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Open-Label Extension Study to Evaluate the Safety of SD-101 Cream in Participants 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. Identifier: NCT02090283
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 : March 18, 2014
Results First Posted : November 4, 2019
Last Update Posted : November 4, 2019
Amicus Therapeutics
Food and Drug Administration (FDA)
Information provided by (Responsible Party):
Scioderm, Inc.

Brief Summary:

The purpose of this study was to assess the continued safety of topical use of SD-101 cream (6%) in participants with Epidermolysis Bullosa (EB).

Funding Source: FDA Office of Orphan Products Development

Condition or disease Intervention/treatment Phase
Epidermolysis Bullosa Drug: SD-101 dermal cream (6%) Phase 2

Detailed Description:

This was an open-label extension study to assess the continued safety of topically applied SD-101 dermal cream (6%) in participants with Simplex, Recessive Dystrophic, and Junctional non-Herlitz EB.

SD-101 dermal cream (6%) was applied topically, once a day to the entire body for the duration of the study. Participants who successfully completed the entire SD-003 study (NCT02014376) had the option to roll over into the SD-004 study (NCT02090283). The baseline visit occurred at the final visit date for the SD-003 study. The body surface area (BSA) coverage of blisters and lesions assessment made at the final SD-003 study visit were used as the baseline information at the baseline visit for the SD-004 study. Participants returned to the study site for the following 13 visits (36 months) to have BSA assessed. BSA was assessed at all subsequent scheduled study center visits. Scheduled study center visits occurred every 6 months after Month 36 (Month 42, 48, and so on). After completion of Month 36, the next participant visit (Month 39) was a phone call from the site to the participant. Telephone visits occurred every 6 months thereafter (Month 45, 51, and so on) and included assessment of adverse events and concomitant medications only. For female participants of childbearing potential, a urine pregnancy test was performed at Month 6 and every 6 months up to and including the final study visit.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 42 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open Label Extension, Multi-Center, Study to Evaluate the Safety of SD-101 Cream in Subjects With Epidermolysis Bullosa
Actual Study Start Date : March 26, 2014
Actual Primary Completion Date : September 14, 2018
Actual Study Completion Date : September 14, 2018

Arm Intervention/treatment
Experimental: SD-101 Dermal Cream (6%)
All participants applied SD-101 dermal cream topically, once a day, to the entire body for the duration of the study.
Drug: SD-101 dermal cream (6%)
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:
  • SD-101
  • SD-101-6.0
  • Zorblisa

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 54 months) ]
    Treatment-emergent adverse events were defined as adverse events that started or worsened on or after baseline visit, which occurred at the final visit date for the SD-003 study. A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module.

Secondary Outcome Measures :
  1. Change From Baseline In Body Surface Area Index (BSAI) Of Lesional Skin At Month 24 [ Time Frame: Baseline, Month 24 ]
    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, and then summed for all body regions to calculate the BSAI. 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 (final visit from the SD-003 study) in BSAI was assessed every 3 months. Only participants with data available for analysis at the specified time point are presented.

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   6 Months and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Informed consent form signed by the participant or participant's legal representative; if the participant is under the age of 18 but capable of providing assent, signed assent from the participant.
  • Participant (or caretaker) must be willing to comply with all protocol requirements.
  • Participant must have successfully completed the SD-003 study.

Exclusion Criteria:

  • Participants who do not meet the inclusion criteria.
  • Pregnancy or breastfeeding during the study. A urine pregnancy test will be performed at the final visit for SD-003 for female participants of childbearing potential.
  • Females of childbearing potential who are not abstinent and 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 identifier (NCT number): NCT02090283

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United States, California
Palo Alto, California, United States, 94304
United States, Illinois
Chicago, Illinois, United States, 60611
United States, Missouri
Saint Louis, Missouri, United States, 63110
United States, New Jersey
Hackensack, New Jersey, United States, 07601
United States, North Carolina
Chapel Hill, North Carolina, United States, 27516
United States, Texas
San Antonio, Texas, United States, 78218
United States, Washington
Seattle, Washington, United States, 98105
Sponsors and Collaborators
Scioderm, Inc.
Amicus Therapeutics
Food and Drug Administration (FDA)
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Study Director: Medical Monitor Amicus Therapeutics
  Study Documents (Full-Text)

Documents provided by Scioderm, Inc.:
Study Protocol  [PDF] January 25, 2017
Statistical Analysis Plan  [PDF] December 20, 2018

Publications of Results:
Paller A., Browning J., Aslam R., et al. Efficacy and Safety Results From a 24-Month, Open-Label Extension of a Phase 2b Dose-Ranging Study of SD-101 Cream in Patients With Epidermolysis Bullosa. Abstract presented at European Academy of Dermatology and Venereology, 26th Congress, Geneva, Switzerland, September 13-17, 2017.

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Responsible Party: Scioderm, Inc. Identifier: NCT02090283    
Other Study ID Numbers: SD-004
1R01FD005093-01 ( U.S. FDA Grant/Contract )
First Posted: March 18, 2014    Key Record Dates
Results First Posted: November 4, 2019
Last Update Posted: November 4, 2019
Last Verified: October 2019
Keywords provided by Scioderm, Inc.:
Amicus Therapeutics
Scioderm, Inc.
Recessive Dystrophic
Junctional non-Herlitz
Epidermolysis Bullosa
SD-101 6%
Allantoin 6%
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