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Clinical Trial Report

Gene Therapy Trial Report

Summary

A Long-term Treatment With B-VEC for Dystrophic Epidermolysis Bullosa


NCTID NCT04917874 (View at clinicaltrials.gov)
Description
Indication Dystrophic Epidermolysis Bullosa, DEB - Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa, Dominant Dystrophic Epidermolysis Bullosa
Compound Name VYJUVEK/beremagene geperpavec
Sponsor Krystal Biotech, Inc.
Funder Type Industry
Status
Completed
Enrollment Count 47

Therapy Information


Target Gene/Variant COL7A1
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Topical
Drug Product Type Viral vector
Target Tissue/Cell
Delivery System Viral transduction
Vector Type HSV1
Editor Type none
Dose 1 Maximum weekly dose: 1.6E9 PFU (6 months to < 3 years)
Dose 2 Maximum weekly dose: 3.2E9 PFU (over 3 years old)
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase3
Submit Date 2021-06-02
Completion Date 2023-07-31
Last Update 2024-04-09

Participation Criteria


Eligible Age >=2 Months
Standard Ages Child, Adult, Older adult
Eligible Sex ALL

Locations


No.of Trial Sites 6
Locations United States

Regulatory Information


Has US IND True
Recent Updates FDA approved 5/19/23, estimated price $600K/year

Resources/Links