- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04167540
GDNF Gene Therapy for Parkinson's Disease
January 18, 2023 updated by: Brain Neurotherapy Bio, Inc.
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.
Study Overview
Status
Active, not recruiting
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Actual)
11
Phase
- Phase 1
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Amber Van Laar, MD
- Email: avanlaar@brainneubio.com
Study Locations
-
-
California
-
Irvine, California, United States, 92697
- University of California Irvine
-
San Francisco, California, United States, 94103
- University of California San Francisco
-
-
Ohio
-
Columbus, Ohio, United States, 43210
- The Ohio State University Medical Center
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
35 years to 75 years (Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Key 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:
- 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
Key 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
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Non-Randomized
- Interventional Model: Parallel Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Earlier stage PD
|
Bilateral image-guided infusion of AAV2-GDNF into putamen, single dose
|
Experimental: Later stage PD
|
Bilateral image-guided infusion of AAV2-GDNF into putamen, single dose
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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.
|
5 years
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Motor symptoms as assessed by the Movement Disorder Society's Unified Parkinson's Disease Rating Scale (MDS-UPDRS)
Time Frame: 18 months
|
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.
|
18 months
|
Non-motor symptoms of Parkinson's disease as assessed by the Non-Motor Symptom Scale (NMSS)
Time Frame: 18 months
|
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.
|
18 months
|
Brain dopaminergic cell integrity as measured by DaTscan
Time Frame: 18 months
|
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.
|
18 months
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
April 1, 2020
Primary Completion (Anticipated)
December 1, 2023
Study Completion (Anticipated)
June 1, 2027
Study Registration Dates
First Submitted
November 7, 2019
First Submitted That Met QC Criteria
November 14, 2019
First Posted (Actual)
November 19, 2019
Study Record Updates
Last Update Posted (Actual)
January 19, 2023
Last Update Submitted That Met QC Criteria
January 18, 2023
Last Verified
January 1, 2023
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- GDNF-102
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
No
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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