Parkinson's DiseasePhase 1/2NCT05894343
Long-term Follow-up of Glutamic Acid Decarboxylase (GAD) Gene Transfer in Parkinson's Disease
A Phase 1/2 study testing AAV-GAD in people with Parkinson's Disease, run by MeiraGTx, LLC at 6 sites in United States.
Trial summary
The objective of this study is to evaluate the long-term safety of AAV-GAD delivered bilaterally to the subthalamic nuclei (STN) in participants with Parkinson's disease. Read more about this study
- Who is it for?
- Ages 25 to 86 years
- Conditions the study is for
- Parkinson's Disease
- What is being tested?
- AAV-GAD
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 6 sitesUnited States
- Study dates
- 19 May 2023to Oct 2029
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
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 86 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Previously enrolled in Study MGT-GAD-025.
Who cannot join
- None
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
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: Long-term Follow-up of Glutamic Acid Decarboxylase Gene Transfer to the Subthalamic Nuclei in Participants With Parkinson's Disease
The objective of this study is to evaluate the long-term safety of AAV-GAD delivered bilaterally to the subthalamic nuclei (STN) in participants with Parkinson's disease.
Read the detailed description
The duration of individual participation in this study will be approximately 54 months for participants who received AAV-GAD in Study MGT-GAD-025 and approximately 60 months for participants who received sham surgery in Study MGT-GAD-025.
- Conditions
- Parkinson's Disease
- Being tested
- AAV-GAD
- 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-GADGenetic
Bilateral infusion of AAV-GAD
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Genetic: AAV-GAD |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with study drug-related adverse events and serious adverse events
Measured: From study start until Month 60 post treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jun 2023
- Study start19 May 2023
- Primary completionOct 2029 (estimated)
- Study completionOct 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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 will be assigned to the follow-up or active treatment group based on the group to which they were randomized in Study MGT-GAD-025.
- Masking
- None (open label)
- Enrolment
- 13 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
- NCT05894343
- Sponsor's protocol ID
- MGT-GAD-026
- Status last verified
- Aug 2026
- First submitted
- 30 May 2023
- Last updated
- 1 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 1 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long-term Follow-up of Glutamic Acid Decarboxylase Gene Transfer to the Subthalamic Nuclei in Participants With Parkinson's Disease
- ClinicalTrials.gov ID
- NCT05894343
- Acronym / sponsor ID
- MGT-GAD-026
- Conditions
- Parkinson's Disease
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 13 participants (actual)
- Lead sponsor
- MeiraGTx, LLCIndustry (pharma, biotech, devices)
- Study start
- 19 May 2023 (actual)
- Primary completion
- Oct 2029 (estimated)
- Study completion
- Oct 2029 (estimated)
- First posted
- 8 Jun 2023 (actual)
- Results
- Not posted in the source record
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 study is to evaluate the long-term safety of AAV-GAD delivered bilaterally to the subthalamic nuclei (STN) in participants with Parkinson's disease.
Detailed description
The duration of individual participation in this study will be approximately 54 months for participants who received AAV-GAD in Study MGT-GAD-025 and approximately 60 months for participants who received sham surgery in Study MGT-GAD-025.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Participants will be assigned to the follow-up or active treatment group based on the group to which they were randomized in Study MGT-GAD-025.
| Arm | Type | Receives |
|---|---|---|
| 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 | Genetic: AAV-GADParticipants 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. |
Who may qualify
- Ages
- 25 to 86 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- Previously enrolled in Study MGT-GAD-025.
Key exclusion criteria (1 listed)
- None
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-GADBilateral infusion of AAV-GAD | Genetic | Active treatment group |
Primary outcome
Number of participants with study drug-related adverse events and serious adverse events
Time frame: From study start until Month 60 post treatment
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Sep 2026.
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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 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 1 inclusion and 1 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: under way, not looking for participants, as of the sponsor's last update on 1 Sep 2026. 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. 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?
This study is still under way. Results are not available in the source record yet.
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?
Number of participants with study drug-related adverse events and serious adverse events (time frame: From study start until Month 60 post treatment). 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 1 Sep 2026.