Study Assessing Long-term Safety and Efficacy of Alpelisib in Patients With PIK3CA-Related Overgr… (NCT04980833) | Clinical Trial Compass
Active — Not RecruitingPhase 2
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)
United States, France, Ireland41 participantsStarted 2022-01-27
Plain-language 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.
Who can participate
Age range2 Years – 100 Years
SexALL
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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…
What they're measuring
1
Prospective period only: Proportion of participants with new or worsening grade ≥3 treatment emergent adverse events (AEs)
Timeframe: 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.