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

A Phase 3, Multi-center, Randomized, Double-Blind, Placebo Controlled Study of the Efficacy and Safety of SD-101 Cream in Patients With Epidermolysis Bullosa

The upcoming trial is for EB patients is a topically applied whole-body treatment of patients with either Simplex, RDEB or Junctional (nH) ages 1 month to 12 years old at study entry. There are only 4 site visits by patients with minimal assessments over a 2-month period, and patients completing this study will have the ability to continue receiving SD-101-6.0 at home in an open-label extension study. The drug product and placebo require no special preparation or storage conditions (room temperature).

This is a Phase 3, multi-center, randomized, double-blind, placebo-controlled, study to compare the efficacy and safety of SD-101-6.0 cream vs. placebo (SD 101-0.0) on wounds in patients with Simplex, Recessive Dystrophic, or Junctional non-Herlitz (JEB-nH) EB.

SD-101-6.0 cream or placebo (SD-101-0.0) will be applied topically, once a day to the entire body for a period of 60 days. Enrolled patients will have 1 target wound selected at baseline. The selected target wound must be at least 21 days old (size 10 to 50 cm2). Photographic confirmation of the target wound location will be collected at baseline, and the picture saved from the first visit will be used to confirm location of the target wound at subsequent visits. The patient will return to the study site for Visit 2 (14 days ±7 days from baseline), Visit 3 (30 days ±7 days from baseline), and Visit 4 (60 days ±7 days from baseline) to have the target wound re-assessed for the level of healing. Once the target wound is healed, it will be considered healed at subsequent visits and no further re-assessment of the target wound will be conducted The ARANZ SilhouetteStar™ will be used to measure the target wound at all visits until the target wound is documented as closed. Assessment of itching will be evaluated at each visit. In addition, body surface area (BSA), and other secondary endpoints will be evaluated at the baseline and Month 2 visits.

Inclusion Criteria:

1. Informed Consent (ICF) form signed by the patient or patient's legal representative. Since all patients with be 12 years old or younger at study entry, signed assent from the patient's legal representative will be required.
2. Patient (or caretaker) must be willing to comply with all protocol requirements.
3. Diagnosis of Simplex, Recessive Dystrophic, or JEB-nH (intermediate) EB.
4. Patient must have 1 target wound (size 10 to 50 cm2).
5. Patients must be between 28 days to 12 years at study entry
6. Target wound must be present for 21 days or more.
7. Patients must have wound burden based on BSA of ≥5% at baseline

Exclusion Criteria:

1. Patients who do not meet the entry criteria outlined above.

2. Selected target wound cannot have clinical evidence of local infection.

3. Use of any investigational drug or approved EB therapy within 30 days before enrollment.

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

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

6. Use of systemic antibiotics within 7 days before enrollment.

7. Current or former malignancy.

8. Participant is one of the following:

   1. Not of childbearing potential (premenarchal or male/not in possession of a uterus).

   2. If of childbearing potential, is nonpregnant (negative urine pregnancy test results at Screening), nonlactating, and practicing 1 of the following medically acceptable methods of birth control:

      • Abstinence from heterosexual intercourse as a lifestyle choice.
      • Hormonal methods such as oral, implantable, injectable, or transdermal contraceptives for a minimum of 1 full cycle (based on the participant's usual menstrual cycle period) before product administration.
      • Intrauterine device. If male, is willing to use a highly effective method of contraception throughout the study period.