Trial Outcomes & Findings for To Evaluate the Pharmacokinetic of Diacerein and Rhein After Maximum Use in Patients With Epidermolysis Bullosa (EB) (NCT NCT03472287)
NCT ID: NCT03472287
Last Updated: 2020-02-10
Results Overview
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.
COMPLETED
PHASE1
11 participants
Days 1-10, at select time points per protocol
2020-02-10
Participant Flow
Participant milestones
| Measure |
Cohort 1 (Adolescents, Adults)
Adolescent and adult subjects with EB (aged 12 and older) received diacerein 1% ointment daily for 10 days
|
Cohort 2 (Children)
Children with EB (aged 4 to 11 years, inclusive) received diacerein 1% ointment daily for 10 days
|
|---|---|---|
|
Overall Study
STARTED
|
8
|
3
|
|
Overall Study
COMPLETED
|
8
|
3
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
To Evaluate the Pharmacokinetic of Diacerein and Rhein After Maximum Use in Patients With Epidermolysis Bullosa (EB)
Baseline characteristics by cohort
| Measure |
Cohort 1 (Adolescents, Adults)
n=8 Participants
Adolescent and adult patients with EB (aged 12 and older) received Diacerein 1% Ointment daily for 10 days.
|
Cohort 2 (Children)
n=3 Participants
Children with EB (aged 4 to 11 years, inclusive) received Diacerein 1% Ointment daily for 10 days.
|
Total
n=11 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
26.5 years
STANDARD_DEVIATION 11.74 • n=99 Participants
|
8.3 years
STANDARD_DEVIATION 1.53 • n=107 Participants
|
21.5 years
STANDARD_DEVIATION 13.0 • n=206 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
6 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
5 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
3 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
7 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
8 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Race (NIH/OMB)
White
|
7 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
9 Participants
n=206 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Region of Enrollment
United States
|
8 Participants
n=99 Participants
|
3 Participants
n=107 Participants
|
11 Participants
n=206 Participants
|
|
EB Classification per Cohort
Epidermolysis Bullosa Simplex (EBS)
|
4 participants
n=99 Participants
|
3 participants
n=107 Participants
|
7 participants
n=206 Participants
|
|
EB Classification per Cohort
Dystrophic Epidermolysis Bullosa (DEB)
|
4 participants
n=99 Participants
|
0 participants
n=107 Participants
|
4 participants
n=206 Participants
|
|
EB Classification per Cohort
Junctional Epidermolysis Bullosa (JEB)
|
0 participants
n=99 Participants
|
0 participants
n=107 Participants
|
0 participants
n=206 Participants
|
PRIMARY outcome
Timeframe: Days 1-10, at select time points per protocolPopulation: Cohort 1: 4 adolescent/adult subjects with EBS, 4 adolescent/adult subjects with DEB; Cohort 2: 3 child subjects with EBS
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.
Outcome measures
| Measure |
Cohort 1 (Adolescents, Adults)
n=8 Participants
Adolescent/adult subjects with EB (aged 12 years and older) received diacerein 1% ointment daily for 10 days.
|
Cohort 2 (Children)
n=3 Participants
Children with EB (ages 4-11 yrs, inclusive) were administered diacerein 1% ointment topically for 10 days.
|
|---|---|---|
|
Detectable Plasma Concentrations of Diacerein and Rhein
Rhein Below the limit of quantification:EBS
|
3 Participants
|
2 Participants
|
|
Detectable Plasma Concentrations of Diacerein and Rhein
Rhein Above Lower limit of quantification:DEB
|
4 Participants
|
0 Participants
|
|
Detectable Plasma Concentrations of Diacerein and Rhein
Diacerein Above Lower limit of quantification:EBS
|
0 Participants
|
0 Participants
|
|
Detectable Plasma Concentrations of Diacerein and Rhein
Diacerein Above Lower limit of quantification:DEB
|
0 Participants
|
0 Participants
|
|
Detectable Plasma Concentrations of Diacerein and Rhein
Rhein Below the limit of quantification:DEB
|
0 Participants
|
0 Participants
|
|
Detectable Plasma Concentrations of Diacerein and Rhein
Rhein Above Lower limit of quantification:EBS
|
1 Participants
|
1 Participants
|
|
Detectable Plasma Concentrations of Diacerein and Rhein
Diacerein Below the limit of quantification:EBS
|
4 Participants
|
3 Participants
|
|
Detectable Plasma Concentrations of Diacerein and Rhein
Diacerein Below the limit of quantification:DEB
|
4 Participants
|
0 Participants
|
Adverse Events
Cohort 1 (Adolescents, Adults)
Cohort 2 (Children)
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Cohort 1 (Adolescents, Adults)
n=8 participants at risk
Adolescent/adult patients with EB (aged 12 and older) received Diacerein 1% Ointment daily for 10 days
|
Cohort 2 (Children)
n=3 participants at risk
Children with EB (aged 4 to 11 years, inclusive) received Diacerein 1% Ointment daily for 10 days
|
|---|---|---|
|
General disorders
Pyrexia
|
12.5%
1/8 • Number of events 1 • Baseline to 30 days after last dose of study drug application (40 days)
|
0.00%
0/3 • Baseline to 30 days after last dose of study drug application (40 days)
|
Additional Information
Dr. Mary Spellman/Chief Medical Officer
Castle Creek Pharmaceuticals, LLC
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place