Ireland
--:--IST
Active, not recruiting Phase 2

A Study of AAV2-GDNF in Adults With Moderate Parkinson's Disease (REGENERATE-PD)

NCT06285643 · tracked via the Priya Life Science Germany tracker
Sponsor
Phase
Phase 2
Started
2024-06-11
Last updated
2026-10-01

Condition(s) studied

Parkinson Disease

Investigational drug(s) / intervention(s)

AAV2-GDNF gene therapy →control surgery

AAV2-GDNF gene therapy: Bilateral image-guided infusion of AAV2-GDNF into putamen, single dose

control surgery: Bilateral partial burr/twist holes without dural penetration

Study 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.

Eligibility

Sex
ALL
Min age
45 Years
Max age
75 Years
Healthy volunteers
No
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

Primary outcome measure(s)

  • Change from baseline to Month 18 on PD Motor Diary — 18 months
    Normalized Good ON time per Hauser Diary

Trial sites (46)

FacilityCityRegionStatus
Mayo Clinic Phoenix Arizona
UCI Alpha Clinic (Neurology) Irvine California
Loma Linda University (Neurology) Loma Linda California
Loma Linda University (Surgical) Loma Linda California
University of California San Francisco (Neurology) San Francisco California
University of San Francisco (Surgical) San Francisco California
CenExel Rocky Mountain Clinical Research (Neurology) Englewood Colorado
Georgetown University (Neurology) Washington D.C. District of Columbia
Parkinson's Disease and Movement Disorders Center of Boca Raton (Neurology) Boca Raton Florida
University of Florida Gainesville Florida
University of Miami Miami Florida
Orlando Health Neuroscience Institute Orlando Florida
Parkinson's Disease Treatment Center of Southwest Florida (Neurology) Port Charlotte Florida
Emory University (Surgical) Atlanta Georgia
Rush University Medical Center (Neurology) Chicago Illinois
University of Kansas Medical Center (Neurology) Kansas City Kansas
Massachusetts General Hospital (Neurology) Boston Massachusetts
Massachusetts General Hospital (Surgical) Boston Massachusetts
Michigan State University (Neurology) East Lansing Michigan
Quest Research Institute (Neurology) Farmington Hills Michigan
Cleveland Clinic Lou Ruvo (Neurology) Las Vegas Nevada
Columbia University New York New York
University of North Carolina (Neurology) Chapel Hill North Carolina
Duke Neurosurgery (Surgical) Durham North Carolina
Duke University (Neurology) Durham North Carolina
Ohio State University (Surgical) Columbus Ohio
University of Pennsylvania (Neurology) Philadelphia Pennsylvania
Vanderbilt University (Neurology) Nashville Tennessee
Texas Movement Disorder Specialists Georgetown Texas
Houston Methodist (Neurology) Houston Texas
Houston Methodist (Surgical) Houston Texas
University of Utah Salt Lake City Utah
EvergreenHealth Research Department (Neurology) Kirkland Washington
University of Wisconsin (Neurology) Madison Wisconsin
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

+ 6 more sites — see the full list on the official registry below.

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06285643 on ClinicalTrials.gov ↗ ← All trials in Germany