NCT02286622

A Single Dose Evaluation of the Effects of Renal 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 ESRD 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 ESRD to that of 8 healthy matched control subjects (age, body mass index \[BMI\], and gender). All subjects with ESRD will be on hemodialysis (HD). Dosing of deflazacort followed by PK evaluation of 21 desacetyl DFZ and, if data permits, deflazacort, will only be performed on a non-HD day. On Day 1, that will be scheduled on a non-HD day for subjects with ESRD, 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. Renal Impaired Cohort: Eight (8) subjects with ESRD on HD. Healthy Match Control Cohort: Eight (8) healthy subjects with estimated creatinine clearance (CLcr) ≥ 90 mL/min. Subjects will be matched for age \[± 15 years\], BMI \[± 15 %\], and gender \[1:1\] to the subjects in the ESRD cohort. Each subject will receive a single oral dose of 18 mg (3 X 6 mg tablets) deflazacort, following an overnight fast. Study drug will be administered orally with approximately 240 mL of water.

Conditions

• Renal 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