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AAV2-GDNF for Advanced Parkinson s Disease

NCT01621581 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 25

Last updated 2026-04-14

No results posted yet for this study

Summary

Background:

\- Glial cell line-derived neurotrophic factor (GDNF) is a chemical that may help protect and strengthen brain cells that produce dopamine. Dopamine is a chemical that affects brain function. People with Parkinson's disease (PD) have problems producing dopamine in the brain. Researchers want to see if gene transfer can help deliver GDNF into the area of the brain that is damaged by PD. The gene transferred in this study, called AAV2-GDNF, may help produce GDNF to protect the damaged brain cells.

Objectives:

\- To test the safety and effectiveness of AAV2-GDNF gene transfer for advanced PD.

Eligibility:

\- Individuals at least 18 years of age who have advanced PD that is not well controlled by medications.

Design:

* Participants will be in the study for about 5 years. There will be 18 outpatient study visits and a 3-day stay in the hospital. There may also be overnight stays for followup visits.
* Participants will be screened with a physical exam and medical history. Blood samples will be collected. Tests of PD symptoms and mood and memory will be given. Imaging studies will be used to find the right part of the brain to infuse the gene. The screening visit will take place up to 60 days before surgery.
* Participants will have a baseline visit about a month before the surgery. For 1 week before the baseline visit, participants will keep a diary on any motor problems. The visit will involve movement tests given before and after taking a regular dose of levodopa.
* Participants will have surgery to infuse AAV2-GDNF into the brain. The surgery will also include a lumbar puncture (spinal tap) to collect cerebrospinal fluid. After surgery, participants will recover in the hospital for at least 2 days.
* Participants will have another lumbar puncture 6 and 18 months after surgery. This will be an outpatient visit.
* Participants will have regular followup visits after the surgery. These visits will include neurological tests and movement studies. Visits with a neurosurgeon will take place 1, 2, and 4 weeks after surgery. Additional visits will take place every 3 months for the first 3 years, and then at longer intervals for up to 5 years.

Conditions

Interventions

GENETIC

Convection enhanced delivery/AAV2-GDNF

Adeno-Associated Virus Encoding Glial Cell Line-Derived Neurotrophic Factor (AAV2-GDNF) Administered via Bilateral Stereotactic Convection-Enhanced Delivery

Sponsors & Collaborators

  • National Institute of Neurological Disorders and Stroke (NINDS)

    lead NIH

Principal Investigators

  • John D Heiss, M.D. · National Institute of Neurological Disorders and Stroke (NINDS)

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2013-03-13
Primary Completion
2022-02-04
Completion
2022-02-04
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01621581 on ClinicalTrials.gov