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

• Register: EUCTR
• Last refreshed on: 2 October 2017
• Main ID: EUCTR2014-002288-14-AT
• Date of registration: 02/07/2014
• Prospective Registration: Yes
• Primary sponsor: Scioderm, An Amicus Therapeutics Company
• Public title: An Investigation into the Efficacy and Safety of SD-101 Cream in Patients with Epidermolysis Bullosa
• Scientific title: 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 - Study of Effectiveness and Safety of SD-101 in Subjects with Epidermolysis Bullosa/ESSENCE Study
• Date of first enrolment: 08/08/2014
• Target sample size: 150
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2014-002288-14
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no Number of treatment arms in the trial: 2
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Australia, Austria, Belgium, Bulgaria, France, Germany, Israel, Italy, Lithuania, Netherlands, Poland, Romania, Serbia, Spain, United Kingdom, United States

Contacts

• Name: Jamie L. Gault
• Affiliation: Scioderm, An Amicus Therapeutics Company

• Name: Jamie L. Gault
• Affiliation: Scioderm, An Amicus Therapeutics Company

Key inclusion & exclusion criteria

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.


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

Health Condition(s) or Problem(s) studied

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

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

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-
Pharmaceutical form of the placebo: Cream
Route of administration of the placebo: Topical use (Noncurrent)

Primary Outcome(s)

Secondary Objective: Not Applicable

Timepoint(s) of evaluation of this end point: The primary efficacy analysis will compare the proportion of patients achieving this endpoint within 3 months.

Primary end point(s): Co-Primary Efficacy Endpoints
The co-primary efficacy endpoints for this study are
• Time to complete target wound closure within 3 months
• The proportion of patients experiencing complete closure of their target wound within 3 months

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 Outcome(s)

Timepoint(s) of evaluation of this end point: The secondary measures of efficacy include:
• Change in lesional skin based on BSA estimates at month 3, compared
to baseline.
• Change in itching assessed at Day 7, compared to baseline.
• Change in pain assessed at Day 7, compared to baseline.
•Proportion of patients experiencing complete would closure at 2 and 1
months.
•Total body wound burden at Month 3

Secondary end point(s): Secondary Efficacy Endpoints
The secondary measures of efficacy include:
• Proportion of patients experiencing complete closure of their target
wound within 2 months
• Proportion of patients experiencing complete closure of their target
wound within 1 month
• Change in lesional skin based on BSAI estimates at Month 3,
compared to Baseline
• Estimation of Total Body Wound Burden at Month 3, compared to
Baseline
• Change in itching assessed at Day 7, compared to Baseline
• Change in pain assessed at Day 7, compared to Baseline

Secondary ID(s)

SD-005

Source(s) of Monetary Support

Scioderm, INC.