Recruiting

Phase 2

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

Sponsor:

AskBio Inc

Code:

NCT06285643

Conditions

Parkinson Disease

Eligibility Criteria

Sex: All

Age: 45 - 70+

Healthy Volunteers: Not accepted

Interventions

AAV2-GDNF gene therapy

control surgery

Study Details

Brief 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.

Conditions

Parkinson Disease

Study ID

NCT06285643

Start date

Jun 11, 2024

Status verified date

Jun, 2026

Completion date

Aug 31, 2028

Anticipated

Primary completion date

Aug 31, 2028

Anticipated

Eligibility Criteria

Sex: All

Age: 45 - 70+

Healthy Volunteers: Not accepted

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 Design

Enrollment

127 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: AAV2-GDNF

sham comparator: Control Surgery

Interventions

AAV2-GDNF gene therapy

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

control surgery

Bilateral partial burr/twist holes without dural penetration

Primary outcome measure

Change from baseline to Month 18 on PD Motor Diary [ Time Frame: 18 months ]

Central Contacts and Locations

Central contacts

Nisha Chhabria, MD

919-388-1040 askfirst@askbio.com

Gayathri Palety, MD

919-388-1040 askfirst@askbio.com

Locations

UCI Alpha Clinic (Neurology)

Recruiting

Irvine, California, United States, 92697

Contacts

949-824-3990 alphaclinic@uci.edu

Loma Linda University (Neurology)

Recruiting

Loma Linda, California, United States, 92354

Contacts

Julie Lacanlale

jlacanlale@llu.edu

University of California San Francisco (Neurology)

Recruiting

San Francisco, California, United States, 94143

Contacts

Aaron Daley

Aaron.Daley@ucsf.edu

CenExel Rocky Mountain Clinical Research (Neurology)

Recruiting

Englewood, Colorado, United States, 80113

Contacts

720-776-0092

Georgetown University (Neurology)

Recruiting

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

Contacts

Luke Lovelace

LL928@georgetown.edu

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

Recruiting

Boca Raton, Florida, United States, 33486

Contacts

Lauren Salmon

561-392-1818 lsalmon@parkinsonscenter.org

Orlando Health Neuroscience Institute

Recruiting

Orlando, Florida, United States, 32806

Contacts

Diana Mondragon

321-841-1324 Diana.Mondragon@orlandohealth.com

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

Recruiting

Port Charlotte, Florida, United States, 33980

Contacts

Brianna Krause

941-743-4987 brianna.krause@igrsus.com

Rush University Medical Center (Neurology)

Recruiting

Chicago, Illinois, United States, 60612

Contacts

Neepa J. Patel, MD

312-563-2900

University of Kansas Medical Center (Neurology)

Recruiting

Kansas City, Kansas, United States, 66103

Contacts

Sarina Fay

sfay2@kumc.edu

Massachusetts General Hospital (Neurology)

Recruiting

Boston, Massachusetts, United States, 02114

Contacts

Nisha Kabir

nkabir@mgh.harvard.edu

Michigan State University (Neurology)

Recruiting

East Lansing, Michigan, United States, 48824

Contacts

Leigh Whittet

517-884-2275 kmetleig@msu.edu,

Quest Research Institute (Neurology)

Recruiting

Farmington Hills, Michigan, United States, 48334

Contacts

Denise Morgott

denise.morgott@questri.com

Cleveland Clinic Lou Ruvo (Neurology)

Recruiting

Las Vegas, Nevada, United States, 89106

Contacts

Liliana Dumitrescu

702-483-6016 DUMITRL@ccf.org

Columbia University

Recruiting

New York, New York, United States, 10032

Contacts

Natasha Desai

347-882-4315 nd2528@cumc.columbia.edu

University of North Carolina (Neurology)

Recruiting

Chapel Hill, North Carolina, United States, 27514

Contacts

Hans Nettesheim

Hans_Nettesheim@med.unc.edu

Duke University (Neurology)

Recruiting

Durham, North Carolina, United States, 27710

Contacts

Karen White-Tong

919-668-2905 karen.white@duke.edu

University of Pennsylvania (Neurology)

Recruiting

Philadelphia, Pennsylvania, United States, 19104

Contacts

Neda Almassi

215-829-7289 neda.almassi@pennmedicine.upenn.edu

Vanderbilt University (Neurology)

Recruiting

Nashville, Tennessee, United States, 37232

Contacts

Haley Kling

615-208-3974 haley.kling@vumc.org

Houston Methodist (Neurology)

Recruiting

Houston, Texas, United States, 77030

Contacts

Daniel Arnaud Dominguez

darnauddominguez@houstonmethodist.org

EvergreenHealth Research Department (Neurology)

Recruiting

Kirkland, Washington, United States, 98034

Contacts

Gowri Rajendran

grajendran@evergreenhealthcare.org

University of Wisconsin (Neurology)

Recruiting

Madison, Wisconsin, United States, 53705

Contacts

Amy Nagel

anagel@neurology.wisc.edu

More Information

Sponsor

AskBio Inc

Last update posted

Jun 29, 2026

Last verified

Jun, 2026

Keywords

Gene therapy

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by AskBio Inc on 2026-06-29.