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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 (RMAT)
Recent Updates FDA approved 5/19/23, estimated price $600K/year

Resources/Links