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

Primary Purpose

Parkinson's Disease

Status
Terminated
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Bilateral surgical infusion of AAV-GAD into the subthalamic nucleus
Sponsored by
Neurologix, Inc.
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Parkinson's Disease focused on measuring 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

Eligibility Criteria

30 Years - 75 Years (Adult, Older Adult)All SexesDoes not accept 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

Sites / Locations

  • Stanford University
  • University of Colorado
  • Massachusetts General Hospital
  • Henry Ford Health Systems - Franklin Pointe Medical
  • University of Rochester
  • Wake Forest University Health Science Center
  • The Ohio State University

Outcomes

Primary Outcome Measures

Evaluate a change in UPDRS scores

Secondary Outcome Measures

Full Information

First Posted
March 24, 2008
Last Updated
February 20, 2012
Sponsor
Neurologix, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT00643890
Brief Title
Study of AAV-GAD Gene Transfer Into the Subthalamic Nucleus for Parkinson's Disease
Official Title
Phase 2 Safety and Efficacy Study Evaluating Glutamic Acid Decarboxylase Gene Transfer to Subthalamic Nuclei in Subjects With Advanced Parkinson's Disease
Study Type
Interventional

2. Study Status

Record Verification Date
February 2012
Overall Recruitment Status
Terminated
Why Stopped
Due to financial reasons
Study Start Date
August 2008 (undefined)
Primary Completion Date
December 2010 (Actual)
Study Completion Date
undefined (undefined)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Neurologix, Inc.

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
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

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
44 (Anticipated)

8. Arms, Groups, and Interventions

Intervention Type
Genetic
Intervention Name(s)
Bilateral surgical infusion of AAV-GAD into the subthalamic nucleus
Intervention Description
One-time bilateral administration of rAAV-GAD at 1X10^12 vector genomes in 35 uL.
Primary Outcome Measure Information:
Title
Evaluate a change in UPDRS scores

10. Eligibility

Sex
All
Minimum Age & Unit of Time
30 Years
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Facility Information:
Facility Name
Stanford University
City
Stanford
State/Province
California
ZIP/Postal Code
94305-5401
Country
United States
Facility Name
University of Colorado
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
Massachusetts General Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Facility Name
Henry Ford Health Systems - Franklin Pointe Medical
City
Southfield
State/Province
Michigan
ZIP/Postal Code
48034
Country
United States
Facility Name
University of Rochester
City
Rochester
State/Province
New York
ZIP/Postal Code
14642
Country
United States
Facility Name
Wake Forest University Health Science Center
City
Winston-Salem
State/Province
North Carolina
ZIP/Postal Code
27157
Country
United States
Facility Name
The Ohio State University
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43210
Country
United States

12. IPD Sharing Statement

Citations:
PubMed Identifier
17586305
Citation
Kaplitt MG, Feigin A, Tang C, Fitzsimons HL, Mattis P, Lawlor PA, Bland RJ, Young D, Strybing K, Eidelberg D, During MJ. Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial. Lancet. 2007 Jun 23;369(9579):2097-105. doi: 10.1016/S0140-6736(07)60982-9.
Results Reference
background
PubMed Identifier
18042721
Citation
Feigin A, Kaplitt MG, Tang C, Lin T, Mattis P, Dhawan V, During MJ, Eidelberg D. Modulation of metabolic brain networks after subthalamic gene therapy for Parkinson's disease. Proc Natl Acad Sci U S A. 2007 Dec 4;104(49):19559-64. doi: 10.1073/pnas.0706006104. Epub 2007 Nov 27.
Results Reference
background
PubMed Identifier
21419704
Citation
LeWitt PA, Rezai AR, Leehey MA, Ojemann SG, Flaherty AW, Eskandar EN, Kostyk SK, Thomas K, Sarkar A, Siddiqui MS, Tatter SB, Schwalb JM, Poston KL, Henderson JM, Kurlan RM, Richard IH, Van Meter L, Sapan CV, During MJ, Kaplitt MG, Feigin A. AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial. Lancet Neurol. 2011 Apr;10(4):309-19. doi: 10.1016/S1474-4422(11)70039-4.
Results Reference
result
PubMed Identifier
28405611
Citation
Niethammer M, Tang CC, LeWitt PA, Rezai AR, Leehey MA, Ojemann SG, Flaherty AW, Eskandar EN, Kostyk SK, Sarkar A, Siddiqui MS, Tatter SB, Schwalb JM, Poston KL, Henderson JM, Kurlan RM, Richard IH, Sapan CV, Eidelberg D, During MJ, Kaplitt MG, Feigin A. Long-term follow-up of a randomized AAV2-GAD gene therapy trial for Parkinson's disease. JCI Insight. 2017 Apr 6;2(7):e90133. doi: 10.1172/jci.insight.90133.
Results Reference
derived

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

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