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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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
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
Results First Posted : August 2, 2019
Last Update Posted : September 27, 2019
Sponsor:
Collaborator:
Amicus Therapeutics
Information provided by (Responsible Party):
Scioderm, Inc.

Brief Summary:
The study aimed to assess the long-term safety of topical use of Zorblisa (SD-101-6.0) in participants with Epidermolysis Bullosa (EB).

Condition or disease Intervention/treatment Phase
Epidermolysis Bullosa Drug: SD-101-6.0 cream Phase 3

Detailed Description:

This was an open label, multi-center extension study to assess the long-term safety of topically applied SD-101-6.0 in participants with simplex, recessive dystrophic, and junctional non-Herlitz EB. SD-101-6.0 was applied topically once a day to the entire body. The planned duration of treatment with SD-101-6.0 for Study SD-006 was up to 48 months, with a safety follow-up period of 30 days; however, the study was terminated early by the sponsor. The maximum study duration completed by at least 1 participant, treatment and safety follow-up, was 37 months.

Participants who successfully completed Study SD-005 had the option to rollover into Study SD-006. The screening/baseline visit (Visit 1) occurred at Visit 5 (approximately 90 days from baseline) of Study SD-005. The Body Surface Area (BSA) assessments of lesional skin and wound burden performed at Visit 5 (approximately 90 days from baseline) for Study SD-005 were utilized as the baseline assessments for Study SD-006. Participants returned for follow-up visits at Month 1 then every 3 months.

At each visit, assessments included 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 was used as the baseline area size of the target wound for Study SD-006. These unhealed target wounds from Study SD-005 were assessed via ARANZ SilhouetteStar™ at each subsequent scheduled visit until the target wound was documented as closed. Closed wounds were assessed for scarring.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 152 participants
Allocation: N/A
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 9, 2015
Actual Primary Completion Date : September 3, 2018
Actual Study Completion Date : September 3, 2018


Arm Intervention/treatment
Experimental: Experimental: SD-101-6.0 cream
All participants (or their caregivers) applied SD-101-6.0 cream topically once a day to the entire body for a period of up to 36 months.
Drug: SD-101-6.0 cream
SD-101 is a white, crystalline powder that is formulated within an odorless, soft, white cream base. SD-101-6.0 cream contains allantoin, a diureide glyoxylic acid, at a concentration of 6% and other excipients.
Other Names:
  • Zorblisa
  • SD-101




Primary Outcome Measures :
  1. Number Of Participants With Treatment Emergent Adverse Events (TEAEs) [ Time Frame: From baseline to 30 days after last application of study drug (up to a maximum of 37 months) ]
    TEAEs were defined as adverse events that started or worsened on or after baseline visit.


Secondary Outcome Measures :
  1. Change From Baseline In Body Surface Area Index (BSAI) Of Lesional Skin Up To Month 30 [ Time Frame: Baseline, up to Month 30 ]
    Lesional skin was defined as areas that contained any of the following: blisters, erosions, ulcerations, scabbing, bullae, or eschars, as well as areas that were weeping, sloughing, oozing, crusted, or denuded. The percentage, ranging from 0% to 100%, of affected body surface area (BSA) was recorded for each defined body region (that is, head/neck, upper limbs, trunk [includes groin], and lower limbs), multiplied by the weighting factor, then summed for all body regions to calculate the BSAI that would range from 0% to 100%. The BSA for lesional skin was to be assessed by the same study physician on each visit for a particular participant. The mean change from baseline in BSAI was assessed every 3 months. Only participants with data available for analysis at each time point are presented.

  2. Change From Baseline In BSAI Of Total Body Wound Burden Up To Month 30 [ Time Frame: Baseline, up to Month 30 ]

    A wound was defined as an open area on the skin (that is, epidermal covering disrupted). Total body wound burden was calculated using BSAI; the percentage, ranging from 0% to 100%, of affected BSA was recorded for each defined body region (that is, head/neck, upper limbs, trunk [includes groin], and lower limbs), multiplied by the weighting factor, then summed for all body regions to calculate the BSAI that would range from 0% to 100%. The BSAI for total body wound burden was to be assessed by the same study physician at each visit for a particular participant.

    The mean change from baseline in total body wound burden was assessed every 3 months. Only participants with data available for analysis at each time point are presented.




Information from the National Library of Medicine

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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 participant or participant's legal representative; if the participant was under the age of 18 but capable of providing assent, signed assent from the participant.
  • Participant (or caretaker) must have been willing to comply with all protocol requirements.
  • Participants who completed the SD-005 study (on study drug at Visit 5, approximately 90 days from baseline).

Exclusion Criteria:

  • Participants who did not meet the entry criteria outlined above.
  • Pregnancy or breastfeeding during the study. (A urine pregnancy test was performed at the final visit for Study SD-005 for female participants of childbearing potential and repeated at screening/baseline visit of Study SD-006 if these visits did not occur on the same day).
  • Female participants of childbearing potential who were 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


Locations
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United States, Arizona
Phoenix, Arizona, United States, 85016
United States, California
Redwood City, California, United States, 94063
United States, Colorado
Aurora, Colorado, United States, 80045
United States, District of Columbia
Washington, District of Columbia, United States, 20016
United States, Illinois
Chicago, Illinois, United States, 60611
United States, Minnesota
Minneapolis, Minnesota, United States, 55455
United States, Missouri
Columbia, Missouri, United States, 65212
Saint Louis, Missouri, United States, 63110
United States, New York
East Setauket, New York, United States, 11733
United States, North Carolina
Chapel Hill, North Carolina, United States, 27516
United States, Ohio
Cincinnati, Ohio, United States, 45229-3039
United States, Pennsylvania
Philadelphia, Pennsylvania, United States, 19104
United States, South Carolina
Charleston, South Carolina, United States, 29425-5780
United States, Texas
San Antonio, Texas, United States, 78218
United States, Washington
Seattle, Washington, United States, 98105
Australia, New South Wales
Kogarah, New South Wales, Australia, 2217
Australia, Victoria
Parkville, Victoria, Australia, 3052
Austria
Salzburg, Austria, 5020
France
Nice, France, 1-06202
Paris, France, 75015
Toulouse, France
Germany
Freiburg im Breisgau, Germany, 79104
Hannover, Germany, 30173
Israel
Tel Aviv, Israel
Lithuania
Kaunas, Lithuania, LT-50009
Netherlands
Groningen, Netherlands, 9713 GZ
Poland
Warsaw, Poland
Serbia
Belgrade, Serbia
Spain
Madrid, Spain
United Kingdom
London, United Kingdom, WC1N 3JH
Sponsors and Collaborators
Scioderm, Inc.
Amicus Therapeutics
Investigators
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Study Director: Medical Monitor Amicus Therapeutics
  Study Documents (Full-Text)

Documents provided by Scioderm, Inc.:
Study Protocol  [PDF] November 10, 2016
Statistical Analysis Plan  [PDF] August 22, 2018

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Responsible Party: Scioderm, Inc.
ClinicalTrials.gov Identifier: NCT02670330    
Other Study ID Numbers: SD-006
2014-005679-96 ( EudraCT Number )
First Posted: February 1, 2016    Key Record Dates
Results First Posted: August 2, 2019
Last Update Posted: September 27, 2019
Last Verified: September 2019
Keywords provided by Scioderm, Inc.:
Amicus Therapeutics
Scioderm, Inc.
Simplex
Recessive Dystrophic
Junctional non-Herlitz
Epidermolysis Bullosa
SD-101 6%
SD-101-6.0
Allantoin 6%
Zorblisa
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