Trial Search Results

Safety and Efficacy of Diacerein 1% Ointment Topical Formulation Compared to Placebo for 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 epidermis. Diacerein 1% Ointment is a topical ointment that is being developed for the treatment of EBS. Diacerein in the topical formulation is hydrolyzed to rhein in the epidermis and dermis following administration. Diacerein and rhein have been shown to inhibit the in vitro and in vivo production and activity of interleukin-1β (IL-1β) and other pro-inflammatory cytokines.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.

Stanford is currently not accepting patients for this trial.

Lead Sponsor:

Castle Creek Pharmaceuticals, LLC

Stanford Investigator(s):

Intervention(s):

  • Drug: Diacerein 1% Ointment Topical Formulation
  • Drug: Vehicle

Phase:

Phase 2

Eligibility


Inclusion Criteria:

   - Subject is male or female at least 4 years of age at Visit 1

   - Subject has a documented genetic mutation consistent with EBS. A blood or saliva
   sample will be collected for genetic confirmation if no documented gene mutation data
   is available. 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 (plantar areas where >25% of the area has
      hyperkeratosis that has been present for greater than 12 weeks cannot be included
      as part of the Assessment Area

      - Generalized: arms, legs, torso, hands and feet (scalp, groin and areas where, in
      the investigator's opinion, the study medication might become occluded cannot be
      included as part of the Assessment Area)

   - 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 (e.g., medicated cleansers, CBD oil, MediHoney, Silvadine cream 1%, the
   Investigator should consult the Medical Monitor regarding therapies not specified in
   the protocol

   - Subject/caregiver agrees to follow topical product application instructions during the
   treatment period

   - If the subject is a woman of childbearing potential, she has a negative urine
   pregnancy test and agrees to use an approved effective method of birth control, as
   defined by this protocol, for the duration of the study.

   - Subject is non-pregnant, non-lactating and is not planning for pregnancy during the
   study period

   - Subject is in good general health and free of any known disease state or physical
   condition which, in the investigator's opinion, might impair evaluation of the EBS
   lesions or which exposes the subject to an unacceptable risk by study participation

   - Subject is willing and able to follow all study instructions and to attend all study
   visits

   - Subject/caregiver is able to comprehend and willing to sign an Informed Consent and/or
   Assent Form.

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 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 2 (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, for
   treatment of EBS lesions unless on a stable regimen (i.e., the same dosing regimen)
   for at least 4 weeks prior to Visit 1. Note: As needed (PRN) use of
   acetaminophen/paracetamol or NSAIDs within the 4 weeks prior to Visit 1 are permitted
   provided the treatment was unrelated to EBS symptom relief.

   - Subject has used any systemic diuretics or cardiac glycosides or any systemic product
   that, in the opinion of the investigator, might put the subject at undue risk by study
   participation or interferes with the study medication application or the study
   assessments within 30 days prior to Visit 1

   - Subject has used any topical product containing allantoin on the EBS lesions to be
   treated within 30 days prior to Visit 1

   - 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 (HbA1c ≥6.5%); Note: controlled diabetes
   (HbA1c < 6.5%) is also considered exclusionary

   - Subject has a history of cardiac, hepatic (ALT and or AST >2x ULN, Total bilirubin
   >1.5x ULN at Visit 1), or renal disease (eGFR<30 ml/min/1.73 m^2) that, in the opinion
   of the investigator, might put the subject at undue risk by study participation or
   interferes with the study medication application or the study assessments

   - Subject has a non-EBS skin disease (e.g., psoriasis, atopic dermatitis, eczema, sun
   damage, etc.), or condition (e.g., sunburn) that, in the opinion of the investigator,
   might put the subject at undue risk by study participation or interferes with the
   study medication application or the study assessments

   - Subject has a history of sensitivity to any of the ingredients in the study
   medications

   - Subject has participated in an investigational drug trial/device in which
   administration of an investigational study medication occurred within 30 days prior to
   Visit 1.

Ages Eligible for Study

4 Years - N/A

Genders Eligible for Study

All

Not currently accepting new patients for this trial

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Elidia Tafoya
650-743-8520
Not Recruiting