Parkinson's DiseasePhase 1NCT04167540
GDNF Gene Therapy for Parkinson's Disease
A Phase 1 study testing AAV2-GDNF in people with Parkinson's Disease, run by Brain Neurotherapy Bio, Inc. at 3 sites in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 35 to 75 years
- Conditions the study is for
- Parkinson's Disease
- What is being tested?
- AAV2-GDNF
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 11 participants
- Where
- 3 sitesUnited States
- Study dates
- 1 Apr 2020to Jun 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 35 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
3 sites in 1 country.
United States3 sites
The Ohio State University Medical Center
Columbus, Ohio, 43210
University of California Irvine
Irvine, California, 92697
University of California San Francisco
San Francisco, California, 94103
About this study
Official title: Open-Label Safety Study of Glial Cell Line-Derived Neurotrophic Factor Gene Transfer (AAV2- GDNF) in Parkinson's Disease
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.
- Conditions
- Parkinson's Disease
- Being tested
- AAV2-GDNF
- Keywords
- PD, Parkinson's disease, Neurotrophic factor, Growth factor, Glial cell line-derived neurotrophic factor, GDNF, AAV, Gene therapy
- Medical subject headings
- Parkinson Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AAV2-GDNFBiological
Bilateral image-guided infusion of AAV2-GDNF into putamen, single dose
| Study group | Role | What they receive |
|---|---|---|
| Earlier stage PD | Experimental | Not described Receives: Biological: AAV2-GDNF |
| Later stage PD | Experimental | Not described Receives: Biological: AAV2-GDNF |
What is being measured
The primary outcome is the main question the study is designed to answer.
The incidence of Treatment-Emergent Adverse Events (TEAE) assessed clinically by physical and neurological examinations
Evaluation of the safety and tolerability through the assessment of incidence of TEAE, identified by MedDRA preferred term and grouped by MedDRA System Organ Class, as well as clinically meaningful changes in clinical exams or laboratory assays.
Measured: 5 years
Secondary outcomes (3)
Motor symptoms as assessed by the Movement Disorder Society's Unified Parkinson's Disease Rating Scale (MDS-UPDRS)
Change from baseline in the MDS-UPDRS. The MDS-UPDRS contains 4 subscales: Part I, non-motor aspects of experiences of daily living (13 items); Part II, motor aspects of experiences of daily living (13 items); Part III, motor examination (33 scores based on 18 items); Part IV, motor complications (6 items). The rating for each item, or sub-item, is from 0 (normal) to 4 (severe). The total score for each Part is obtained from the sum of the corresponding item scores.
Measured: 18 months
Non-motor symptoms of Parkinson's disease as assessed by the Non-Motor Symptom Scale (NMSS)
Change from baseline in the NMSS. The NMSS evaluates 9 domains of non-motor of severity and frequency of PD symptoms associated with cardiovascular health, sleep and fatigue, mood and cognition, perceptual problems and hallucinations, attention and memory, gastrointestinal tract, urinary, sexual function, and a miscellaneous domain for other common non-motor conditions. Severity x frequency scores range 0-108, with 0 being less severe and less frequent.
Measured: 18 months
Brain dopaminergic cell integrity as measured by DaTscan
Percentage and absolute changes in Ioflupane retention as a marker for dopamine transporter protein expressed by dopamine producing cells within the brain. Measured by quantitative analysis of DaTscan SPECT imaging.
Measured: 18 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Nov 2019
- Study start1 Apr 2020
- Primary completion17 Oct 2023
- Study completionJun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Parkinson's DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Model description
- 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.
- Masking
- None (open label)
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- Brain Neurotherapy Bio, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- AskBio Inc, California Institute for Regenerative Medicine (CIRM)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04167540
- Sponsor's protocol ID
- GDNF-102
- Status last verified
- Sep 2025
- First submitted
- 7 Nov 2019
- Last updated
- 19 Sep 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open-Label Safety Study of Glial Cell Line-Derived Neurotrophic Factor Gene Transfer (AAV2- GDNF) in Parkinson's Disease
- ClinicalTrials.gov ID
- NCT04167540
- Acronym / sponsor ID
- GDNF-102
- Conditions
- Parkinson's Disease
- Keywords
- PD, Parkinson's disease, Neurotrophic factor, Growth factor, Glial cell line-derived neurotrophic factor, GDNF, AAV, Gene therapy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 11 participants (actual)
- Lead sponsor
- Brain Neurotherapy Bio, Inc.Industry (pharma, biotech, devices)
- Collaborators
- AskBio Inc, California Institute for Regenerative Medicine (CIRM)
- Study start
- 1 Apr 2020 (actual)
- Primary completion
- 17 Oct 2023 (actual)
- Study completion
- Jun 2027 (estimated)
- First posted
- 19 Nov 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- 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.
| Arm | Type | Receives |
|---|---|---|
| Earlier stage PD | Experimental | Biological: AAV2-GDNF |
| Later stage PD | Experimental | Biological: AAV2-GDNF |
Who may qualify
- Ages
- 35 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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:
- Responsiveness to levodopa
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AAV2-GDNFBilateral image-guided infusion of AAV2-GDNF into putamen, single dose | Biological | Earlier stage PD, Later stage PD |
Primary outcome
The incidence of Treatment-Emergent Adverse Events (TEAE) assessed clinically by physical and neurological examinations
Time frame: 5 years
Evaluation of the safety and tolerability through the assessment of incidence of TEAE, identified by MedDRA preferred term and grouped by MedDRA System Organ Class, as well as clinically meaningful changes in clinical exams or laboratory assays.
3 secondary outcomes
Motor symptoms as assessed by the Movement Disorder Society's Unified Parkinson's Disease Rating Scale (MDS-UPDRS)
Time frame: 18 months
Non-motor symptoms of Parkinson's disease as assessed by the Non-Motor Symptom Scale (NMSS)
Time frame: 18 months
Brain dopaminergic cell integrity as measured by DaTscan
Time frame: 18 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Sep 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing AAV2-GDNF in people with Parkinson's Disease, run by Brain Neurotherapy Bio, Inc. at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 19 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The incidence of Treatment-Emergent Adverse Events (TEAE) assessed clinically by physical and neurological examinations (time frame: 5 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Sep 2025.