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Trial record 1 of 1 for:    NCT03154333
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Safety and Efficacy of Diacerein 1% Ointment for Subjects With Epidermolysis Bullosa Simplex (EBS)

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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03154333
Recruitment Status : Terminated (Futility)
First Posted : May 16, 2017
Results First Posted : November 5, 2019
Last Update Posted : November 5, 2019
Sponsor:
Information provided by (Responsible Party):
Castle Creek Pharmaceuticals, LLC

Brief Summary:
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 purpose of this study is to compare the efficacy of diacerein 1% ointment to vehicle ointment when applied once-daily for 8 weeks in subjects with EBS.

Condition or disease Intervention/treatment Phase
Epidermolysis Bullosa Simplex Drug: diacerein 1% ointment Drug: A placebo ointment Phase 2

Detailed Description:

The study is an international, multicenter, randomized, double-blind, vehicle-controlled, parallel group study to evaluate the safety and efficacy of diacerein 1% ointment for the treatment of subjects with EBS. Participants were assigned to study groups receiving either diacerein 1% ointment or vehicle ointment for 8 weeks, followed by an 8 week follow-up period. Approximately 80 subjects were to be randomized to one of the 2 treatment groups.

The primary objective of this study is to compare the efficacy of diacerein 1% ointment to vehicle ointment based on reduction in body surface area (BSA) of EBS lesions being treated when applied once-daily for 8 weeks in subjects with EBS. The secondary objectives are to compare the effects of diacerein 1% ointment to vehicle ointment in subjects with EBS in changes in Investigator Global Assessment (IGA) scores, pain, pruritus, mobility, and safety and tolerability.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 54 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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 Epidermolysis Bullosa Simplex
Actual Study Start Date : June 1, 2017
Actual Primary Completion Date : October 15, 2018
Actual Study Completion Date : October 31, 2018


Arm Intervention/treatment
Experimental: diacerein 1% ointment
diacerein 1% ointment will be used for 8 weeks
Drug: diacerein 1% ointment
diacerein 1% ointment administered topically
Other Name: CCP-020

Placebo Comparator: vehicle ointment
vehicle ointment will be used for 8 weeks
Drug: A placebo ointment
vehicle ointment administered topically




Primary Outcome Measures :
  1. Proportion of Subjects Who Achieved ≥ 60% Reduction in Body Surface Area (BSA) of EBS Lesions Within Assessment Area [ Time Frame: Baseline to Week 8 ]
    Analysis of the proportion of subjects who achieved a ≥60% reduction in Body Surface Area (BSA) of EBS lesions within Assessment Area from Baseline to Week 8


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

    The investigator's global assessment (IGA) is a five-point scale that is used for overall clinical assessment of severity of disease and classifies EBS-involved skin with a score ranging from 0-4. Success on the IGA was defined as ≥2-point reduction from Baseline to Visit 6 (Week 8).

    IGA Scoring:

    0 = Clear; 1 = Near Clear; 2 = Mild; 3 = Moderate; 4 = Severe

    Minimum score = 0 Maximum score = 4; higher score = worse outcome




Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   4 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03154333


Locations
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Sponsors and Collaborators
Castle Creek Pharmaceuticals, LLC
Investigators
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Study Director: Mary Spellman, MD Castle Creek Pharmaceuticals
  Study Documents (Full-Text)

Documents provided by Castle Creek Pharmaceuticals, LLC:
Statistical Analysis Plan  [PDF] August 31, 2018
Study Protocol  [PDF] January 2, 2018


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Responsible Party: Castle Creek Pharmaceuticals, LLC
ClinicalTrials.gov Identifier: NCT03154333    
Other Study ID Numbers: CCP-020-301
First Posted: May 16, 2017    Key Record Dates
Results First Posted: November 5, 2019
Last Update Posted: November 5, 2019
Last Verified: October 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Castle Creek Pharmaceuticals, LLC:
Epidermolysis
bullosa
simplex
EBS
Additional relevant MeSH terms:
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Epidermolysis Bullosa
Epidermolysis Bullosa Simplex
Skin Abnormalities
Congenital Abnormalities
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Skin Diseases, Vesiculobullous
Diacetylrhein
Anti-Inflammatory Agents