Open-Label Extension Study to Evaluate the Safety of SD-101 Cream in Participants With Epidermolysis Bullosa
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|ClinicalTrials.gov 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
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|
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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||42 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
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.
- 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.
- 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.
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): NCT02090283
|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|
|Study Director:||Medical Monitor||Amicus Therapeutics|