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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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9 participants in 2 patient groups

Active Treatment

Experimental group

Treatment:

Biological: AAV2-GDNF gene therapy

Placebo Surgery

Sham Comparator group

Treatment:

Procedure: Sham (Placebo) Surgery

Sponsor of this trial

Lead Sponsor

Brain Neurotherapy Bio