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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
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