- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02384460
ESSENCE Study: Efficacy and Safety of SD-101 Cream in Participants With Epidermolysis Bullosa
April 1, 2020 updated by: Scioderm, Inc.
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
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).
Study Overview
Status
Completed
Conditions
Intervention / Treatment
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.
Study Type
Interventional
Enrollment (Actual)
169
Phase
- Phase 3
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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New South Wales
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Kogarah, New South Wales, Australia, 2217
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Victoria
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Parkville, Victoria, Australia, 3050
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Parkville, Victoria, Australia, 3052
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Salzburg, Austria, 5020
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Nice, France, 0-06200
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Paris, France, 75015
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Toulouse, France
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Freiburg, Germany
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Hannover, Germany
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Tel Aviv, Israel
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Milano, Italy, 20122
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Kaunas, Lithuania
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Groningen, Netherlands, 9713 GZ
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Koszykowa
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Warszawa, Koszykowa, Poland
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Belgrade, Serbia
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Madrid, Spain
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London, United Kingdom, WC1N 3JH
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Arizona
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Phoenix, Arizona, United States, 85016
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California
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Redwood City, California, United States, 94063
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Colorado
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Aurora, Colorado, United States, 80045
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District of Columbia
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Washington, District of Columbia, United States, 20016
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Florida
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Miami, Florida, United States, 33136
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Miami, Florida, United States, 33155
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Illinois
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Chicago, Illinois, United States, 60611
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Minnesota
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Minneapolis, Minnesota, United States, 55455
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Missouri
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Columbia, Missouri, United States, 65212
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Saint Louis, Missouri, United States, 63110
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New Hampshire
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Lebanon, New Hampshire, United States, 03756
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New Jersey
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Hackensack, New Jersey, United States, 07601
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New York
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East Setauket, New York, United States, 11733
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New York, New York, United States, 10032
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North Carolina
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Chapel Hill, North Carolina, United States, 27516
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Ohio
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Cincinnati, Ohio, United States, 45229-3039
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Pennsylvania
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Philadelphia, Pennsylvania, United States, 19104
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South Carolina
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Charleston, South Carolina, United States, 29425-5780
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Texas
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Austin, Texas, United States, 78723
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San Antonio, Texas, United States, 78218
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Washington
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Seattle, Washington, United States, 98105
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
1 month and older (Child, Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
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.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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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
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applied topically once a day for 90 days
Other Names:
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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
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applied topically once a day for 90 days
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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Time To Complete Target Wound Closure Within 3 Months
Time Frame: From baseline to Month 3 visit
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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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From baseline to Month 3 visit
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The Percentage Of Participants Experiencing Complete Closure Of Their Target Wound Within 3 Months
Time Frame: From baseline to Month 3 visit
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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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From baseline to Month 3 visit
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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Percentage Of Participants Experiencing Complete Closure Of Their Target Wound At Month 1 And Month 2 Visits
Time Frame: From baseline to Month 1 and Month 2 visits
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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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From baseline to Month 1 and Month 2 visits
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Change From Baseline In Body Surface Area Index (BSAI) Of Lesional Skin At Month 3 Visit
Time Frame: Baseline, Month 3 visit
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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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Baseline, Month 3 visit
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Change From Baseline In BSAI Of Total Body Wound Burden At Month 3 Visit
Time Frame: Baseline, Month 3 visit
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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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Baseline, Month 3 visit
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Change From Baseline In Itching Score At Day 7
Time Frame: Baseline, Day 7
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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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Baseline, Day 7
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Change From Baseline In Pain Score At Day 7
Time Frame: Baseline, Day 7
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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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Baseline, Day 7
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Collaborators
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
General Publications
- 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.
- 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.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
March 11, 2015
Primary Completion (Actual)
July 5, 2017
Study Completion (Actual)
July 5, 2017
Study Registration Dates
First Submitted
February 13, 2015
First Submitted That Met QC Criteria
March 9, 2015
First Posted (Estimate)
March 10, 2015
Study Record Updates
Last Update Posted (Actual)
April 9, 2020
Last Update Submitted That Met QC Criteria
April 1, 2020
Last Verified
April 1, 2020
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- SD-005
- 2014-002288-14 (EudraCT Number)
- R01-005095-01 (Other Identifier: Orphan Product Grant)
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Epidermolysis Bullosa
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Castle Creek Pharmaceuticals, LLCCompletedDystrophic Epidermolysis Bullosa | Epidermolysis Bullosa Simplex | Junctional Epidermolysis Bullosa | Epidermolysis Bullosa (EB)United States
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Krystal Biotech, Inc.CompletedDystrophic Epidermolysis Bullosa | Recessive Dystrophic Epidermolysis Bullosa | Dominant Dystrophic Epidermolysis Bullosa | DEB - Dystrophic Epidermolysis BullosaUnited States
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Krystal Biotech, Inc.CompletedDystrophic Epidermolysis Bullosa | Recessive Dystrophic Epidermolysis Bullosa | Dominant Dystrophic Epidermolysis BullosaUnited States
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Castle Creek Biosciences, LLC.TerminatedEpidermolysis Bullosa Dystrophica, RecessiveUnited States
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Instituto de Investigación Hospital Universitario...Instituto de Salud Carlos III; Universidad Carlos III Madrid (TERMeG); St John... and other collaboratorsUnknownEpidermolysis Bullosa Dystrophica, RecessiveSpain
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Phoenicis TherapeuticsNot yet recruitingDystrophic Epidermolysis BullosaUnited States
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Thomas Jefferson UniversityOnconova Therapeutics, Inc.RecruitingRecessive Dystrophic Epidermolysis BullosaUnited States
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