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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT02670330




Registration number
NCT02670330
Ethics application status
Date submitted
28/07/2015
Date registered
1/02/2016
Date last updated
2/10/2018

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 - Zorblisa (SD-101-6.0) cream

Experimental: Experimental: ZORBLISA (SD-101 cream ) - All subjects will apply ZORBLISA (SD-101-6.0%) cream topically, once a day to the entire body for a period of up to 1440 days (48 months).


Treatment: Drugs: Zorblisa (SD-101-6.0) cream
applied topically once a day to the entire body for 1440 days (48 months).

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Demonstrate continued long term safety of Zorblisa assessed via monitoring of local tolerability at the application sites, occurrence of adverse events and physical examinations. Results will be summarized - The primary objective is to demonstrate the longterm safety of ZORBLISA in patients, with Simplex, Recessive Dystrophic, and Junctional non-Herlitz Epidermolysis Bullosa. Results will be summarized using descriptive statistics. Summary statistics to be used include proportion of patients with complete closure of their target lesion at each visit, mean and standard deviation of BSAI compared with baseline at each visit, and incidence rates of specific adverse events. Additional evaluations may be made to compare responses of patients in this extension study with their responses in SD-005. For those patients randomized to SD-101-6.0 in SD-005, the use of summary statistics as described in the previous paragraph will provide an understanding of the long term effect of SD-101-6.0 in these patients. For those patients randomized to the control arm in SD-005, summary statistics will be used to compare their response to SD-101-6.0 in SD-006 with their response to control in SD-005.
Timepoint [1] 0 0
up to 1440 days
Secondary outcome [1] 0 0
Changes in Body Surface Area (BSA) of lesional skin and wound burden using BSAI - The secondary objectives are to assess the efficacy of ZORBLISA in terms of the change in Body Surface Area (BSA) of lesional skin and wound burden; as well as closure of unhealed target wounds from the SD-005 study. Change in lesional skin based on BSA estimates compared to Baseline will be measured using the Body Surface Area Index (BSAI). Change in total body wound coverage based on BSA estimates compared to Baseline will be measured using the BSAI.
Timepoint [1] 0 0
Months 1, 3, 6, 9 12,15,18, 21, 24, 27, 30, 33, 36, 39, 42, 25, and 48

Eligibility
Key inclusion criteria
- Informed Consent form signed by the subject or subject's legal representative; if the
subject is under the age of 18 but capable of providing assent, signed assent from the
subject.

- Subject (or caretaker) must be willing to comply with all protocol requirements.

- Patients who completed the SD-005 study (on study drug at Visit 5).
Minimum age
1 Month
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Patients who do not meet the entry criteria outlined above.

- Pregnancy or breastfeeding during the study. (A urine pregnancy test will be performed
at the final visit for SD-005 for female patients of childbearing potential and
repeated at Visit 1 if these visits do no occur on the same day)

- Females of childbearing potential who are not abstinent or not practicing a medically
acceptable method of contraception

Study design
Purpose of the study
Treatment
Allocation to intervention
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
Not Applicable
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Terminated
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Premier Specialists Pty Ltd. - Kogarah
Recruitment hospital [2] 0 0
The Royal Children's Hospital - 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
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
Warszawa
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 aim is to assess the long-term safety of topical use of ZORBLISA in patients with
Epidemolysis Bullosa.
Trial website
https://clinicaltrials.gov/show/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/show/NCT02670330