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

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ClinicalTrials.gov Identifier: NCT02014376
Recruitment Status : Completed
First Posted : December 18, 2013
Results First Posted : January 13, 2020
Last Update Posted : January 13, 2020
Sponsor:
Collaborator:
Amicus Therapeutics
Information provided by (Responsible Party):
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).

Condition or disease Intervention/treatment Phase
Epidermolysis Bullosa Drug: SD-101 dermal cream (3%) Drug: SD-101 Dermal Cream (6%) Drug: Vehicle (SD-101 0%) Phase 2

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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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 48 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
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
Actual Study Start Date : January 6, 2014
Actual Primary Completion Date : June 24, 2014
Actual Study Completion Date : June 24, 2014


Arm Intervention/treatment
Experimental: SD-101 Dermal Cream (6%)
SD-101 dermal cream (6%) applied topically once daily over the entire body for 90 days.
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

Experimental: SD-101 Dermal Cream (3%)
SD-101 dermal cream (3%) applied topically once daily over the entire body for 90 days.
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

Placebo Comparator: Vehicle (0%)
Vehicle dermal cream (SD-101 0%) applied topically once daily over the entire body for 90 days.
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 :
  1. Participants With Documented Complete Closure Of The Target Wound Within 1 Month After Initiation Of Treatment [ Time Frame: 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 :
  1. Participants With Documented Complete Closure Of The Target Wound Within 2 And 3 Months After Initiation Of Treatment [ Time Frame: 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.

  2. Percentage Change From Baseline In Lesional Skin Based On Body Surface Area Index (BSAI) Measurements At Month 3 [ Time Frame: 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.

  3. Participants Experiencing A Change From Baseline In Itching At Day 7 [ Time Frame: 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.

  4. Change From Baseline In Pain At Day 7 [ Time Frame: 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.

  5. Participants With Scarring At Week 2, Month 1, Month 2, And Month 3 [ Time Frame: 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).



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Ages Eligible for Study:   6 Months and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02014376


Locations
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United States, California
Palo Alto, California, United States, 94304
United States, Illinois
Chicago, Illinois, United States, 60611
United States, Missouri
Saint Louis, Missouri, United States, 63110
United States, New Jersey
Hackensack, New Jersey, United States, 07601
United States, North Carolina
Chapel Hill, North Carolina, United States, 27516
United States, Texas
San Antonio, Texas, United States, 78218
United States, Washington
Seattle, Washington, United States, 98105
Sponsors and Collaborators
Scioderm, Inc.
Amicus Therapeutics
Investigators
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Study Director: Medical Monitor Amicus Therapeutics
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Responsible Party: Scioderm, Inc.
ClinicalTrials.gov Identifier: NCT02014376    
Other Study ID Numbers: SD-003
First Posted: December 18, 2013    Key Record Dates
Results First Posted: January 13, 2020
Last Update Posted: January 13, 2020
Last Verified: January 2020
Keywords provided by Scioderm, Inc.:
SD-101 6%
SD-101-6.0
SD-101 3%
SD-101-3.0
Epidermolysis Bullosa
Simplex
Recessive Dystrophic
Junctional non-Herlitz
Amicus Therapeutics
Scioderm, Inc.
Allantoin 6%
Allantoin 3%
Zorblisa
Additional relevant MeSH terms:
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Epidermolysis Bullosa
Skin Abnormalities
Congenital Abnormalities
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Skin Diseases, Vesiculobullous