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Gene Therapy Trial Report

Summary

GDNF Gene Therapy for Multiple System Atrophy


NCTID NCT04680065 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Multiple System Atrophy
Disease Ontology Term DOID:4752
Compound Name AB-1005
Compound Description AAV2-GDNF
Sponsor Brain Neurotherapy Bio, Inc.
Funder Type Industry
Recruitment Status
Active not recruiting
Enrollment Count 9 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant GDNF
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Overexpression of protective allele/gene
Route of Administration Intraparenchymal
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV2
Editor Type none
Dose 1 Undisclosed dose 1

Study Record Dates


Current Stage Phase1
Submit Date 2020-12-15
Completion Date 2028-08
Last Update 2026-01-30

Participation Criteria


Eligible Age 35 Years - 75 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
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.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 7
Locations United States

Regulatory Information


Has US IND True
FDA Designations
Recent Updates Phase 1 enrollment completed September 2025

Resources/Links