The primary objective is to compare the efficacy and safety of SD-101-6.0vs. SD- 101-0.0 (placebo) in patients with Simplex, RecessiveDystrophic, or Junctional non Herlitz Epidermolysis Bullosa.The primary endpoint is the complete closure of the…
ID
Source
Brief title
Condition
- Epidermal and dermal conditions
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Primary Efficacy Endpoints
The 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
Secondary outcome
Key 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 based on BSAI 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
Background summary
Epidermolysis Bullosa (EB) is a rare group of inherited disorders that
typically manifest at birth as blistering and lesion formation on the skin and,
in some cases, the epithelial lining of other organs, in response to little or
no apparant trauma. In consequence, the skin is extremely fragile which can
result in shearing of the skin, causing a high risk of infection. All forms of
EB are both debilitating and life threatening. There are no standard of care
products available to treat the dermal manifestations of EB, and there is no
approved drug for EB in either Europe or United States. There have been
numerous studies published on potential treatments for skin manifestations
associated with EB. No controlled studies showed clinical benefit of any
therapy. Newer exploratory treatments including skin grafts, bioengineered skin
products, and gene therapy have been unsuccessful to date. In an open label
study patients treated with SD-101 cream showed significant improvements in the
complete healing of lesions, clinically meaningful reductions in the extent of
total skin surface involvement with active disease, and reduced pain and
itching. The aim of the study is to evaluate the efficacy and safety of SD-101
cream vs Placebo in patients with EB.
Study 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.The primary
endpoint is the complete closure of the patient*s target wound within 2 months.
Study design
Phase 3, multi-center, randomized, double-blind, placebo controlled, study to
assess the efficacy and safety of SD-101 vs placebo on lesions in patients with
Simplex, Recessive Dystrophic, or Junctional non-Hertlitz Epidermolysis Bullosa.
Topical application of study cream (i.e. SD-101 or placebo) during 90 days,
once daily.
Intervention
Topical application of SD-101 skin cream for 90 days (once daily)
Study burden and risks
In a previous study with SD-101, some cases of mild redness of the skin have
been reported after application of SD-101 cream. Furthermore, patients will be
asked to complete pain and itching scales, and diaries. All patients will have
to use study cream (SD-101 or placebo) once daily for 90 days and visit the
hospital/clinic more frequently.
Creekstone Drive Suite 160 4601
Durham NC 27703
US
Creekstone Drive Suite 160 4601
Durham NC 27703
US
Listed location countries
Age
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
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.
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| EudraCT | EUCTR2014-002288-14-NL |
| CCMO | NL49780.042.14 |