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GDNF Gene Therapy for Multiple System Atrophy

NCT04680065 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 9

Last updated 2026-01-30

No results posted yet for this 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.

Conditions

Interventions

BIOLOGICAL

AAV2-GDNF gene therapy

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

PROCEDURE

Sham (Placebo) Surgery

Bilateral partial burr/twist holes without dural penetration

Sponsors & Collaborators

  • Brain Neurotherapy Bio, Inc.

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
35 Years
Max Age
75 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-10-03
Primary Completion
2026-08-31
Completion
2028-08-31
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04680065 on ClinicalTrials.gov