AAV2-GDNF: Bilateral image-guided infusion of AAV2-GDNF into putamen, single dose
Study 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.
Eligibility
Sex
ALL
Min age
35 Years
Max age
75 Years
Healthy volunteers
No
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:
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
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
Primary outcome measure(s)
The incidence of Treatment-Emergent Adverse Events (TEAE) assessed clinically by physical and neurological examinations — 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.
Trial sites (3)
Facility
City
Region
Status
University of California Irvine
Irvine
California
University of California San Francisco
San Francisco
California
The Ohio State University Medical Center
Columbus
Ohio
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This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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