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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03154333




Registration number
NCT03154333
Ethics application status
Date submitted
10/05/2017
Date registered
16/05/2017

Titles & IDs
Public title
Safety and Efficacy of Diacerein 1% Ointment for Subjects With Epidermolysis Bullosa Simplex (EBS)
Scientific title
An International, Multicenter, Randomized, Double-Blind, Parallel-Group Phase 2 Study Evaluating the Safety and Efficacy of Diacerein 1% Ointment Topical Formulation in Subjects With Epidermolysis Bullosa Simplex
Secondary ID [1] 0 0
CCP-020-301
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Epidermolysis Bullosa Simplex 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 - diacerein 1% ointment
Treatment: Drugs - A placebo ointment

Experimental: diacerein 1% ointment - diacerein 1% ointment will be used for 8 weeks

Placebo comparator: vehicle ointment - vehicle ointment will be used for 8 weeks


Treatment: Drugs: diacerein 1% ointment
diacerein 1% ointment administered topically

Treatment: Drugs: A placebo ointment
vehicle ointment administered topically
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Proportion of Subjects Who Achieved = 60% Reduction in Body Surface Area (BSA) of EBS Lesions Within Assessment Area
Timepoint [1] 0 0
Baseline to Week 8
Secondary outcome [1] 0 0
The Proportion of Subjects Who Achieved Success on the Investigator's Global Assessment (IGA)
Timepoint [1] 0 0
Baseline to Week 8

Eligibility
Key inclusion criteria
Key

* Subject is at least 4 years of age at Screening
* Subject has a documented genetic mutation consistent with EBS. Gene mutations acceptable for inclusion are as follows: KRT5, KRT14, PLEC1, TGM5, PKP1, DSP, FERMT1, EXPH5, DST, KLHL24.
* Subject has an Assessment Area of EBS lesions to be treated, that is =2% body surface area (BSA) and the EBS lesions are in one or both of the following body areas:

* Localized: plantar and/or palmar areas
* Generalized: arms, legs, torso, hands and feet
* Subject's EBS lesions in the Assessment Area have an Investigator's Global Assessment (IGA) score of =3
* Subject/caregiver agrees to not use any topical therapies other than the study medication that might influence the status of the EBS lesions during the duration of the study
* Subject is non-pregnant as confirmed by a negative urine pregnancy screen, non-lactating and is not planning for pregnancy during the study period
* If the subject is a woman of childbearing potential, agrees to use an approved effective method of birth control
* Subject is in good general health and free of any known disease state or physical condition which might impair evaluation of the EBS lesions or which exposes the subject to an unacceptable risk by study participation

Key
Minimum age
4 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Subject has EBS lesions to be treated that are infected
* Subject has used any diacerein containing product within 6 months prior to Screening
* Subject has used systemic immunotherapy or cytotoxic chemotherapy within 60 days prior to Screening
* Subject has used systemic steroidal therapy or has used topical steroidal therapy on the EBS lesions to be treated within 30 days prior to Baseline
* Subject has evidence of a systemic infection or has used systemic antibiotics within 7 days prior to Screening
* Subject is currently using systemic analgesics and/or anti-histamine therapy, for treatment of EBS lesions unless on a stable regimen (i.e., the same dosing regimen) for at least 4 weeks prior to Screening
* Subject has used any systemic diuretics or cardiac glycosides or any systemic product that might put the subject at undue risk
* Subject has used any topical product containing allantoin on the EBS lesions to be treated within 30 days prior to Screening
* Subject has a current malignancy, or a history of treatment for a malignancy within 2 years prior to Screening
* Subject currently has diabetes mellitus (HbA1c =6.5%) or controlled diabetes (HbA1c < 6.5%)
* Subject has a history of cardiac, hepatic (ALT and or AST >2x ULN, Total bilirubin >1.5x ULN at Screening), or renal disease (eGFR<30 ml/min/1.73 m^2)
* Subject has a non-EBS skin disease or condition (e.g., sunburn) that might put the subject at undue risk by study participation or interferes with the study medication application or the study assessments

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
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
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
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
1/06/2017
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
31/10/2018
Sample size
Target
Accrual to date
Final
54
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
Premier Specialists Pty Ltd; The Church - Kogarah
Recruitment postcode(s) [1] 0 0
2217 - Kogarah
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
Illinois
Country [5] 0 0
United States of America
State/province [5] 0 0
Minnesota
Country [6] 0 0
United States of America
State/province [6] 0 0
Missouri
Country [7] 0 0
United States of America
State/province [7] 0 0
New York
Country [8] 0 0
United States of America
State/province [8] 0 0
North Carolina
Country [9] 0 0
United States of America
State/province [9] 0 0
Ohio
Country [10] 0 0
United States of America
State/province [10] 0 0
South Carolina
Country [11] 0 0
United States of America
State/province [11] 0 0
Texas
Country [12] 0 0
Austria
State/province [12] 0 0
Salzburg
Country [13] 0 0
France
State/province [13] 0 0
Cedex
Country [14] 0 0
France
State/province [14] 0 0
Nice
Country [15] 0 0
Germany
State/province [15] 0 0
Freiburg
Country [16] 0 0
Israel
State/province [16] 0 0
Tel Aviv
Country [17] 0 0
Netherlands
State/province [17] 0 0
Groningen
Country [18] 0 0
United Kingdom
State/province [18] 0 0
England
Country [19] 0 0
United Kingdom
State/province [19] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Castle Creek Pharmaceuticals, LLC
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Mary Spellman, MD
Address 0 0
Castle Creek Pharmaceuticals
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

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT03154333