An Investigation into the Efficacy and Safety of SD-101 Cream in Patients with Epidermolysis Bullosa

Phase 1

Conditions: Epidermolysis Bullosa
MedDRA version: 19.0Level: PTClassification code 10014989Term: Epidermolysis bullosaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]

Registration Number: EUCTR2014-002288-14-AT

Lead Sponsor: Scioderm, An Amicus Therapeutics Company

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 150

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.
Are the trial subjects under 18? yes
Number of subjects for this age range: 100
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 50
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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 clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: The primary objective is to compare the efficacy and safety of SD-101-6.0vs. SD- 101-0.0 (placebo) in patients with Simplex, Recessive Dystrophic, or Junctional non Herlitz Epidermolysis Bullosa. ;Secondary Objective: Not Applicable;Primary end point(s): Co-Primary Efficacy Endpoints<br>The co-primary efficacy endpoints for this study are<br>• Time to complete target wound closure within 3 months<br>• The proportion of patients experiencing complete closure of their target wound within 3 months;Timepoint(s) of evaluation of this end point: The primary efficacy analysis will compare the proportion of patients achieving this endpoint within 3 months.

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Secondary Efficacy Endpoints<br>The secondary measures of efficacy include:<br>• Proportion of patients experiencing complete closure of their target<br>wound within 2 months<br>• Proportion of patients experiencing complete closure of their target<br>wound within 1 month<br>• Change in lesional skin based on BSAI estimates at Month 3,<br>compared to Baseline<br>• Estimation of Total Body Wound Burden at Month 3, compared to<br>Baseline<br>• Change in itching assessed at Day 7, compared to Baseline<br>• Change in pain assessed at Day 7, compared to Baseline;Timepoint(s) of evaluation of this end point: The secondary measures of efficacy include:<br>• Change in lesional skin based on BSA estimates at month 3, compared<br>to baseline.<br>• Change in itching assessed at Day 7, compared to baseline.<br>• Change in pain assessed at Day 7, compared to baseline.<br>•Proportion of patients experiencing complete would closure at 2 and 1<br>months.<br>•Total body wound burden at Month 3