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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
  Purpose
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.

Condition Intervention Phase
Epidermolysis Bullosa Simplex Drug: Diacerein 1% Ointment Topical Formulation Drug: Vehicle Phase 2

Study Type: Interventional
Study Design: 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 (EBS)

Resource links provided by NLM:


Further study details as provided by Castle Creek Pharmaceuticals, LLC:

Primary Outcome Measures:
  • 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.


Secondary Outcome Measures:
  • 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.


Estimated Enrollment: 80
Actual Study Start Date: May 31, 2017
Estimated Study Completion Date: May 2018
Estimated Primary Completion Date: May 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Diacerein 1% ointment
Diacerein 1% ointment topical formulation will be used for 8 weeks
Drug: Diacerein 1% Ointment Topical Formulation
Diacerein 1% Ointment administered topically
Placebo Comparator: A placebo topical ointment
Placebo topical ointment will be used for 8 weeks
Drug: Vehicle
Place ointment administered topically

Detailed Description:

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.

  Eligibility

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.


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

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
  Contacts and Locations
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
United States, California
Stanford University Recruiting
Palo Alto, California, United States, 94304
Contact: Elidia Tafoya    650-743-8520    etafoya@stanford.edu   
Principal Investigator: Joyce Teng, MD         
United States, Colorado
Children's Hospital Colorado Recruiting
Aurora, Colorado, United States, 80045
Contact: Kathleen Peoples    720-777-4708    Kathleen.Peoples@childrenscolorado.org   
Principal Investigator: Anna Bruckner, MD         
United States, Illinois
Northwestern University Recruiting
Chicago, Illinois, United States, 60611
Contact: Rema Zebda    312-227-6484    rema.zebda@northwestern.edu   
Principal Investigator: Amy Paller, MD         
United States, Missouri
University of Missouri Healthcare Recruiting
Columbia, Missouri, United States, 65212
Contact: Hope Gole    573-882-8030    golehk@health.missouri.edu   
Principal Investigator: Jonathan Dyer, MD         
United States, North Carolina
University of North Carolina - Chapel Hill Recruiting
Chapel Hill, North Carolina, United States, 27516
Contact: Laura Keck    984-974-3682    laura_mcleod@med.unc.edu   
Principal Investigator: Aida Lugo-Somolinos, MD         
United States, Texas
Texas Dermatology and Laser Specialists Recruiting
San Antonio, Texas, United States, 78218
Contact: Jenna Encina    210-852-2779    jenna@texasdls.com   
Principal Investigator: John Browning, MD         
Germany
University Medical Center Freiburg Recruiting
Freiburg, Germany, 79104
Contact: Aurelia Winter    +49 (0) 761 270-67141    aurelia.winter@uniklinik-freiburg.de   
Principal Investigator: Cristina Has, MD         
Israel
Tel Aviv Sourasky Medical Center Recruiting
Tel Aviv, Israel, 6423906
Contact: Udi Ben Dor    +972-3-6973768    udibd@tlvmc.gov.il   
Principal Investigator: Eli Sprecher         
Netherlands
University Medical Center Groningen ; Polikliniek Dermatologie Recruiting
Groningen, Netherlands, AB20;30,001
Contact: Ena Sokol    +31 (0)50 361 2520    e.sokol@umcg.nl   
Principal Investigator: Marcel Jonkman, MD         
Sponsors and Collaborators
Castle Creek Pharmaceuticals, LLC
Investigators
Study Director: Greg Licholai, MD Castle Creek Pharmaceuticals
  More Information

Additional Information:
Responsible Party: Castle Creek Pharmaceuticals, LLC
ClinicalTrials.gov Identifier: NCT03154333     History of Changes
Other Study ID Numbers: CCP-020-301
First Submitted: May 10, 2017
First Posted: May 16, 2017
Last Update Posted: October 2, 2017
Last Verified: September 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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
blister
EBS
topical
ointment
lesion

Additional relevant MeSH terms:
Epidermolysis Bullosa
Epidermolysis Bullosa Simplex
Skin Abnormalities
Congenital Abnormalities
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Skin Diseases, Vesiculobullous
Diacetylrhein
Anti-Inflammatory Agents