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

Primary Purpose

Epidermolysis Bullosa

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
SD-101 dermal cream (3%)
SD-101 Dermal Cream (6%)
Vehicle (SD-101 0%)
Sponsored by
Scioderm, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Epidermolysis Bullosa focused on measuring Epidermolysis Bullosa, Simplex, Recessive Dystrophic, Junctional non-Herlitz, Amicus Therapeutics, Scioderm, Inc., SD-101 6%, SD-101-6.0, Allantoin 6%, SD-101 3%, SD-101-3.0, Allantoin 3%, Zorblisa

Eligibility Criteria

6 Months - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Placebo Comparator

Arm Label

SD-101 Dermal Cream (6%)

SD-101 Dermal Cream (3%)

Vehicle (0%)

Arm Description

SD-101 dermal cream (6%) applied topically once daily over the entire body for 90 days.

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

Vehicle dermal cream (SD-101 0%) applied topically once daily over the entire body for 90 days.

Outcomes

Primary Outcome Measures

Participants With Documented Complete Closure Of The Target Wound Within 1 Month After Initiation Of Treatment
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

Participants With Documented Complete Closure Of The Target Wound Within 2 And 3 Months After Initiation Of Treatment
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.
Percentage Change From Baseline In Lesional Skin Based On Body Surface Area Index (BSAI) Measurements At 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 Experiencing A Change From Baseline In Itching At 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.
Change From Baseline In Pain At 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.
Participants With Scarring At 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).

Full Information

First Posted
December 12, 2013
Last Updated
January 7, 2020
Sponsor
Scioderm, Inc.
Collaborators
Amicus Therapeutics
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1. Study Identification

Unique Protocol Identification Number
NCT02014376
Brief Title
Study of Effectiveness and Safety of SD-101 in Participants With Epidermolysis Bullosa
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
Study Type
Interventional

2. Study Status

Record Verification Date
January 2020
Overall Recruitment Status
Completed
Study Start Date
January 6, 2014 (Actual)
Primary Completion Date
June 24, 2014 (Actual)
Study Completion Date
June 24, 2014 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Scioderm, Inc.
Collaborators
Amicus Therapeutics

4. Oversight

Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Epidermolysis Bullosa
Keywords
Epidermolysis Bullosa, Simplex, Recessive Dystrophic, Junctional non-Herlitz, Amicus Therapeutics, Scioderm, Inc., SD-101 6%, SD-101-6.0, Allantoin 6%, SD-101 3%, SD-101-3.0, Allantoin 3%, Zorblisa

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
48 (Actual)

8. Arms, Groups, and Interventions

Arm Title
SD-101 Dermal Cream (6%)
Arm Type
Experimental
Arm Description
SD-101 dermal cream (6%) applied topically once daily over the entire body for 90 days.
Arm Title
SD-101 Dermal Cream (3%)
Arm Type
Experimental
Arm Description
SD-101 dermal cream (3%) applied topically once daily over the entire body for 90 days.
Arm Title
Vehicle (0%)
Arm Type
Placebo Comparator
Arm Description
Vehicle dermal cream (SD-101 0%) applied topically once daily over the entire body for 90 days.
Intervention Type
Drug
Intervention Name(s)
SD-101 dermal cream (3%)
Other Intervention Name(s)
SD-101, SD-101-3.0, Zorblisa
Intervention Description
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.
Intervention Type
Drug
Intervention Name(s)
SD-101 Dermal Cream (6%)
Other Intervention Name(s)
SD-101, SD-101-6.0, Zorblisa
Intervention Description
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.
Intervention Type
Drug
Intervention Name(s)
Vehicle (SD-101 0%)
Other Intervention Name(s)
Placebo Comparator, SD-101-0.0
Intervention Description
A white, crystalline powder that is formulated within an odorless, soft, white cream base. It contains no allantoin, only excipients.
Primary Outcome Measure Information:
Title
Participants With Documented Complete Closure Of The Target Wound Within 1 Month After Initiation Of Treatment
Description
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.
Time Frame
Baseline to 1 Month
Secondary Outcome Measure Information:
Title
Participants With Documented Complete Closure Of The Target Wound Within 2 And 3 Months After Initiation Of Treatment
Description
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.
Time Frame
Baseline to Month 2 and Month 3
Title
Percentage Change From Baseline In Lesional Skin Based On Body Surface Area Index (BSAI) Measurements At Month 3
Description
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.
Time Frame
Baseline, Month 3
Title
Participants Experiencing A Change From Baseline In Itching At Day 7
Description
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.
Time Frame
Baseline, Day 7
Title
Change From Baseline In Pain At Day 7
Description
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.
Time Frame
Baseline, Day 7
Title
Participants With Scarring At Week 2, Month 1, Month 2, And Month 3
Description
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).
Time Frame
Week 2, Month 1, Month 2, and Month 3

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Months
Accepts Healthy Volunteers
No
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Monitor
Organizational Affiliation
Amicus Therapeutics
Official's Role
Study Director
Facility Information:
City
Palo Alto
State/Province
California
ZIP/Postal Code
94304
Country
United States
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
City
Hackensack
State/Province
New Jersey
ZIP/Postal Code
07601
Country
United States
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27516
Country
United States
City
San Antonio
State/Province
Texas
ZIP/Postal Code
78218
Country
United States
City
Seattle
State/Province
Washington
ZIP/Postal Code
98105
Country
United States

12. IPD Sharing Statement

Learn more about this trial

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

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