PIK3CA-related Overgrowth Spectrum (PROS) Clinical Trial
Official title:
EPIK-P2: 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)
This is a prospective Phase II multi-center study with an upfront 16-week, randomized, double-blind, placebo-controlled period, and extension periods, to assess the efficacy, safety and pharmacokinetics of alpelisib in pediatric and adult participants with PIK3CA-related overgrowth spectrum (PROS).
This study consists of a screening period of up to 42 days, core period of 24 weeks, extension period of 24 weeks and long-term extension period of up to approximately 5 years. The study will enroll adult participants (Group 1), 6-17 years old pediatric participants (Group 2), two exploratory sets of 2-5 years old pediatric participants (Group 3 treated with granules and Group 4 treated with film-coated tablets (FCT)) ) and an exploratory group of 6 to 17 years old pediatric participants (Group 5; treated with FCT [at a higher starting dose than Group 2]). Eligible participants aged ≥6 years old will be randomized in a 2:1 ratio to alpelisib or matching placebo. Both age groups (group 1 and group 2) will be enrolled in the study in parallel. In the core period, participants will receive treatment in blinded fashion, with an upfront 16-week placebo-controlled period. After Week 16 those participants who were randomized to receive placebo will be switched to active treatment with alpelisib. Those participants who were randomized to receive alpelisib will continue their active treatment. Participants in Group 4 will be enrolled before Group 3. Group 5 will be open to enrollment after enrollment of Group 2 has been completed. All participants will receive alpelisib in an open-label setting. Group 3 will be enrolled later, after the completion of the primary analysis when the efficacy, safety and PK data will be available from the participants in Groups 1 and 2 in addition to the data from Group 4 and 5 as available, in order to select the recommended dose for participants in Group 3. The planned duration of alpelisib treatment in the study will be up to 5 years after randomization/treatment start for all age groups. Participant may be discontinued from treatment with alpelisib earlier due to unacceptable toxicity, confirmed disease progression, death, and/or any other reason at the discretion of the investigator or the participant. ;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
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Retrospective Chart Review Study of Patients With PIK3CA-Related Overgrowth Spectrum Who Have Received Alpelisib
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