Phase 3 Trial Evaluating Safety and Effectiveness of SD-101 Cream for Treating Epidermolysis Bullosa in Children Aged 1 Month to 12 Years

What this Trial Is About

Researchers are studying Epidermolysis Bullosa EB in children aged 1 month to 12 years with Simplex, Recessive Dystrophic, or Junctional non-Herlitz JEB-nH types. This Phase 3, multi-center trial compares the safety and effectiveness of SD-101-6.0 cream, a topical treatment, versus placebo applied to wounds. The study aims to evaluate wound healing and itching over two months. Participants will apply either SD-101-6.0 cream or placebo once daily to the entire body for 60 days. Each patient has one target wound between 10 and 50 cm2, present for at least 21 days. The wound is photographed and measured with a specialized device at baseline and follow-up visits. If the wound heals earlier, no further assessments are done on it. Patients will attend four visits over two months. During the study, researchers will assess wound closure, changes in wound burden, itching, and body surface area affected by wounds. Assessments include photographic documentation, wound measurements, and itch evaluations at each visit. After completing the study, participants may continue to receive SD-101-6.0 cream at home in an open-label extension. The entire study period involves minimal visits and limited assessments to reduce burden on patients.

Efficacy and Safety Study to Evaluate SD-101 in Epidermolysis Bullosa