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

Phase 3

Completed

Conditions: Genetic skin disorder
10014982
inherited connective tissue disease

Registration Number: NL-OMON44714

Lead Sponsor: Scioderm Inc.

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Completed

Sex: Not specified

Target Recruitment: 14

Inclusion Criteria

1. Informed Consent form signed by the patient or patient's legal representative; also, if the patient is under the age of majority but capable of providing assent, signed assent from the patient.
2. Patient (or caretaker) must be willing to comply with all protocol requirements.
3. Diagnosis of Simplex, Recessive Dystrophic, or Junctional non-Herlitz EB.
4. Patient must have 1 target wound (size 10 to 50 cm2).
5. Patients 1 month and older.
6. Target wound must be present for 21 days or more

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 within the 30 days before enrollment.
4. Use of immunotherapy or cytotoxic chemotherapy within the 60 days before enrollment.
5. Use of systemic or topical steroidal therapy within the 30 days before enrollment. (Inhaled steroids and ophthalmic drops containing steroids are allowed)
6. Use of systemic antibiotics within the 7 days before enrollment.
7. Current or former malignancy.
8. Arterial or venous disorder resulting in ulcerated lesions.
9.Pregnancy or breastfeeding during the study. (A urine pregnancy test will be performed at screening and every 30 days until the final visit for female patients of childbearing potential)
10. Females of childbearing potential who are not abstinent and not practicing a medically acceptable method of contraception.

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Primary Efficacy Endpoints<br /><br>The primary efficacy endpoints for this study are<br /><br>* Time to complete target wound closure within 3 months<br /><br>* The proportion of patients experiencing complete closure of their<br /><br>target wound within 3 months</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Key Secondary Efficacy Endpoints<br /><br>The secondary measures of efficacy include:<br /><br>* Proportion of patients experiencing complete closure of their target<br /><br>wound within 2 months<br /><br>* Proportion of patients experiencing complete closure of their target<br /><br>wound within 1 month<br /><br>* Change in lesional skin based on BSAI estimates at Month 3,<br /><br>compared to Baseline<br /><br>* Estimation of Total Body Wound Burden based on BSAI at Month 3, compared to<br /><br>Baseline<br /><br>* Change in itching assessed at Day 7, compared to Baseline<br /><br>* Change in pain assessed at Day 7, compared to Baseline</p><br>