Clinical trial NCT02090283: Open-Label Extension Study to Evaluate the Safety of SD-101 Cream in Participants With Epidermolysis Bullosa
Official title: An Open Label Extension, Multi-Center, Study to Evaluate the Safety of SD-101 Cream in Subjects With Epidermolysis Bullosa
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Epidermolysis Bullosa
Keywords: Amicus Therapeutics
Scioderm, Inc.
Recessive Dystrophic
Junctional non-Herlitz
Epidermolysis Bullosa
Simplex
SD-101 6%
SD-101-6.0
Allantoin 6%
Zorblisa
Lead sponsor: Scioderm, Inc.
Sponsor class: INDUSTRY
Collaborators: name: Amicus Therapeutics
name: Food and Drug Administration (FDA)
First posted: 2014-03-18
Enrollment: 42
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study was to assess the continued safety of topical use of SD-101 cream (6%) in participants with Epidermolysis Bullosa (EB).

Funding Source: FDA Office of Orphan Products Development

Detailed description: This was an open-label extension study to assess the continued safety of topically applied SD-101 dermal cream (6%) in participants with Simplex, Recessive Dystrophic, and Junctional non-Herlitz EB.

SD-101 dermal cream (6%) was applied topically, once a day to the entire body for the duration of the study. Participants who successfully completed the entire SD-003 study (NCT02014376) had the option to roll over into the SD-004 study (NCT02090283). The baseline visit occurred at the final visit date for the SD-003 study. The body surface area (BSA) coverage of blisters and lesions assessment made at the final SD-003 study visit were used as the baseline information at the baseline visit for the SD-004 study. Participants returned to the study site for the following 13 visits (36 months) to have BSA assessed. BSA was assessed at all subsequent scheduled study center visits. Scheduled study center visits occurred every 6 months after Month 36 (Month 42, 48, and so on). After completion of Month 36, the next participant visit (Month 39) was a phone call from the site to the participant. Telephone visits occurred every 6 months thereafter (Month 45, 51, and so on) and included assessment of adverse events and concomitant medications only. For female participants of childbearing potential, a urine pregnancy test was performed at Month 6 and every 6 months up to and including the final study visit.

Eligibility criteria: Inclusion Criteria:

* Informed consent form signed by the participant or participant's legal representative; if the participant is under the age of 18 but capable of providing assent, signed assent from the participant.
* Participant (or caretaker) must be willing to comply with all protocol requirements.
* Participant must have successfully completed the SD-003 study.

Exclusion Criteria:

* Participants who do not meet the inclusion criteria.
* Pregnancy or breastfeeding during the study. A urine pregnancy test will be performed at the final visit for SD-003 for female participants of childbearing potential.
* Females of childbearing potential who are not abstinent and 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 54 months)
Secondary outcomes:
measure: Change From Baseline In Body Surface Area Index (BSAI) Of Lesional Skin At Month 24
timeFrame: Baseline, Month 24
Other outcomes:
Locations:
city: Palo Alto
country: United States
state: California
city: Chicago
country: United States
state: Illinois
city: Saint Louis
country: United States
state: Missouri
city: Hackensack
country: United States
state: New Jersey
city: Chapel Hill
country: United States
state: North Carolina
city: San Antonio
country: United States
state: Texas
city: Seattle
country: United States
state: Washington
Study officials:
affiliation: Amicus Therapeutics
name: Medical Monitor
role: STUDY_DIRECTOR
References:
citation: Paller A., Browning J., Aslam R., et al. Efficacy and Safety Results From a 24-Month, Open-Label Extension of a Phase 2b Dose-Ranging Study of SD-101 Cream in Patients With Epidermolysis Bullosa. Abstract presented at European Academy of Dermatology and Venereology, 26th Congress, Geneva, Switzerland, September 13-17, 2017.