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

• Register: EUCTR
• Last refreshed on: 24 September 2018
• Main ID: EUCTR2014-005679-96-ES
• Date of registration: 09/09/2016
• Prospective Registration: Yes
• Primary sponsor: Scioderm, INC
• Public title: An Investigation into the Long-term Safety of Zorblisa (SD-101-6.0) cream used by Patients with Epidermolysis Bullosa.
• Scientific title: An Open Label, Multi-center, Extension Study to Evaluate the Long-term Safety of Zorblisa (SD-101-6.0) in Patients with Epidermolysis Bullosa - Study to Evaluate the Long-term Safety of Zorblisa in Patients with Epidermolysis Bullosa
• Date of first enrolment: 14/11/2016
• Target sample size: 130
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2014-005679-96
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no; Randomised: no; Open: yes; Single blind: no; Double blind: no; Parallel group: no; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: no; Other: no; Number of treatment arms in the trial: 1
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Austria, France, Germany, Italy, Lithuania, Netherlands, Poland, Spain, United Kingdom, United States

Contacts

• Name: Jamie Gault
• Affiliation: Scioderm, INC.

• Name: Jamie Gault
• Affiliation: Scioderm, INC.

Key inclusion & exclusion criteria

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.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Epidermolysis Bullosa
MedDRA version: 19.0 Level: PT Classification code 10014989 Term: Epidermolysis bullosa System Organ Class: 10010331 - Congenital, familial and genetic disorders

Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]

Intervention(s)

Product Name: Zorblisa
Product Code: SD-101
Pharmaceutical Form: Cream
INN or Proposed INN: ALLANTOIN
CAS Number: 97-59-6
Other descriptive name: ALLANTOIN
Concentration unit: % (W/V) percent weight/volume
Concentration type: equal
Concentration number: 6-

Primary Outcome(s)

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.

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

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

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

Secondary Outcome(s)

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
- Change in total body wound coverage based on BSA estimates at Months 1, 3, 6, 9 and 12 compared to Baseline
- 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
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.

Secondary ID(s)

SD-006

2014-005679-96-AT

Source(s) of Monetary Support

Scioderm, INC.