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

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
127

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • 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

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
AskBio Inc

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
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)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
June 11, 2024

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
August 31, 2028

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
August 31, 2028

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
February 14, 2024

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 23, 2024

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
February 29, 2024

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
December 8, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 2, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
April 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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