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

This study is currently recruiting participants.
Verified September 2017 by Castle Creek Pharmaceuticals, LLC
Sponsor:
ClinicalTrials.gov Identifier:
NCT03154333
First Posted: May 16, 2017
Last Update Posted: October 2, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
Castle Creek Pharmaceuticals, LLC
May 10, 2017
May 16, 2017
October 2, 2017
May 31, 2017
May 2018   (Final data collection date for primary outcome measure)
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.
Same as current
Complete list of historical versions of study NCT03154333 on ClinicalTrials.gov Archive Site
  • 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.
Same as current
Not Provided
Not Provided
 
Safety and Efficacy of Diacerein 1% Ointment Topical Formulation Compared to Placebo for Subjects With Epidermolysis Bullosa Simplex (EBS)
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 (EBS)
Epidermolysis bullosa simplex (EBS) is a rare genetic skin disease characterized by fragility of the skin and mucous membranes resulting in painful blisters and erosions after minor trauma. The simplex form is classified by skin blister development in the basal and suprabasal epidermis. Diacerein 1% Ointment is a topical ointment that is being developed for the treatment of EBS with the potential to block activity of interleukin-1β (IL-1β), a pro-inflammatory cytokine, and reduce the auto-inflammatory effects in the skin of patients with EBS. The purpose of this study is to compare the efficacy of Diacerein 1% Ointment to Control Ointment when applied once-daily for 8 weeks in subjects with EBS.

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.

Interventional
Phase 2
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Epidermolysis Bullosa Simplex
  • Drug: Diacerein 1% Ointment Topical Formulation
    Diacerein 1% Ointment administered topically
  • Drug: Vehicle
    Place ointment administered topically
  • Experimental: Diacerein 1% ointment
    Diacerein 1% ointment topical formulation will be used for 8 weeks
    Intervention: Drug: Diacerein 1% Ointment Topical Formulation
  • Placebo Comparator: A placebo topical ointment
    Placebo topical ointment will be used for 8 weeks
    Intervention: Drug: Vehicle
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
80
May 2018
May 2018   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  • Subject has a clinical diagnosis of EBS
  • Subject has a laboratory confirmed diagnosis of EBS or has a blood sample collected for genetic confirmation (laboratory confirmation is required for randomization)
  • 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)
  • Subject's EBS lesions to be treated have an Investigator's Global Assessment (IGA) score of ≥3
  • Be able to comply with all other study requirement

Exclusion Criteria:

  • Subject has EBS lesions to be treated that are infected (i.e., EBS lesions that require therapy to treat an infection)
  • Subject has another member of her/his immediate family (i.e., living in the same household) enrolled in this study
  • Subject has used any diacerein containing product within 6 months prior to Visit 1
  • Subject has used systemic immunotherapy or cytotoxic chemotherapy within 60 days prior to Visit 1
  • 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)
  • Subject has evidence of a systemic infection or has used systemic antibiotics within 7 days prior to Visit 1
  • Subject is currently using systemic analgesics and/or anti-histamine therapy, unless on a stable regimen
  • 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)
  • Subject currently has diabetes mellitus
Sexes Eligible for Study: All
4 Years and older   (Child, Adult, Senior)
No
Germany,   Israel,   Netherlands,   United States
 
 
NCT03154333
CCP-020-301
Yes
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Plan to Share IPD: No
Castle Creek Pharmaceuticals, LLC
Castle Creek Pharmaceuticals, LLC
Not Provided
Study Director: Greg Licholai, MD Castle Creek Pharmaceuticals
Castle Creek Pharmaceuticals, LLC
September 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP