An International Study Evaluating the Safety and Efficacy of Diacerein 1% Ointment Topical Formulation in Subjects with Epidermolysis Bullosa Simplex (EBS)
- Conditions
- Epidermolysis Bullosa Simplex (EBS)MedDRA version: 20.0 Level: PT Classification code 10014989 Term: Epidermolysis bullosa System Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]
- Registration Number
- EUCTR2016-004427-24-FR
- Lead Sponsor
- Castle Creek Pharmaceuticals, LLC
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Not specified
- Target Recruitment
- 80
1.Subject is male or female at least 4 years of age
2.Subject has a clinical diagnosis of EBS
3.Subject has a laboratory confirmed diagnosis of EBS or has a blood sample collected for genetic confirmation (laboratory confirmation is required for randomization)
4.Subject has a total lesion surface area of EBS lesions to be treated, that is =2% and =30% body surface area (BSA) and the EBS lesions are in one or both of the following body areas:
-Localized: plantar and/or palmar areas (plantar areas where >25% of the area has hyperkeratosis that has been present for greater than 12 weeks are excluded)
-Generalized: arms, legs, torso, hands and feet (face, scalp, groin and areas where, in the investigator’s opinion, the study medication might become occluded are excluded)
5.Subject’s EBS lesions to be treated have an Investigator’s Global Assessment (IGA) score of =3
6.Subject/caregiver agrees to not use any topical therapies other than the study medication and the investigator approved bland non-medicated emollient/moisturizer that, in the investigators opinion, might influence the status of the EBS lesions during the duration of the study (e.g., bleach cleansers, bleach baths, topical antiseptics, topical disinfectants, etc.)
7.Subject/caregiver agrees to not apply any other topical products to the EBS lesions during the treatment period (Note: routine cleansing products and sunscreens are permitted)
8.If the subject is a woman of childbearing potential, she has a negative urine pregnancy test and agrees to use an approved effective method of birth control, as defined by this protocol, for the duration of the study.
9.Subject is non-pregnant, non-lactating and is not planning for pregnancy during the study period
10.Subject is in good general health and free of any known disease state or physical condition which, in the investigator’s opinion, might impair evaluation of the EBS lesions or which exposes the subject to an unacceptable risk by study participation
11.Subject is willing and able to follow all study instructions and to attend all study visits
12.Subject/caregiver is able to comprehend and willing to sign an Informed Consent and/or Assent Form.
Are the trial subjects under 18? yes
Number of subjects for this age range: 40
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 20
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 20
1.Subject has EBS lesions to be treated that are infected (i.e., EBS lesions that require therapy to treat an infection)
2.Subject has another member of her/his immediate family (i.e., living in the same household) enrolled in this study
3.Subject has used any diacerein containing product within 6 months prior to Visit 1
4.Subject has used systemic immunotherapy or cytotoxic chemotherapy within 60 days prior to Visit 1
5.Subject has used systemic steroidal therapy or has used topical steroidal therapy on the EBS lesions to be treated within 30 days prior to Visit 1 (Note: inhaled and ophthalmic products containing steroids are allowed)
6.Subject has evidence of a systemic infection or has used systemic antibiotics within 7 days prior to Visit 1
7.Subject is currently using systemic analgesics and/or anti-histamine therapy, unless on a stable regimen (i.e., the same dosing regimen, an as needed” [PRN] use is not a stable regimen) for at least 4 weeks prior to Visit 1
8.Subject has used any systemic diuretics or cardiac glycosides or any systemic product that, in the opinion of the investigator, might put the subject at undue risk by study participation or interferes with the study medication application or the study assessments within 30 days prior to Visit 1
9.Subject has used any topical product containing allantoin on the EBS lesions to be treated within 30 days prior to Visit 1
10.Subject has a current malignancy, or a history of treatment for a malignancy within 2 years prior to Visit 1 (Note: does not include non-melanoma skin cancer)
11.Subject currently has diabetes mellitus
12.Subject has a history of cardiac, hepatic, or renal disease that, in the opinion of the investigator, might put the subject at undue risk by study participation or interferes with the study medication application or the study assessments
13.Subject has a non-EBS skin disease (e.g., psoriasis, atopic dermatitis, eczema, sun damage, etc.), or condition (e.g., sunburn) that, in the opinion of the investigator, might put the subject at undue risk by study participation or interferes with the study medication application or the study assessments
14.Subject has a history of sensitivity to any of the ingredients in the study medications
15.Subject has participated in an investigational drug trial in which administration of an investigational study medication occurred within 30 days prior to Visit 1.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To compare the efficacy of Diacerein 1% Ointment to Control Ointment when applied once-daily in subjects with EBS;<br> Secondary Objective: To compare the effects of Diacerein 1% Ointment to Control Ointment in subjects with EBS in:<br> •Safety and tolerability<br> •Pruritus <br> •Pain<br> •Mobility<br> •Reduction in Body Surface Area (BSA) of EBS lesions<br> •Reduction in Lesion Surface Area (LSA) of the reference lesion.<br> ;Primary end point(s): A statistically significant difference by the Cochran-Mantel-Haenszel (CMH) test between the Diacerein 1% Ointment and the Control Ointment treatment groups in terms of the proportion of subjects who achieve success in the IGA at Visit 8 (Week 16), with success defined as an IGA grade of 0 (Clear) or 1 (Near Clear) with at least a 2-grade improvement.;Timepoint(s) of evaluation of this end point: Baseline (D1) and Visit 8 (D113)
- Secondary Outcome Measures
Name Time Method <br> Secondary end point(s): To compare the effects of Diacerein 1% Ointment to Control Ointment in subjects with EBS from Visit 2 (Week 0, D1) to Visit 8 (Week 16, D113) in:<br> • Safety and tolerability<br> • Pruritus <br> • Pain<br> • Mobility<br> • Reduction in BSA of EBS lesions<br> • Reduction in Lesion Surface Area of the Reference Lesion.<br> ;Timepoint(s) of evaluation of this end point: Baseline (D1) and Visit 8 (D113)