Type 1 Gaucher Disease (Peripheral/Non-neuronopathic Manifestations)
Conditions
Brief summary
Incidence and severity of TEAEs and SAEs, including clinically significant changes in vital signs, clinical laboratory parameter assessments, 12-lead ECGs, results of abdominal and bone MRIs, physical and neurological examinations, and waist circumference and weight over time.
Detailed description
Change and percent change from baseline in spleen volume (MN), Change from baseline in platelet count, Change from baseline in GCase enzyme activity and protein levels and GluSph levels, Time from LY3884961 to ERT/SRT discontinuation., Time from discontinuation of ERT/SRT to re-initiation of ERT/SRT.
Interventions
Sponsors
Prevail Therapeutics Inc.
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence and severity of TEAEs and SAEs, including clinically significant changes in vital signs, clinical laboratory parameter assessments, 12-lead ECGs, results of abdominal and bone MRIs, physical and neurological examinations, and waist circumference and weight over time. | — |
Secondary
| Measure | Time frame |
|---|---|
| Change and percent change from baseline in spleen volume (MN), Change from baseline in platelet count, Change from baseline in GCase enzyme activity and protein levels and GluSph levels, Time from LY3884961 to ERT/SRT discontinuation., Time from discontinuation of ERT/SRT to re-initiation of ERT/SRT. | — |
Countries
Germany, Spain
Outcome results
None listed