A Double-blind Study to Evaluate the Safety of Glutamic Acid Decarboxylase Gene Transfer in Parkinson's Participants

MeiraGTx

Status and phase: Completed; Phase 2; Phase 1

Conditions: Parkinson's Disease

Treatments: Genetic: AAV-GAD High Dose; Genetic: AAV-GAD Low Dose; Procedure: Sham Surgery

Study type: Interventional

Funder types: Industry

Identifiers

NCT05603312

MGT-GAD-025

Details and patient eligibility

About

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.

Full 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.

Enrollment: 14 patients

Sex: All

Ages: 25 to 85 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Confirmed Parkinson's Disease
• Levodopa responsiveness for at least 12 months
• UPDRS Part 3 score of ≥25 points in the "off" state

Exclusion criteria

• 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 design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

---

14 participants in 3 patient groups

AAV-GAD Low Dose

Experimental group

Description:

Eligible participants received bilateral infusion of AAV-GAD low dose into the STN

Treatment:

Genetic: AAV-GAD Low Dose

AAV-GAD High Dose

Experimental group

Description:

Eligible participants received bilateral infusion of AAV-GAD high dose into the STN

Treatment:

Genetic: AAV-GAD High Dose

Sham Surgery

Sham Comparator group

Description:

Eligible participants underwent a sham surgical procedure

Treatment:

Procedure: Sham Surgery