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

December 2, 2025 updated by: AskBio Inc

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

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

127

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • Germany
      • Berlin, Germany
        • Active, not recruiting
        • Charité - Universitätsmedizin Berlin (Surgical)
      • Marburg, Germany
      • Tübingen, Germany
      • Tübingen, Germany
        • Active, not recruiting
        • Universitätsklinikum Tübingen (Surgical)
      • Würzburg, Germany
        • Recruiting
        • Universitätsklinikum Würzburg (Neurology)
        • Contact:
  • Poland
      • Gdansk, Poland
        • Recruiting
        • Copernicus Podmiot Leczniczy, Szpital św. Wojciecha (Neurology)
        • Contact:
      • Katowice, Poland
        • Recruiting
        • Specjalistyczna Praktyka Lekarska Prof. Grzegorz Opala (Neurology)
        • Contact:
      • Warsaw, Poland
        • Active, not recruiting
        • Mazowiecki Szpital Bródnowski (Surgical)
      • Warsaw, Poland
      • Warsaw, Poland
  • United Kingdom
      • Bristol, United Kingdom
      • Cardiff, United Kingdom
        • Active, not recruiting
        • University Hospital of Wales (Surgical)
  • United States
    • California
      • Irvine, California, United States, 92697
        • Recruiting
        • UCI Alpha Clinic (Neurology)
        • Contact:
      • Loma Linda, California, United States, 92354
        • Recruiting
        • Loma Linda University (Neurology)
        • Contact:
      • Loma Linda, California, United States, 92354
        • Active, not recruiting
        • Loma Linda University (Surgical)
      • San Francisco, California, United States, 94143
        • Recruiting
        • University of California San Francisco (Neurology)
        • Contact:
      • San Francisco, California, United States, 94143
        • Active, not recruiting
        • University of San Francisco (Surgical)
    • Colorado
      • Englewood, Colorado, United States, 80113
        • Recruiting
        • CenExel Rocky Mountain Clinical Research (Neurology)
        • Contact:
          • Phone Number: 720-776-0092
    • District of Columbia
      • Washington D.C., District of Columbia, United States, 20057
    • Florida
      • Boca Raton, Florida, United States, 33486
        • Recruiting
        • Parkinson's Disease and Movement Disorders Center of Boca Raton (Neurology)
        • Contact:
      • Port Charlotte, Florida, United States, 33980
        • Active, not recruiting
        • Parkinson's Disease Treatment Center of Southwest Florida (Neurology)
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Active, not recruiting
        • Emory University (Surgical)
    • Illinois
      • Chicago, Illinois, United States, 60612
        • Recruiting
        • Rush University Medical Center (Neurology)
        • Contact:
          • Neepa J. Patel, MD
          • Phone Number: 312-563-2900
    • Kansas
      • Kansas City, Kansas, United States, 66103
        • Recruiting
        • University of Kansas Medical Center (Neurology)
        • Contact:
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Active, not recruiting
        • Massachusetts General Hospital (Surgical)
      • Boston, Massachusetts, United States, 02114
    • Michigan
      • East Lansing, Michigan, United States, 48824
        • Recruiting
        • Michigan State University (Neurology)
        • Contact:
      • Farmington Hills, Michigan, United States, 48334
    • Nevada
      • Las Vegas, Nevada, United States, 89106
        • Recruiting
        • Cleveland Clinic Lou Ruvo (Neurology)
        • Contact:
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27514
      • Durham, North Carolina, United States, 27705
        • Active, not recruiting
        • Duke Neurosurgery (Surgical)
      • Durham, North Carolina, United States, 27710
        • Recruiting
        • Duke University (Neurology)
        • Contact:
    • Ohio
      • Columbus, Ohio, United States, 43210
        • Active, not recruiting
        • Ohio State University (Surgical)
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
    • Tennessee
      • Nashville, Tennessee, United States, 37232
        • Recruiting
        • Vanderbilt University (Neurology)
        • Contact:
    • Texas
      • Houston, Texas, United States, 77030
        • Active, not recruiting
        • Houston Methodist (Surgical)
      • Houston, Texas, United States, 77030
    • Washington
      • Kirkland, Washington, United States, 98034
    • Wisconsin
      • Madison, Wisconsin, United States, 53705

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: AAV2-GDNF
Drug: AAV2-GDNF gene therapy
Bilateral image-guided infusion of AAV2-GDNF into putamen, single dose
Other Names:
  • Biological
Sham Comparator: Control Surgery
Procedure: control surgery
Bilateral partial burr/twist holes without dural penetration

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change from baseline to Month 18 on PD Motor Diary
Time Frame: 18 months
Normalized Good ON time per Hauser Diary
18 months

Secondary Outcome Measures

Outcome Measure
Time Frame
Change from baseline in the MDS-UPDRS parts 2 and 3, and the PD Motor Diary
Time Frame: 18 months
18 months
Change from baseline in the MDS-UPDRS part 1 and PDQ-39
Time Frame: 18 months
18 months
Change in Levodopa equivalent daily dose
Time Frame: 18 months
18 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

AskBio Inc

Collaborators

Bayer

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 11, 2024

Primary Completion (Estimated)

August 31, 2028

Study Completion (Estimated)

August 31, 2028

Study Registration Dates

First Submitted

February 14, 2024

First Submitted That Met QC Criteria

February 23, 2024

First Posted (Actual)

February 29, 2024

Study Record Updates

Last Update Posted (Actual)

December 8, 2025

Last Update Submitted That Met QC Criteria

December 2, 2025

Last Verified

April 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ASK-PD5-CS201

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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