GBA1-associated Parkinson’s disease
Conditions
Brief summary
Time to clinically meaningful progression, Incidence of treatment-emergent adverse events (TEAEs) by frequency, severity, and seriousness, Change from baseline in clinical laboratory tests, vital signs, and electrocardiograms (ECGs), Incidence of positive responses (Yes) to Item 4 or 5 on the Columbia-Suicide Severity Rating Scale (C-SSRS)
Detailed description
Change from baseline to Week 78 in a composite battery of cognitive tests: • Hopkins Verbal Learning Test-Revised, Delayed Recall score (HVLT-R, DR) • Letter-Number Sequencing (LNS) test • Semantic Verbal Fluency • Symbol Digit Modalities Test (SDMT), Change from baseline to Week 78 in a collection of clinical outcome assessments and patient-reported outcomes. Time to: • Initiation or modification of symptomatic therapy
Interventions
DRUGTablets matching VQ-101
DRUGVQ-101
Sponsors
Vanqua Bio Inc.
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Time to clinically meaningful progression, Incidence of treatment-emergent adverse events (TEAEs) by frequency, severity, and seriousness, Change from baseline in clinical laboratory tests, vital signs, and electrocardiograms (ECGs), Incidence of positive responses (Yes) to Item 4 or 5 on the Columbia-Suicide Severity Rating Scale (C-SSRS) | — |
Secondary
| Measure | Time frame |
|---|---|
| Change from baseline to Week 78 in a composite battery of cognitive tests: • Hopkins Verbal Learning Test-Revised, Delayed Recall score (HVLT-R, DR) • Letter-Number Sequencing (LNS) test • Semantic Verbal Fluency • Symbol Digit Modalities Test (SDMT), Change from baseline to Week 78 in a collection of clinical outcome assessments and patient-reported outcomes. Time to: • Initiation or modification of symptomatic therapy | — |
Outcome results
None listed