Open Label Extension Study to Evaluate the Long-term Safety of Zorblisa (SD-101-6.0) in Patients With Epidermolysis Bullosa
This trial is active, not recruiting.
|Treatment||zorblisa (sd-101-6.0) cream|
|Collaborator||Amicus Therapeutics, Inc.|
|Start date||May 2015|
|End date||September 2017|
|Trial size||130 participants|
|Trial identifier||NCT02670330, 2014-005679-96, SD-006|
The aim is to assess the long-term safety of topical use of ZORBLISA in patients with Epidemolysis Bullosa.
|United States||No locations recruiting|
|Other countries||No locations recruiting|
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|Redwood City, CA||Stanford University-Dept of Dermatology||no longer recruiting|
|Aurora, CO||University of Colorado School of Medicine||no longer recruiting|
|Chicago, IL||Ann & Robert H. Lurie Children's Hospital of Chicago||no longer recruiting|
|St. Louis, MO||Washington University-Dermatology||no longer recruiting|
|Chapel Hill, NC||UNC Dermatology and Skin Cancer Center||no longer recruiting|
|Cincinatti, OH||Cincinnati Children's Hospital Medical Center||no longer recruiting|
|San Antonio, TX||Texas Dermatology and Laser Specialists||no longer recruiting|
|Salzburg, Austria||EB House Austria, General Hospital Salzburg/Salzburger Landesklinikum (SALK), Paracelsus Medical University Salzburg (PMU)||no longer recruiting|
|Nice, France||Hopital Archet 2 Service de Dermatologie||no longer recruiting|
|Paris, France||University of Paris Sorbonne Descartes, IMAGINE Institute, Necker-Enfants Malades Medical School||no longer recruiting|
|Freiburg, Germany||Medical Center- University of Freiburg, Department of Dermatology, Hauptstr. 7||no longer recruiting|
|Milano, Italy||Universita degli Studi di Milano, Fondazione IRCCS Ca' Granda Ospedale Maggiore||no longer recruiting|
|Groningen, Netherlands||University Medical Center Groningen, Hanzeplein 1 Department Dermatology||no longer recruiting|
|Warszawa, Poland||Department of Immunodermatology, Medical University of Warsaw||no longer recruiting|
|London, United Kingdom||Clinical Research Facilities (CRF) unit, Great Ormond Street Hospital NHS Foundation Trust||no longer recruiting|
|Endpoint classification||safety study|
|Intervention model||single group assignment|
All subjects will apply ZORBLISA (SD-101-6.0%) cream topically, once a day to the entire body for a period of up to 630 days.
Demonstrate continued long Term Safety of Zorblisa assessed via monitoring of local tolerability at the application sites, occurrence of adverse events and physical examinations. Results will be summarized
time frame: up to 630 days
Changes in Body Surface Area (BSA) of lesional skin and wound burden using BSAI
time frame: Months 1, 3, 6, 9 12,15,18 and 21
Male or female participants at least 1 month old.
- 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.
- Subject (or caretaker) must be willing to comply with all protocol requirements.
- Patients who completed the SD-005 study (on study drug at Visit 5).
- Patients who do not meet the entry criteria outlined above.
- 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)
- Females of childbearing potential who are not abstinent or not practicing a medically acceptable method of contraception
|Official 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|
|Description||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 apparent 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. In some EB subtypes, high mortality occurs before the age of 1 (Junctional Herlitz), and others in adolescences to early adulthood, typically due to infection or failure to thrive. In addition, children surviving into their 20's and 30's are also at risk for development of a virulent form of squamous cell carcinoma, which is in many cases fatal. 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 the United States. There have been numerous studies published on potential treatments for skin manifestations associated with EB, including vitamin E therapy, systemic phenytoin, topical nonsteroidal agents, cyproheptadine, tetracycline, and dapsone. 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. 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. ZORBLISA will be applied topically, once a day, to the entire body for a period of 630 days. Patients who successfully completed study SD-005 (on study drug at Visit 5) will have the option to roll over into the SD-006 study. The baseline visit (Visit 1) will occur at Visit 5 for SD-005. The Body Surface Area (BSA) assessments of lesional skin and wound burden performed at the Visit 5 for SD-005 will be utilized as the baseline assessment for SD-006. Patients will return for follow-up visits at months 1, 3, 6, 9 ,12, 15, 18 and 21 after Visit 1. At each visit, assessments will include BSA of lesional skin and wound burden. For target wounds that are not closed by the end of Study SD-005, the ARANZ picture and calculation of target wound area at the final visit for Study SD-005 will used as the baseline area size of the target wound for SD-006. These unhealed target wounds from SD-005 will be assessed via ARANZ at each subsequent scheduled visit until the target wound is documented as closed. Closed wounds will be assessed for scarring.|
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