Epidermolysis Bullosa Clinical Trial
Official title:
An Open Label Multi-Center Extension Study to Evaluate the Long-term Safety of Zorblisaâ„¢ (SD-101-6.0) in Patients With Epidermolysis Bullosa
Verified date | September 2019 |
Source | Scioderm, Inc. |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
The study aimed to assess the long-term safety of topical use of Zorblisa (SD-101-6.0) in participants with Epidermolysis Bullosa (EB).
Status | Terminated |
Enrollment | 152 |
Est. completion date | September 3, 2018 |
Est. primary completion date | September 3, 2018 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 1 Month and older |
Eligibility |
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. |
Country | Name | City | State |
---|---|---|---|
n/a |
Lead Sponsor | Collaborator |
---|---|
Scioderm, Inc. | Amicus Therapeutics |
United States, Australia, Austria, France, Germany, Israel, Lithuania, Netherlands, Poland, Serbia, Spain, United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Number Of Participants With Treatment Emergent Adverse Events (TEAEs) | TEAEs were defined as adverse events that started or worsened on or after baseline visit. | From baseline to 30 days after last application of study drug (up to a maximum of 37 months) | |
Secondary | Change From Baseline In Body Surface Area Index (BSAI) Of Lesional Skin 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. | Baseline, up to Month 30 | |
Secondary | Change From Baseline In BSAI Of Total Body Wound Burden 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. |
Baseline, up to Month 30 |
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