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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT02384460
Registration number
NCT02384460
Ethics application status
Date submitted
13/02/2015
Date registered
10/03/2015
Date last updated
9/04/2020
Titles & IDs
Public title
ESSENCE Study: Efficacy and Safety of SD-101 Cream in Participants With Epidermolysis Bullosa
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Scientific 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
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Secondary ID [1]
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2014-002288-14
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Secondary ID [2]
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SD-005
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Epidermolysis Bullosa
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Condition category
Condition code
Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Skin
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Dermatological conditions
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Skin
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Other skin conditions
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - SD-101-6.0 cream
Treatment: Drugs - Placebo (SD-101-0.0) cream
Experimental: SD-101-6.0 cream - SD-101-6.0 cream applied topically, once a day to the entire body for a period of 90 days
Placebo Comparator: Placebo (SD-101-0.0) cream - SD-101-0.0 (placebo) cream applied topically, once a day to the entire body for a period of 90 days
Treatment: Drugs: SD-101-6.0 cream
applied topically once a day for 90 days
Treatment: Drugs: Placebo (SD-101-0.0) cream
applied topically once a day for 90 days
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Time To Complete Target Wound Closure Within 3 Months
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Assessment method [1]
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Target wounds were monitored at each study visit for complete closure, defined as skin re-epithelialization without drainage. Time to target wound closure was measured from the date of the first administration of the study drug to the date of target wound closure. Participants were censored if they did not have a response within 3 months, or withdrew earlier before the confirmation of their target wound closing. This primary end point displays the mean time to complete target wound closure, analyzed using a Kaplan-Meier approach.
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Timepoint [1]
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From baseline to Month 3 visit
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Primary outcome [2]
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The Percentage Of Participants Experiencing Complete Closure Of Their Target Wound Within 3 Months
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Assessment method [2]
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Target wounds were monitored at each study visit for complete closure, defined as skin re-epithelialization without drainage. Participants were considered responders if they experienced complete wound closure at the Week 2 or Months 1, 2, or 3 visits. If a target wound was documented to have closed at a given visit, it was considered closed at all subsequent visits. This primary end point displays the percentage of participants from the ITT population who had complete target wound closure by the end of the study period (that is, 3 months). Analysis was performed on participants with post-baseline wound closure data.
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Timepoint [2]
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From baseline to Month 3 visit
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Secondary outcome [1]
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Percentage Of Participants Experiencing Complete Closure Of Their Target Wound At Month 1 And Month 2 Visits
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Assessment method [1]
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Target wounds were monitored at each study visit for complete closure, defined as skin re-epithelialization without drainage. The percentage of participants who completed target wound closure at the Month 1 and Month 2 study visits is displayed. If a target wound was documented to have closed at a given visit, it was considered closed at all subsequent visits.
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Timepoint [1]
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From baseline to Month 1 and Month 2 visits
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Secondary outcome [2]
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Change From Baseline In Body Surface Area Index (BSAI) Of Lesional Skin At Month 3 Visit
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Assessment method [2]
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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. BSAI was calculated as a percentage, ranging from 0% to 100%, of affected body surface area, 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.
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Timepoint [2]
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Baseline, Month 3 visit
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Secondary outcome [3]
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Change From Baseline In BSAI Of Total Body Wound Burden At Month 3 Visit
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Assessment method [3]
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Total body wound burden was calculated using BSAI. A wound defined as an open area on the skin (that is, epidermal covering disrupted). BSAI was calculated as a percentage, ranging from 0% to 100%, of affected body surface area, recorded for each defined body region (that is, head/neck, upper limbs, lower limbs, trunk [includes groin]), and multiplied by the weighting factor, then summed for all body regions.
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Timepoint [3]
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Baseline, Month 3 visit
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Secondary outcome [4]
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Change From Baseline In Itching Score At Day 7
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Assessment method [4]
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Itching was assessed using the 5-point Itch Man Pruritus Assessment Tool. For participants up to 5 years of age, itching was assessed using caretaker's response and participants 6 years of age and older self-reported their itching assessments based on the following scores: 0=Comfortable, no itch; 1=itches a little, does not interfere with activity; 2=itches more, sometimes interferes with activity; 3=itches a lot, difficult to be still, concentrate; 4=itches most terribly, impossible to sit still or concentrate. Itching scores were categorized into 3 groups based on improvement; Improved or No Itching, Not Improved, and Missing. An itching score reduction from baseline greater than or equal to 1 point on the scale was classed as improved.
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Timepoint [4]
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Baseline, Day 7
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Secondary outcome [5]
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Change From Baseline In Pain Score At Day 7
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Assessment method [5]
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Change in pain assessed at Day 7 compared to baseline was measured using the Face, Legs, Activity, Cry, and Consolability (FLACC) behavioral scale for participants 1 month to 3 years of age. Each of the 5 FLACC categories was scored from 0 to 2, which resulted in a total score between 0 and 10 with 0=Relaxed and comfortable, 1 to 3=Mild discomfort, 4 to 6=Moderate pain, and 7 to 10=Severe discomfort/pain. For participants 4 years of age and older, the "Wong Faces Pain Scale" was used. This scale shows a series of faces ranging from a happy face at 0, which represents "no hurt," to a crying face at 10, which represents "hurts worst". Pain scores were categorized into 3 groups based on improvement: Improved or No Pain, Not Improved, and Missing. A pain score reduction from baseline greater than or equal to 2 points on the scale was classed as improved.
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Timepoint [5]
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Baseline, Day 7
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Eligibility
Key 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.
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Minimum age
1
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key 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.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
11/03/2015
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
5/07/2017
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Sample size
Target
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Accrual to date
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Final
169
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Recruitment in Australia
Recruitment state(s)
NSW,VIC
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Recruitment hospital [1]
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- Kogarah
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Recruitment hospital [2]
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- Parkville
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Recruitment postcode(s) [1]
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2217 - Kogarah
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Recruitment postcode(s) [2]
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3050 - Parkville
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Recruitment postcode(s) [3]
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3052 - Parkville
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Recruitment outside Australia
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United States of America
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Arizona
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California
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Colorado
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District of Columbia
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Florida
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Illinois
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Minnesota
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Missouri
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New York
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Ohio
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Texas
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Washington
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Austria
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Salzburg
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France
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Nice
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France
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Paris
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France
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Toulouse
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Germany
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Freiburg
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Germany
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Hannover
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Israel
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Tel Aviv
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Italy
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Milano
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Groningen
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Poland
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Koszykowa
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Serbia
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Belgrade
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Madrid
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United Kingdom
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London
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Scioderm, Inc.
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Address
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Other collaborator category [1]
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Commercial sector/Industry
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Name [1]
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Amicus Therapeutics
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Address [1]
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Ethics approval
Ethics application status
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Summary
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).
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Trial website
https://clinicaltrials.gov/ct2/show/NCT02384460
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
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Medical Monitor
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Address
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Amicus Therapeutics
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT02384460
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