A Randomized, Double-blind, Vehicle-controlled Phase 2 Study to Evaluate Efficacy, Safety, and Tolerability of Topically Applied TCP-25 in Patients with Epidermolysis Bullosa

Ki vehet részt

• Male or female patients with documented diagnosis of DEB or JEB, confirmed by genetic testing and/or by a skin biopsy with immunofluorescence mapping. • Patients ≥4 years old. Note: Initially, patients ≥12 years will be enrolled. Enrollment will be open to 4 to 11 year old pediatric patients (both inclusive), after a DMC reviews and provides a positive opinion regarding the safety and tolerability of the IMP in at least 2 patients 12 to 18 years old who have completed at least 4 weeks of IMP use and in all patients > 18years. • The patient has the presence of a well-matched index wound pair which will be included in two respective treatment areas, with a minimum distance of at least 5 cm (2 inches) between the treatment areas

Ki nem vehet részt

• The patient has any subtype of EB other than DEB or JEB. • The patient is currently being treated or planned to be treated with systemic antibiotics. Note: Use of preventive and/or anti-inflammatory antibiotic treatment, including doxycycline, on an established treatment regimen (stable dose for ≥6 weeks before the Baseline Visit) is permitted. Use of topical antibiotics on the index wounds within 7 days before the Baseline Visit is prohibited. • Use of systemic corticosteroids >0.2 mg/kg prednisone dose equivalent per day within 30 days or use of topical corticosteroids on index wounds within 7 days before the Screening Visit. Note: Corticosteroids for inhalation, ophthalmic, or intranasal use are permitted. • The patient has a history of any of the following treatments: a. Local topical Filsuvez on the study treatment areas within 1 month before the Screening Visit, b. Local topical gene therapy with Vyjuvek on the study treatment areas within 3 months before the Screening Visit, c. Systemic mesenchymal stem cell treatment, including Ebesenar, within 6 months before the Screening Visit, or d. Cell-gene (graft) therapy, Zevaskyn, for the treatment of EB affecting the treatment areas any time before the Screening Visit