Phase 3
N=169
ESSENCE Study: Efficacy and Safety of SD-101 Cream in Participants With Epidermolysis Bullosa
Epidermolysis Bullosa
Bottom Line
View on ClinicalTrials.gov: NCT02384460 ↗Enrolled (actual)
169
Serious AEs
7.1%
Results posted
Oct 2018
Primary outcome: Primary: Time To Complete Target Wound Closure Within 3 Months — 41.6; 53.6 days — p=0.985
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- SD-101-6.0 cream (Drug); Placebo (SD-101-0.0) cream (Drug)
- Age
- Pediatric, Adult, Older Adult · 0+ yrs
- Sex
- All
- Sponsor
- Scioderm, Inc.
- Primary completion
- Jul 2017
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Time To Complete Target Wound Closure Within 3 Months |
41.6; 53.6 | 0.985 |
| PRIMARY The Percentage Of Participants Experiencing Complete Closure Of Their Target Wound Within 3 Months |
49.4; 53.6 | 0.39 |
| SECONDARY Percentage Of Participants Experiencing Complete Closure Of Their Target Wound At Month 1 And Month 2 Visits |
31.6; 22.6; 43.0; 42.9 | 0.212 |
| SECONDARY Change From Baseline In Body Surface Area Index (BSAI) Of Lesional Skin At Month 3 Visit |
-4.637; -5.319 | 0.706 |
| SECONDARY Change From Baseline In BSAI Of Total Body Wound Burden At Month 3 Visit |
-3.050; -2.922 | 0.9 |
| SECONDARY Change From Baseline In Itching Score At Day 7 |
-0.5; -0.3 | 0.262 |
| SECONDARY Change From Baseline In Pain Score At Day 7 |
-0.3; -0.6 | 0.098 |
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).
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.
Data sourced from ClinicalTrials.gov (NCT02384460). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.