NCT02286609

A Single Dose Evaluation of the Effects of Moderate Hepatic Impairment on Deflazacort Pharmacokinetics

A Single Dose Evaluation of the Effects of Moderate (Child-Pugh Grade B) Hepatic Impairment on Deflazacort Pharmacokinetics

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 16 (actual)
• Sponsor: PTC Therapeutics · Industry
• Sex: All
• Age: 18 Years – 80 Years
• Healthy volunteers: Accepted

Summary

This is a non-randomized, open-label, single-dose study to compare the PK of 21-desacetyl-DFZ and, if data permits, deflazacort in 8 subjects with moderate hepatic impairment (based on the Child Pugh classification, Grade B) to that of 8 healthy matched control subjects (age, body mass index \[BMI\], and gender).

Detailed description

This is a non-randomized, open-label, single-dose study to compare the PK of 21-desacetyl-DFZ and, if data permits, deflazacort in 8 subjects with moderate hepatic impairment (based on the Child Pugh classification, Grade B) to that of 8 healthy matched control subjects (age, body mass index \[BMI\], and gender). On Day 1, a single oral dose of deflazacort will be administered followed by serial blood sampling for 24 hours to assess the PK of 21-desacetyl-DFZ and, if data permits, deflazacort. Safety will be monitored throughout the study by repeated clinical and laboratory evaluations. Subjects will return to the Clinical Research Unit (CRU) 3 days (± 1 day) following study drug administration to determine if any adverse events (AEs) have occurred since the last study visit. Subjects who terminate the study early will be contacted if the Principal Investigator (PI) deems necessary. A total of sixteen (16) adult male and female subjects will be enrolled. Hepatic Impaired Cohort: Eight (8) subjects with moderate hepatic insufficiency (a score of 7 to 9, on the Child-Pugh scale). Healthy Match Control Cohort: Eight (8) healthy subjects. Subjects will be matched for age \[± 15 years\], BMI \[± 15 %\], and gender \[1:1\] to the subjects in the moderate hepatic impaired cohort.

Conditions

• Hepatic Impairment

Interventions

Type	Name	Description
DRUG	Deflazacort	Deflazacort, a glucocorticoid with anti-inflammatory and immunosuppressive effects, is used in treating a variety of diseases. Pharmacologically it is an inactive pro-drug which is metabolized immediately to the active metabolite, 21 desacetyl-DFZ. The elimination of this metabolite is primarily via the urine in humans. Its potency is approximately 70 to 90% of prednisone and 6 mg of deflazacort has approximately the same anti-inflammatory potency as 5 mg of prednisolone or prednisone.

Timeline

• Start date: 2014-12-01
• Primary completion: 2015-02-01
• Completion: 2015-02-01
• First posted: 2014-11-10
• Last updated: 2017-08-18

Locations

2 sites across 1 country: United States