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AAV-GAD · 2 trials · 1 indication
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
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AAV-GAD is an investigational gene therapy being studied for the treatment of Parkinson's Disease. It is designed to deliver the glutamic acid decarboxylase gene to the brain, with the goal of producing GABA, a neurotransmitter that may help regulate the overactive neurons associated with Parkinson's symptoms. It is currently in Phase 1 clinical development.
AAV-GAD targets the glutamic acid decarboxylase gene, which encodes the enzyme responsible for synthesizing GABA, an inhibitory neurotransmitter. By delivering this gene to the brain, the therapy aims to increase GABA production and reduce the excessive neuronal activity that contributes to Parkinson's Disease symptoms.
AAV-GAD is being developed by MeiraGTx Holdings plc, a biopharmaceutical company traded on the NASDAQ under the ticker symbol MGTX. The company is conducting clinical trials to evaluate the safety and efficacy of this gene therapy for Parkinson's Disease.
AAV-GAD is currently in Phase 1 clinical development. It is an investigational therapy and has not been approved by the FDA. The drug has received a Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA, which is intended to expedite the development and review of regenerative medicine therapies.
AAV-GAD has been studied in two Phase 1 clinical trials. The first, NCT05603312, was a double-blind study evaluating safety in 14 participants with Parkinson's Disease and has been completed. The second, NCT05894343, is an active long-term follow-up study of 13 participants. Both trials were conducted in the United States.
Yes, AAV-GAD is also referred to as glutamic acid decarboxylase gene transfer. The clinical trials for this therapy are titled to reflect this, such as 'A Double-blind Study to Evaluate the Safety of Glutamic Acid Decarboxylase Gene Transfer in Parkinson's Participants' and 'Long-term Follow-up of Glutamic Acid Decarboxylase (GAD) Gene Transfer in Parkinson's Disease'.