RecruitingPhase 2NCT06285643

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


Sponsor

AskBio Inc

Enrollment

127 participants

Start Date

Jun 11, 2024

Study Type

INTERVENTIONAL

Conditions

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

Min Age: 45 YearsMax Age: 75 Years

Inclusion Criteria10

  • 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

Exclusion Criteria9

  • 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

Interventions

DRUGAAV2-GDNF gene therapy

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

PROCEDUREcontrol surgery

Bilateral partial burr/twist holes without dural penetration


Locations(39)

UCI Alpha Clinic (Neurology)

Irvine, California, United States

Loma Linda University (Neurology)

Loma Linda, California, United States

Loma Linda University (Surgical)

Loma Linda, California, United States

University of California San Francisco (Neurology)

San Francisco, California, United States

University of San Francisco (Surgical)

San Francisco, California, United States

CenExel Rocky Mountain Clinical Research (Neurology)

Englewood, Colorado, United States

Georgetown University (Neurology)

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

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

Boca Raton, Florida, United States

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

Port Charlotte, Florida, United States

Emory University (Surgical)

Atlanta, Georgia, United States

Rush University Medical Center (Neurology)

Chicago, Illinois, United States

University of Kansas Medical Center (Neurology)

Kansas City, Kansas, United States

Massachusetts General Hospital (Neurology)

Boston, Massachusetts, United States

Massachusetts General Hospital (Surgical)

Boston, Massachusetts, United States

Michigan State University (Neurology)

East Lansing, Michigan, United States

Quest Research Institute (Neurology)

Farmington Hills, Michigan, United States

Cleveland Clinic Lou Ruvo (Neurology)

Las Vegas, Nevada, United States

University of North Carolina (Neurology)

Chapel Hill, North Carolina, United States

Duke Neurosurgery (Surgical)

Durham, North Carolina, United States

Duke University (Neurology)

Durham, North Carolina, United States

Ohio State University (Surgical)

Columbus, Ohio, United States

University of Pennsylvania (Neurology)

Philadelphia, Pennsylvania, United States

Vanderbilt University (Neurology)

Nashville, Tennessee, United States

Houston Methodist (Neurology)

Houston, Texas, United States

Houston Methodist (Surgical)

Houston, Texas, United States

EvergreenHealth Research Department (Neurology)

Kirkland, Washington, United States

University of Wisconsin (Neurology)

Madison, Wisconsin, 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

View Full Details on ClinicalTrials.gov

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NCT06285643


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