Clinical Research Directory

Targeting Collagen VII Antibodies With IV IgG in Dystrophic Epidermolysis Bullosa

The study objective is to see if IV IgG treatment in Recessive Dystrophic Epidermolysis Bullosa (RDEB) skin in conjunction with VYJUVEK treatment improves wound healing and affects the levels of C7 and HSV-1 antibody levels in serum. Fewer wounds, more rapidly healing wounds, and decreased C7 and HSV-1 antibodies could improve quality of life.

A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa

The purpose of this study is to determine whether administration of FCX-007 in addition to standard of care improves wound healing as compared to standard of care alone (control) in children, adolescents, and adults with Recessive Dystrophic Epidermolysis Bullosa. Funding Source - FDA OOPD

GMEB-SASS: A Gene-Modified Skin Substitute for RDEB Treatment

This study is being done to find out if a new type of skin graft, called GMEB-SASS, is safe and effective for helping wounds heal in people with RDEB (Recessive Dystrophic Epidermolysis Bullosa). The GMEB-SASS graft contains two types of living skin cells: keratinocytes and fibroblasts. It is made in a laboratory using a small sample of the patient's own skin. To help the patient's skin cells produce a missing protein called type VII collagen, scientists grow the patient's cells in the lab and use a virus-like tool (called a retroviral vector) to give the cells the correct instructions. This allows the cells to make the normal protein that is missing in people with RDEB. The graft is designed to be permanent, and the goal is to improve wound healing by replacing damaged skin cells with healthy ones.

A Study Comparing KB803 and Matched Placebo in Patients With Dystrophic Epidermolysis Bullosa

KB803-EYE-01 is a Phase 3 double-blind, randomized, placebo-controlled, crossover study to evaluate the safety and efficacy of KB803 versus matched placebo in pediatric and adult subjects with recurrent corneal abrasions due to dystrophic epidermolysis bullosa (DEB).

Pilot Study of ELK-003 Eye Drops for Treating Ocular Manifestations of Epidermolysis Bullosa

This study consists of two phases: an Observational Phase to evaluate the natural history of ocular manifestations in subjects with Dystrophic and Junctional Epidermolysis Bullosa, followed by a Treatment Phase to assess the effects of ELK-003 eye drops. Each subject will serve as their own control by comparing ocular manifestations documented during the Observational Phase to those recorded during the Treatment Phase.

A Phase 3b Study for the Treatment of Dystrophic Epidermolysis Bullosa (DEB) in New and Previously EB-101 Treated Patients

To evaluate and further characterize the safety of EB-101 (LZRSE-Col7A1 gene-corrected keratinocyte sheets with type VII collagen \[C7\] expression) for the treatment of large, chronic DEB wounds in new and previously EB-101 treated patients 12 months and older.

Targeting Collagen VII Antibodies in Bullous Diseases Using Efgartigimod IV (VYVGART)

The study objective is to see if IV Efgartigimod and Vyjuvek treatment in Recessive Dystrophic Epidermolysis Bullosa (RDEB) and IV Efgartigimod treatment in Epidermolysis Bullosa Acquisita (EBA) improves wound healing and affects the levels of C7 antibody levels in serum. Fewer wounds, more rapidly healing wounds, and decreased C7 antibodies could improve quality of life.

Long-Term Follow-up Protocol

The main objective of this prospective, observational, long-term follow-up (LTFU) study is to evaluate the long-term safety profile of the gene therapy products evaluated by Krystal Biotech, Inc. which have a shared backbone of HSV-1, in participants who received at least one dose of investigational product (IP).