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Open Label Extension Study to Evaluate the Long-term Safety of Zorblisa (SD-101-6.0) in Patients With Epidermolysis Bullosa

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ClinicalTrials.gov Identifier: NCT02670330
Recruitment Status : Terminated (The Sponsor voluntarily recalled SD-101 and terminated the study due to GMP deficiencies identified during an FDA inspection at the site of the manufacturer.)
First Posted : February 1, 2016
Last Update Posted : October 2, 2018
Sponsor:
Collaborator:
Amicus Therapeutics
Information provided by (Responsible Party):
Scioderm, Inc.

Brief Summary:
The aim is to assess the long-term safety of topical use of ZORBLISA in patients with Epidemolysis Bullosa.

Condition or disease Intervention/treatment Phase
Epidermolysis Bullosa Drug: Zorblisa (SD-101-6.0) cream Not Applicable

Detailed 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 1440 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, 21, 24, 27, 30, 33, 36, 39, 42, and 48 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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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 152 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : June 18, 2015
Actual Primary Completion Date : June 4, 2018
Actual Study Completion Date : September 3, 2018


Arm Intervention/treatment
Experimental: Experimental: ZORBLISA (SD-101 cream )
All subjects will apply ZORBLISA (SD-101-6.0%) cream topically, once a day to the entire body for a period of up to 1440 days (48 months).
Drug: Zorblisa (SD-101-6.0) cream
applied topically once a day to the entire body for 1440 days (48 months).
Other Names:
  • Zorblisa
  • SD-101




Primary Outcome Measures :
  1. 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 1440 days ]
    The primary objective is to demonstrate the longterm safety of ZORBLISA in patients, with Simplex, Recessive Dystrophic, and Junctional non-Herlitz Epidermolysis Bullosa. Results will be summarized using descriptive statistics. Summary statistics to be used include proportion of patients with complete closure of their target lesion at each visit, mean and standard deviation of BSAI compared with baseline at each visit, and incidence rates of specific adverse events. Additional evaluations may be made to compare responses of patients in this extension study with their responses in SD-005. For those patients randomized to SD-101-6.0 in SD-005, the use of summary statistics as described in the previous paragraph will provide an understanding of the long term effect of SD-101-6.0 in these patients. For those patients randomized to the control arm in SD-005, summary statistics will be used to compare their response to SD-101-6.0 in SD-006 with their response to control in SD-005.


Secondary Outcome Measures :
  1. Changes in Body Surface Area (BSA) of lesional skin and wound burden using BSAI [ Time Frame: Months 1, 3, 6, 9 12,15,18, 21, 24, 27, 30, 33, 36, 39, 42, 25, and 48 ]
    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. Change in lesional skin based on BSA estimates compared to Baseline will be measured using the Body Surface Area Index (BSAI). Change in total body wound coverage based on BSA estimates compared to Baseline will be measured using the BSAI.



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Ages Eligible for Study:   1 Month and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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).

Exclusion Criteria:

  • 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 and repeated at Visit 1 if these visits do no occur on the same day)
  • Females of childbearing potential who are not abstinent or not practicing a medically acceptable method of contraception

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02670330


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Sponsors and Collaborators
Scioderm, Inc.
Amicus Therapeutics
Investigators
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Study Director: Medical Monitor Amicus Therapeutics

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Responsible Party: Scioderm, Inc.
ClinicalTrials.gov Identifier: NCT02670330     History of Changes
Other Study ID Numbers: SD-006
2014-005679-96 ( EudraCT Number )
First Posted: February 1, 2016    Key Record Dates
Last Update Posted: October 2, 2018
Last Verified: September 2018

Keywords provided by Scioderm, Inc.:
Epidermolysis Bullosa
Simplex
Recessive Dystrophic
Junctional non-Herlitz

Additional relevant MeSH terms:
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Epidermolysis Bullosa
Skin Abnormalities
Congenital Abnormalities
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Skin Diseases, Vesiculobullous