lymphatic malformations associated with PIK3CA mutation
Conditions
Brief summary
Response (yes/no) at Week 24 (confirmatory phase) of Stage 2, defined by achieving at least 20% reduction in the sum of target lesion volumes (1 to 3 lesions), assessed by MRI by a blinded independent review committee (BIRC) at Week 24, provided that none of the individual target lesions has at least 20% increase from baseline and in absence of progression of non-target lesions and without new lesions.
Detailed description
Response (yes/no) at Week 24 (confirmatory phase) of Stage 2 defined by achieving at least a 1-point improvement compared to baseline on the patient global impression of severity (PGI-S) scale., Response at Week 24 of Stage 2, defined by achieving at least 20% reduction in the sum of target lesion volumes (1 to 3 lesions), assessed by MRI by a BIRC at Week 24, provided that none of the individual target lesions has at least 20% increase from baseline and in absence of progression of non-target lesions and without new lesions.
Interventions
DRUGBYL719
DRUGPlacebo to BYL719 125 mg film-coated tablets
DRUGPlacebo to BYL719 200 mg film-coated tablets
DRUGPlacebo to BYL719 50 mg film-coated tablets
Sponsors
Novartis Pharma AG
Eligibility
Sex/Gender
All
Age
0 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Response (yes/no) at Week 24 (confirmatory phase) of Stage 2, defined by achieving at least 20% reduction in the sum of target lesion volumes (1 to 3 lesions), assessed by MRI by a blinded independent review committee (BIRC) at Week 24, provided that none of the individual target lesions has at least 20% increase from baseline and in absence of progression of non-target lesions and without new lesions. | — |
Secondary
| Measure | Time frame |
|---|---|
| Response (yes/no) at Week 24 (confirmatory phase) of Stage 2 defined by achieving at least a 1-point improvement compared to baseline on the patient global impression of severity (PGI-S) scale., Response at Week 24 of Stage 2, defined by achieving at least 20% reduction in the sum of target lesion volumes (1 to 3 lesions), assessed by MRI by a BIRC at Week 24, provided that none of the individual target lesions has at least 20% increase from baseline and in absence of progression of non-target lesions and without new lesions. | — |
Countries
Belgium, France, Germany, Italy, Netherlands, Spain
Outcome results
None listed