Gene therapy trial hopes to ease Parkinson's symptoms

NCT ID NCT06285643

First seen Jun 27, 2026 · Last updated Jun 30, 2026 · Updated 2 times

Summary

This Phase 2 study tests whether a single dose of AAV2-GDNF gene therapy, delivered directly into the brain, can improve motor function in adults with moderate Parkinson's disease. About 127 participants will receive either the gene therapy or a sham surgery. The main goal is to see if the treatment increases 'good on time' without troublesome side effects over 18 months.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance
AAV2-GDNF gene therapy
What this could lead to
If successful, this could lead to a new gene therapy that helps control Parkinson's symptoms and reduces the need for daily medications.
What could go wrong
This is an early Phase 2 trial with only 127 participants, so results may not apply to everyone. The sham surgery control group helps test safety, but there are risks from the surgical procedure and the gene therapy itself.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • CenExel Rocky Mountain Clinical Research (Neurology)

    RECRUITING

    Englewood, Colorado, 80113, United States

    Contact Phone: •••-•••-••••

  • Charité - Universitätsmedizin Berlin (Surgical)

    ACTIVE_NOT_RECRUITING

    Berlin, Germany

  • Cleveland Clinic Lou Ruvo (Neurology)

    RECRUITING

    Las Vegas, Nevada, 89106, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Columbia University

    RECRUITING

    New York, New York, 10032, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

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

    RECRUITING

    Gdansk, Poland

    Contact Email: •••••@•••••

  • Duke Neurosurgery (Surgical)

    ACTIVE_NOT_RECRUITING

    Durham, North Carolina, 27705, United States

  • Duke University (Neurology)

    RECRUITING

    Durham, North Carolina, 27710, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Emory University (Surgical)

    ACTIVE_NOT_RECRUITING

    Atlanta, Georgia, 30322, United States

  • EvergreenHealth Research Department (Neurology)

    RECRUITING

    Kirkland, Washington, 98034, United States

    Contact Email: •••••@•••••

  • Georgetown University (Neurology)

    RECRUITING

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

    Contact Email: •••••@•••••

  • Houston Methodist (Neurology)

    RECRUITING

    Houston, Texas, 77030, United States

    Contact Email: •••••@•••••

  • Houston Methodist (Surgical)

    ACTIVE_NOT_RECRUITING

    Houston, Texas, 77030, United States

  • Loma Linda University (Neurology)

    RECRUITING

    Loma Linda, California, 92354, United States

    Contact Email: •••••@•••••

  • Loma Linda University (Surgical)

    ACTIVE_NOT_RECRUITING

    Loma Linda, California, 92354, United States

  • Massachusetts General Hospital (Neurology)

    RECRUITING

    Boston, Massachusetts, 02114, United States

    Contact Email: •••••@•••••

  • Massachusetts General Hospital (Surgical)

    ACTIVE_NOT_RECRUITING

    Boston, Massachusetts, 02114, United States

  • Mayo Clinic

    NOT_YET_RECRUITING

    Phoenix, Arizona, 85054, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Mazowiecki Szpital Bródnowski (Neurology)

    RECRUITING

    Warsaw, Poland

    Contact Email: •••••@•••••

  • Mazowiecki Szpital Bródnowski (Surgical)

    ACTIVE_NOT_RECRUITING

    Warsaw, Poland

  • Michigan State University (Neurology)

    RECRUITING

    East Lansing, Michigan, 48824, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • North Bristol NHS Trust - Southmead Hospital (Neurology)

    RECRUITING

    Bristol, United Kingdom

    Contact Email: •••••@•••••

  • Ohio State University (Surgical)

    ACTIVE_NOT_RECRUITING

    Columbus, Ohio, 43210, United States

  • Orlando Health Neuroscience Institute

    RECRUITING

    Orlando, Florida, 32806, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

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

    RECRUITING

    Port Charlotte, Florida, 33980, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

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

    RECRUITING

    Boca Raton, Florida, 33486, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Philipps-Universität Marburg (Neurology)

    RECRUITING

    Marburg, Germany

    Contact Email: •••••@•••••

  • Quest Research Institute (Neurology)

    RECRUITING

    Farmington Hills, Michigan, 48334, United States

    Contact Email: •••••@•••••

  • Rush University Medical Center (Neurology)

    RECRUITING

    Chicago, Illinois, 60612, United States

    Contact Phone: •••-•••-••••

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

    RECRUITING

    Warsaw, Poland

    Contact Email: •••••@•••••

  • Specjalistyczna Praktyka Lekarska Prof. Grzegorz Opala (Neurology)

    RECRUITING

    Katowice, Poland

    Contact Email: •••••@•••••

  • Texas Movement Disorder Specialists

    NOT_YET_RECRUITING

    Georgetown, Texas, 78628, United States

    Contact Email: •••••@•••••

  • UCI Alpha Clinic (Neurology)

    RECRUITING

    Irvine, California, 92697, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • University Hospital of Wales (Surgical)

    ACTIVE_NOT_RECRUITING

    Cardiff, United Kingdom

  • University of California San Francisco (Neurology)

    RECRUITING

    San Francisco, California, 94143, United States

    Contact Email: •••••@•••••

  • University of Florida

    NOT_YET_RECRUITING

    Gainesville, Florida, 32608, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • University of Kansas Medical Center (Neurology)

    RECRUITING

    Kansas City, Kansas, 66103, United States

    Contact Email: •••••@•••••

  • University of Miami

    NOT_YET_RECRUITING

    Miami, Florida, 33125, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • University of North Carolina (Neurology)

    RECRUITING

    Chapel Hill, North Carolina, 27514, United States

    Contact Email: •••••@•••••

  • University of Pennsylvania (Neurology)

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • University of San Francisco (Surgical)

    ACTIVE_NOT_RECRUITING

    San Francisco, California, 94143, United States

  • University of Utah

    NOT_YET_RECRUITING

    Salt Lake City, Utah, 84132, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • University of Wisconsin (Neurology)

    RECRUITING

    Madison, Wisconsin, 53705, United States

    Contact Email: •••••@•••••

  • Universitätsklinikum Tübingen (Neurology)

    RECRUITING

    Tübingen, Germany

    Contact Email: •••••@•••••

  • Universitätsklinikum Tübingen (Surgical)

    ACTIVE_NOT_RECRUITING

    Tübingen, Germany

  • Universitätsklinikum Würzburg (Neurology)

    RECRUITING

    Würzburg, Germany

    Contact Email: •••••@•••••

  • Vanderbilt University (Neurology)

    RECRUITING

    Nashville, Tennessee, 37232, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

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