Study of Effectiveness and Safety of SD-101 in Participants With Epidermolysis Bullosa
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
1 other identifier
interventional
48
1 country
7
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).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_2
Started Jan 2014
Shorter than P25 for phase_2
7 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
December 12, 2013
CompletedFirst Posted
Study publicly available on registry
December 18, 2013
CompletedStudy Start
First participant enrolled
January 6, 2014
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 24, 2014
CompletedStudy Completion
Last participant's last visit for all outcomes
June 24, 2014
CompletedResults Posted
Study results publicly available
January 13, 2020
CompletedJanuary 13, 2020
January 1, 2020
6 months
December 12, 2013
November 13, 2019
January 7, 2020
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
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.
Baseline to 1 Month
Secondary Outcomes (5)
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
Percentage Change From Baseline In Lesional Skin Based On Body Surface Area Index (BSAI) Measurements At Month 3
Baseline, Month 3
Participants Experiencing A Change From Baseline In Itching At Day 7
Baseline, Day 7
Change From Baseline In Pain At Day 7
Baseline, Day 7
Participants With Scarring At Week 2, Month 1, Month 2, And Month 3
Week 2, Month 1, Month 2, and Month 3
Study Arms (3)
SD-101 Dermal Cream (6%)
EXPERIMENTALSD-101 dermal cream (6%) applied topically once daily over the entire body for 90 days.
SD-101 Dermal Cream (3%)
EXPERIMENTALSD-101 dermal cream (3%) applied topically once daily over the entire body for 90 days.
Vehicle (0%)
PLACEBO COMPARATORVehicle dermal cream (SD-101 0%) applied topically once daily over the entire body for 90 days.
Interventions
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.
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.
A white, crystalline powder that is formulated within an odorless, soft, white cream base. It contains no allantoin, only excipients.
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Scioderm, Inc.lead
- Amicus Therapeuticscollaborator
Study Sites (7)
Unknown Facility
Palo Alto, California, 94304, United States
Unknown Facility
Chicago, Illinois, 60611, United States
Unknown Facility
St Louis, Missouri, 63110, United States
Unknown Facility
Hackensack, New Jersey, 07601, United States
Unknown Facility
Chapel Hill, North Carolina, 27516, United States
Unknown Facility
San Antonio, Texas, 78218, United States
Unknown Facility
Seattle, Washington, 98105, United States
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Patient Advocacy
- Organization
- Amicus Therapeutics, Inc.
Study Officials
- STUDY DIRECTOR
Medical Monitor
Amicus Therapeutics
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- GT60
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- TRIPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
December 12, 2013
First Posted
December 18, 2013
Study Start
January 6, 2014
Primary Completion
June 24, 2014
Study Completion
June 24, 2014
Last Updated
January 13, 2020
Results First Posted
January 13, 2020
Record last verified: 2020-01