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A Study of AAV2-GDNF in Adults With Moderate Parkinson's Disease (REGENERATE-PD)
NCT06285643 · AskBio Inc
In plain English
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Official title
A Phase 2, Randomized, Double-blind, Sham Surgery-controlled Study of the Efficacy and Safety of Intraputaminal AAV2-GDNF in the Treatment of Adults With Moderate Stage Parkinson's Disease
About this study
The objective of this randomized, surgically controlled, double-blinded, Phase 2 study is to evaluate the safety and efficacy of AAV2-GDNF delivered to the putamen in subjects with moderate Parkinson's Disease.
Eligibility criteria
Inclusion Criteria:
Age
1. Male and female adults 45-75 years of age inclusive, at the time of signing of informed consent Type of Subject and Disease Characteristics
2. Diagnosed with Parkinson's disease in the past 4-10 years (inclusive) as defined by the following:
1. Presence of bradykinesia PLUS any of the following:
* Rigidity
* Rest tremor
* Postural instability
2. Presence of motor fluctuations as measured by the PD Motor Diary
3. Stable anti-parkinsonian medication regimen for \>/= 4 weeks prior to screening
4. Must demonstrate responsiveness to levodopa therapy
Exclusion Criteria:
* Known history or current evidence of medical, genetic, or neurological conditions that may provide an alternative to idiopathic PD diagnosis
* Presence or history of significant vascular and/or cardiovascular disease
* Presence of significant cognitive impairment, poorly controlled depression/anxiety
* Presence or history of psychosis or impulse control disorder
* History of malignancy other than treated cutaneous squamous or basal cell carcinomas
* Presence of clinically relevant conditions that could compromise surgical suitability and/or subject safety
* Contraindication to magnetic resonance imaging and/or use gadolinium-based contrast agents
* Prior history of brain surgery including, but no limited: DBS, pallidotomy focused ultrasound thalamotomy, or other experimental neurosurgical procedure
* Chronic immunosuppressive therapy
Study design
Enrollment target: 127 participants
Allocation: randomized
Masking: quadruple
Age groups: adult, older_adult
Timeline
Starts: 2024-06-11
Estimated completion: 2028-08-31
Last updated: 2025-12-08
Interventions
Drug: AAV2-GDNF gene therapyProcedure: control surgery
Primary outcomes
- • Change from baseline to Month 18 on PD Motor Diary (18 months)
Sponsor
AskBio Inc · industry
With: Bayer
Contacts & investigators
ContactNisha Chhabria, MD · contact · askfirst@askbio.com · 919-388-1040
ContactAmber Van Laar, MD · contact · askfirst@askbio.com · 919-388-1040
All locations (39)
UCI Alpha Clinic (Neurology)Recruiting
Irvine, California, United States
Loma Linda University (Neurology)Recruiting
Loma Linda, California, United States
Loma Linda University (Surgical)Active Not Recruiting
Loma Linda, California, United States
University of California San Francisco (Neurology)Recruiting
San Francisco, California, United States
University of San Francisco (Surgical)Active Not Recruiting
San Francisco, California, United States
CenExel Rocky Mountain Clinical Research (Neurology)Recruiting
Englewood, Colorado, United States
Georgetown University (Neurology)Recruiting
Washington D.C., District of Columbia, United States
Parkinson's Disease and Movement Disorders Center of Boca Raton (Neurology)Recruiting
Boca Raton, Florida, United States
Parkinson's Disease Treatment Center of Southwest Florida (Neurology)Active Not Recruiting
Port Charlotte, Florida, United States
Emory University (Surgical)Active Not Recruiting
Atlanta, Georgia, United States
Rush University Medical Center (Neurology)Recruiting
Chicago, Illinois, United States
University of Kansas Medical Center (Neurology)Recruiting
Kansas City, Kansas, United States
Massachusetts General Hospital (Neurology)Recruiting
Boston, Massachusetts, United States
Massachusetts General Hospital (Surgical)Active Not Recruiting
Boston, Massachusetts, United States
Michigan State University (Neurology)Recruiting
East Lansing, Michigan, United States
Quest Research Institute (Neurology)Recruiting
Farmington Hills, Michigan, United States
Cleveland Clinic Lou Ruvo (Neurology)Recruiting
Las Vegas, Nevada, United States
University of North Carolina (Neurology)Recruiting
Chapel Hill, North Carolina, United States
Duke Neurosurgery (Surgical)Active Not Recruiting
Durham, North Carolina, United States
Duke University (Neurology)Recruiting
Durham, North Carolina, United States
Ohio State University (Surgical)Active Not Recruiting
Columbus, Ohio, United States
University of Pennsylvania (Neurology)Recruiting
Philadelphia, Pennsylvania, United States
Vanderbilt University (Neurology)Recruiting
Nashville, Tennessee, United States
Houston Methodist (Neurology)Recruiting
Houston, Texas, United States
Houston Methodist (Surgical)Active Not Recruiting
Houston, Texas, United States
EvergreenHealth Research Department (Neurology)Recruiting
Kirkland, Washington, United States
University of Wisconsin (Neurology)Recruiting
Madison, Wisconsin, United States
Charité - Universitätsmedizin Berlin (Surgical)Active Not Recruiting
Berlin, Germany
Philipps-Universität Marburg (Neurology)Recruiting
Marburg, Germany
Universitätsklinikum Tübingen (Neurology)Recruiting
Tübingen, Germany
Universitätsklinikum Tübingen (Surgical)Active Not Recruiting
Tübingen, Germany
Universitätsklinikum Würzburg (Neurology)Recruiting
Würzburg, Germany
Copernicus Podmiot Leczniczy, Szpital św. Wojciecha (Neurology)Recruiting
Gdansk, Poland
Specjalistyczna Praktyka Lekarska Prof. Grzegorz Opala (Neurology)Recruiting
Katowice, Poland
Mazowiecki Szpital Bródnowski (Neurology)Recruiting
Warsaw, Poland
Mazowiecki Szpital Bródnowski (Surgical)Active Not Recruiting
Warsaw, Poland
Samodzielny Publiczny Szpital Kliniczny im. Prof. W. Orłowskiego CMKP (Neurology)Recruiting
Warsaw, Poland
North Bristol NHS Trust - Southmead Hospital (Neurology)Recruiting
Bristol, United Kingdom
University Hospital of Wales (Surgical)Active Not Recruiting
Cardiff, United Kingdom