Trial Outcomes & Findings for To Evaluate the Pharmacokinetic of Diacerein and Rhein After Maximum Use in Patients With Epidermolysis Bullosa (EB) (NCT 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.

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

11 participants

Primary outcome timeframe

Days 1-10, at select time points per protocol

Results posted on

2020-02-10

Participant Flow

Participant milestones

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

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=5 Participants	8.3 years STANDARD_DEVIATION 1.53 • n=7 Participants	21.5 years STANDARD_DEVIATION 13.0 • n=5 Participants
Sex: Female, Male Female	5 Participants n=5 Participants	1 Participants n=7 Participants	6 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants	2 Participants n=7 Participants	5 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	7 Participants n=5 Participants	1 Participants n=7 Participants	8 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) White	7 Participants n=5 Participants	2 Participants n=7 Participants	9 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment United States	8 Participants n=5 Participants	3 Participants n=7 Participants	11 Participants n=5 Participants
EB Classification per Cohort Epidermolysis Bullosa Simplex (EBS)	4 participants n=5 Participants	3 participants n=7 Participants	7 participants n=5 Participants
EB Classification per Cohort Dystrophic Epidermolysis Bullosa (DEB)	4 participants n=5 Participants	0 participants n=7 Participants	4 participants n=5 Participants
EB Classification per Cohort Junctional Epidermolysis Bullosa (JEB)	0 participants n=5 Participants	0 participants n=7 Participants	0 participants n=5 Participants

PRIMARY outcome

Timeframe: Days 1-10, at select time points per protocol

Population: Cohort 1: 4 adolescent/adult subjects with EBS, 4 adolescent/adult subjects with DEB; Cohort 2: 3 child subjects with EBS

Outcome measures

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 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 Rhein Above Lower limit of quantification:DEB	4 Participants	0 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
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

Adverse Events

Cohort 1 (Adolescents, Adults)

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Cohort 2 (Children)

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

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

Phone: 862 286 0400

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place