e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel resection) at time of informed consent.
* Participant with uncontrolled diabetes mellitus (Type I or II) at time of informed consent.
Other inclusion/exclusion criteria may apply
Primary outcome measure(s)
Prospective period only: Proportion of participants with new or worsening grade ≥3 treatment emergent adverse events (AEs) — From date of first interventional dose administration in the prospective period (Day 1) to 30 days after last dose of study drug, assessed up to 5 years. Incidence of new or worsening grade ≥3 treatment emergent AEs (by system organ class and preferred term)
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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