GDNF Gene Therapy for Multiple System Atrophy

Brain Neurotherapy Bio

Status and phase

Enrolling

Phase 1

Conditions

Multiple System Atrophy

Treatments

Procedure: Sham (Placebo) Surgery

Biological: AAV2-GDNF gene therapy

Study type

Interventional

Funder types

Industry

Identifiers

NCT04680065

MSA-101

Details and patient eligibility

About

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.

Enrollment

9 estimated patients

Sex

All

Ages

35 to 75 years old

Volunteers

No Healthy Volunteers

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.