GDNF Gene Therapy for Parkinson's Disease

NCT ID: NCT04167540

Last Updated: 2025-09-19

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

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE1

Total Enrollment

11 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-04-01

Study Completion Date

2027-06-30

Brief Summary

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The objective of this Phase 1b investigation is to evaluate the safety and potential clinical effect of AAV2-GDNF delivered to the putamen in subjects with either a recent or a long-standing diagnosis of PD.

Detailed Description

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Conditions

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Parkinson's Disease

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

A total of 12 study participants will be administered the investigational product in this Phase 1b trial. Participants will be enrolled into cohorts, based upon the duration and stage of their PD. Six (6) participants will be dosed in each cohort.
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Earlier stage PD

Group Type EXPERIMENTAL

AAV2-GDNF

Intervention Type BIOLOGICAL

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

Later stage PD

Group Type EXPERIMENTAL

AAV2-GDNF

Intervention Type BIOLOGICAL

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

Interventions

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AAV2-GDNF

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

Intervention Type BIOLOGICAL

Eligibility Criteria

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Inclusion Criteria

* Male and female adults 35-75 years of age (inclusive)
* Diagnosed with Parkinson's disease
* Modified Hoehn and Yahr stage I-III OFF medication
* Time since receiving a clinical diagnosis of PD and disease severity consistent with one of the following:

1. EITHER: Less than 5 years since clinical diagnosis of PD and mild to moderate UPDRS III OFF score
2. OR: At least 4 years since clinical diagnosis of PD and moderate to severe UPDRS III OFF score
* Responsiveness to levodopa

Exclusion Criteria

* Atypical parkinsonism
* Severe dyskinesia
* Presence of dementia, psychosis, substance abuse or qualify as "severe depression"
* Prior brain surgery (i.e. deep brain stimulator or DBS implantation) or other brain imaging abnormalities
* Receiving an investigational drug
* History of cancer or poorly controlled medical conditions that would increase surgical risk
* Inability to tolerate laying flat in an MRI or allergy to gadolinium
Minimum Eligible Age

35 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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AskBio Inc

INDUSTRY

Sponsor Role collaborator

California Institute for Regenerative Medicine (CIRM)

OTHER

Sponsor Role collaborator

Brain Neurotherapy Bio, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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University of California Irvine

Irvine, California, United States

Site Status

University of California San Francisco

San Francisco, California, United States

Site Status

The Ohio State University Medical Center

Columbus, Ohio, United States

Site Status

Countries

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United States

Other Identifiers

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GDNF-102

Identifier Type: -

Identifier Source: org_study_id

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