Safety and Efficacy of Diacerein 1% Ointment for Subjects With Epidermolysis Bullosa Simplex (EBS)

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03154333
• Date of registration: 10/05/2017
• Prospective Registration: Yes
• Primary sponsor: Castle Creek Pharmaceuticals, LLC
• 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
• Date of first enrolment: June 1, 2017
• Target sample size: 54
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT03154333
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

Australia, Austria, France, Germany, Israel, Netherlands, United Kingdom, United States

Contacts

• Name: Mary Spellman, MD
• Affiliation: Castle Creek Pharmaceuticals

Key inclusion & exclusion criteria

Key Inclusion Criteria:

- 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 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



Age minimum: 4 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Epidermolysis Bullosa Simplex

Intervention(s)

Drug: A placebo ointment

Drug: diacerein 1% ointment

Primary Outcome(s)

Proportion of Subjects Who Achieved = 60% Reduction in Body Surface Area (BSA) of EBS Lesions Within Assessment Area [Time Frame: Baseline to Week 8]

Secondary Outcome(s)

The Proportion of Subjects Who Achieved Success on the Investigator's Global Assessment (IGA) [Time Frame: Baseline to Week 8]

Secondary ID(s)

CCP-020-301

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 05/11/2019
• URL: https://clinicaltrials.gov/ct2/show/results/NCT03154333