Gene Therapy for Parkinson's Disease

Recruiting in Palo Alto (17 mi)

+5 other locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: MeiraGTx, LLC

No Placebo Group

Trial Summary

What is the purpose of this trial?The objective of this clinical trial is 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.

Eligibility Criteria

This trial is for people with confirmed Parkinson's Disease who have responded to Levodopa treatment for at least a year and have moderate to severe symptoms when not medicated. It's not suitable for those with past brain surgery, atypical Parkinson’s, significant medical or psychiatric issues, previous brain infections or injuries, noticeable one-sided neurological problems, or cognitive impairment.

Inclusion Criteria

I have responded to Levodopa treatment for over a year.

I have been diagnosed with Parkinson's Disease.

You have a score of 25 or more on a test that measures symptoms of Parkinson's disease when you are not taking your medication.

Exclusion Criteria

You have significant problems with memory and thinking, as measured by a specific test called the Montreal Cognitive Assessment (MoCA) scoring 20 or less.

Your Beck Depression Inventory-II score is 20 or higher.

I have been diagnosed with atypical Parkinson's disease.

+4 more

Participant Groups

The study tests the safety of two doses of a gene therapy called AAV-GAD delivered directly into a part of the brain affected by Parkinson's. Participants are randomly assigned to receive either the actual gene therapy or a pretend surgery without active treatment.

3Treatment groups

Experimental Treatment

Placebo Group

Group I: AAV-GAD Dose 2 treatment groupExperimental Treatment1 Intervention

Eligible participants will receive bilateral infusion of AAV-GAD Dose 2 into the STN

Group II: AAV-GAD Dose 1 treatment groupExperimental Treatment1 Intervention

Eligible participants will receive bilateral infusion of AAV-GAD Dose 1 into the STN

Group III: Sham treatment groupPlacebo Group1 Intervention

Eligible participants will undergo a sham surgical procedure