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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
GDNF Gene Therapy for Multiple System Atrophy
NCTID
NCT04680065
(View at clinicaltrials.gov)
Description
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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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
Recruiting
Enrollment Count
9
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
Target Tissue/Cell
Delivery System
Viral transduction
Vector Type
AAV2
Editor Type
none
Dose 1
Undisclosed dose 1
Dose 2
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1
Submit Date
2020-12-15
Completion Date
2028-12
Last Update
2025-02-10
Participation Criteria
Eligible Age
35 Years - 75 Years
Standard Ages
Adult, Older adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
7
Locations
United States
Regulatory Information
Has US IND
True
FDA Designations
Recent Updates
First patient randomized 11/17/23
Resources/Links
News and Press Releases
AskBio Announces First Patient Randomized in Phase 1 Trial of AB-1005 (AAV2-GDNF) Gene Therapy for Multiple System Atrophy-Parkinsonian Type (MSA-P)