Parkinson's DiseasePhase 2NCT00643890
Study of AAV-GAD Gene Transfer Into the Subthalamic Nucleus for Parkinson's Disease
A Phase 2 study testing Bilateral surgical infusion of AAV-GAD into the subthalamic nucleus in people with Parkinson's Disease, run by Neurologix, Inc. at 7 sites in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 30 to 75 years
- Conditions the study is for
- Parkinson's Disease
- What is being tested?
- Bilateral surgical infusion of AAV-GAD into the subthalamic nucleus
- Type of treatment
- Injection or infusion
- How many people
- 44 participants
- Where
- 7 sitesUnited States
- Study dates
- Aug 2008to Dec 2010
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 30 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 9Show fewer
- Secondary Parkinsonism
- Atypical Parkinson's disease
- History of substance abuse
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
United States7 sites
University of Colorado
Aurora, Colorado, 80045
Massachusetts General Hospital
Boston, Massachusetts, 02114
The Ohio State University
Columbus, Ohio, 43210
University of Rochester
Rochester, New York, 14642
Henry Ford Health Systems - Franklin Pointe Medical
Southfield, Michigan, 48034
Stanford University
Stanford, California, 94305-5401
Wake Forest University Health Science Center
Winston-Salem, North Carolina, 27157
About this study
Official title: Phase 2 Safety and Efficacy Study Evaluating Glutamic Acid Decarboxylase Gene Transfer to Subthalamic Nuclei in Subjects With Advanced Parkinson's Disease
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.
- Conditions
- Parkinson's Disease
- Being tested
- Bilateral surgical infusion of AAV-GAD into the subthalamic nucleus
- 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
- Medical subject headings
- Parkinson Disease, Ganglion Cysts, Movement Disorders, Basal Ganglia Diseases, Central Nervous System Diseases, Parkinsonian Disorders, Neurodegenerative Diseases, Brain Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Bilateral surgical infusion of AAV-GAD into the subthalamic nucleusGenetic
One-time bilateral administration of rAAV-GAD at 1X10^12 vector genomes in 35 uL.
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluate a change in UPDRS scores
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Mar 2008 (estimated)
- Study startAug 2008
- Primary completionDec 2010
Why it stopped: Due to financial reasons
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Parkinson's DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 44 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Neurologix, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00643890
- Sponsor's protocol ID
- NRGX-GAD-02
- Status last verified
- Feb 2012
- First submitted
- 24 Mar 2008
- Last updated
- 22 Feb 2012
- Results posted
- No
- Data monitoring committee
- Yes
Publications
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.
PubMed 17586305Feigin 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.
PubMed 18042721LeWitt 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.
PubMed 21419704Niethammer 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.
PubMed 28405611
Study at a glance
From the registry record, last updated by the sponsor on 22 Feb 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 2 Safety and Efficacy Study Evaluating Glutamic Acid Decarboxylase Gene Transfer to Subthalamic Nuclei in Subjects With Advanced Parkinson's Disease
- ClinicalTrials.gov ID
- NCT00643890
- Acronym / sponsor ID
- NRGX-GAD-02
- 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
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Due to financial reasons
- Enrolment
- 44 participants (estimated)
- Lead sponsor
- Neurologix, Inc.Industry (pharma, biotech, devices)
- Study start
- Aug 2008
- Primary completion
- Dec 2010 (actual)
- First posted
- 26 Mar 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
Who may qualify
- Ages
- 30 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (9 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Bilateral surgical infusion of AAV-GAD into the subthalamic nucleusOne-time bilateral administration of rAAV-GAD at 1X10^12 vector genomes in 35 uL. | Genetic | – |
Primary outcome
Evaluate a change in UPDRS scores
Results and safety
The study was stopped early (reason given: Due to financial reasons). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Feb 2012.
- Background: PubMed 17586305Kaplitt 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.
- Background: PubMed 18042721Feigin 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 publication: PubMed 21419704LeWitt 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.
- Derived from this study: PubMed 28405611Niethammer 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.
Related trials for Parkinson's Disease
Enrolling by invitationObservational study
Early Molecular Biomarkers for Differentiating Parkinsonian Syndromes
PARKINSON DISEASE (Disorder), Atypical Parkinsonism
Active, not recruitingPhase 1/2
Clinical Safety and Efficacy of Umbilical Cord Mesenchymal Stromal Cells-derived Exosomes in Patients With Parkinson's Disease
Parkinson Disease (PD)
Active, not recruitingObservational study
Adaptive DBS to Offset STN Dysregulation During Exercise in PD
Parkinson Disease
Not yet recruiting
Compulsive Eating and Brain's Reward System in Different Pathologies
PARKINSON DISEASE (Disorder), Bipolar 1 Disorder
Not yet recruiting
Evaluating the Effect of the Zeen Mobility Device on Clinical Practicality, Safety, and Impact (Via Assessments of Walking, Standing Tolerance, Balance, Endurance, Effort, and Participation in Daily Tasks) in an Outpatient Rehabilitation Setting for Individuals With Mobility Impairments.
Stroke, Parkinson's Disease (PD)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Bilateral surgical infusion of AAV-GAD into the subthalamic nucleus in people with Parkinson's Disease, run by Neurologix, Inc. at 7 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 22 Feb 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Due to financial reasons). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluate a change in UPDRS scores. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00643890, which also lists 4 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Feb 2012.