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

NCT ID: NCT06285643

Last Updated: 2025-11-14

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

87 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-06-11

Study Completion Date

2027-11-30

Brief Summary

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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.

Detailed Description

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Conditions

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Parkinson Disease

Keywords

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Gene therapy

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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AAV2-GDNF

Group Type EXPERIMENTAL

AAV2-GDNF Gene therapy

Intervention Type DRUG

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

Control Surgery

Group Type SHAM_COMPARATOR

Control Surgery

Intervention Type PROCEDURE

Bilateral partial burr/twist holes without dural penetration

Interventions

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AAV2-GDNF Gene therapy

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

Intervention Type DRUG

Control Surgery

Bilateral partial burr/twist holes without dural penetration

Intervention Type PROCEDURE

Other Intervention Names

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Biological

Eligibility Criteria

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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 regiment 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
Minimum Eligible Age

45 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Bayer

INDUSTRY

Sponsor Role collaborator

AskBio Inc

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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UCI Alpha Clinic (Neurology)

Irvine, California, United States

Site Status RECRUITING

Loma Linda University (Neurology)

Loma Linda, California, United States

Site Status RECRUITING

Loma Linda University (Surgical)

Loma Linda, California, United States

Site Status ACTIVE_NOT_RECRUITING

University of California San Francisco (Neurology)

San Francisco, California, United States

Site Status RECRUITING

University of San Francisco (Surgical)

San Francisco, California, United States

Site Status ACTIVE_NOT_RECRUITING

CenExel Rocky Mountain Clinical Research (Neurology)

Englewood, Colorado, United States

Site Status RECRUITING

Georgetown University (Neurology)

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

Site Status RECRUITING

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

Boca Raton, Florida, United States

Site Status RECRUITING

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

Port Charlotte, Florida, United States

Site Status ACTIVE_NOT_RECRUITING

Emory University (Surgical)

Atlanta, Georgia, United States

Site Status ACTIVE_NOT_RECRUITING

Rush University Medical Center (Neurology)

Chicago, Illinois, United States

Site Status RECRUITING

University of Kansas Medical Center (Neurology)

Kansas City, Kansas, United States

Site Status RECRUITING

Massachusetts General Hospital (Neurology)

Boston, Massachusetts, United States

Site Status RECRUITING

Massachusetts General Hospital (Surgical)

Boston, Massachusetts, United States

Site Status ACTIVE_NOT_RECRUITING

Michigan State University (Neurology)

East Lansing, Michigan, United States

Site Status RECRUITING

Quest Research Institute (Neurology)

Farmington Hills, Michigan, United States

Site Status RECRUITING

Cleveland Clinic Lou Ruvo (Neurology)

Las Vegas, Nevada, United States

Site Status RECRUITING

University of North Carolina (Neurology)

Chapel Hill, North Carolina, United States

Site Status RECRUITING

Duke Neurosurgery (Surgical)

Durham, North Carolina, United States

Site Status ACTIVE_NOT_RECRUITING

Duke University (Neurology)

Durham, North Carolina, United States

Site Status RECRUITING

Ohio State University (Surgical)

Columbus, Ohio, United States

Site Status ACTIVE_NOT_RECRUITING

University of Pennsylvania (Neurology)

Philadelphia, Pennsylvania, United States

Site Status RECRUITING

Vanderbilt University (Neurology)

Nashville, Tennessee, United States

Site Status RECRUITING

Houston Methodist (Neurology)

Houston, Texas, United States

Site Status RECRUITING

Houston Methodist (Surgical)

Houston, Texas, United States

Site Status ACTIVE_NOT_RECRUITING

EvergreenHealth Research Department (Neurology)

Kirkland, Washington, United States

Site Status RECRUITING

University of Wisconsin (Neurology)

Madison, Wisconsin, United States

Site Status RECRUITING

Charité - Universitätsmedizin Berlin (Surgical)

Berlin, , Germany

Site Status ACTIVE_NOT_RECRUITING

Philipps-Universität Marburg (Neurology)

Marburg, , Germany

Site Status RECRUITING

Universitätsklinikum Tübingen (Neurology)

Tübingen, , Germany

Site Status RECRUITING

Universitätsklinikum Tübingen (Surgical)

Tübingen, , Germany

Site Status ACTIVE_NOT_RECRUITING

Universitätsklinikum Würzburg (Neurology)

Würzburg, , Germany

Site Status RECRUITING

Copernicus Podmiot Leczniczy, Szpital św. Wojciecha (Neurology)

Gdansk, , Poland

Site Status RECRUITING

Specjalistyczna Praktyka Lekarska Prof. Grzegorz Opala (Neurology)

Katowice, , Poland

Site Status RECRUITING

Mazowiecki Szpital Bródnowski (Neurology)

Warsaw, , Poland

Site Status RECRUITING

Mazowiecki Szpital Bródnowski (Surgical)

Warsaw, , Poland

Site Status ACTIVE_NOT_RECRUITING

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

Warsaw, , Poland

Site Status RECRUITING

North Bristol NHS Trust - Southmead Hospital (Neurology)

Bristol, , United Kingdom

Site Status RECRUITING

University Hospital of Wales (Surgical)

Cardiff, , United Kingdom

Site Status ACTIVE_NOT_RECRUITING

Countries

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United States Germany Poland United Kingdom

Central Contacts

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Nisha Chhabria, MD

Role: CONTACT

Phone: 919-388-1040

Email: [email protected]

Ellie Hu, MD

Role: CONTACT

Phone: 919-388-1040

Email: [email protected]

Facility Contacts

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Role: primary

Julie Lacanlale

Role: primary

Aaron Daley

Role: primary

Role: primary

Luke Lovelace

Role: primary

Lauren Salmon

Role: primary

Neepa J. Patel, MD

Role: primary

Sarina Fay

Role: primary

Nisha Kabir

Role: primary

Leigh Whittet

Role: primary

Denise Morgott

Role: primary

Liliana Dumitrescu

Role: primary

Hans Nettesheim

Role: primary

Karen White-Tong

Role: primary

Neda Almassi

Role: primary

Haley Kling

Role: primary

Daniel Arnaud Dominguez

Role: primary

Gowri Rajendran

Role: primary

Amy Nagel

Role: primary

Stefanie Spriewald

Role: primary

Gabriele König

Role: primary

Christine Leypold

Role: primary

Anna Roszmann

Role: primary

Magdalena Łyniewska

Role: primary

Joanna Kralka

Role: primary

Marta Piaścik-Gromada

Role: primary

Dominika Kruszynska

Role: primary

Related Links

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https://mytomorrows.com/ask-bio/regenerate-pd/en-us/

Nurse Navigator Service for Potential Participants

Other Identifiers

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ASK-PD5-CS201

Identifier Type: -

Identifier Source: org_study_id