Study of Effectiveness and Safety of SD-101 in Participants With Epidermolysis Bullosa
Condition: Epidermolysis Bullosa
MeSH terms: Epidermolysis Bullosa
Intervention: SD-101 dermal cream (3%); SD-101 Dermal Cream (6%); Vehicle (SD-101 0%)
Intervention description: SD-101 is a white, crystalline powder that is formulated within an odorless, soft, white cream base. SD-101-3.0 cream contains allantoin, a diureide glyoxylic acid, at a concentration of 3% and other excipients.; SD-101 is a white, crystalline powder that is formulated within an odorless, soft, white cream base. SD-101-6.0 cream contains allantoin, a diureide glyoxylic acid, at a concentration of 6% and other excipients.; A white, crystalline powder that is formulated within an odorless, soft, white cream base. It contains no allantoin, only excipients.
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Enrollment: 48.0
Sex: All
Minimum age: 6 Months
Healthy volunteers: No
Keywords: Epidermolysis Bullosa; Simplex; Recessive Dystrophic; Junctional non-Herlitz; Amicus Therapeutics; Scioderm, Inc.; SD-101 6%; SD-101-6.0; Allantoin 6%; SD-101 3%; SD-101-3.0; Allantoin 3%; Zorblisa

Summary:

      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).
    

Eligibility criteria:

        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.