Clinical trial NCT02670330: Open Label Extension Study to Evaluate the Long-term Safety of Zorblisa (SD-101-6.0) in Patients With Epidermolysis Bullosa
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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Epidermolysis Bullosa
Keywords: Amicus Therapeutics
Scioderm, Inc.
Simplex
Recessive Dystrophic
Junctional non-Herlitz
Epidermolysis Bullosa
SD-101 6%
SD-101-6.0
Allantoin 6%
Zorblisa
Lead sponsor: Scioderm, Inc.
Sponsor class: INDUSTRY
Collaborators: name: Amicus Therapeutics
First posted: 2016-02-01
Enrollment: 152
Sex: ALL
Minimum age (years): 1
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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).

Detailed 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.

Eligibility criteria: 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.

Primary outcomes:
measure: Number Of Participants With Treatment Emergent Adverse Events (TEAEs)
timeFrame: From baseline to 30 days after last application of study drug (up to a maximum of 37 months)
Secondary outcomes:
measure: Change From Baseline In Body Surface Area Index (BSAI) Of Lesional Skin Up To Month 30
timeFrame: Baseline, up to Month 30
measure: Change From Baseline In BSAI Of Total Body Wound Burden Up To Month 30
timeFrame: Baseline, up to Month 30
Other outcomes:
Locations:
city: Phoenix
country: United States
state: Arizona
city: Redwood City
country: United States
state: California
city: Aurora
country: United States
state: Colorado
city: Washington
country: United States
state: District of Columbia
city: Chicago
country: United States
state: Illinois
city: Minneapolis
country: United States
state: Minnesota
city: Columbia
country: United States
state: Missouri
city: Saint Louis
country: United States
state: Missouri
city: East Setauket
country: United States
state: New York
city: Chapel Hill
country: United States
state: North Carolina
city: Cincinnati
country: United States
state: Ohio
city: Philadelphia
country: United States
state: Pennsylvania
city: Charleston
country: United States
state: South Carolina
city: San Antonio
country: United States
state: Texas
city: Seattle
country: United States
state: Washington
city: Kogarah
country: Australia
state: New South Wales
city: Parkville
country: Australia
state: Victoria
city: Salzburg
country: Austria
city: Nice
country: France
city: Paris
country: France
city: Toulouse
country: France
city: Freiburg im Breisgau
country: Germany
city: Hannover
country: Germany
city: Tel Aviv
country: Israel
city: Kaunas
country: Lithuania
city: Groningen
country: Netherlands
city: Warsaw
country: Poland
city: Belgrade
country: Serbia
city: Madrid
country: Spain
city: London
country: United Kingdom
Study officials:
affiliation: Amicus Therapeutics
name: Medical Monitor
role: STUDY_DIRECTOR
References: