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A pharmacokinetic (PK) study in 16-20 EB subjects to be allocated to two cohorts. Cohort 1 to include 8-10 subjects (ages 12 yrs and older); Cohort 2 to include 8-10 subjects (ages 6 months-11 yrs, inclusive). Cohort 2 only included subjects 4 yrs and older. Serial PK blood sampling collected on Days 1 and 10. Analyses were performed to determine the concentrations of diacerein and rhein.
This was an open-label, single-period study that intended to include subjects with EB consisting of infants/ children (ages 6 months to 11 years, inclusive) and adolescents/ adults (ages 12 years and older). The study included 2 cohorts.
Cohort 1 was to include adolescent/adult subjects with EB (ages 12 years and older);
Cohort 2 was to include infants/children with EB (ages 4 to 11 years, inclusive).
For both cohorts, the study intended to enroll subjects with epidermolysis bullosa simplex (EBS), dystrophic epidermolysis bullosa (DEB), and junctional epidermolysis bullosa (JEB) with lesions encompassing >/=2% BSA. The diacerein application area was >/=5% BSA and included lesioned and non-lesioned skin (if lesions accounted for <5% BSA); however, topical administration was \
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cohort 1 (Adolescents, Adults) | Experimental | Adolescent and adult subjects with EB (aged 12 years and older) received diacerein 1% ointment daily for 10 days. |
|
| Cohort 2 (Children) | Experimental | Children with EB (aged 4 to 11 years, inclusive) received diacerein 1% ointment daily for 10 days. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Diacerein 1% Ointment | Drug | Diacerein 1% Ointment administered topically |
|
| Measure | Description | Time Frame |
|---|---|---|
| Detectable Plasma Concentrations of Diacerein and Rhein | Bioanalytical analyses were performed to determine concentrations levels of diacerein and rhein in plasma using validated bioanalytical methods. For Cohort 1, blood samples were taken at pre-dose and 0.5, 1, 2, 3, 4, 6, and 8 hours post-dose. For Cohort 2, blood samples were taken at pre-dose and 1, 2, 4, 6, and 8 hours post-dose. Trough PK samples were collected on any 2 available days from Days 3 through 9 for Cohort 1 only. Summary statistics for each scheduled time were only reported if at least 50% of subjects had quantifiable concentrations. | Days 1-10, at select time points per protocol |
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Key Inclusion Criteria:
Key Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Mary Spellman | Castle Creek Pharmaceuticals, LLC | Study Chair |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Stanford School of Medicine | Palo Alto | California | 94304 | United States | ||
| Northwestern University Medical Center |
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| ID | Title | Description |
|---|---|---|
| FG000 | Cohort 1 (Adolescents, Adults) | Adolescent and adult subjects with EB (aged 12 and older) received diacerein 1% ointment daily for 10 days |
| FG001 | Cohort 2 (Children) | Children with EB (aged 4 to 11 years, inclusive) received diacerein 1% ointment daily for 10 days |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| SAP | No | Yes | No | Statistical Analysis Plan | Jan 7, 2019 | Jul 10, 2019 |
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| Chicago |
| Illinois |
| 60654 |
| United States |
| UNC Dermatology | Chapel Hill | North Carolina | 27599 | United States |
| COMPLETED |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | Cohort 1 (Adolescents, Adults) | Adolescent and adult patients with EB (aged 12 and older) received Diacerein 1% Ointment daily for 10 days. |
| BG001 | Cohort 2 (Children) | Children with EB (aged 4 to 11 years, inclusive) received Diacerein 1% Ointment daily for 10 days. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Region of Enrollment | Count of Participants | Participants |
| ||||||||||||||||
| EB Classification per Cohort | Number | participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Detectable Plasma Concentrations of Diacerein and Rhein | Bioanalytical analyses were performed to determine concentrations levels of diacerein and rhein in plasma using validated bioanalytical methods. For Cohort 1, blood samples were taken at pre-dose and 0.5, 1, 2, 3, 4, 6, and 8 hours post-dose. For Cohort 2, blood samples were taken at pre-dose and 1, 2, 4, 6, and 8 hours post-dose. Trough PK samples were collected on any 2 available days from Days 3 through 9 for Cohort 1 only. Summary statistics for each scheduled time were only reported if at least 50% of subjects had quantifiable concentrations. | Cohort 1: 4 adolescent/adult subjects with EBS, 4 adolescent/adult subjects with DEB; Cohort 2: 3 child subjects with EBS | Posted | Count of Participants | Participants | Days 1-10, at select time points per protocol |
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Baseline to 30 days after last dose of study drug application (40 days)
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Cohort 1 (Adolescents, Adults) | Adolescent/adult patients with EB (aged 12 and older) received Diacerein 1% Ointment daily for 10 days | 0 | 8 | 0 | 8 | 1 | 8 |
| EG001 | Cohort 2 (Children) | Children with EB (aged 4 to 11 years, inclusive) received Diacerein 1% Ointment daily for 10 days | 0 | 3 | 0 | 3 | 0 | 3 |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pyrexia | General disorders | MedDRA (19.0), 2019 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Mary Spellman/Chief Medical Officer | Castle Creek Pharmaceuticals, LLC | 862 286 0400 | mspellman@castlecreekpharma.com |
| SAP_000.pdf |
| Prot | Yes | No | No | Study Protocol | Feb 7, 2018 | Jul 17, 2019 | Prot_001.pdf |
| ID | Term |
|---|---|
| D004820 | Epidermolysis Bullosa |
| D016110 | Epidermolysis Bullosa Simplex |
| D016108 | Epidermolysis Bullosa Dystrophica |
| D016109 | Epidermolysis Bullosa, Junctional |
| ID | Term |
|---|---|
| D012868 | Skin Abnormalities |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D012873 | Skin Diseases, Genetic |
| D030342 | Genetic Diseases, Inborn |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D012872 | Skin Diseases, Vesiculobullous |
| D003095 | Collagen Diseases |
| D003240 | Connective Tissue Diseases |
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| ID | Term |
|---|---|
| C025292 | diacerein |
| D009824 | Ointments |
| ID | Term |
|---|---|
| D004304 | Dosage Forms |
| D004364 | Pharmaceutical Preparations |
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| Male |
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| Not Hispanic or Latino |
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| Unknown or Not Reported |
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| Asian |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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| Dystrophic Epidermolysis Bullosa (DEB) |
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| Junctional Epidermolysis Bullosa (JEB) |
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| Rhein Below the limit of quantification:DEB |
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| Rhein Above Lower limit of quantification:EBS |
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| Rhein Above Lower limit of quantification:DEB |
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| Diacerein Below the limit of quantification:EBS |
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| Diacerein Below the limit of quantification:DEB |
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| Diacerein Above Lower limit of quantification:EBS |
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| Diacerein Above Lower limit of quantification:DEB |
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