Targeting Collagen VII Antibodies in Bullous Diseases Using Efgartigimod IV (VYVGART)
M. Peter Marinkovich
Summary
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.
Eligibility
- Age range
- 12+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * For DEB patients (aged 12 years or older): DEB confirmed with mutation analysis and correlated by phenotype, and treatment of at least 1 wound treated with topical gene therapy (VYJUVEK). Presence of C7 antibodies above normal cutoff on ELISA. * For (classic) EBA patients (aged 18 years or older): EBA confirmed with positive histopathology (DIF), C7 antibodies above normal cutoff on ELISA, and having at least 1 skin lesion. * The participant has a Karnofsky performance status of at least 60% at screening. * Contraceptive use by reproductive male and female patients shoul…
Interventions
- BiologicalEfgartigimod
Dosage: 10mg/kg Frequency: Once a week Duration: 25 weeks
Location
- Stanford UniversityRedwood City, California