Epidermolysis BullosaPhase 3NCT02384460
ESSENCE Study: Efficacy and Safety of SD-101 Cream in Participants With Epidermolysis Bullosa
A Phase 3 study testing SD-101-6.0 cream in people with Epidermolysis Bullosa, run by Scioderm, Inc. at 38 sites in 13 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 1 month and older
- Conditions the study is for
- Epidermolysis Bullosa
- What is being tested?
- SD-101-6.0 cream
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 169 participants
- Where
- 38 sitesin 13 countries
- Study dates
- 11 Mar 2015to 5 Jul 2017
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 month and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Show all 10Show fewer
- 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.
Trial locations and contacts
38 sites in 13 countries.
38 sites in 13 countries
United States21 sites
Study site
Aurora, Colorado, 80045
Study site
Austin, Texas, 78723
Study site
Chapel Hill, North Carolina, 27516
Study site
Charleston, South Carolina, 29425-5780
Study site
Chicago, Illinois, 60611
Study site
Cincinnati, Ohio, 45229-3039
Study site
Columbia, Missouri, 65212
Study site
East Setauket, New York, 11733
Study site
Hackensack, New Jersey, 07601
Study site
Lebanon, New Hampshire, 03756
Study site
Miami, Florida, 33136
Study site
Miami, Florida, 33155
Study site
Minneapolis, Minnesota, 55455
Study site
New York, New York, 10032
Study site
Philadelphia, Pennsylvania, 19104
Study site
Phoenix, Arizona, 85016
Study site
Redwood City, California, 94063
Study site
San Antonio, Texas, 78218
Study site
Seattle, Washington, 98105
Study site
St Louis, Missouri, 63110
Study site
Washington D.C., District of Columbia, 20016
Australia3 sites
Study site
Kogarah, New South Wales, 2217
Study site
Parkville, Victoria, 3050
Study site
Parkville, Victoria, 3052
France3 sites
Study site
Nice, 0-06200
Study site
Paris, 75015
Study site
Toulouse
About this study
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
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).
Read the 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.
- Conditions
- Epidermolysis Bullosa
- Being tested
- SD-101-6.0 cream, Placebo (SD-101-0.0) cream
- Keywords
- Amicus Therapeutics, Scioderm, Inc., Simplex, Recessive Dystrophic, Junctional non-Herlitz, Epidermolysis Bullosa, SD-101 6%, SD-101-6.0, Allantoin 6%, Zorblisa
- Medical subject headings
- Epidermolysis Bullosa
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SD-101-6.0 creamDrug
Also called SD-101
applied topically once a day for 90 days
Placebo (SD-101-0.0) creamDrug
Also called placebo
applied topically once a day for 90 days
| Study group | Role | What they receive |
|---|---|---|
| SD-101-6.0 cream | Experimental | SD-101-6.0 cream applied topically, once a day to the entire body for a period of 90 days Receives: Drug: SD-101-6.0 cream |
| Placebo (SD-101-0.0) cream | Placebo comparator | SD-101-0.0 (placebo) cream applied topically, once a day to the entire body for a period of 90 days Receives: Drug: Placebo (SD-101-0.0) cream |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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
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.
Measured: 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
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.
Measured: Baseline, Month 3 visit
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.
Measured: Baseline, Month 3 visit
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.
Measured: Baseline, Day 7
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.
Measured: Baseline, Day 7
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Mar 2015 (estimated)
- Study start11 Mar 2015
- Primary completion5 Jul 2017
- Study completion5 Jul 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Epidermolysis BullosaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 169 participants (actual)
Sponsor and investigators
- Lead sponsor
- Scioderm, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Amicus Therapeutics
- Responsible party
- Sponsor
- Investigators
- Medical Monitor, Study director, Amicus Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02384460
- Sponsor's protocol ID
- SD-005
- Other IDs
- 2014-002288-14, R01-005095-01, 2014
- Status last verified
- Apr 2020
- First submitted
- 13 Feb 2015
- Last updated
- 9 Apr 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
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.
PubMed 32693833Paller 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.
PubMed 32576219
Study at a glance
From the registry record, last updated by the sponsor on 9 Apr 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02384460
- Acronym / sponsor ID
- SD-005
- Conditions
- Epidermolysis Bullosa
- Keywords
- Amicus Therapeutics, Scioderm, Inc., Simplex, Recessive Dystrophic, Junctional non-Herlitz, Epidermolysis Bullosa, SD-101 6%, SD-101-6.0
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 169 participants (actual)
- Lead sponsor
- Scioderm, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Amicus Therapeutics
- Study start
- 11 Mar 2015 (actual)
- Primary completion
- 5 Jul 2017 (actual)
- Study completion
- 5 Jul 2017 (actual)
- First posted
- 10 Mar 2015 (estimated)
- Results
- Posted 16 Oct 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| SD-101-6.0 cream | Experimental | Drug: SD-101-6.0 creamSD-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 comparator | Drug: Placebo (SD-101-0.0) creamSD-101-0.0 (placebo) cream applied topically, once a day to the entire body for a period of 90 days |
Who may qualify
- Ages
- 1 month and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (10 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SD-101-6.0 creamAlso: SD-101applied topically once a day for 90 days | Drug | SD-101-6.0 cream |
| Placebo (SD-101-0.0) creamAlso: placeboapplied topically once a day for 90 days | Drug | Placebo (SD-101-0.0) cream |
Primary outcome
Time To Complete Target Wound Closure Within 3 Months
Time frame: From baseline to Month 3 visit
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
Time frame: From baseline to Month 3 visit
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.
5 secondary outcomes
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
Change From Baseline In Body Surface Area Index (BSAI) Of Lesional Skin At Month 3 Visit
Time frame: Baseline, Month 3 visit
Change From Baseline In BSAI Of Total Body Wound Burden At Month 3 Visit
Time frame: Baseline, Month 3 visit
Change From Baseline In Itching Score At Day 7
Time frame: Baseline, Day 7
Change From Baseline In Pain Score At Day 7
Time frame: Baseline, Day 7
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Apr 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 32693833Murrell 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.
- Derived from this study: PubMed 32576219Paller 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Monitor study director
Amicus Therapeutics
38 sites in 13 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing SD-101-6.0 cream in people with Epidermolysis Bullosa, run by Scioderm, Inc. at 38 sites in 13 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 9 Apr 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 38 sites in United States, Australia, France, Germany, Austria and 8 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 16 Oct 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Time To Complete Target Wound Closure Within 3 Months (time frame: From baseline to Month 3 visit). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02384460, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Apr 2020.