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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/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
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
Secondary ID [1] 0 0
2014-002288-14
Secondary ID [2] 0 0
SD-005
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Epidermolysis Bullosa 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Skin 0 0 0 0
Dermatological conditions
Skin 0 0 0 0
Other skin conditions

Intervention/exposure
Study type
Interventional
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
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Time To Complete Target Wound Closure Within 3 Months
Timepoint [1] 0 0
From baseline to Month 3 visit
Primary outcome [2] 0 0
The Percentage Of Participants Experiencing Complete Closure Of Their Target Wound Within 3 Months
Timepoint [2] 0 0
From baseline to Month 3 visit
Secondary outcome [1] 0 0
Percentage Of Participants Experiencing Complete Closure Of Their Target Wound At Month 1 And Month 2 Visits
Timepoint [1] 0 0
From baseline to Month 1 and Month 2 visits
Secondary outcome [2] 0 0
Change From Baseline In Body Surface Area Index (BSAI) Of Lesional Skin At Month 3 Visit
Timepoint [2] 0 0
Baseline, Month 3 visit
Secondary outcome [3] 0 0
Change From Baseline In BSAI Of Total Body Wound Burden At Month 3 Visit
Timepoint [3] 0 0
Baseline, Month 3 visit
Secondary outcome [4] 0 0
Change From Baseline In Itching Score At Day 7
Timepoint [4] 0 0
Baseline, Day 7
Secondary outcome [5] 0 0
Change From Baseline In Pain Score At Day 7
Timepoint [5] 0 0
Baseline, Day 7

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.
Minimum age
1 Month
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
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
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
11/03/2015
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
5/07/2017
Sample size
Target
Accrual to date
Final
169
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
- Kogarah
Recruitment hospital [2] 0 0
- Parkville
Recruitment postcode(s) [1] 0 0
2217 - Kogarah
Recruitment postcode(s) [2] 0 0
3050 - Parkville
Recruitment postcode(s) [3] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Colorado
Country [4] 0 0
United States of America
State/province [4] 0 0
District of Columbia
Country [5] 0 0
United States of America
State/province [5] 0 0
Florida
Country [6] 0 0
United States of America
State/province [6] 0 0
Illinois
Country [7] 0 0
United States of America
State/province [7] 0 0
Minnesota
Country [8] 0 0
United States of America
State/province [8] 0 0
Missouri
Country [9] 0 0
United States of America
State/province [9] 0 0
New Hampshire
Country [10] 0 0
United States of America
State/province [10] 0 0
New Jersey
Country [11] 0 0
United States of America
State/province [11] 0 0
New York
Country [12] 0 0
United States of America
State/province [12] 0 0
North Carolina
Country [13] 0 0
United States of America
State/province [13] 0 0
Ohio
Country [14] 0 0
United States of America
State/province [14] 0 0
Pennsylvania
Country [15] 0 0
United States of America
State/province [15] 0 0
South Carolina
Country [16] 0 0
United States of America
State/province [16] 0 0
Texas
Country [17] 0 0
United States of America
State/province [17] 0 0
Washington
Country [18] 0 0
Austria
State/province [18] 0 0
Salzburg
Country [19] 0 0
France
State/province [19] 0 0
Nice
Country [20] 0 0
France
State/province [20] 0 0
Paris
Country [21] 0 0
France
State/province [21] 0 0
Toulouse
Country [22] 0 0
Germany
State/province [22] 0 0
Freiburg
Country [23] 0 0
Germany
State/province [23] 0 0
Hannover
Country [24] 0 0
Israel
State/province [24] 0 0
Tel Aviv
Country [25] 0 0
Italy
State/province [25] 0 0
Milano
Country [26] 0 0
Lithuania
State/province [26] 0 0
Kaunas
Country [27] 0 0
Netherlands
State/province [27] 0 0
Groningen
Country [28] 0 0
Poland
State/province [28] 0 0
Koszykowa
Country [29] 0 0
Serbia
State/province [29] 0 0
Belgrade
Country [30] 0 0
Spain
State/province [30] 0 0
Madrid
Country [31] 0 0
United Kingdom
State/province [31] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Scioderm, Inc.
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
Amicus Therapeutics
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medical Monitor
Address 0 0
Amicus Therapeutics
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results are available at https://clinicaltrials.gov/study/NCT02384460