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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
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
Secondary ID [1] 0 0
2014-005679-96
Secondary ID [2] 0 0
SD-006
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Epidermolysis Bullosa 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Skin 0 0 0 0
Dermatological conditions
Skin 0 0 0 0
Other skin conditions

Intervention/exposure
Study type
Interventional
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.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number Of Participants With Treatment Emergent Adverse Events (TEAEs)
Timepoint [1] 0 0
From baseline to 30 days after last application of study drug (up to a maximum of 37 months)
Secondary outcome [1] 0 0
Change From Baseline In Body Surface Area Index (BSAI) Of Lesional Skin Up To Month 30
Timepoint [1] 0 0
Baseline, up to Month 30
Secondary outcome [2] 0 0
Change From Baseline In BSAI Of Total Body Wound Burden Up To Month 30
Timepoint [2] 0 0
Baseline, up to Month 30

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).
Minimum age
1 Month
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
9/06/2015
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
3/09/2018
Sample size
Target
Accrual to date
Final
152
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
- Kogarah
Recruitment hospital [2] 0 0
- Parkville
Recruitment postcode(s) [1] 0 0
2217 - Kogarah
Recruitment postcode(s) [2] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Colorado
Country [4] 0 0
United States of America
State/province [4] 0 0
District of Columbia
Country [5] 0 0
United States of America
State/province [5] 0 0
Illinois
Country [6] 0 0
United States of America
State/province [6] 0 0
Minnesota
Country [7] 0 0
United States of America
State/province [7] 0 0
Missouri
Country [8] 0 0
United States of America
State/province [8] 0 0
New York
Country [9] 0 0
United States of America
State/province [9] 0 0
North Carolina
Country [10] 0 0
United States of America
State/province [10] 0 0
Ohio
Country [11] 0 0
United States of America
State/province [11] 0 0
Pennsylvania
Country [12] 0 0
United States of America
State/province [12] 0 0
South Carolina
Country [13] 0 0
United States of America
State/province [13] 0 0
Texas
Country [14] 0 0
United States of America
State/province [14] 0 0
Washington
Country [15] 0 0
Austria
State/province [15] 0 0
Salzburg
Country [16] 0 0
France
State/province [16] 0 0
Nice
Country [17] 0 0
France
State/province [17] 0 0
Paris
Country [18] 0 0
France
State/province [18] 0 0
Toulouse
Country [19] 0 0
Germany
State/province [19] 0 0
Freiburg im Breisgau
Country [20] 0 0
Germany
State/province [20] 0 0
Hannover
Country [21] 0 0
Israel
State/province [21] 0 0
Tel Aviv
Country [22] 0 0
Lithuania
State/province [22] 0 0
Kaunas
Country [23] 0 0
Netherlands
State/province [23] 0 0
Groningen
Country [24] 0 0
Poland
State/province [24] 0 0
Warsaw
Country [25] 0 0
Serbia
State/province [25] 0 0
Belgrade
Country [26] 0 0
Spain
State/province [26] 0 0
Madrid
Country [27] 0 0
United Kingdom
State/province [27] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Scioderm, Inc.
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
Amicus Therapeutics
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

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).
Trial website
https://clinicaltrials.gov/study/NCT02670330
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medical Monitor
Address 0 0
Amicus Therapeutics
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT02670330