NCT04167540Active, not recruitingPhase 1
GDNF Gene Therapy for Parkinson's Disease
Open-Label Safety Study of Glial Cell Line-Derived Neurotrophic Factor Gene Transfer (AAV2- GDNF) in Parkinson's Disease
Source: ClinicalTrials.gov · Last updated 19 Sept 2025
Brief summary (registry, in English)
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.
- Status (registry)
- Active, not recruiting
- Phase (registry)
- Phase 1
- Study type
- Interventional
- Enrollment
- 11 (actual)
- First submitted
- 7 Nov 2019
- Start date
- 1 Apr 2020
- Primary completion date
- 17 Oct 2023
- Lead sponsor
- Brain Neurotherapy Bio, Inc.
- Diseases
- Parkinson disease
- NCT number
- NCT04167540
What you commit to
- Study type: Interventional · Phase 1
- Participants enrolled: 11
- From 1 Apr 2020 to the primary completion date, 17 Oct 2023
- 3 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Prior brain surgery (i.e. deep brain stimulator or DBS implantation) or other brain imaging abnormalities
- Receiving an investigational drug
Other illness
- Presence of dementia, psychosis, substance abuse or qualify as "severe depression"
- 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
Other
- Atypical parkinsonism
- Severe dyskinesia
Who the study is looking for
- 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:
- EITHER: Less than 5 years since clinical diagnosis of PD and mild to moderate UPDRS III OFF score
- OR: At least 4 years since clinical diagnosis of PD and moderate to severe UPDRS III OFF score
- Responsiveness to levodopa
Age in the registry: 35 years to 75 years · Sex: All · Does not accept healthy volunteers
Sites (3)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| The Ohio State University Medical Center Coordinator of this site? Confirm its status | Columbus | United States | Unknown status |
| University of California, Irvine Coordinator of this site? Confirm its status | Irvine | United States | Unknown status |
| University of California San Francisco Coordinator of this site? Confirm its status | San Francisco | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.