Helen Bronte-Stewart
Safety and Efficacy Study of GAD Gene Transfer Therapy in Parkinson's disease
Contact Information
Stanford University School of Medicine 300 Pasteur Drive Stanford, CA 94305Brief
The purpose of this study is to determine the potential effectiveness and safety of a new type of treatment for Parkinson?s disease (PD) called gene transfer.
Recruiting Status:
RecruitingStanford Recruiting Status:
RecruitingCondition(s):
Intervention(s):
- Gene Transfer: AAV-GAD
Phase:
Phase 2Eligibility
Ages Eligible for Study:
30 years to 75 yearsGenders Eligible for Study:
Male and FemaleHealth of Volunteers:
People with the conditions listed in this trial can participate as controls.Key Inclusion Criteria:
1. Age 30 years to 75 years.
2. Typical features of PD present for at least 5 years, and levodopa responsiveness demonstrated for at least 12 months.
3. Progressive Parkinsonian disability with demonstrated benefit from continuing use of levodopa (and from other PD medications, if part of the treatment regimen).
Key Exclusion Criteria:
1. Any past history of brain surgery for PD.
2. History of any serious cerebral insult, such as head injury, toxic exposures, or central nervous system (CNS) infection.
3. Ongoing treatment with dopamine-blocking drugs.
4. Evidence for other significant medical or psychiatric disorders, such as psychosis, frequent hallucinations, severe depression, significant cognitive decline in recent months, systemic organ failure, or bleeding diathesis.
Additional Study Details
Official Title:
Phase 2 Safety and Efficacy Study Evaluating Glutamic Acid Decarboxylase Gene Transfer to Subthalamic Nucleus in Subjects with Advanced Parkinson's DiseaseAnticipated start date:
8/3/2009Lead Sponsor:
Wake Forest UniversityInvestigator(s):
Study Type:
InterventionalPurpose:
TreatmentAllocation:
RandomizedMasking:
Double BlindControl:
noneAssignment:
Single GroupEndpoints:
Safety/EfficacyPrimary Outcomes:
- The primary endpoint will be change in ?off?-state-determined UPDRS Part 3 (Motor section) scores evaluated between baseline and 6 months in the rAAV-GAD-treated group and compared to subjects receiving sham-surgery (burr holes only).
Secondary Outcomes:
- Secondary endpoints will also comparisons with baseline and 6 months for UPDRS Part 2 (Activities of daily living), the BPRS, the Hagell-Widner Dyskinesia Scale, a battery of neuropsychological testing and home PD motor function diaries.
Total Number to be Enrolled:
44Total Number to be Enrolled at Stanford:
5More Information
Publications About this Study:
- 17586305: 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.
- 18042721: Modulation of metabolic brain networks after subthalamic gene therapy for Parkinson's disease.
Secondary ID(s):
- IRB 16229
- SPO 45203
Locations & Contacts
Stanford Locations & Contacts:
Stanford University School of Medicine 300 Pasteur Drive Stanford, CA 94305Non-Stanford Locations:
The Stanford website does not have any locations outside of Stanford listed for this trial. You may want to check clinicaltrials.gov for posible additional locations.
This listing was last updated:
9/24/2009PLEASE NOTE:
Study Coordinators and Research Nurses cannot give medical advice over the phone. Telephone numbers are provided for obtaining additional information on specific clinical research trials only. If you have specific questions which require clinical expertise, please call your primary care physician. If you do not have a primary care physician please feel free to call the SHC Physician Referral Service at (800) 756-9000 or send an email.
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