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An Investigation into the Long-term Safety of Zorblisa (SD-101-6.0) creamused by Patients with Epidermolysis Bullosa.

Phase 1
Conditions
Epidermolysis Bullosa
MedDRA version: 20.0Level: 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-IT
Lead Sponsor
SCIODERM, INC
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
ot Recruiting
Sex
All
Target Recruitment
152
Inclusion Criteria

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:
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) yes
F.1.3.1 Number of subjects for this age range 0

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 and repeated at Visit 1 if these visits do not occur on the same day)
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
NameTimeMethod
Main Objective: The primary objective is to demonstrate the long-term safety of<br>ZORBLISA in patients, with Simplex, Recessive Dystrophic and Junctional<br>non-Herlitz Epidermolysis Bullosa.;Secondary Objective: The secondary objectives are to assess the efficacy of ZORBLISA in<br>terms of the change in Body Surface Area (BSA) of lesional skin and<br>wound burden; as well as closure of unhealed target wounds from the<br>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 tolerabilty at the application sites, occurrence of adverse events and physical examinations at Months 1,3,6,9,12,15,18, and 21
Secondary Outcome Measures
NameTimeMethod
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, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and 48 compared to Baseline.<br>- Change in total corporal lesions based on BSA estimates at Months 1, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and 48 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. The unhealed target wound from SD-005 will be assessed at each subsequent scheduled visit until the target wound is documented as closed.
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