Study of AAV-GAD Gene Transfer Into the Subthalamic Nucleus for Parkinson's Disease

Phase 2 Safety and Efficacy Study Evaluating Glutamic Acid Decarboxylase Gene Transfer to Subthalamic Nuclei in Subjects With Advanced Parkinson's Disease

Status

Terminated

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT00643890

Enrollment

Registered

2008-03-26

Start date

2008-08-31

Completion date

Unknown

Last updated

2012-02-22

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Parkinson's Disease

Keywords

Keywords:, Parkinson's disease, Gene transfer, Gene therapy, Subthalamic nucleus, Adeno-associated virus, Study placed in the following topic categories:, Ganglion Cysts, Movement Disorders, Parkinson Disease, Basal Ganglia Diseases, Central Nervous System Diseases, Parkinsonian Disorders, Neurodegenerative Diseases, Brain Diseases, Additional relevant MeSH terms:, Nervous System Diseases

Brief summary

The purpose of this study is to determine the safety and efficacy of AAV-GAD gene transfer into the subthalamic nucleus (STN) region of the brain. This study involves the treatment of subjects with medically refractory Parkinson's disease (PD). The gene transfer product, a disabled virus with a gene called GAD, will be infused into the STN bilaterally using stereotactic surgical techniques. The overall goal of this approach is to normalize the activity of the STN and reduce the motor symptoms of PD. Because the change in UPDRS demonstrated a positive outcome, the sham surgery subjects from the blinded portion of the study will be invited to crossover into the Open-label Arm portion of the study. The Open-label Arm will further evaluate the safety and efficacy of AAV-GAD gene transfer into the subthalamic nucleus (STN) region of the brain.

Interventions

GENETICBilateral surgical infusion of AAV-GAD into the subthalamic nucleus

One-time bilateral administration of rAAV-GAD at 1X10\^12 vector genomes in 35 uL.

Study design

Allocation

RANDOMIZED

Primary purpose

TREATMENT

Masking

QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)

Eligibility

Sex/Gender

ALL

Age

30 Years to 75 Years

Healthy volunteers

Inclusion criteria

* Idiopathic Parkinson's disease * Duration of disease for at least 5 years * Levodopa responsiveness for at least 12 months * UPDRS Part 3 score ≥ 25 or more in off state

Exclusion criteria

* Past history of brain surgery for PD * Beck Depression Inventory Score ≥ 20 * Any history of cerebral insult or central nervous system infection * Cognitive impairment score \< 130 on the Mattis Dementia Rating Scale * Focal neurological deficits * Evidence of significant medical or psychiatric disorders * Secondary Parkinsonism * Atypical Parkinson's disease * History of substance abuse

Design outcomes

Primary

Measure	Time frame
Evaluate a change in UPDRS scores	—

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Mar 29, 2026