An Investigation into the Long-term Safety of Zorblisa (SD-101-6.0) cream used by 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: EUCTR2014-005679-96-ES
• Lead Sponsor: Scioderm, INC

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2016-09-09
• Last Update Posted: 24 September 2018

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 130

Inclusion Criteria

1. Informed Consent form signed by the patient or patient's legal
representative; if the patient is under the age of 18 but
capable of providing assent, signed assent from the patient.
2. Patient (or caretaker) must be willing to comply with all protocol
requirements.
3. Patient who completed the SD-005 study (on study drug at Visit 5)
Are the trial subjects under 18? yes
Number of subjects for this age range: 130
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 39
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 in the Inclusion criteria.
2. Pregnancy or breastfeeding during the study. (A urine pregnancy test will be performed at the final visit for SD-005 for female patients of childbearing potential.)
3. Females of childbearing potential who are not abstinent or 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 demonstrate the long-term safety of ZORBLISA in patients, with Simplex, Recessive Dystrophic and Junctional non-Herlitz Epidermolysis Bullosa.;Secondary Objective: The secondary objectives are to assess the efficacy of ZORBLISA in terms of the change in Body Surface Area (BSA) of lesional skin and wound burden; as well as closure of unhealed target wounds from the SD-005 study;Primary end point(s): The primary endpoint is the long-term safety of ZORBLISA in patients with Simplex, Recessive Dystrophic, and Junctional non-Herlitz Epidermolysis Bullosa;Timepoint(s) of evaluation of this end point: Safety is assessed via monitoring of local tolerability at the application sites, occurrence of adverse events and physical examinations at Months 1, 3, 6, 9 and 12

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): The secondary endpoints include the change from baseline in BSA coverage of lesional skin and wound burden; as well as closure of unhealed target wounds from the SD-005 study.;Timepoint(s) of evaluation of this end point: - Change in lesional skin based on BSA estimates at Months 1, 3, 6, 9 and 12 compared to Baseline<br>- Change in total body wound coverage based on BSA estimates at Months 1, 3, 6, 9 and 12 compared to Baseline<br>- For target wounds that are not closed by the end of Study SD-005, the target wound area at the final visit for Study SD-005 will be used as the baseline<br>area size of the target wound for SD-006. Target wounds will be assessed at each subsequent visit until the wound is documented as closed.