Clinical Trial: Safety and Efficacy of Diacerein 1% Ointment Topical Formulation Compared to Placebo for Subjects With Epidermolysis Bullosa Simplex (EBS)

Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Interventional

Official 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 The proposed study is an international, randomized, double-blind, parallel-group Phase 2 study. Participants will be subject to up to 6-week screening period, then be assigned to treat EBS lesions once daily with either Diacerein 1% ointment topical formulation or placebo control ointment for 8 weeks, followed by an 8 week follow-up period. Approximately 80 subjects are planned to be randomized to one of the 2 treatment groups in this study at approximately 18 international investigational centers.

The objective of this study is to compare the efficacy of Diacerein 1% Ointment to Control Ointment when applied once-daily in subjects with EBS using an Investigator Global Assessment to determine overall treatment success. The study will also compare the effects of Diacerein 1% Ointment to Control Ointment in subjects with EBS in terms of safety and tolerability, patient-reported effects on pruritus, pain, mobility, as well as reduction in body surface area of EBS lesions, and reduction in lesion surface area of the referenced lesion.


Sponsor: Castle Creek Pharmaceuticals, LLC

Current Primary Outcome: Efficacy of Diacerein 1% Ointment compared to Control Ointment [ Time Frame: Change from Visit 8 (Week 16) compared to Visit 2 (Week 0) ]

Statistically significant difference between Diacerein 1% ointment and Control ointment in terms of the proportion of subject who achieve treatment success defined as an Investigator's Global Assessment (IGA) grade 0 or 1 at Visit 8 (Week 16) compared to Visit 2 (Week 0) with at least a 2-point reduction in the IGA score.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Number of patients with adverse events based on assessment of safety measures [ Time Frame: Change from Visit 8 (Week 16) compared to Visit 2 (Week 0) ]
    Number of patients with adverse events using the following safety measure assessments: demographics\medical history, adverse events and SAEs, vital signs, physical examination, clinical laboratory, and urine pregnancy tests.
  • Pruritus Intensity assessed using the Numeric Rating Scale [ Time Frame: Change from Visit 8 (Week 16) compared to Visit 2 (Week 0) ]
    Pruritus Intensity will be assessed using the Numeric Rating Scale. The Pruritus Intensity is the subject's, or caregiver's if appropriate, assessment of the average overall intensity of pruritus experienced by the subject over the previous 24 hours on all EBS lesions
  • Pain Intensity assessed using the Numeric Rating Scale or Face Diagram [ Time Frame: Change from Visit 8 (Week 16) compared to Visit 2 (Week 0) ]
    Pain Intensity will be assessed using the Numeric Rating Scale or Face Diagram to describe the pain intensity.
  • Mobility Assessment using a series of age-appropriate questions in the eDiary [ Time Frame: Change from Visit 8 (Week 16) compared to Visit 2 (Week 0) ]
    The Mobility Assessment is the subject/caregiver's assessment of the subject's degree of mobility over the previous 24 hours. The subject/caregiver will report the degree of mobility by answering a series of age-appropriate questions in the eDiary.
  • Reduction in BSA of EBS lesions based on the investigator's assessment [ Time Frame: Change from Visit 8 (Week 16) compared to Visit 2 (Week 0) ]
    The BSA will be determined by the investigator and 1% BSA is defined as the area of the subject's hand held flat, including the thumb and fingers held together.
  • Lesion Surface Area of the Reference Lesion based on the investigator's assessment or photographs [ Time Frame: Change from Visit 8 (Week 16) compared to Visit 2 (Week 0) ]
    The Lesion Surface Area will measure the length and width of the Reference Lesion using a ruler. Length will be measured to the nearest millimeter. Width (the length of the longest axis perpendicular to the length) will be measured to the nearest millimeter.


Original Secondary Outcome: Same as current

Information By: Castle Creek Pharmaceuticals, LLC

Dates:
Date Received: May 10, 2017
Date Started: May 2017
Date Completion: May 2018
Last Updated: May 12, 2017
Last Verified: May 2017