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

March 26, 2008 updated by: Neurologix, Inc.

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

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.

Study Type

Interventional

Enrollment

12

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • New York
      • Manhasset, New York, United States, 11030
        • North Shore University Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

25 years to 75 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Safety

Secondary Outcome Measures

Outcome Measure
Improvement in brain metabolism measured by PET scans
Improvement in standard clinical rating scales

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Neurologix, Inc.

Collaborators

Weill Medical College of Cornell University
North Shore University Hospital

Investigators

  • Principal Investigator: Michael G Kaplitt, MD PhD, Weill Medical College of Cornell University
  • Principal Investigator: Matthew J During, MD, Weill Medical College of Cornell University

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

August 1, 2003

Study Completion

August 1, 2005

Study Registration Dates

First Submitted

September 15, 2005

First Submitted That Met QC Criteria

September 15, 2005

First Posted (Estimate)

September 19, 2005

Study Record Updates

Last Update Posted (Estimate)

March 27, 2008

Last Update Submitted That Met QC Criteria

March 26, 2008

Last Verified

September 1, 2005

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 0902-478

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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