GDNF Gene Therapy for Multiple System Atrophy

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE1

Total Enrollment

9 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-10-03

Study Completion Date

2028-08-31

Brief Summary

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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.

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Detailed Description

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Conditions

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Multiple System Atrophy

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Up to 9 study participants meeting eligibility criteria will be randomized in a 2:1 fashion to receive either the investigational medicinal product or sham surgery in this Phase 1 trial.

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Active Treatment

Group Type EXPERIMENTAL

AAV2-GDNF gene therapy

Intervention Type BIOLOGICAL

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

Placebo Surgery

Group Type SHAM_COMPARATOR

Sham (Placebo) Surgery

Intervention Type PROCEDURE

Bilateral partial burr/twist holes without dural penetration

Interventions

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AAV2-GDNF gene therapy

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

Intervention Type BIOLOGICAL

Sham (Placebo) Surgery

Bilateral partial burr/twist holes without dural penetration

Intervention Type PROCEDURE

Eligibility Criteria

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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

Minimum Eligible Age

35 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No