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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT02670330
Registration number
NCT02670330
Ethics application status
Date submitted
28/07/2015
Date registered
1/02/2016
Date last updated
27/09/2019
Titles & IDs
Public title
Open Label Extension Study to Evaluate the Long-term Safety of Zorblisa (SD-101-6.0) in Patients With Epidermolysis Bullosa
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Scientific title
An Open Label Multi-Center Extension Study to Evaluate the Long-term Safety of Zorblisa™ (SD-101-6.0) in Patients With Epidermolysis Bullosa
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Secondary ID [1]
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2014-005679-96
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Secondary ID [2]
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SD-006
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Epidermolysis Bullosa
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Condition category
Condition code
Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Skin
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Dermatological conditions
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Skin
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Other skin conditions
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - SD-101-6.0 cream
Experimental: Experimental: SD-101-6.0 cream - All participants (or their caregivers) applied SD-101-6.0 cream topically once a day to the entire body for a period of up to 36 months.
Treatment: Drugs: SD-101-6.0 cream
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.
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Number Of Participants With Treatment Emergent Adverse Events (TEAEs)
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Assessment method [1]
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TEAEs were defined as adverse events that started or worsened on or after baseline visit.
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Timepoint [1]
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From baseline to 30 days after last application of study drug (up to a maximum of 37 months)
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Secondary outcome [1]
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Change From Baseline In Body Surface Area Index (BSAI) Of Lesional Skin Up To Month 30
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Assessment method [1]
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Lesional skin was defined as areas that contained any of the following: blisters, erosions, ulcerations, scabbing, bullae, or eschars, as well as areas that were weeping, sloughing, oozing, crusted, or denuded. The percentage, ranging from 0% to 100%, of affected body surface area (BSA) was recorded for each defined body region (that is, head/neck, upper limbs, trunk \[includes groin\], and lower limbs), multiplied by the weighting factor, then summed for all body regions to calculate the BSAI that would range from 0% to 100%. The BSA for lesional skin was to be assessed by the same study physician on each visit for a particular participant. The mean change from baseline in BSAI was assessed every 3 months. Only participants with data available for analysis at each time point are presented.
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Timepoint [1]
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Baseline, up to Month 30
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Secondary outcome [2]
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Change From Baseline In BSAI Of Total Body Wound Burden Up To Month 30
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Assessment method [2]
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A wound was defined as an open area on the skin (that is, epidermal covering disrupted). Total body wound burden was calculated using BSAI; the percentage, ranging from 0% to 100%, of affected BSA was recorded for each defined body region (that is, head/neck, upper limbs, trunk \[includes groin\], and lower limbs), multiplied by the weighting factor, then summed for all body regions to calculate the BSAI that would range from 0% to 100%. The BSAI for total body wound burden was to be assessed by the same study physician at each visit for a particular participant.
The mean change from baseline in total body wound burden was assessed every 3 months. Only participants with data available for analysis at each time point are presented.
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Timepoint [2]
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Baseline, up to Month 30
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Eligibility
Key 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 protocol requirements.
* Participants who completed the SD-005 study (on study drug at Visit 5, approximately 90 days from baseline).
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Minimum age
1
Month
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
* Participants who did not meet the entry criteria outlined above.
* Pregnancy or breastfeeding during the study. (A urine pregnancy test was performed at the final visit for Study SD-005 for female participants of childbearing potential and repeated at screening/baseline visit of Study SD-006 if these visits did not occur on the same day).
* Female participants of childbearing potential who were not abstinent or not practicing a medically acceptable method of contraception.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
NA
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Stopped early
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
9/06/2015
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
3/09/2018
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Sample size
Target
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Accrual to date
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Final
152
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Recruitment in Australia
Recruitment state(s)
NSW,VIC
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Recruitment hospital [1]
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- Kogarah
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Recruitment hospital [2]
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- Parkville
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Recruitment postcode(s) [1]
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2217 - Kogarah
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Recruitment postcode(s) [2]
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3052 - Parkville
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Recruitment outside Australia
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United States of America
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Arizona
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California
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Colorado
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District of Columbia
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Illinois
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Missouri
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North Carolina
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Ohio
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Pennsylvania
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South Carolina
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Texas
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Washington
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Austria
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Salzburg
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France
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Nice
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France
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Paris
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France
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Toulouse
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Germany
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Freiburg im Breisgau
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Germany
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Hannover
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Israel
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Tel Aviv
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Lithuania
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Kaunas
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Groningen
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Poland
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Warsaw
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Serbia
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Belgrade
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Spain
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Madrid
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United Kingdom
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London
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Scioderm, Inc.
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Address
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Other collaborator category [1]
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Commercial sector/industry
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Name [1]
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Amicus Therapeutics
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Address [1]
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Ethics approval
Ethics application status
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Summary
Brief summary
The study aimed to assess the long-term safety of topical use of Zorblisa (SD-101-6.0) in participants with Epidermolysis Bullosa (EB).
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Trial website
https://clinicaltrials.gov/study/NCT02670330
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Medical Monitor
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Address
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Amicus Therapeutics
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Contact person for scientific queries
No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Type
Other Details
Attachment
Study protocol
https://cdn.clinicaltrials.gov/large-docs/30/NCT02670330/Prot_000.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/30/NCT02670330/SAP_001.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT02670330
Download to PDF