Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03562494 | VY-AADC02 for Parkinson's Disease With Motor Fluctuations (RESTORE-1) | PHASE1 | COMPLETED | 14 | — | — | Oct 17, 2018 | Oct 30, 2024 | Dec 10, 2024 | 11 | United States |
| NCT01973543 | Safety Study of AADC Gene Therapy (VY-AADC01) for Parkinson's Disease | PHASE1 | COMPLETED | 15 | — | — | Oct 1, 2013 | Jan 24, 2020 | Mar 3, 2020 | 2 | United States |
Safety and Tolerability of AADC Gene Transfer assessed by Adverse Events and Serious Adverse Events.
| Arm | Type | Description |
|---|---|---|
| VY-AADC02 (NBIb-1817) | EXPERIMENTAL | Single administration of up to 3.6 x 10\^12 vector genomes (vg) of VY-AADC02 |
| Sham (Placebo) Surgery | PLACEBO_COMPARATOR | Sham surgical procedure |
| VY-AADC01 Dose 1 | EXPERIMENTAL | 7.5 x 10\^11 vector genomes of VY-AADC01; Single dose, neurosurgically infused, bilaterally into the striatum. |
| VY-AADC01 Dose 2 | EXPERIMENTAL | 1.5 x 10\^12 vector genomes of VY-AADC01; Single dose, neurosurgically infused, bilaterally into the striatum. |
| VY-AADC01 Dose 3 | EXPERIMENTAL | 4.7x 10\^12 vector genomes of VY-AADC01; Single dose, neurosurgically infused, bilaterally into the striatum. |
| Name | Type | Description |
|---|---|---|
| VY-AADC02 | BIOLOGICAL | Adeno-associated viral vector serotype 2 encoding human aromatic L-amino acid decarboxylase (AAV2- hAADC) infusion into the brain |
| Sham (Placebo) Surgery | OTHER | Bilateral partial burr/twist holes without dura penetration |
| VY-AADC01 | BIOLOGICAL | Neurosurgical delivery of VY-AADC01 to the brain. |
Key Inclusion Criteria: 1. Males and females, 40 to 75 years of age (inclusive) 2. Diagnosis of PD, consistent with United Kingdom Brain Bank Criteria 3. Motor responsiveness to dopaminergic therapy, demonstrated by improvement in Movement Disorder Society - Unified Parkinson's Disease Rating Scale...