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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT05894343 | Long-term Follow-up of Glutamic Acid Decarboxylase (GAD) Gene Transfer in Parkinson's Disease | PHASE1 | ACTIVE NOT_RECRUITING | 13 | — | — | May 19, 2023 | Oct 1, 2029 | Aug 17, 2025 | 6 | United States |
| NCT05603312 | A Double-blind Study to Evaluate the Safety of Glutamic Acid Decarboxylase Gene Transfer in Parkinson's Participants | PHASE1 | COMPLETED | 14 | — | — | Oct 5, 2022 | Sep 6, 2024 | Sep 5, 2025 | 6 | United States |
The primary outcome measure is the safety of treatment with AAV-GAD, assessed by the absence of IMP-related treatment-emergent adverse events.
The primary outcome measure is the safety of treatment with AAV-GAD, assessed by the absence of IMP-related serious treatment-emergent adverse events.
| Arm | Type | Description |
|---|---|---|
| Follow-up group | NO_INTERVENTION | Participants who were randomized to immediate treatment in Study MGT-GAD-025 will transition directly to a long-term follow-up schedule to complete an additional 54 months of follow-up. All study participants are to be followed for 60 months after vector administration. |
| Active treatment group | EXPERIMENTAL | Participants who were randomized to sham surgery in Study MGT-GAD-025 will transition to an active treatment schedule, including open-label bilateral treatment, upon confirmation of continued eligibility. Upon completion of the treatment period, participants will enter the long-term follow-up schedule to complete a total of 60 months of follow-up. |
| AAV-GAD Low Dose | EXPERIMENTAL | Eligible participants received bilateral infusion of AAV-GAD low dose into the STN |
| AAV-GAD High Dose | EXPERIMENTAL | Eligible participants received bilateral infusion of AAV-GAD high dose into the STN |
| Sham Surgery | SHAM_COMPARATOR | Eligible participants underwent a sham surgical procedure |
| Name | Type | Description |
|---|---|---|
| AAV-GAD | GENETIC | Bilateral infusion of AAV-GAD |
| AAV-GAD Low Dose | GENETIC | Bilateral infusion of AAV-GAD low dose |
| AAV-GAD High Dose | GENETIC | Bilateral infusion of AAV-GAD high dose |
| Sham Surgery | PROCEDURE | Sham infusion |
Inclusion Criteria: * Previously enrolled in Study MGT-GAD-025. Exclusion Criteria: * None