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

Scioderm

Status and phase

Terminated

Phase 3

Conditions

Epidermolysis Bullosa

Treatments

Drug: SD-101-6.0 cream

Study type

Interventional

Funder types

Industry

Identifiers

NCT02670330

SD-006

2014-005679-96 (EudraCT Number)

Details and patient eligibility

About

The study aimed to assess the long-term safety of topical use of Zorblisa (SD-101-6.0) in participants with Epidermolysis Bullosa (EB).

Full 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.

Enrollment

152 patients

Sex

All

Ages

1+ month old

Volunteers

No Healthy Volunteers

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.