BridgeMD
A research study to find out if the study treatment alpelisib (BYL719) is safe for a long period of time for who has confirmed diagnosis of PIK3CA-related overgrowth spectrum (PROS)
EU CTIS ID: 2023-508522-95-00
What this study is testing
Prospective period only - To assess the long-term safety and tolerability of alpelisib over time.
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Patients who had previously participated in EPIK-P1
- Signed informed consent form (ICF) 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 cutoff date of 09-Mar-2020 -If participants discontinue treatment for safety reasons prior to the first visit for the prospective period of the current study and if the treatment cannot be restarted, but they consent for the retrospective period, only the retrospective data will be abstracted -If participants discontinue treatment for any other reason, including worsening of disease, prior to the first visit for the prospective period of the current study, and they consent to restart treatment as considered beneficial by the investigator, they are -eligible for both periods
You likely can't join if
- 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 AEs -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 (QTcF) prolongation (>500 ms or >60 ms change from baseline)
- For participants in the prospective period 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.
- For participants in the prospective period Participant with uncontrolled diabetes mellitus (Type I or II) at time of informed consent.
The study team makes the final eligibility decision.
Where it's taking place
- United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 65+ years, 0-17 years, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.