A Study of AAV2-GDNF in Adults With Moderate Parkinson's Disease (REGENERATE-PD)
REGENERATE-PD
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
1 other identifier
interventional
127
4 countries
46
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_2 parkinson-disease
Started Jun 2024
Longer than P75 for phase_2 parkinson-disease
46 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
February 14, 2024
CompletedFirst Posted
Study publicly available on registry
February 29, 2024
CompletedStudy Start
First participant enrolled
June 11, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 31, 2028
ExpectedStudy Completion
Last participant's last visit for all outcomes
August 31, 2028
October 1, 2026
September 1, 2026
4.2 years
February 14, 2024
September 30, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Change from baseline to Month 18 on PD Motor Diary
Normalized Good ON time per Hauser Diary
18 months
Study Arms (2)
AAV2-GDNF
EXPERIMENTALControl Surgery
SHAM COMPARATORInterventions
Bilateral image-guided infusion of AAV2-GDNF into putamen, single dose
Eligibility Criteria
You may qualify if:
- Age
- Male and female adults 45-75 years of age inclusive, at the time of signing of informed consent Type of Subject and Disease Characteristics
- Diagnosed with Parkinson's disease in the past 4-10 years (inclusive) as defined by the following:
- Presence of bradykinesia PLUS any of the following:
- Rigidity
- Rest tremor
- Postural instability
- Presence of motor fluctuations as measured by the PD Motor Diary
- Stable anti-parkinsonian medication regimen for \>/= 4 weeks prior to screening
- Must demonstrate responsiveness to levodopa therapy
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- AskBio Inclead
- Bayercollaborator
Study Sites (46)
Mayo Clinic
Phoenix, Arizona, 85054, United States
UCI Alpha Clinic (Neurology)
Irvine, California, 92697, United States
Loma Linda University (Neurology)
Loma Linda, California, 92354, United States
Loma Linda University (Surgical)
Loma Linda, California, 92354, United States
University of California San Francisco (Neurology)
San Francisco, California, 94143, United States
University of San Francisco (Surgical)
San Francisco, California, 94143, United States
CenExel Rocky Mountain Clinical Research (Neurology)
Englewood, Colorado, 80113, United States
Georgetown University (Neurology)
Washington D.C., District of Columbia, 20057, United States
Parkinson's Disease and Movement Disorders Center of Boca Raton (Neurology)
Boca Raton, Florida, 33486, United States
University of Florida
Gainesville, Florida, 32608, United States
University of Miami
Miami, Florida, 33125, United States
Orlando Health Neuroscience Institute
Orlando, Florida, 32806, United States
Parkinson's Disease Treatment Center of Southwest Florida (Neurology)
Port Charlotte, Florida, 33980, United States
Emory University (Surgical)
Atlanta, Georgia, 30322, United States
Rush University Medical Center (Neurology)
Chicago, Illinois, 60612, United States
University of Kansas Medical Center (Neurology)
Kansas City, Kansas, 66103, United States
Massachusetts General Hospital (Neurology)
Boston, Massachusetts, 02114, United States
Massachusetts General Hospital (Surgical)
Boston, Massachusetts, 02114, United States
Michigan State University (Neurology)
East Lansing, Michigan, 48824, United States
Quest Research Institute (Neurology)
Farmington Hills, Michigan, 48334, United States
Cleveland Clinic Lou Ruvo (Neurology)
Las Vegas, Nevada, 89106, United States
Columbia University
New York, New York, 10032, United States
University of North Carolina (Neurology)
Chapel Hill, North Carolina, 27514, United States
Duke Neurosurgery (Surgical)
Durham, North Carolina, 27705, United States
Duke University (Neurology)
Durham, North Carolina, 27710, United States
Ohio State University (Surgical)
Columbus, Ohio, 43210, United States
University of Pennsylvania (Neurology)
Philadelphia, Pennsylvania, 19104, United States
Vanderbilt University (Neurology)
Nashville, Tennessee, 37232, United States
Texas Movement Disorder Specialists
Georgetown, Texas, 78628, United States
Houston Methodist (Neurology)
Houston, Texas, 77030, United States
Houston Methodist (Surgical)
Houston, Texas, 77030, United States
University of Utah
Salt Lake City, Utah, 84132, United States
EvergreenHealth Research Department (Neurology)
Kirkland, Washington, 98034, United States
University of Wisconsin (Neurology)
Madison, Wisconsin, 53705, United States
Charité - Universitätsmedizin Berlin (Surgical)
Berlin, Germany
Philipps-Universität Marburg (Neurology)
Marburg, Germany
Universitätsklinikum Tübingen (Neurology)
Tübingen, Germany
Universitätsklinikum Tübingen (Surgical)
Tübingen, Germany
Universitätsklinikum Würzburg (Neurology)
Würzburg, Germany
Copernicus Podmiot Leczniczy, Szpital św. Wojciecha (Neurology)
Gdansk, Poland
Specjalistyczna Praktyka Lekarska Prof. Grzegorz Opala (Neurology)
Katowice, Poland
Mazowiecki Szpital Bródnowski (Neurology)
Warsaw, Poland
Mazowiecki Szpital Bródnowski (Surgical)
Warsaw, Poland
Samodzielny Publiczny Szpital Kliniczny im. Prof. W. Orłowskiego CMKP (Neurology)
Warsaw, Poland
North Bristol NHS Trust - Southmead Hospital (Neurology)
Bristol, United Kingdom
University Hospital of Wales (Surgical)
Cardiff, United Kingdom
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
February 14, 2024
First Posted
February 29, 2024
Study Start
June 11, 2024
Primary Completion (Estimated)
August 31, 2028
Study Completion (Estimated)
August 31, 2028
Last Updated
October 1, 2026
Record last verified: 2026-09