Parkinson’s Disease with at Least One GBA1 Mutation
Conditions
Brief summary
Incidence and severity of treatment-emergent AEs and SAE, Incidence of procedure or treatment-emergent safety findings, Treatment emergent and change from baseline in immunogenicity of adeno-associated viruses serotype 9 and glucocerebrosidase
Detailed description
Change from baseline in glycolipid and glucocerebrosidase levels, as well as glucocerebrosidase enzyme activity in blood, Change from baseline in glycolipid and glucocerebrosidase levels, as well as glucocerebrosidase enzyme activity in cerebrospinal fluid
Interventions
DRUGLY3884961
Sponsors
Prevail Therapeutics Inc.
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence and severity of treatment-emergent AEs and SAE, Incidence of procedure or treatment-emergent safety findings, Treatment emergent and change from baseline in immunogenicity of adeno-associated viruses serotype 9 and glucocerebrosidase | — |
Secondary
| Measure | Time frame |
|---|---|
| Change from baseline in glycolipid and glucocerebrosidase levels, as well as glucocerebrosidase enzyme activity in blood, Change from baseline in glycolipid and glucocerebrosidase levels, as well as glucocerebrosidase enzyme activity in cerebrospinal fluid | — |
Countries
Netherlands
Outcome results
None listed