Study of Effectiveness and Safety of SD-101 in Participants With Epidermolysis Bullosa

Phase 2

Completed

Conditions: Epidermolysis Bullosa

Interventions: Drug: SD-101 Dermal Cream (6%)
Drug: SD-101 dermal cream (3%)
Drug: Vehicle (SD-101 0%)

Registration Number: NCT02014376

Lead Sponsor: Scioderm, Inc.

Brief 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).

Detailed Description: This was a Phase 2b, multi-center, randomized, double-blind, vehicle-controlled study to assess the efficacy and safety of SD-101 dermal cream (3% or 6%) on skin lesions in participants with Simplex, Recessive Dystrophic, or Junctional non-Herlitz EB.

SD-101 cream \[containing 3% or 6% or vehicle (0%)\] was applied topically, once a day to the entire body for a period of 90 days. Eligible participants had a target wound assessed at baseline. Selected target wound had to be at least a certain age and within a prespecified size range at study entry. 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 at Week 2, Month 1, Month 4, and Month 5 (at 14, 30, 60, and 90 days, respectively) to have the target wound previously identified at baseline re-assessed for the level of healing. In addition, changes in itching, pain, body surface area coverage of blisters and lesions, and scaring of the healed target wound were also assessed at each visit.

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 48

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.

Study & Design

Study Type: INTERVENTIONAL

Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
SD-101 Dermal Cream (6%)	SD-101 Dermal Cream (6%)	SD-101 dermal cream (6%) applied topically once daily over the entire body for 90 days.
SD-101 Dermal Cream (3%)	SD-101 dermal cream (3%)	SD-101 dermal cream (3%) applied topically once daily over the entire body for 90 days.
Vehicle (0%)	Vehicle (SD-101 0%)	Vehicle dermal cream (SD-101 0%) applied topically once daily over the entire body for 90 days.

Primary Outcome Measures

Name	Time	Method
Participants With Documented Complete Closure Of The Target Wound Within 1 Month After Initiation Of Treatment	Baseline to 1 Month	The ARANZ SilhouetteStar™, a wound imaging, measurement, and documentation system providing accurate wound assessment, was used to measure the target wound at all visits. Information captured included photographic images, quantitative measures, and other target wound assessment data input to the device by the clinician, all obtained with no contact to the participant's skin. Information about the target wound's measurement history was available on this system so that the serial progression of the target wound status could also be calculated and presented.

Secondary Outcome Measures

Name	Time	Method
Participants Experiencing A Change From Baseline In Itching At Day 7	Baseline, Day 7	The Itch Man Pruritus Assessment Tool was used to measure the intensity of itching. Itching was assessed and reported at Baseline and Day 7. For participants 6 months to 5 years of age, itching was assessed using the caretaker's response, while in participants 6 years and older, itching was self-reported.
Percentage Change From Baseline In Lesional Skin Based On Body Surface Area Index (BSAI) Measurements At Month 3	Baseline, Month 3	The BSAI is a global measure of disease "spread" with weighting factors. Lesional skin consisted of area(s) that could contain any of the following: blisters, bullae, erosions, ulcerations, scabbing and eschars, as well as areas that are weeping, sloughing, oozing, crusted and denuded. The percentage, ranging from 0% to 100%, of affected body surface area was recorded for each defined body region (head/neck, upper limbs, trunk \[includes groin\], and lower limbs), multiplied by the weighting factor, and then summed for all body regions to calculate the BSAI. The BSAI affected with blisters and wounds was calculated at baseline and Month 3 to assess the total affected area. Percentage change from baseline was calculated as follows: Percentage change from baseline = 100\*(Post-baseline value minus Baseline value) divided by Baseline value. Mean percentage change from baseline in BSAI is reported. Only participants with data available for analysis at the specified time point are presented.
Participants With Scarring At Week 2, Month 1, Month 2, And Month 3	Week 2, Month 1, Month 2, and Month 3	In the event of a healed wound, where complete closure was confirmed, the extent of scarring was assessed as "Present" or "Absent" at all post-baseline visits (Week 2 and Months 1, 2, and 3).
Participants With Documented Complete Closure Of The Target Wound Within 2 And 3 Months After Initiation Of Treatment	Baseline to Month 2 and Month 3	The ARANZ SilhouetteStar™, a wound imaging, measurement, and documentation system providing accurate wound assessment, was used to measure the target wound at all visits. Information captured included photographic images, quantitative measures, and other target wound assessment data input to the device by the clinician, all obtained with no contact to the participant's skin. Information about the target wound's measurement history was available on this system so that the serial progression of the target wound status could also be calculated and presented.
Change From Baseline In Pain At Day 7	Baseline, Day 7	Pain was assessed at Baseline and Day 7. The presence and intensity of pain was assessed using the Face, Legs, Activity, Cry, Consolability (FLACC) Pain Scale for participants 6 months to 3 years of age. For participants aged 4 years and older, the Wong Faces Pain Scale was used. Scores were attributed for each of the 5 categories in the FLACC scale from 0 to 2, which resulted in a total score between 0 and 10. The Wong Faces Pain scale used 1 item to rate pain on a 0 to 10 scale. Higher score values indicated more pain.