ESSENCE Study: Efficacy and Safety of SD-101 Cream in Participants With Epidermolysis Bullosa

• Status: Completed
• Trial end date: 2017-07-05
• Target enrollment: 0
• Participant gender: All

Summary

The aim was to assess the efficacy and safety of SD-101-6.0 cream versus Placebo (SD-101-0.0) cream in the treatment of skin lesions in participants with Epidermolysis Bullosa. Funding Source - United States Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD).

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Scioderm, Inc.
• Collaborators: Amicus Therapeutics; Amicus Therapeutics, Inc.

Criteria

Inclusion Criteria:

• Informed Consent form signed by the participant or participant's legal representative; if the participant was under the age of 18 but capable of providing assent, signed assent from the participant.

• Participant (or caretaker) must have been willing to comply with all protocol requirements.

• Diagnosis of Simplex, Recessive Dystrophic, or Junctional non-Herlitz EB.

• Participant must have had 1 target wound (size 10 to 50 cm^2) at study entry.

• Participants 1 month and older.

• Target wound must have been present for at least 21 days.

Exclusion Criteria:

• Participants who did not meet the entry criteria outlined above.

• Selected target wound did not have clinical evidence of local infection.

• Use of any investigational drug within the 30 days before enrollment.

• Use of immunotherapy or cytotoxic chemotherapy within the 60 days before enrollment.

• Use of systemic or topical steroidal therapy within the 30 days before enrollment. (Inhaled steroids and ophthalmic drops containing steroids were allowed).

• Use of systemic antibiotics within the 7 days before enrollment.

• Current or former malignancy.

• Arterial or venous disorder resulting in ulcerated lesions.

• Pregnancy or breastfeeding during the study. (A urine pregnancy test was performed at screening and every 30 days until the final visit for female participants of childbearing potential).

• Females of childbearing potential who were not abstinent and not practicing a medically acceptable method of contraception.