This is a Phase 2, double-blind, randomized, vehicle-controlled study designed to evaluate efficacy, safety, and tolerability of topically applied TCP-25 gel in patients with confirmed DEB or JEB. The study will implement intrasubject randomization, ie, a pair of matching index wounds will be randomly assigned to be treated with a local application of either TCP 25 gel or vehicle gel.
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 3 patients 12 to 18 years old who have completed at least 4 weeks of IMP use.
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.
Parallel
TCP-25 gel
DrugTopical gel
Vehicle (placebo)
DrugPlacebo gel
Open wound area
Frequency, intensity, and seriousness of Adverse Events in all treated patients until end of treatment
Change in procedural pain using the Wong Baker FACES® Pain Rating Scale (0 min -10 max)
Wong Baker FACES pain rating scale uses 6 graphically or cartoon-depicted faces with varying facial expressions that represent no pain (no hurt) (Face 0) to worst pain/hurt that can be imagined (Face 10).
Change from baseline in clinical impression of overall treatment area
TCP-25 plasma concentrations
