Parkinson's DiseasePhase 1/2NCT05603312
A Double-blind Study to Evaluate the Safety of Glutamic Acid Decarboxylase Gene Transfer in Parkinson's Participants
A Phase 1/2 study testing AAV-GAD Low Dose and AAV-GAD High Dose in people with Parkinson's Disease, run by MeiraGTx, LLC at 6 sites in United States.
Trial summary
The objective of this clinical trial was to evaluate the safety and tolerability of adeno-associated virus (AAV)-mediated delivery of glutamic acid decarboxylase (GAD) gene transfer into the subthalamic nuclei (STN) of participants with Parkinson's Disease. Read more about this study
- Who is it for?
- Ages 25 to 85 years
- Conditions the study is for
- Parkinson's Disease
- What is being tested?
- AAV-GAD Low Dose and AAV-GAD High Dose
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 14 participants
- Where
- 6 sitesUnited States
- Study dates
- 5 Oct 2022to 6 Sep 2024
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 25 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Confirmed Parkinson's Disease
- Levodopa responsiveness for at least 12 months
- UPDRS Part 3 score of ≥25 points in the "off" state
Who cannot join
- History of brain surgery to treat Parkinson's Disease
- Any history of cerebral insult or central nervous system infection
- Atypical Parkinson's Disease
- Focal or lateralized neurologic deficits
- Evidence of significant medical or psychiatric disorders
- Cognitive impairment as defined by the Montreal Cognitive Assessment (MoCA) ≤ 20
- Beck Depression Inventory-II score of ≥ 20
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United States6 sites
West Virginia University
Morgantown, West Virginia, 26506
Yale University - Yale New Haven Hospital
New Haven, Connecticut, 06510
Weill Cornell Medicine
New York, New York, 10021
Columbia University Irving Medical Center
New York, New York, 10032
Thomas Jefferson University
Philadelphia, Pennsylvania, 19107
Henry Ford West Bloomfield Hospital
West Bloomfield, Michigan, 48322
About this study
Official title: A Randomized, Double-Blind, Sham-Controlled Study to Evaluate the Safety and Tolerability of Glutamic Acid Decarboxylase Gene Transfer to the Subthalamic Nuclei in Participants With Parkinson's Disease
The objective of this clinical trial was to evaluate the safety and tolerability of adeno-associated virus (AAV)-mediated delivery of glutamic acid decarboxylase (GAD) gene transfer into the subthalamic nuclei (STN) of participants with Parkinson's Disease.
Read the detailed description
The planned length of participation in the study for each participant was approximately 7 months, including a screening period of up to 40 days, randomization, surgery, and a follow-up period of 26 weeks.
- Conditions
- Parkinson's Disease
- Being tested
- AAV-GAD Low Dose, AAV-GAD High Dose, Sham Surgery
- Medical subject headings
- Parkinson Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AAV-GAD Low DoseGenetic
Bilateral infusion of AAV-GAD low dose
AAV-GAD High DoseGenetic
Bilateral infusion of AAV-GAD high dose
Sham SurgeryProcedure or surgery
Sham infusion
| Study group | Role | What they receive |
|---|---|---|
| AAV-GAD Low Dose | Experimental | Eligible participants received bilateral infusion of AAV-GAD low dose into the STN Receives: Genetic: AAV-GAD Low Dose |
| AAV-GAD High Dose | Experimental | Eligible participants received bilateral infusion of AAV-GAD high dose into the STN Receives: Genetic: AAV-GAD High Dose |
| Sham Surgery | Sham comparator | Eligible participants underwent a sham surgical procedure Receives: Procedure: Sham Surgery |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Adverse Events Related to the Treatment
The primary outcome measure is the safety of treatment with AAV-GAD, assessed by the absence of IMP-related treatment-emergent adverse events.
Measured: Baseline to Week 26
Incidence of Serious Adverse Events Related to the Treatment
The primary outcome measure is the safety of treatment with AAV-GAD, assessed by the absence of IMP-related serious treatment-emergent adverse events.
Measured: Baseline to Week 26
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Nov 2022
- Study start5 Oct 2022
- Primary completion6 Sep 2024
- Study completion6 Sep 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Parkinson's DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Participants are enrolled in one of three groups in parallel for the duration of the study.
- Masking
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- Enrolment
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- MeiraGTx, LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05603312
- Sponsor's protocol ID
- MGT-GAD-025
- Status last verified
- Sep 2025
- First submitted
- 12 Oct 2022
- Last updated
- 5 Sep 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Sham-Controlled Study to Evaluate the Safety and Tolerability of Glutamic Acid Decarboxylase Gene Transfer to the Subthalamic Nuclei in Participants With Parkinson's Disease
- ClinicalTrials.gov ID
- NCT05603312
- Acronym / sponsor ID
- MGT-GAD-025
- Conditions
- Parkinson's Disease
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 14 participants (actual)
- Lead sponsor
- MeiraGTx, LLCIndustry (pharma, biotech, devices)
- Study start
- 5 Oct 2022 (actual)
- Primary completion
- 6 Sep 2024 (actual)
- Study completion
- 6 Sep 2024 (actual)
- First posted
- 2 Nov 2022 (actual)
- Results
- Posted 5 Sep 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The objective of this clinical trial was to evaluate the safety and tolerability of adeno-associated virus (AAV)-mediated delivery of glutamic acid decarboxylase (GAD) gene transfer into the subthalamic nuclei (STN) of participants with Parkinson's Disease.
Detailed description
The planned length of participation in the study for each participant was approximately 7 months, including a screening period of up to 40 days, randomization, surgery, and a follow-up period of 26 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, investigator, outcomes assessor)
- Number of arms
- 3
- Model description
- Participants are enrolled in one of three groups in parallel for the duration of the study.
| Arm | Type | Receives |
|---|---|---|
| AAV-GAD Low Dose | Experimental | Genetic: AAV-GAD Low DoseEligible participants received bilateral infusion of AAV-GAD low dose into the STN |
| AAV-GAD High Dose | Experimental | Genetic: AAV-GAD High DoseEligible participants received bilateral infusion of AAV-GAD high dose into the STN |
| Sham Surgery | Sham comparator | Procedure: Sham SurgeryEligible participants underwent a sham surgical procedure |
Who may qualify
- Ages
- 25 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Confirmed Parkinson's Disease
- Levodopa responsiveness for at least 12 months
- UPDRS Part 3 score of ≥25 points in the "off" state
Key exclusion criteria (7 listed)
- History of brain surgery to treat Parkinson's Disease
- Any history of cerebral insult or central nervous system infection
- Atypical Parkinson's Disease
- Focal or lateralized neurologic deficits
- Evidence of significant medical or psychiatric disorders
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AAV-GAD Low DoseBilateral infusion of AAV-GAD low dose | Genetic | AAV-GAD Low Dose |
| AAV-GAD High DoseBilateral infusion of AAV-GAD high dose | Genetic | AAV-GAD High Dose |
| Sham SurgerySham infusion | Procedure or surgery | Sham Surgery |
Primary outcome
Incidence of Adverse Events Related to the Treatment
Time frame: Baseline to Week 26
The primary outcome measure is the safety of treatment with AAV-GAD, assessed by the absence of IMP-related treatment-emergent adverse events.
Incidence of Serious Adverse Events Related to the Treatment
Time frame: Baseline to Week 26
The primary outcome measure is the safety of treatment with AAV-GAD, assessed by the absence of IMP-related serious treatment-emergent adverse events.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Sep 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing AAV-GAD Low Dose and AAV-GAD High Dose in people with Parkinson's Disease, run by MeiraGTx, LLC at 6 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 5 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 5 Sep 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Adverse Events Related to the Treatment (time frame: Baseline to Week 26). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Sep 2025.