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Completed

NCT Number: NCT00195143

Safety Study of Subthalamic Nucleus Gene Therapy for Parkinson's Disease

The purpose of this study is to determine the safety of using a modified virus to transfer a gene called GAD into a region of the brain called the subthalamic nucleus in patients with advanced Parkinson's disease. The overall goal of this approach is to ultimately normalize the flow of information in several brain regions responsible for movement, to ultimately improve function in patients with this disorder. The current study is primarily designed to evaluate the safety of this approach, but patients are also being monitored for possible signs of effectiveness as well.

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Key information

Conditions

Parkinson's Disease Basal Ganglia Diseases Brain Diseases Central Nervous System Diseases Movement Disorders Nervous System Diseases Neurodegenerative Diseases Parkinson Disease Parkinsonian Disorders Synucleinopathies

Age range

25 year–75 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

North Shore University Hospital

Manhasset, New York, 11030, United States

About this study

This study involves treatment of patients with medically refractory Parkinson's disease (PD) with gene therapy. The patients are chosen from a population of patients who would normally be candidates for standard deep brain stimulation (DBS) surgery for PD. These patients respond to medical therapy, but develop substantially reduced responses over time, often with severe fluctuations in their condition between a functional and severely non-functional state. Some patients also develop dose-limiting side effects from medication, including involuntary movements called dyskinesias and nightmares. When there are no medical contraindications, DBS is often performed in these patients to try to quiet hyperactive brain regions such as the subthalamic nucleus (STN). In PD, the STN is overactive due to a loss of GABA inputs to this region, which normally reduces neuronal firing. In turn, the STN drives other brain regions, including the globus pallidus (GPi) and substantia nigra (SNr), which are also hyperactive and which also have reduced GABA inputs. The goal of this gene therapy trial is to introduce the gene for glutamic acid decarboxylase (GAD) into the STN using an adeno-associated virus (AAV) vector, in order to permit the STN to produce it's own GABA, as well as release GABA into the GPi and SNr targets, which also have reduced GABA inputs. This is anticipated to restore a more normal pattern of information flow from this basal ganglia circuit to the thalamus and higher cortical structures in order to reduce the motor symptoms of PD, while eliminating complications arising from inserting DBS electrodes and batteries into the human body.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Duration of disease: Greater than 5 years
  • Idiopathic Parkinson's disease
  • Parkinson's medication stable for 3 months
  • Absence of dementia
  • Hoehn and Yahr rating: 3 or greater and/or UPDRS: 30 or more in "off" state and/or Complications of l-dopa therapy limiting effective use

Exclusion criteria

  • Poor candidate for any surgery
  • Significant dementia
  • Secondary parkinsonism
  • Severe autonomic symptoms
  • Atypical Parkinson's disease
  • History of substance abuse

Treatment and study plan

Surgical infusion of AAV-GAD into the subthalamic nucleus

Genetic

Primary outcomes

  1. Safety

Secondary outcomes

  1. Improvement in brain metabolism measured by PET scans

  2. Improvement in standard clinical rating scales

Sponsors and collaborators

Lead sponsor

Neurologix, Inc.

Industry

Collaborators

  • North Shore University Hospital
  • Weill Medical College of Cornell University

Registry information

Official study title

Phase I Study of Subthalamic GAD Gene Transfer in Medically Refractory Parkinson's Disease Patients

Important dates

Study start
2003
Study completion
2005
First posted
Sep 19, 2005
Registry last updated
Mar 27, 2008

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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