Clinical trial NCT02384460: ESSENCE Study: Efficacy and Safety of SD-101 Cream in Participants With Epidermolysis Bullosa
Official title: A Phase 3, Multi-center, Randomized, Double-Blind, Placebo Controlled Study of the Efficacy and Safety of SD-101 Cream in Patients With Epidermolysis Bullosa
Study type: INTERVENTIONAL
Overall status: COMPLETED
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: 2015-03-10
Enrollment: 169
Sex: ALL
Minimum age (years): 1
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The aim was to assess the efficacy and safety of SD-101-6.0 cream versus Placebo (SD-101-0.0) cream in the treatment of skin lesions in participants with Epidermolysis Bullosa. Funding Source - United States Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD).

Detailed description: This was a Phase 3, multi-center, randomized, double-blind, placebo-controlled study to assess the efficacy and safety of SD-101-6.0 cream versus placebo (SD-101-0.0) cream on skin lesions in participants with Simplex, Recessive Dystrophic, or Junctional non-Herlitz Epidermolysis Bullosa. Epidermolysis Bullosa is a rare group of inherited disorders that typically manifest at birth as blistering and lesion formation on the skin in response to little or no apparent trauma. In this study, SD-101-6.0 cream or placebo (SD-101-0.0) cream was to be applied topically, once a day to the entire body for a period of 90 days. Participants had 1 target wound selected at baseline by the investigator. The selected target wound was required to have been present for at least 21 days. Photographic confirmation of the target wound location was collected at baseline, and the picture saved from the first visit was used to confirm location of the target wound at subsequent visits. The participant returned to the study site for Visit 2 (approximately 14 days from baseline), Visit 3 (approximately 30 days from baseline), Visit 4 (approximately 60 days from baseline), and Visit 5 (approximately 90 days from baseline) to have the target wound assessed for the level of healing. In addition, itching, pain, body surface area, target wound closure, and scarring of healed target wound were assessed at each visit. The ARANZ SilhouetteStar™ was used to measure the target wound at all visits.

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.
* Diagnosis of Simplex, Recessive Dystrophic, or Junctional non-Herlitz EB.
* Participant must have had 1 target wound (size 10 to 50 cm\^2) at study entry.
* Participants 1 month and older.
* Target wound must have been present for at least 21 days.

Exclusion Criteria:

* Participants who did not meet the entry criteria outlined above.
* Selected target wound did not have clinical evidence of local infection.
* Use of any investigational drug within the 30 days before enrollment.
* Use of immunotherapy or cytotoxic chemotherapy within the 60 days before enrollment.
* Use of systemic or topical steroidal therapy within the 30 days before enrollment. (Inhaled steroids and ophthalmic drops containing steroids were allowed).
* Use of systemic antibiotics within the 7 days before enrollment.
* Current or former malignancy.
* Arterial or venous disorder resulting in ulcerated lesions.
* Pregnancy or breastfeeding during the study. (A urine pregnancy test was performed at screening and every 30 days until the final visit for female participants of childbearing potential).
* Females of childbearing potential who were not abstinent and not practicing a medically acceptable method of contraception.

Primary outcomes:
measure: Time To Complete Target Wound Closure Within 3 Months
timeFrame: From baseline to Month 3 visit
measure: The Percentage Of Participants Experiencing Complete Closure Of Their Target Wound Within 3 Months
timeFrame: From baseline to Month 3 visit
Secondary outcomes:
measure: Percentage Of Participants Experiencing Complete Closure Of Their Target Wound At Month 1 And Month 2 Visits
timeFrame: From baseline to Month 1 and Month 2 visits
measure: Change From Baseline In Body Surface Area Index (BSAI) Of Lesional Skin At Month 3 Visit
timeFrame: Baseline, Month 3 visit
measure: Change From Baseline In BSAI Of Total Body Wound Burden At Month 3 Visit
timeFrame: Baseline, Month 3 visit
measure: Change From Baseline In Itching Score At Day 7
timeFrame: Baseline, Day 7
measure: Change From Baseline In Pain Score At Day 7
timeFrame: Baseline, Day 7
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: Miami
country: United States
state: Florida
city: Miami
country: United States
state: Florida
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: Lebanon
country: United States
state: New Hampshire
city: Hackensack
country: United States
state: New Jersey
city: East Setauket
country: United States
state: New York
city: New York
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: Austin
country: United States
state: Texas
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: Parkville
country: Australia
state: Victoria
city: Salzburg
country: Austria
city: Nice
country: France
city: Paris
country: France
city: Toulouse
country: France
city: Freiburg
country: Germany
city: Hannover
country: Germany
city: Tel Aviv
country: Israel
city: Milano
country: Italy
city: Kaunas
country: Lithuania
city: Groningen
country: Netherlands
city: Warszawa
country: Poland
state: Koszykowa
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:
citation: Murrell DF, Paller AS, Bodemer C, Browning J, Nikolic M, Barth JA, Lagast H, Krusinska E, Reha A; ESSENCE Study Group. Wound closure in epidermolysis bullosa: data from the vehicle arm of the phase 3 ESSENCE Study. Orphanet J Rare Dis. 2020 Jul 21;15(1):190. doi: 10.1186/s13023-020-01435-3.
citation: Paller AS, Browning J, Nikolic M, Bodemer C, Murrell DF, Lenon W, Krusinska E, Reha A, Lagast H, Barth JA; ESSENCE Study Group. Efficacy and tolerability of the investigational topical cream SD-101 (6% allantoin) in patients with epidermolysis bullosa: a phase 3, randomized, double-blind, vehicle-controlled trial (ESSENCE study). Orphanet J Rare Dis. 2020 Jun 23;15(1):158. doi: 10.1186/s13023-020-01419-3.