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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
The Safety and Efficacy of Intravenous EXG110 in Patients With Fabry Disease
NCTID
NCT06819514
(View at clinicaltrials.gov)
Description
A phase 1/2, multicenter, open-label, Dose-escalation study to evaluate the safety and efficacy of intravenous EXG110 in patients with Fabry disease
(Show More)
Development Status
Active
Indication
Fabry Disease
Disease Ontology Term
DOID:14499
Compound Name
EXG110
Sponsor
Hangzhou Jiayin Biotech Ltd
Funder Type
Industry
Recruitment Status
Not yet recruiting
Enrollment Count
16
Results Posted
Not Available
Therapy Information
Target Gene/Variant
GLA
Therapy Type
Gene transfer
Therapy Route
In-vivo
Mechanism of Action
Functional gene replacement
Route of Administration
Intravenous
Drug Product Type
Viral vector
Target Tissue/Cell
Delivery System
Viral transduction
Vector Type
proprietary AAV
Editor Type
none
Dose 1
Undisclosed low dose
Dose 2
Undisclosed high dose
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2025-01-22
Completion Date
2028-03-15
Last Update
2025-02-11
Participation Criteria
Eligible Age
>=18 Years
Standard Ages
Adult, Older adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
3
Locations
China
Regulatory Information
Has US IND
False
FDA Designations
Orphan Drug Designation
Recent Updates
First patient has been dosed in China, company plans to initiate US clinical trials
Resources/Links
News and Press Releases
US FDA Grants Orphan Drug Designation to EXG110, a Novel Gene Therapy for Fabry Disease
Related NCTID
Phase Not Applicable: NCT06539624