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Gene Therapy Trial Report

Summary

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


NCTID NCT04917874 (View at clinicaltrials.gov)
Description
Development Status Approved
Indication Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Disease Ontology Term DOID:4959
Compound Name VYJUVEK
Compound Alias beremagene geperpavec
Sponsor Krystal Biotech, Inc.
Funder Type Industry
Recruitment Status
Completed
Enrollment Count 47 (ACTUAL)
Results Posted Not Available

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
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)

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
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: * Willing and able to give consent/assent * Clinical diagnosis of epidermolysis bullosa * Confirmation of diagnosis (either DDEB or RDEB) by genetic testing including COL7A1. * Age: 2 months of age and older at the time of informed consent/assent * Women of childbearing age must be willing to use reliable birth control method throughout the treatment duration and for 3 months following the last treatment application Exclusion Criteria: * Diseases or conditions that could interfere with the assessment of safety of the study treatment and compliance of the participant with study visits/procedures, as determined by the Investigator * Pregnant or nursing women * Active infection in the area that will undergo administration, that the Investigator believes will negatively impact the IP application * Known allergy to any of the constituents of the product * Concurrent skin transplant or mesh skin grafting; if the participant is currently having transplantation or grafting surgeries they must wait until the transplant has begun to heal and the graft has absorbed prior to initiating B-VEC therapy * Participation in an interventional gene therapy clinical trial within the past three (3) months (not including B-VEC administration)
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 6
Locations United States

Regulatory Information


Has US IND True
FDA Designations Fast Track, Orphan Drug Designation, Priority Review, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
Recent Updates FDA approved 5/19/23, estimated price $600K/year

Resources/Links