Back to resultsESSENCE Study: Efficacy and Safety of SD-101 Cream in Participants With Epidermolysis Bullosa
Primary Purpose
Epidermolysis Bullosa
Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
SD-101-6.0 cream
Placebo (SD-101-0.0) cream
Sponsored by
About this trial
This is an interventional treatment trial for Epidermolysis Bullosa focused on measuring Amicus Therapeutics, Scioderm, Inc., Simplex, Recessive Dystrophic, Junctional non-Herlitz, Epidermolysis Bullosa, SD-101 6%, SD-101-6.0, Allantoin 6%, Zorblisa
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.
Sites / Locations
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
SD-101-6.0 cream
Placebo (SD-101-0.0) cream
Arm Description
SD-101-6.0 cream applied topically, once a day to the entire body for a period of 90 days
SD-101-0.0 (placebo) cream applied topically, once a day to the entire body for a period of 90 days
Outcomes
Primary Outcome Measures
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.
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.
Secondary Outcome Measures
Percentage Of Participants Experiencing Complete Closure Of Their Target Wound At Month 1 And Month 2 Visits
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.
Change From Baseline In Body Surface Area Index (BSAI) Of Lesional Skin At Month 3 Visit
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.
Change From Baseline In BSAI Of Total Body Wound Burden At Month 3 Visit
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.
Change From Baseline In Itching Score At Day 7
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.
Change From Baseline In Pain Score At Day 7
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.
Full Information
NCT ID
NCT02384460
First Posted
February 13, 2015
Last Updated
April 1, 2020
Sponsor
Scioderm, Inc.
Collaborators
Amicus Therapeutics
1. Study Identification
Unique Protocol Identification Number
NCT02384460
Brief Title
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
2. Study Status
Record Verification Date
April 2020
Overall Recruitment Status
Completed
Study Start Date
March 11, 2015 (Actual)
Primary Completion Date
July 5, 2017 (Actual)
Study Completion Date
July 5, 2017 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Scioderm, Inc.
Collaborators
Amicus Therapeutics
4. Oversight
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
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
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
169 (Actual)
8. Arms, Groups, and Interventions
Arm Title
SD-101-6.0 cream
Arm Type
Experimental
Arm Description
SD-101-6.0 cream applied topically, once a day to the entire body for a period of 90 days
Arm Title
Placebo (SD-101-0.0) cream
Arm Type
Placebo Comparator
Arm Description
SD-101-0.0 (placebo) cream applied topically, once a day to the entire body for a period of 90 days
Intervention Type
Drug
Intervention Name(s)
SD-101-6.0 cream
Other Intervention Name(s)
SD-101
Intervention Description
applied topically once a day for 90 days
Intervention Type
Drug
Intervention Name(s)
Placebo (SD-101-0.0) cream
Other Intervention Name(s)
placebo
Intervention Description
applied topically once a day for 90 days
Primary Outcome Measure Information:
Title
Time To Complete Target Wound Closure Within 3 Months
Description
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.
Time Frame
From baseline to Month 3 visit
Title
The Percentage Of Participants Experiencing Complete Closure Of Their Target Wound Within 3 Months
Description
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.
Time Frame
From baseline to Month 3 visit
Secondary Outcome Measure Information:
Title
Percentage Of Participants Experiencing Complete Closure Of Their Target Wound At Month 1 And Month 2 Visits
Description
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.
Time Frame
From baseline to Month 1 and Month 2 visits
Title
Change From Baseline In Body Surface Area Index (BSAI) Of Lesional Skin At Month 3 Visit
Description
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.
Time Frame
Baseline, Month 3 visit
Title
Change From Baseline In BSAI Of Total Body Wound Burden At Month 3 Visit
Description
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.
Time Frame
Baseline, Month 3 visit
Title
Change From Baseline In Itching Score At Day 7
Description
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.
Time Frame
Baseline, Day 7
Title
Change From Baseline In Pain Score At Day 7
Description
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.
Time Frame
Baseline, Day 7
10. Eligibility
Sex
All
Minimum Age & Unit of Time
1 Month
Accepts Healthy Volunteers
No
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Monitor
Organizational Affiliation
Amicus Therapeutics
Official's Role
Study Director
Facility Information:
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85016
Country
United States
City
Redwood City
State/Province
California
ZIP/Postal Code
94063
Country
United States
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20016
Country
United States
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
City
Miami
State/Province
Florida
ZIP/Postal Code
33155
Country
United States
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States
City
Columbia
State/Province
Missouri
ZIP/Postal Code
65212
Country
United States
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
City
Lebanon
State/Province
New Hampshire
ZIP/Postal Code
03756
Country
United States
City
Hackensack
State/Province
New Jersey
ZIP/Postal Code
07601
Country
United States
City
East Setauket
State/Province
New York
ZIP/Postal Code
11733
Country
United States
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27516
Country
United States
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229-3039
Country
United States
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
City
Charleston
State/Province
South Carolina
ZIP/Postal Code
29425-5780
Country
United States
City
Austin
State/Province
Texas
ZIP/Postal Code
78723
Country
United States
City
San Antonio
State/Province
Texas
ZIP/Postal Code
78218
Country
United States
City
Seattle
State/Province
Washington
ZIP/Postal Code
98105
Country
United States
City
Kogarah
State/Province
New South Wales
ZIP/Postal Code
2217
Country
Australia
City
Parkville
State/Province
Victoria
ZIP/Postal Code
3050
Country
Australia
City
Parkville
State/Province
Victoria
ZIP/Postal Code
3052
Country
Australia
City
Salzburg
ZIP/Postal Code
5020
Country
Austria
City
Nice
ZIP/Postal Code
0-06200
Country
France
City
Paris
ZIP/Postal Code
75015
Country
France
City
Toulouse
Country
France
City
Freiburg
Country
Germany
City
Hannover
Country
Germany
City
Tel Aviv
Country
Israel
City
Milano
ZIP/Postal Code
20122
Country
Italy
City
Kaunas
Country
Lithuania
City
Groningen
ZIP/Postal Code
9713 GZ
Country
Netherlands
City
Warszawa
State/Province
Koszykowa
Country
Poland
City
Belgrade
Country
Serbia
City
Madrid
Country
Spain
City
London
ZIP/Postal Code
WC1N 3JH
Country
United Kingdom
12. IPD Sharing Statement
Citations:
PubMed Identifier
32693833
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.
Results Reference
derived
PubMed Identifier
32576219
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.
Results Reference
derived
Learn more about this trial
ESSENCE Study: Efficacy and Safety of SD-101 Cream in Participants With Epidermolysis Bullosa
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