NCT06285643

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.

Trial Health

80
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
127

participants targeted

Target at P75+ for phase_2 parkinson-disease

Timeline
28mo left

Started Jun 2024

Longer than P75 for phase_2 parkinson-disease

Geographic Reach
4 countries

39 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress45%
Jun 2024Aug 2028

First Submitted

Initial submission to the registry

February 14, 2024

Completed
15 days until next milestone

First Posted

Study publicly available on registry

February 29, 2024

Completed
3 months until next milestone

Study Start

First participant enrolled

June 11, 2024

Completed
4.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 31, 2028

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 31, 2028

Last Updated

December 8, 2025

Status Verified

April 1, 2025

Enrollment Period

4.2 years

First QC Date

February 14, 2024

Last Update Submit

December 2, 2025

Conditions

Keywords

Gene therapy

Outcome Measures

Primary Outcomes (1)

  • Change from baseline to Month 18 on PD Motor Diary

    Normalized Good ON time per Hauser Diary

    18 months

Study Arms (2)

AAV2-GDNF

EXPERIMENTAL
Drug: AAV2-GDNF gene therapy

Control Surgery

SHAM COMPARATOR
Procedure: control surgery

Interventions

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

Also known as: Biological
AAV2-GDNF

Bilateral partial burr/twist holes without dural penetration

Control Surgery

Eligibility Criteria

Age45 Years - 75 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (39)

UCI Alpha Clinic (Neurology)

Irvine, California, 92697, United States

RECRUITING

Loma Linda University (Neurology)

Loma Linda, California, 92354, United States

RECRUITING

Loma Linda University (Surgical)

Loma Linda, California, 92354, United States

ACTIVE NOT RECRUITING

University of California San Francisco (Neurology)

San Francisco, California, 94143, United States

RECRUITING

University of San Francisco (Surgical)

San Francisco, California, 94143, United States

ACTIVE NOT RECRUITING

CenExel Rocky Mountain Clinical Research (Neurology)

Englewood, Colorado, 80113, United States

RECRUITING

Georgetown University (Neurology)

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

RECRUITING

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

Boca Raton, Florida, 33486, United States

RECRUITING

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

Port Charlotte, Florida, 33980, United States

ACTIVE NOT RECRUITING

Emory University (Surgical)

Atlanta, Georgia, 30322, United States

ACTIVE NOT RECRUITING

Rush University Medical Center (Neurology)

Chicago, Illinois, 60612, United States

RECRUITING

University of Kansas Medical Center (Neurology)

Kansas City, Kansas, 66103, United States

RECRUITING

Massachusetts General Hospital (Neurology)

Boston, Massachusetts, 02114, United States

RECRUITING

Massachusetts General Hospital (Surgical)

Boston, Massachusetts, 02114, United States

ACTIVE NOT RECRUITING

Michigan State University (Neurology)

East Lansing, Michigan, 48824, United States

RECRUITING

Quest Research Institute (Neurology)

Farmington Hills, Michigan, 48334, United States

RECRUITING

Cleveland Clinic Lou Ruvo (Neurology)

Las Vegas, Nevada, 89106, United States

RECRUITING

University of North Carolina (Neurology)

Chapel Hill, North Carolina, 27514, United States

RECRUITING

Duke Neurosurgery (Surgical)

Durham, North Carolina, 27705, United States

ACTIVE NOT RECRUITING

Duke University (Neurology)

Durham, North Carolina, 27710, United States

RECRUITING

Ohio State University (Surgical)

Columbus, Ohio, 43210, United States

ACTIVE NOT RECRUITING

University of Pennsylvania (Neurology)

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

Vanderbilt University (Neurology)

Nashville, Tennessee, 37232, United States

RECRUITING

Houston Methodist (Neurology)

Houston, Texas, 77030, United States

RECRUITING

Houston Methodist (Surgical)

Houston, Texas, 77030, United States

ACTIVE NOT RECRUITING

EvergreenHealth Research Department (Neurology)

Kirkland, Washington, 98034, United States

RECRUITING

University of Wisconsin (Neurology)

Madison, Wisconsin, 53705, United States

RECRUITING

Charité - Universitätsmedizin Berlin (Surgical)

Berlin, Germany

ACTIVE NOT RECRUITING

Philipps-Universität Marburg (Neurology)

Marburg, Germany

RECRUITING

Universitätsklinikum Tübingen (Neurology)

Tübingen, Germany

RECRUITING

Universitätsklinikum Tübingen (Surgical)

Tübingen, Germany

ACTIVE NOT RECRUITING

Universitätsklinikum Würzburg (Neurology)

Würzburg, Germany

RECRUITING

Copernicus Podmiot Leczniczy, Szpital św. Wojciecha (Neurology)

Gdansk, Poland

RECRUITING

Specjalistyczna Praktyka Lekarska Prof. Grzegorz Opala (Neurology)

Katowice, Poland

RECRUITING

Mazowiecki Szpital Bródnowski (Neurology)

Warsaw, Poland

RECRUITING

Mazowiecki Szpital Bródnowski (Surgical)

Warsaw, Poland

ACTIVE NOT RECRUITING

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

Warsaw, Poland

RECRUITING

North Bristol NHS Trust - Southmead Hospital (Neurology)

Bristol, United Kingdom

RECRUITING

University Hospital of Wales (Surgical)

Cardiff, United Kingdom

ACTIVE NOT RECRUITING

Related Links

MeSH Terms

Conditions

Parkinson Disease

Interventions

Biological Products

Condition Hierarchy (Ancestors)

Parkinsonian DisordersBasal Ganglia DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesMovement DisordersSynucleinopathiesNeurodegenerative Diseases

Intervention Hierarchy (Ancestors)

Complex Mixtures

Central Study Contacts

Nisha Chhabria, MD

CONTACT

Amber Van Laar, MD

CONTACT

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 14, 2024

First Posted

February 29, 2024

Study Start

June 11, 2024

Primary Completion (Estimated)

August 31, 2028

Study Completion (Estimated)

August 31, 2028

Last Updated

December 8, 2025

Record last verified: 2025-04

Locations