Study of Effectiveness and Safety of SD-101 in Participants With Epidermolysis Bullosa

A Phase 2b, Multi-Center, Randomized, Double-Blind, Vehicle-Controlled Study of the Effectiveness and Safety of SD-101 Cream in Subjects With Epidermolysis Bullosa

The purpose of this study was to assess whether the topical use of SD-101 cream (3% or 6%) was effective in treating wounds in participants with Simplex, Recessive Dystrophic, or Junctional non-Herlitz Epidermolysis Bullosa (EB).

This was a Phase 2b, multi-center, randomized, double-blind, vehicle-controlled study to assess the efficacy and safety of SD-101 dermal cream (3% or 6%) on skin lesions in participants with Simplex, Recessive Dystrophic, or Junctional non-Herlitz EB.

SD-101 cream [containing 3% or 6% or vehicle (0%)] was applied topically, once a day to the entire body for a period of 90 days. Eligible participants had a target wound assessed at baseline. Selected target wound had to be at least a certain age and within a prespecified size range at study entry. Photographic confirmation of the target wound location was collected at baseline, and the picture saved from the first visit was used to confirm location of the target wound at subsequent visits. The participant returned to the study site at Week 2, Month 1, Month 4, and Month 5 (at 14, 30, 60, and 90 days, respectively) to have the target wound previously identified at baseline re-assessed for the level of healing. In addition, changes in itching, pain, body surface area coverage of blisters and lesions, and scaring of the healed target wound were also assessed at each visit.

Inclusion Criteria:

• Informed Consent form signed by the participant or the participant's legal representative; if the participant is under the age of majority but capable of providing assent, signed assent from the participant.
• Participant (or caretaker) was willing to comply with all protocol requirements.
• Diagnosis of Simplex, Recessive Dystrophic, or Junctional non-Herlitz EB.
• Participants 6 months of age and older.
• Participants had 1 target wound within a prespecified size range at study entry.
• Target wound was at least 21 days or older.

Exclusion Criteria:

• Participants who did not meet all the entry criteria outlined in inclusion criteria.
• Selected target wound had clinical evidence of local infection.
• Use of any investigational drug within 30 days before enrollment.
• Use of immunotherapy or cytotoxic chemotherapy within 60 days before enrollment.
• Use of systemic or topical steroidal therapy within 30 days before enrollment (inhaled steroids and ophthalmic drops containing steroids were allowed).
• Use of systemic antibiotics within 7 days before enrollment.
• Current or former malignancy.
• Arterial or venous disorder resulting in ulcerated wounds.
• Diabetes mellitus.
• Pregnancy or breastfeeding during the study (a urine pregnancy test was performed at screening for female participants of childbearing potential).
• Females of childbearing potential who were not abstinent and not practicing a medically acceptable method of contraception.
• Known history of cardiac, hepatic, or renal disease.