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
Results First Posted : August 2, 2019
Last Update Posted : September 27, 2019
|Condition or disease||Intervention/treatment||Phase|
|Epidermolysis Bullosa||Drug: SD-101-6.0 cream||Phase 3|
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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||152 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
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.
- 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.
- 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.
- 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.
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
|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|
|Parkville, Victoria, Australia, 3052|
|Salzburg, Austria, 5020|
|Nice, France, 1-06202|
|Paris, France, 75015|
|Freiburg im Breisgau, Germany, 79104|
|Hannover, Germany, 30173|
|Tel Aviv, Israel|
|Kaunas, Lithuania, LT-50009|
|Groningen, Netherlands, 9713 GZ|
|London, United Kingdom, WC1N 3JH|
|Study Director:||Medical Monitor||Amicus Therapeutics|