Randomized, Double-Blind, Placebo-controlled Safety Study of Glial Cell Line-Derived Neurotrophic Factor Gene Transfer (AAV2-GDNF) in Multiple System Atrophy

Brain Neurotherapy Bio, Inc.7 sites in 1 country9 target enrollmentOctober 3, 2023

ConditionsMultiple System Atrophy

InterventionsAAV2-GDNF gene therapy Sham (Placebo) Surgery

DrugsAAV2-GDNF gene therapy

Overview

Phase: Phase 1
Intervention: AAV2-GDNF gene therapy
Conditions: Multiple System Atrophy
Sponsor: Brain Neurotherapy Bio, Inc.
Enrollment: 9
Locations: 7
Primary Endpoint: The incidence of Treatment-Emergent Adverse Events (TEAE) and Serious Adverse Events (SAE) assessed clinically by physical and neurological examinations
Status: Active, Not Recruiting
Last Updated: 3 months ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief 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.

Registry

clinicaltrials.gov

Start Date

October 3, 2023

End Date

August 1, 2028

Last Updated

3 months ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

Brain Neurotherapy Bio, Inc.

Responsible Party

Sponsor

Eligibility Criteria

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.

Arms & Interventions

Active Treatment

Intervention: AAV2-GDNF gene therapy

Placebo Surgery

Intervention: Sham (Placebo) Surgery

Outcomes

Primary Outcomes

The incidence of Treatment-Emergent Adverse Events (TEAE) and Serious Adverse Events (SAE) assessed clinically by physical and neurological examinations

Time Frame: 3 years

Number of TEAE and SAE's reported post-treatment.

Secondary Outcomes

MSA symptoms/signs as assessed by the Unified Multiple System Atrophy Rating Scale (UMSARS)(12 months)
Change in the quality of life as measured by Multiple System Atrophy Quality of Life (MSA-QoL)(12 months)
Change in striatal dopamine transporter binding as measured by [123-I] Ioflupane(12 months)