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SD-101 dermal

Phase 2

Epidermolysis Bullosa | Small molecule | Rare Disease |Amicus Therapeutics, Inc.|Last Updated: Jan 13, 2020

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials1
Total Enrollment48

FDA Designations

No designations recorded

Clinical trial landscape

SD-101 dermal · 1 trial · 1 indication

Phase 2 1
NCT02014376Study of Effectiveness and Safety of SD-101 in Participants With Epidermolysis BullosaEpidermolysis Bullosa
COMPLETED48 Analytics
PHASE2COMPLETED
Study of Effectiveness and Safety of SD-101 in Participants With Epidermolysis Bullosa
Epidermolysis BullosaUnlock trial analytics

Study Endpoints

Primary Endpoints

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

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
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Study Design & Arms

AllocationRANDOMIZED
MaskingTRIPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
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

NameTypeDescription
SD-101 dermal cream (3%)DRUGSD-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 Dermal Cream (6%)DRUGSD-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.
Vehicle (SD-101 0%)DRUGA white, crystalline powder that is formulated within an odorless, soft, white cream base. It contains no allantoin, only excipients.
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Eligibility Criteria

Age Range6 Months to N/A
SexALL
Healthy VolunteersNo
Study Sites7

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 ...

Countries:United States
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Frequently asked questions about SD-101 dermal

What is SD-101 dermal used for?

SD-101 dermal is an investigational small molecule being studied for the treatment of Epidermolysis Bullosa, a rare genetic skin disorder. It is applied to the skin and is currently in Phase 2 clinical development. The drug has completed one clinical trial in the United States.

Who makes SD-101 dermal?

SD-101 dermal is being developed by Amicus Therapeutics, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol FOLD. The company is conducting clinical research on this investigational therapy for Epidermolysis Bullosa.

What phase is SD-101 dermal in?

SD-101 dermal is in Phase 2 clinical development. It is an investigational drug, meaning it has not been approved by regulatory authorities. One Phase 2 clinical trial for the treatment of Epidermolysis Bullosa has been completed.

What clinical trials is SD-101 dermal in?

SD-101 dermal has one completed clinical trial, identified as NCT02014376, titled "Study of Effectiveness and Safety of SD-101 in Participants With Epidermolysis Bullosa." This Phase 2 study enrolled 48 participants in the United States and was randomized, double-blind, and placebo-controlled.

Is SD-101 dermal the same as SD-101?

SD-101 dermal is a specific formulation of the drug SD-101, developed by Amicus Therapeutics for topical application in treating Epidermolysis Bullosa. The clinical trial NCT02014376 evaluated this dermal formulation in patients with the condition.