An Investigation into the Efficacy and Safety of SD-101 Cream in Patients with Epidermolysis Bullosa
- Conditions
- Epidermolysis BullosaMedDRA version: 17.0Level: PTClassification code 10014989Term: Epidermolysis bullosaSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]
- Registration Number
- EUCTR2014-002288-14-IT
- Lead Sponsor
- Scioderm, INC.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 90
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 5 to 50 crrr').
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: 90
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 29
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
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. Diabetes mellitus.
10.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)
11. Females of childbearing potential who are not abstinent and not practicing a medically acceptable method of contraception.
12.History of cardiac, hepatic, or renal disease.
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 evaluate 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. The primary endpoint is the complete closure of the patient's target wound.;Secondary Objective: Not Applicable;Primary end point(s): The primary endpoint is the complete closure of the patient's target wound. Complete target<br>wound closure is defined as skin reepithelialization without drainage. ;Timepoint(s) of evaluation of this end point: The primary efficacy analysis will compare the proportion of patients achieving this endpoint within one month of initiation of study treatment.
- Secondary Outcome Measures
Name Time Method Secondary end point(s): The secondary endpoints include the estimation of body surface area (BSA) coverage of lesional skin, and assessment of itching, pain, and scarring.<br>;Timepoint(s) of evaluation of this end point: The secondary measures of efficacy include:<br>• Change in lesional skin based on body surface area (BSA)measurements at month 3, compared to baseline.<br>• Change in itching assessed at week 1 (Day 7), compared to baseline.<br>• Change in pain assessed at week 1 (Day 7), compared to baseline.<br>• Assessment of scarring of healed target wound.<br>• Assessments of changes in itching and pain at Days 1-6, week 2, months 1 and 2<br>• Reduction in lesional skin based on body surface area (BSA) measurements at months 1 and 2, compared to baseline.<br>• Total target wound closure at months 2 and 3.