ESSENCE Study: Efficacy and Safety of SD-101 Cream in Participants With Epidermolysis Bullosa
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
3 other identifiers
interventional
169
12 countries
37
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).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_3
Started Mar 2015
37 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
February 13, 2015
CompletedFirst Posted
Study publicly available on registry
March 10, 2015
CompletedStudy Start
First participant enrolled
March 11, 2015
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 5, 2017
CompletedStudy Completion
Last participant's last visit for all outcomes
July 5, 2017
CompletedResults Posted
Study results publicly available
October 16, 2018
CompletedApril 9, 2020
April 1, 2020
2.3 years
February 13, 2015
September 19, 2018
April 1, 2020
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
Time To Complete Target Wound Closure Within 3 Months
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.
From baseline to Month 3 visit
The Percentage Of Participants Experiencing Complete Closure Of Their Target Wound Within 3 Months
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.
From baseline to Month 3 visit
Secondary Outcomes (5)
Percentage Of Participants Experiencing Complete Closure Of Their Target Wound At Month 1 And Month 2 Visits
From baseline to Month 1 and Month 2 visits
Change From Baseline In Body Surface Area Index (BSAI) Of Lesional Skin At Month 3 Visit
Baseline, Month 3 visit
Change From Baseline In BSAI Of Total Body Wound Burden At Month 3 Visit
Baseline, Month 3 visit
Change From Baseline In Itching Score At Day 7
Baseline, Day 7
Change From Baseline In Pain Score At Day 7
Baseline, Day 7
Study Arms (2)
SD-101-6.0 cream
EXPERIMENTALSD-101-6.0 cream applied topically, once a day to the entire body for a period of 90 days
Placebo (SD-101-0.0) cream
PLACEBO COMPARATORSD-101-0.0 (placebo) cream applied topically, once a day to the entire body for a period of 90 days
Interventions
applied topically once a day for 90 days
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Scioderm, Inc.lead
- Amicus Therapeuticscollaborator
Study Sites (38)
Unknown Facility
Phoenix, Arizona, 85016, United States
Unknown Facility
Redwood City, California, 94063, United States
Unknown Facility
Aurora, Colorado, 80045, United States
Unknown Facility
Washington D.C., District of Columbia, 20016, United States
Unknown Facility
Miami, Florida, 33136, United States
Unknown Facility
Miami, Florida, 33155, United States
Unknown Facility
Chicago, Illinois, 60611, United States
Unknown Facility
Minneapolis, Minnesota, 55455, United States
Unknown Facility
Columbia, Missouri, 65212, United States
Unknown Facility
St Louis, Missouri, 63110, United States
Unknown Facility
Lebanon, New Hampshire, 03756, United States
Unknown Facility
Hackensack, New Jersey, 07601, United States
Unknown Facility
East Setauket, New York, 11733, United States
Unknown Facility
New York, New York, 10032, United States
Unknown Facility
Chapel Hill, North Carolina, 27516, United States
Unknown Facility
Cincinnati, Ohio, 45229-3039, United States
Unknown Facility
Philadelphia, Pennsylvania, 19104, United States
Unknown Facility
Charleston, South Carolina, 29425-5780, United States
Unknown Facility
Austin, Texas, 78723, United States
Unknown Facility
San Antonio, Texas, 78218, United States
Unknown Facility
Seattle, Washington, 98105, United States
Unknown Facility
Kogarah, New South Wales, 2217, Australia
Unknown Facility
Parkville, Victoria, 3050, Australia
Unknown Facility
Parkville, Victoria, 3052, Australia
Unknown Facility
Salzburg, 5020, Austria
Unknown Facility
Nice, 0-06200, France
Unknown Facility
Paris, 75015, France
Unknown Facility
Toulouse, France
Unknown Facility
Freiburg im Breisgau, Germany
Unknown Facility
Hanover, Germany
Unknown Facility
Tel Aviv, Israel
Unknown Facility
Milan, 20122, Italy
Unknown Facility
Kaunas, Lithuania
Unknown Facility
Groningen, 9713 GZ, Netherlands
Unknown Facility
Warsaw, Koszykowa, Poland
Unknown Facility
Belgrade, Serbia
Unknown Facility
Madrid, Spain
Unknown Facility
London, WC1N 3JH, United Kingdom
Related Publications (2)
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.
PMID: 32693833DERIVEDPaller 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.
PMID: 32576219DERIVED
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Patient Advocacy
- Organization
- Amicus Therapeutics, Inc.
Study Officials
- STUDY DIRECTOR
Medical Monitor
Amicus Therapeutics
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
February 13, 2015
First Posted
March 10, 2015
Study Start
March 11, 2015
Primary Completion
July 5, 2017
Study Completion
July 5, 2017
Last Updated
April 9, 2020
Results First Posted
October 16, 2018
Record last verified: 2020-04