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Clinical Trial Report

Gene Therapy Trial Report

Summary

A Clinical Study to Evaluate the Safety and Efficacy of LY-M003 Injection in Patients with Wilson Disease


NCTID NCT06650319 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Wilson Disease
Disease Ontology Term DOID:893
Compound Name LY-M003
Sponsor Chaohui Yu
Funder Type Other
Recruitment Status
Recruiting
Enrollment Count 10
Results Posted Not Available

Therapy Information


Target Gene/Variant ATP7B
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 transdution
Vector Type AAV8
Editor Type
Dose 1 Undisclosed dose escalation, 3 levels
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Early phase1
Submit Date 2024-10-15
Completion Date 2030-03-30
Last Update 2025-02-13

Participation Criteria


Eligible Age 18 Years - 60 Years
Standard Ages Adult
Sexes Eligible for Study ALL

Locations


No.of Trial Sites 1
Locations China

Regulatory Information


Has US IND False
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates

Resources/Links


Resources/Links

No External Links Available.