Open Label Extension Study to Evaluate the Long-term Safety of Zorblisa (SD-101-6.0) in Patients With Epidermolysis Bullosa

Epidermolysis Bullosa Clinical Trial

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT02670330
• Other study ID #: SD-006
• Secondary ID: 2014-005679-96
• Status: Terminated
• Phase: Phase 3
• Start date: June 9, 2015
• Est. completion date: September 3, 2018

Study information

• Verified date: September 2019
• Source: Scioderm, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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.

Recruitment information / eligibility

• 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.

Related Conditions & MeSH terms

• Epidermolysis Bullosa

Intervention

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.

Locations

Country	Name	City	State
n/a

Sponsors (2)

Lead Sponsor	Collaborator
Scioderm, Inc.	Amicus Therapeutics

Countries where clinical trial is conducted

United States,  Australia,  Austria,  France,  Germany,  Israel,  Lithuania,  Netherlands,  Poland,  Serbia,  Spain,  United Kingdom, 

Outcome

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