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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| AAV-GAD Low Dose | Experimental | Eligible participants received bilateral infusion of AAV-GAD low dose into the STN |
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| AAV-GAD High Dose | Experimental | Eligible participants received bilateral infusion of AAV-GAD high dose into the STN |
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| Sham Surgery | Sham Comparator | Eligible participants underwent a sham surgical procedure |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| AAV-GAD Low Dose | Genetic | Bilateral infusion of AAV-GAD low dose |
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| Measure | Description | Time Frame |
|---|---|---|
| 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. | 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. | Baseline to Week 26 |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Yale University - Yale New Haven Hospital | New Haven | Connecticut | 06510 | United States | ||
| Henry Ford West Bloomfield Hospital |
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Participants were recruited from 6 medical centers in the United States (US). A total of 14 participants were enrolled in the study.
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| ID | Title | Description |
|---|---|---|
| FG000 | AAV-GAD Low Dose | Bilateral infusion of AAV-GAD Low Dose into the STN |
| FG001 | AAV-GAD High Dose | Bilateral infusion of AAV-GAD High Dose into the STN |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Sep 22, 2023 |
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Participants are enrolled in one of three groups in parallel for the duration of the study.
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| AAV-GAD High Dose |
| Genetic |
Bilateral infusion of AAV-GAD high dose |
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| Sham Surgery | Procedure | Sham infusion |
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| West Bloomfield |
| Michigan |
| 48322 |
| United States |
| Weill Cornell Medicine | New York | New York | 10021 | United States |
| Columbia University Irving Medical Center | New York | New York | 10032 | United States |
| Thomas Jefferson University | Philadelphia | Pennsylvania | 19107 | United States |
| West Virginia University | Morgantown | West Virginia | 26506 | United States |
| FG002 | Sham Surgery | Sham infusion |
| COMPLETED |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | AAV-GAD Low Dose | Bilateral infusion of AAV-GAD Low Dose into the STN |
| BG001 | AAV-GAD High Dose | Bilateral infusion of AAV-GAD High Dose into the STN |
| BG002 | Sham Surgery | Sham infusion |
| BG003 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Region of Enrollment | Number | participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | Posted | Count of Participants | Participants | Baseline to Week 26 |
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| Primary | 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. | Posted | Count of Participants | Participants | Baseline to Week 26 |
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From the signing of the Informed Consent Form to Week 26
Safety was evaluated by vital signs, neurological and physical examinations, electrocardiograms, radiographic imaging, and clinical laboratory tests.
Treatment-related AEs were reported in 1 participant. This participant (high-dose group) experienced mild confusion and a mild headache perioperatively. These events started on the day of surgery and resolved the same day and 11 days later, respectively. All other AEs and all SAEs were assessed by the Investigator as unrelated to the study drug.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | AAV-GAD Low Dose | Bilateral infusion of AAV-GAD Low Dose into the STN | 0 | 5 | 2 | 5 | 5 | 5 |
| EG001 | AAV-GAD High Dose | Bilateral infusion of AAV-GAD High Dose into the STN | 0 | 5 | 0 | 5 | 5 | 5 |
| EG002 | Sham Surgery | Sham infusion | 0 | 4 | 1 | 4 | 4 | 4 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Catheter site haemorrhage | General disorders | Systematic Assessment |
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| Freezing phenomenon | Nervous system disorders | Systematic Assessment |
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| Urinary retention | Renal and urinary disorders | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Palpitations | Cardiac disorders | Systematic Assessment |
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| Eye swelling | Eye disorders | Systematic Assessment |
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| Periorbital oedema | Eye disorders | Systematic Assessment |
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| Vision blurred | Eye disorders | Systematic Assessment |
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| Dry mouth | Gastrointestinal disorders | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | Systematic Assessment |
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| Fatigue | General disorders | Systematic Assessment |
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| Gait disturbance | General disorders | Systematic Assessment |
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| Peripheral swelling | General disorders | Systematic Assessment |
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| Acute sinusitis | Infections and infestations | Systematic Assessment |
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| Urinary tract infection | Infections and infestations | Systematic Assessment |
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| Arthropod bite | Injury, poisoning and procedural complications | Systematic Assessment |
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| Confusion postoperative | Injury, poisoning and procedural complications | Systematic Assessment |
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| Eye contusion | Injury, poisoning and procedural complications | Systematic Assessment |
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| Fall | Injury, poisoning and procedural complications | Systematic Assessment |
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| Computerised tomogram abnormal | Investigations | Systematic Assessment |
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| Haematocrit decreased | Investigations | Systematic Assessment |
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| Red blood cells urine positive | Investigations | Systematic Assessment |
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| Reflex test abnormal | Investigations | Systematic Assessment |
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| Back pain | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| Muscle rigidity | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| Muscular weakness | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| Pain in extremity | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| Torticollis | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| Balance disorder | Nervous system disorders | Systematic Assessment |
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| Cognitive disorder | Nervous system disorders | Systematic Assessment |
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| Dyskinesia | Nervous system disorders | Systematic Assessment |
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| Facial spasm | Nervous system disorders | Systematic Assessment |
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| Head discomfort | Nervous system disorders | Systematic Assessment |
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| Headache | Nervous system disorders | Systematic Assessment |
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| Hyperreflexia | Nervous system disorders | Systematic Assessment |
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| Hypoaesthesia | Nervous system disorders | Systematic Assessment |
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| On and off phenomenon | Nervous system disorders | Systematic Assessment |
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| Paraesthesia | Nervous system disorders | Systematic Assessment |
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| Anxiety | Psychiatric disorders | Systematic Assessment |
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| Confusional state | Psychiatric disorders | Systematic Assessment |
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| Hallucination, visual | Psychiatric disorders | Systematic Assessment |
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| Mental status changes | Psychiatric disorders | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
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| Nasal congestion | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
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| Flushing | Vascular disorders | Systematic Assessment |
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| Thrombophlebitis | Vascular disorders | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Program Manager | MeiraGTx, LLC | 646-860-7982 | patients@meiragtx.com |
| Aug 4, 2025 |
| Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D010300 | Parkinson Disease |
| ID | Term |
|---|---|
| D020734 | Parkinsonian Disorders |
| D001480 | Basal Ganglia Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D009069 | Movement Disorders |
| D000080874 | Synucleinopathies |
| D019636 | Neurodegenerative Diseases |
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| Between 18 and 65 years |
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| >=65 years |
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| Male |
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| Not Hispanic or Latino |
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| Unknown or Not Reported |
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| Asian |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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