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

Last updated: June 25, 2026
Sponsor: AskBio Inc
Overall Status: Active - Recruiting

Phase

2

Condition

N/A

Treatment

Control Surgery

AAV2-GDNF gene therapy

AAV2-GDNF Gene therapy

Clinical Study ID

NCT06285643
ASK-PD5-CS201
  • Ages 45-75
  • All Genders

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 Criteria

Inclusion

Inclusion Criteria:

Age

  1. Male and female adults 45-75 years of age inclusive, at the time of signing ofinformed consent Type of Subject and Disease Characteristics

  2. Diagnosed with Parkinson's disease in the past 4-10 years (inclusive) as defined bythe following:

  3. Presence of bradykinesia PLUS any of the following:

  • Rigidity
  • Rest tremor
  • Postural instability
  1. Presence of motor fluctuations as measured by the PD Motor Diary

  2. Stable anti-parkinsonian medication regimen for >/= 4 weeks prior to screening

  3. Must demonstrate responsiveness to levodopa therapy

Exclusion

Exclusion Criteria:

  • Known history or current evidence of medical, genetic, or neurological conditionsthat 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 surgicalsuitability and/or subject safety

  • Contraindication to magnetic resonance imaging and/or use gadolinium-based contrastagents

  • Prior history of brain surgery including, but no limited: DBS, pallidotomy focusedultrasound thalamotomy, or other experimental neurosurgical procedure

  • Chronic immunosuppressive therapy

Study Design

Total Participants: 127
Treatment Group(s): 4
Primary Treatment: Control Surgery
Phase: 2
Study Start date:
June 11, 2024
Estimated Completion Date:
August 31, 2028

Connect with a study center

  • Charité - Universitätsmedizin Berlin (Surgical)

    Berlin,
    Germany

    Site Not Available

  • Philipps-Universität Marburg (Neurology)

    Marburg,
    Germany

    Active - Recruiting

  • Universitätsklinikum Tübingen (Neurology)

    Tübingen,
    Germany

    Active - Recruiting

  • Universitätsklinikum Tübingen (Surgical)

    Tübingen,
    Germany

    Site Not Available

  • Universitätsklinikum Tübingen (Neurology)

    Tübingen 2820860,
    Germany

    Active - Recruiting

  • Universitätsklinikum Würzburg (Neurology)

    Würzburg,
    Germany

    Active - Recruiting

  • Copernicus Podmiot Leczniczy, Szpital św. Wojciecha (Neurology)

    Gdansk,
    Poland

    Active - Recruiting

  • Specjalistyczna Praktyka Lekarska Prof. Grzegorz Opala

    Katowice,
    Poland

    Active - Recruiting

  • Specjalistyczna Praktyka Lekarska Prof. Grzegorz Opala (Neurology)

    Katowice,
    Poland

    Active - Recruiting

  • Mazowiecki Szpital Bródnowski

    Warsaw,
    Poland

    Site Not Available

  • Mazowiecki Szpital Bródnowski (Neurology)

    Warsaw,
    Poland

    Active - Recruiting

  • Mazowiecki Szpital Bródnowski (Surgical)

    Warsaw,
    Poland

    Site Not Available

  • Samodzielny Publiczny Szpital Kliniczny im. Prof. W. Orłowskiego CMKP (Neurology)

    Warsaw,
    Poland

    Active - Recruiting

  • Mazowiecki Szpital Bródnowski (Neurology)

    Warsaw 756135,
    Poland

    Active - Recruiting

  • Samodzielny Publiczny Szpital Kliniczny im. Prof. W. Orłowskiego CMKP (Neurology)

    Warsaw 756135,
    Poland

    Active - Recruiting

  • Mazowiecki Szpital Bródnowski (Neurology)

    Warszawa,
    Poland

    Active - Recruiting

  • Mazowiecki Szpital Bródnowski (Surgical)

    Warszawa,
    Poland

    Site Not Available

  • North Bristol NHS Trust - Southmead Hospital (Neurology)

    Bristol,
    United Kingdom

    Active - Recruiting

  • University Hospital of Wales

    Cardiff,
    United Kingdom

    Site Not Available

  • University Hospital of Wales (Surgical)

    Cardiff,
    United Kingdom

    Site Not Available

  • Mayo Clinic

    Phoenix, Arizona 85054
    United States

    Site Not Available

  • UCI Alpha Clinic (Neurology)

    Irvine, California 92697
    United States

    Active - Recruiting

  • Loma Linda University

    Loma Linda, California 92354
    United States

    Site Not Available

  • Loma Linda University (Neurology)

    Loma Linda, California 92354
    United States

    Active - Recruiting

  • Loma Linda University (Surgical)

    Loma Linda, California 92354
    United States

    Site Not Available

  • University of California San Francisco

    San Francisco, California 94143
    United States

    Site Not Available

  • University of California San Francisco (Neurology)

    San Francisco, California 94143
    United States

    Active - Recruiting

  • University of San Francisco (Surgical)

    San Francisco, California 94143
    United States

    Site Not Available

  • Loma Linda University (Surgical)

    Loma Linda 5367696, California 5332921 92354
    United States

    Site Not Available

  • University of California San Francisco (Neurology)

    San Francisco 5391959, California 5332921 94143
    United States

    Active - Recruiting

  • CenExel Rocky Mountain Clinical Research (Neurology)

    Englewood, Colorado 80113
    United States

    Active - Recruiting

  • Georgetown University (Neurology)

    Washington D.C., District of Columbia 20057
    United States

    Active - Recruiting

  • Parkinson's Disease and Movement Disorders Center of Boca Raton (Neurology)

    Boca Raton, Florida 33486
    United States

    Active - Recruiting

  • University of Florida

    Gainesville, Florida 32608
    United States

    Site Not Available

  • University of Miami

    Miami, Florida 33125
    United States

    Site Not Available

  • Orlando Health Neuroscience Institute

    Orlando, Florida 32806
    United States

    Active - Recruiting

  • Parkinson's Disease Treatment Center of Southwest Florida

    Port Charlotte, Florida 33980
    United States

    Active - Recruiting

  • Parkinson's Disease Treatment Center of Southwest Florida (Neurology)

    Port Charlotte, Florida 33980
    United States

    Active - Recruiting

  • Emory University (Surgical)

    Atlanta, Georgia 30322
    United States

    Site Not Available

  • Rush University Medical Center (Neurology)

    Chicago, Illinois 60612
    United States

    Active - Recruiting

  • University of Kansas Medical Center

    Kansas City, Kansas 66103
    United States

    Active - Recruiting

  • University of Kansas Medical Center (Neurology)

    Kansas City, Kansas 66103
    United States

    Active - Recruiting

  • Massachusetts General Hospital

    Boston, Massachusetts 02114
    United States

    Active - Recruiting

  • Massachusetts General Hospital (Neurology)

    Boston, Massachusetts 02114
    United States

    Active - Recruiting

  • Massachusetts General Hospital (Surgical)

    Boston, Massachusetts 02114
    United States

    Site Not Available

  • Massachusetts General Hospital (Neurology)

    Boston 4930956, Massachusetts 6254926 02114
    United States

    Active - Recruiting

  • Michigan State University

    East Lansing, Michigan 48824
    United States

    Active - Recruiting

  • Michigan State University (Neurology)

    East Lansing, Michigan 48824
    United States

    Active - Recruiting

  • Quest Research Institute

    Farmington Hills, Michigan 48334
    United States

    Site Not Available

  • Quest Research Institute (Neurology)

    Farmington Hills, Michigan 48334
    United States

    Active - Recruiting

  • Cleveland Clinic Lou Ruvo

    Las Vegas, Nevada 89106
    United States

    Site Not Available

  • Cleveland Clinic Lou Ruvo (Neurology)

    Las Vegas, Nevada 89106
    United States

    Active - Recruiting

  • Columbia University

    New York, New York 10032
    United States

    Active - Recruiting

  • University of North Carolina

    Chapel Hill, North Carolina 27514
    United States

    Site Not Available

  • University of North Carolina (Neurology)

    Chapel Hill, North Carolina 27514
    United States

    Active - Recruiting

  • Duke Neurosurgery (Surgical)

    Durham, North Carolina 27705
    United States

    Site Not Available

  • Duke University

    Durham, North Carolina 27710
    United States

    Site Not Available

  • Duke University (Neurology)

    Durham, North Carolina 27710
    United States

    Active - Recruiting

  • Duke Neurosurgery (Surgical)

    Durham 4464368, North Carolina 4482348 27705
    United States

    Site Not Available

  • Ohio State University (Surgical)

    Columbus, Ohio 43210
    United States

    Site Not Available

  • University of Pennsylvania

    Philadelphia, Pennsylvania 19104
    United States

    Active - Recruiting

  • University of Pennsylvania (Neurology)

    Philadelphia, Pennsylvania 19104
    United States

    Active - Recruiting

  • Vanderbilt University

    Nashville, Tennessee 37232
    United States

    Site Not Available

  • Vanderbilt University (Neurology)

    Nashville, Tennessee 37232
    United States

    Active - Recruiting

  • Texas Movement Disorder Specialists

    Georgetown, Texas 78628
    United States

    Site Not Available

  • Houston Methodist (Neurology)

    Houston, Texas 77030
    United States

    Active - Recruiting

  • Houston Methodist (Surgical)

    Houston, Texas 77030
    United States

    Site Not Available

  • Houston Methodist (Neurology)

    Houston 4699066, Texas 4736286 77030
    United States

    Active - Recruiting

  • University of Utah

    Salt Lake City, Utah 84132
    United States

    Site Not Available

  • EvergreenHealth Research Department (Neurology)

    Kirkland, Washington 98034
    United States

    Active - Recruiting

  • University of Wisconsin

    Madison, Wisconsin 53705
    United States

    Active - Recruiting

  • University of Wisconsin (Neurology)

    Madison, Wisconsin 53705
    United States

    Active - Recruiting

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