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SD-101-6.0

Phase 3

Epidermolysis Bullosa | Small molecule | Dermatology |Amicus Therapeutics, Inc.|Last Updated: Apr 9, 2020

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDBiomarker
Total Trials1
Total Enrollment169
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02384460ESSENCE Study: Efficacy and Safety of SD-101 Cream in Participants With Epidermolysis BullosaPHASE3 COMPLETED 169Mar 11, 2015Jul 5, 2017Apr 9, 202038 United States, Australia +11
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Study Endpoints
Primary Endpoints
Time To Complete Target Wound Closure Within 3 Months
From baseline to Month 3 visit

Target wounds were monitored at each study visit for complete closure, defined as skin re-epithelialization without drainage. Time to target wound closure was measured from the date of the first administration of the study drug to the date of target wound closure. Participants were censored if they did not have a response within 3 months, or withdrew earlier before the confirmation of their target wound closing. This primary end point displays the mean time to complete target wound closure, analyzed using a Kaplan-Meier approach.

The Percentage Of Participants Experiencing Complete Closure Of Their Target Wound Within 3 Months
From baseline to Month 3 visit

Target wounds were monitored at each study visit for complete closure, defined as skin re-epithelialization without drainage. Participants were considered responders if they experienced complete wound closure at the Week 2 or Months 1, 2, or 3 visits. If a target wound was documented to have closed at a given visit, it was considered closed at all subsequent visits. This primary end point displays the percentage of participants from the ITT population who had complete target wound closure by the end of the study period (that is, 3 months). Analysis was performed on participants with post-baseline wound closure data.

Secondary Endpoints
Percentage Of Participants Experiencing Complete Closure Of Their Target Wound At Month 1 And Month 2 Visits
From baseline to Month 1 and Month 2 visits
Change From Baseline In Body Surface Area Index (BSAI) Of Lesional Skin At Month 3 Visit
Baseline, Month 3 visit
Change From Baseline In BSAI Of Total Body Wound Burden At Month 3 Visit
Baseline, Month 3 visit
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
SD-101-6.0 creamEXPERIMENTALSD-101-6.0 cream applied topically, once a day to the entire body for a period of 90 days
Placebo (SD-101-0.0) creamPLACEBO_COMPARATORSD-101-0.0 (placebo) cream applied topically, once a day to the entire body for a period of 90 days
Interventions
NameTypeDescription
SD-101-6.0 creamDRUGapplied topically once a day for 90 days
Placebo (SD-101-0.0) creamDRUGapplied topically once a day for 90 days
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Eligibility Criteria
Age Range1 Month — N/A
SexALL
Healthy VolunteersNo
Study Sites38

Inclusion Criteria: * Informed Consent form signed by the participant or participant's legal representative; if the participant was under the age of 18 but capable of providing assent, signed assent from the participant. * Participant (or caretaker) must have been willing to comply with all protoco...

Countries:United StatesAustraliaAustriaFranceGermanyIsraelItalyLithuaniaNetherlandsPolandSerbiaSpainUnited Kingdom
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