PIK3CA-Related Overgrowth Spectrum (PROS)
Conditions
Brief summary
Response (yes/no) defined by achieving at least 20% reduction from baseline in the sum of target lesion volumes (1 to 3 lesions, assessed by MRI by a blinded independent review committee (BIRC)), provided that none of the individual target lesions has ≥ 20% increase from baseline and in absence of progression of non-target lesions and without new lesions.
Detailed description
The key secondary objective is to demonstrate the efficacy of alpelisib based on the comparison of the proportion of participants achieving response at Week 16 with alpelisib versus placebo in Groups 1 or 2.
Interventions
DRUGBYL719
DRUGPlacebo to BYL719 50 mg film-coated tablets (Light Yellow)
Placebo to BYL719 125 mg film-coated tablets (Dark Yellow)
Sponsors
Novartis Pharma AG
Eligibility
Sex/Gender
All
Age
0 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Response (yes/no) defined by achieving at least 20% reduction from baseline in the sum of target lesion volumes (1 to 3 lesions, assessed by MRI by a blinded independent review committee (BIRC)), provided that none of the individual target lesions has ≥ 20% increase from baseline and in absence of progression of non-target lesions and without new lesions. | — |
Secondary
| Measure | Time frame |
|---|---|
| The key secondary objective is to demonstrate the efficacy of alpelisib based on the comparison of the proportion of participants achieving response at Week 16 with alpelisib versus placebo in Groups 1 or 2. | — |
Countries
France, Germany, Italy, Netherlands, Norway, Spain
Outcome results
None listed