The aim is to assess the long-term safety of topical use of ZORBLISA in patients with Epidermolysis Bullosa (EB).
ID
Source
Brief title
Condition
- Epidermal and dermal conditions
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
The primary objective is to demonstrate the longterm safety of ZORBLISA in
patients, with Simplex, Recessive Dystrophic, and Junctional non-Herlitz
Epidermolysis Bullosa.
Secondary outcome
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.
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 is to assess the long-term safety of
topical use of ZORBLISA in patients with Epidermolysis Bullosa (EB).
Study objective
The aim is to assess the long-term safety of topical use of ZORBLISA in
patients with Epidermolysis Bullosa (EB).
Study design
This is an open label, multi-center extension study to assess the long-term
safety of topically applied ZORBLISA in Patients with Simplex, Recessive
Dystrophic, and Junctional non-Herlitz Epidermolysis Bullosa.
Intervention
ZORBLISA will be applied topically, once a day, to the entire body for a period
of 630 days.
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 once daily for 630 days and visit
the hospital/clinic more frequently.
Slater Road, Suite 170 1007
Durham NC 27703
US
Slater Road, Suite 170 1007
Durham NC 27703
US
Listed location countries
Age
Inclusion criteria
1. Informed Consent form signed by the subject or subject's legal representative; if the subject
is under the age of 18 but capable of providing assent, signed assent from the subject.
2. Subject (or caretaker) must be willing to comply with all protocol requirements.
3. Patients who completed the SD-005 study (on study drug at Visit 5).
Exclusion criteria
1. Patients who do not meet the entry criteria outlined above.
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
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 | EUCTR2014-005679-96-NL |
| CCMO | NL52446.042.15 |