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Clinical Trial Report

Gene Therapy Trial Report

Summary

Gene Therapy for Haemophilia A.

NCTID NCT03001830 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Hemophilia A
Disease Ontology Term DOID:12134
Compound Name AAV2/8-HLP-FVIII-V3
Sponsor University College, London
Funder Type Other
Recruitment Status
Active not recruiting
Enrollment Count 14
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 6E11 vg/kg
Dose 2 2E12 vg/kg
Dose 3 4E12 vg/kg
Dose 4 6E12 vg/kg
Dose 5

Study Record Dates

Current Stage Phase1, Phase2
Submit Date 2016-12-09
Completion Date 2029-12
Last Update 2025-03-05

Participation Criteria

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

Locations

No.of Trial Sites 4
Locations United States,United Kingdom

Regulatory Information

Has US IND True
FDA Designations
Recent Updates

Resources/Links

Clinical Publications