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Active, not recruiting Phase 1

GDNF Gene Therapy for Parkinson's Disease

NCT04167540 · tracked via the Priya Life Science USA tracker
Sponsor
Brain Neurotherapy Bio, Inc
Phase
Phase 1
Started
2020-04-01
Last updated
2025-09-19

Condition(s) studied

Parkinson's Disease

Investigational drug(s) / intervention(s)

AAV2-GDNF

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)

FacilityCityRegionStatus
University of California Irvine Irvine California
University of California San Francisco San Francisco California
The Ohio State University Medical Center Columbus Ohio

More Brain Neurotherapy Bio, Inc. trials in the USA

Official registry record

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.

View NCT04167540 on ClinicalTrials.gov ↗ ← All trials in the USA