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Clinical Trial Report

Gene Therapy Trial Report

Summary

ASC618 Gene Therapy in Hemophilia A Patients


NCTID NCT04676048 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Hemophilia A
Disease Ontology Term DOID:12134
Compound Name ASC618
Compound Description HCB-ET3-LCO
Sponsor ASC Therapeutics
Funder Type Industry
Recruitment Status
Recruiting
Enrollment Count 12
Results Posted Not Available

Therapy Information


Target Gene/Variant F8
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 AAV2/8
Editor Type none
Dose 1 Undisclosed dose 1
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2020-12-15
Completion Date 2026-12
Last Update 2023-02-01

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study MALE

Locations


No.of Trial Sites 1
Locations United States

Regulatory Information


Has US IND True
FDA Designations Fast Track, Orphan Drug Designation
Recent Updates First patient dosed January 2024

Resources/Links