A Multi-center Study to Evaluate the Pharmacokinetics of Diacerein and Rhein and the Safety of Diacerein after Maximum Use, Topical Administration of CCP-020 (Diacerein 1% ointment) to Patients with Epidermolysis Bullosa (EB)

Withdrawn

Conditions: Epidermolysis Bullosa
10040789
10014982

Registration Number: NL-OMON46073

Lead Sponsor: Castle Creek Pharmaceuticals, LLC

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Withdrawn

Sex: Not specified

Target Recruitment: 8

Inclusion Criteria

1. Subject/caregiver is able to comprehend and willing to sign an Informed Consent and/or Assent Form.
2. Subject is male or female at least 12 years of age (Cohort 1) or at least 6 months of age to 11 years, inclusive (Cohort 2) at screening.
3. The subject must weigh at least 9 kg (19.8 lbs) at Screening.
4. Subject has a documented genetic mutation consistent with EB. A blood or saliva sample will be collected for genetic confirmation if no documented gene mutation data is available.
5. Subject has EB lesions on * 2% body surface area (BSA) and the EB lesions are in the following body areas:
a. Localized: plantar and/or palmar areas
b. Generalized: arms, legs, torso, hands and feet.
6. Subject/caregiver agrees to not apply any other topical products to the application area during the treatment period
7. 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.
8. Subject is non-pregnant, non-lactating and is not planning for pregnancy during the study period
9. Subject is in good general health and free of any known disease state or physical condition which, in the investigator*s opinion, which exposes the subject to an unacceptable risk by study participation.
10. Subject is willing and able to follow all study instructions and to attend all study visits.

Exclusion Criteria

1. Subject has EB lesions where drug will be applied that are infected (i.e., EB lesions that require anti-microbial therapy to treat an infection)
2. Subject has used any diacerein containing product within 1 month prior to Visit 1
3. Subject has used systemic immunotherapy or cytotoxic chemotherapy within 60 days prior to dosing.
4. Subject has used systemic steroidal therapy or has used topical steroidal therapy on the EB lesions in the application area within 14 days prior to dosing (Note: inhaled, nasal sprays, and ophthalmic products containing steroids are allowed)
5. Subject has evidence of a systemic infection or has used systemic antibiotics within 7 days prior to dosing
6. 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 dosing
7. Subject has a current malignancy, or a history of treatment for a malignancy within 2 years prior to dosing (Note: does not include non-melanoma skin cancer)
8. Subject currently has diabetes mellitus (HbA1c *6.5%) Note: controlled diabetes (HbA1c < 6.5%) is also considered exclusionary
9. 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 m2 [MDRD-adults *18, Bedside Schwartz * children <18]) that, in the opinion of the investigator, might put the subject at undue risk by study participation or interferes with the study medication application of the study assessments
10. Subject has an active non-EB skin disease (e.g., psoriasis, atopic dermatitis, eczema, sun damage, etc.), or condition (e.g., sunburn) that, in the opinion of the investigator, would put the subject at undue risk by study participation or would interfere with the study medication application or the study assessments
11. Subject has a history of sensitivity to any of the ingredients in the study medication
12. Subject has participated in an investigational drug trial in which administration of an investigational study medication occurred within 30 days prior to dosing

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Derived plasma PK parameters (Cmax, Tmax and AUC), if available, for diacerein<br /><br>and rhein will be summarized by treatment using descriptive statistics (sample<br /><br>size, arithmetic and geometric mean, CV%, SD of the arithmetic mean, median,<br /><br>minimum, and maximum)</p><br>

Secondary Outcome Measures