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Completed

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

ClinicalTrials.gov ID NCT02014376
Sponsor Scioderm, Inc.
Information provided by Scioderm, Inc. (Responsible Party)
Last Update Posted 2020-01-13
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Study Overview

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.

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Official Title
A Phase 2b, Multi-Center, Randomized, Double-Blind, Vehicle-Controlled Study of the Effectiveness and Safety of SD-101 Cream in Subjects With Epidermolysis Bullosa
Conditions
Epidermolysis Bullosa
Intervention / Treatment
  • Drug: SD-101 dermal cream (3%)
  • Drug: SD-101 Dermal Cream (6%)
  • Drug: Vehicle (SD-101 0%)
  • Drug: SD-101 dermal cream (3%)
  • Drug: SD-101 Dermal Cream (6%)
  • Drug: Vehicle (SD-101 0%)
Other Study ID Numbers
  • SD-003
Study Start (Actual)
2014-01-06
Primary Completion (Actual)
2014-06-24
Study Completion (Actual)
2014-06-24
Enrollment (Actual)
48
Study Type
Interventional
Phase
Phase 2

Contacts and Locations

This section provides contact details for people who can answer questions about joining this study, and information on where this study is taking place.

To learn more, please see the Contacts and Locations section in How to Read a Study Record(https://clinicaltrials.gov/study-basics/how-to-read-study-record#contacts-and-locations).

Click to view interactive map

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies(https://clinicaltrials.gov/study-basics/learn-about-studies).
Eligibility Criteria
Description

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.
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Ages Eligible for Study
6 Months and older (ChildAdultOlder Adult )
Sexes Eligible for Study
All
Accepts Healthy Volunteers
No

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

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Design Details
Primary Purpose : Treatment
Allocation : Randomized
Interventional Model : Parallel Assignment
Masking : Triple (ParticipantCare ProviderInvestigator)

Arms and Interventions

Participant Group/Arm Intervention/Treatment
Participant Group/Arm Experimental: SD-101 Dermal Cream (6%)
SD-101 dermal cream (6%) applied topically once daily over the entire body for 90 days.
Intervention/Treatment Drug: SD-101 Dermal Cream (6%)
  • SD-101 is a white, crystalline powder that is formulated within an odorless, soft, white cream base. SD-101-6.0 cream contains allantoin, a diureide glyoxylic acid, at a concentration of 6% and other excipients.

  • Other Names:
    • SD-101
    • SD-101-6.0
    • Zorblisa
Participant Group/Arm Experimental: SD-101 Dermal Cream (3%)
SD-101 dermal cream (3%) applied topically once daily over the entire body for 90 days.
Intervention/Treatment Drug: SD-101 dermal cream (3%)
  • SD-101 is a white, crystalline powder that is formulated within an odorless, soft, white cream base. SD-101-3.0 cream contains allantoin, a diureide glyoxylic acid, at a concentration of 3% and other excipients.

  • Other Names:
    • SD-101
    • SD-101-3.0
    • Zorblisa
Participant Group/Arm Placebo Comparator: Vehicle (0%)
Vehicle dermal cream (SD-101 0%) applied topically once daily over the entire body for 90 days.
Intervention/Treatment Drug: Vehicle (SD-101 0%)
  • A white, crystalline powder that is formulated within an odorless, soft, white cream base. It contains no allantoin, only excipients.

  • Other Names:
    • Placebo Comparator
    • SD-101-0.0
Primary Outcome Measures
Outcome Measure Measure Description Time Frame
Participants With Documented Complete Closure Of The Target Wound Within 1 Month After Initiation Of TreatmentThe 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.Baseline to 1 Month
Secondary Outcome Measures
Outcome Measure Measure Description Time Frame
Participants With Documented Complete Closure Of The Target Wound Within 2 And 3 Months After Initiation Of TreatmentThe 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.Baseline to Month 2 and Month 3
Percentage Change From Baseline In Lesional Skin Based On Body Surface Area Index (BSAI) Measurements At Month 3The 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.Baseline, Month 3
Participants Experiencing A Change From Baseline In Itching At Day 7The 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.Baseline, Day 7
Change From Baseline In Pain At Day 7Pain 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.Baseline, Day 7
Participants With Scarring At Week 2, Month 1, Month 2, And Month 3In 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).Week 2, Month 1, Month 2, and Month 3

Collaborators and Investigators

This is where you will find people and organizations involved with this study.
Sponsor
Scioderm, Inc.
Collaborators
  • Amicus Therapeutics
Investigators
  • Study Director:Medical Monitor,Amicus Therapeutics

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Registration Dates
First Submitted
2013-12-12
First Submitted that Met QC Criteria
2013-12-12
First Posted (Estimated)
2013-12-18
Results Reporting Dates
Results First Submitted
2019-11-13
Results First Submitted that Met QC Criteria
2020-01-07
Results First Posted
2020-01-13
Certification/Extension Dates
Certification/Extension First Submitted
2017-10-03
Certification/Extension First Submitted that Met QC Criteria
2017-10-10
Certification/Extension First Posted
2017-10-17
Study Record Updates
Last Update Submitted that met QC Criteria
2020-01-07
Last Update Posted
2020-01-13
Last Verified
2020-01

More Information

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Keywords Provided by Scioderm, Inc.
Additional Relevant MeSH Terms