Phase 2
N=48
Study of Effectiveness and Safety of SD-101 in Participants With Epidermolysis Bullosa
Epidermolysis Bullosa
Bottom Line
View on ClinicalTrials.gov: NCT02014376 ↗Enrolled (actual)
48
Serious AEs
6.3%
Results posted
Jan 2020
Primary outcome: Primary: Participants With Documented Complete Closure Of The Target Wound Within 1 Month After Initiation Of Treatment — 8; 6; 7; 7 Participants — p==0.3699
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- SD-101 dermal cream (3%) (Drug); SD-101 Dermal Cream (6%) (Drug); Vehicle (SD-101 0%) (Drug)
- Age
- Pediatric, Adult, Older Adult · 0+ yrs
- Sex
- All
- Sponsor
- Scioderm, Inc.
- Primary completion
- Jun 2014
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Participants With Documented Complete Closure Of The Target Wound Within 1 Month After Initiation Of Treatment |
8; 6; 7; 7; 10; 10 | =0.3699 |
| SECONDARY Participants With Documented Complete Closure Of The Target Wound Within 2 And 3 Months After Initiation Of Treatment |
9; 7; 7; 6; 9; 10 | — |
| SECONDARY Percentage Change From Baseline In Lesional Skin Based On Body Surface Area Index (BSAI) Measurements At Month 3 |
-28.02; -42.52; -5.75 | — |
| SECONDARY Participants Experiencing A Change From Baseline In Itching At Day 7 |
5; 4; 7; 9; 12; 10 | — |
| SECONDARY Change From Baseline In Pain At Day 7 |
0.50; -0.17; -1.60; 0.91; -0.70; 1.08 | — |
| SECONDARY Participants With Scarring At Week 2, Month 1, Month 2, And Month 3 |
0; 0; 0; 4; 1; 2 | — |
Summary
The purpose of this study was to assess whether the topical use of SD-101 cream (3% or 6%) was effective in treating wounds in participants with Simplex, Recessive Dystrophic, or Junctional non-Herlitz Epidermolysis Bullosa (EB).
Eligibility Criteria
Inclusion Criteria
- Informed Consent form signed by the participant or the participant's legal representative; if the participant is under the age of majority but capable of providing assent, signed assent from the participant.
- Participant (or caretaker) was willing to comply with all protocol requirements.
- Diagnosis of Simplex, Recessive Dystrophic, or Junctional non-Herlitz EB.
- Participants 6 months of age and older.
- Participants had 1 target wound within a prespecified size range at study entry.
- Target wound was at least 21 days or older.
Exclusion Criteria
- Participants who did not meet all the entry criteria outlined in inclusion criteria.
- Selected target wound had clinical evidence of local infection.
- Use of any investigational drug within 30 days before enrollment.
- Use of immunotherapy or cytotoxic chemotherapy within 60 days before enrollment.
- Use of systemic or topical steroidal therapy within 30 days before enrollment (inhaled steroids and ophthalmic drops containing steroids were allowed).
- Use of systemic antibiotics within 7 days before enrollment.
- Current or former malignancy.
- Arterial or venous disorder resulting in ulcerated wounds.
- Diabetes mellitus.
- Pregnancy or breastfeeding during the study (a urine pregnancy test was performed at screening for female participants of childbearing potential).
- Females of childbearing potential who were not abstinent and not practicing a medically acceptable method of contraception.
- Known history of cardiac, hepatic, or renal disease.
Data sourced from ClinicalTrials.gov (NCT02014376). 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.