Ireland
--:--IST
Latest
Astellas Expands Its 330 Million Euro Tralee Biopharma Facility with a Second Aseptic Filling Line to Double Drug-Product Capacity Xeolas Pharmaceuticals Opens 158,000 Sq Ft State-of-the-Art Baldoyle Facility to Scale Specialty Medicine Manufacturing Priya Life Science Partners with Fleming for the 9th Annual Corporate Compliance & Transparency in Life Sciences Summit in Zurich Ireland Has the Capital and the Lessons: Digital Project Management Is How They Become Delivery Dunbar Pharma Brings First Plant-Derived Dronabinol API to UK Market Through IPS Pharma Leveraging Priya Life Science as a Data Tracker: The Ultimate Use Case & Career Guide The €100K Reality Check: Why a Six-Figure Pharma Salary in Ireland Feels Different Than in Switzerland or Germany Ireland's €93.8 Billion Non-EU Pharma Export Engine: 2025 Eurostat Trade Data and Economic Impact Astellas Expands Its 330 Million Euro Tralee Biopharma Facility with a Second Aseptic Filling Line to Double Drug-Product Capacity Xeolas Pharmaceuticals Opens 158,000 Sq Ft State-of-the-Art Baldoyle Facility to Scale Specialty Medicine Manufacturing Priya Life Science Partners with Fleming for the 9th Annual Corporate Compliance & Transparency in Life Sciences Summit in Zurich Ireland Has the Capital and the Lessons: Digital Project Management Is How They Become Delivery Dunbar Pharma Brings First Plant-Derived Dronabinol API to UK Market Through IPS Pharma Leveraging Priya Life Science as a Data Tracker: The Ultimate Use Case & Career Guide The €100K Reality Check: Why a Six-Figure Pharma Salary in Ireland Feels Different Than in Switzerland or Germany Ireland's €93.8 Billion Non-EU Pharma Export Engine: 2025 Eurostat Trade Data and Economic Impact
Active, not recruiting Phase 1

GDNF Gene Therapy for Multiple System Atrophy

NCT04680065 · tracked via the Priya Life Science USA tracker
Sponsor
Brain Neurotherapy Bio, Inc
Phase
Phase 1
Started
2023-10-03
Last updated
2026-01-30

Condition(s) studied

Multiple System Atrophy

Investigational drug(s) / intervention(s)

AAV2-GDNF gene therapy →Sham (Placebo) Surgery

AAV2-GDNF gene therapy: Bilateral image-guided infusion of AAV2-GDNF into putamen, single dose

Sham (Placebo) Surgery: Bilateral partial burr/twist holes without dural penetration

Study summary

The objective of this randomized, double-blinded, placebo-controlled Phase 1 investigation is to evaluate the safety and potential clinical effect of AAV2-GDNF delivered to the putamen in subjects with either a possible or probable diagnosis of Multiple System Atrophy.

Eligibility

Sex
ALL
Min age
35 Years
Max age
75 Years
Healthy volunteers
No
Inclusion Criteria: * Male and female 35-75 years of age (inclusive) * Clinical diagnosis of MSA, parkinsonian type with symptoms onset sporadic, progressive and \> 30 years of age * Less than 5 years from MSA parkinsonian diagnosis with expected survival more than 3 years * Stable anti-parkinsonian medication regimen * Ability to walk a distance of 25 feet with or without an assistive device Exclusion Criteria: * Presence of idiopathic Parkinson's disease (PD) or any PD-related mutation or other neurological diseases * Presence of dementia, psychosis, substance abuse or poorly controlled depression * Prior brain surgery (i.e., deep brain stimulator implantation) or other brain imaging abnormalities * History of cancer or poorly controlled medical conditions that would increase surgical risk * Received investigational agent within 12 weeks * Inability to tolerate laying flat in an MRI and/or allergy to gadolinium NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Primary outcome measure(s)

  • The incidence of Treatment-Emergent Adverse Events (TEAE) and Serious Adverse Events (SAE) assessed clinically by physical and neurological examinations — 3 years
    Number of TEAE and SAE's reported post-treatment.

Trial sites (7)

FacilityCityRegionStatus
University of California Irvine Irvine California
Parkinson's Disease and Movement Disorders Center of Boca Raton Boca Raton Florida
Massachusetts General Hospital Boston Massachusetts
Quest Research Institute Farmington Hills Michigan
NYU Langone Health New York New York
The Ohio State University Medical Center Columbus Ohio
Vanderbilt University Medical Center Nashville Tennessee

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 NCT04680065 on ClinicalTrials.gov ↗ ← All trials in the USA