A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy
|Study Design:||Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Basic Science
|Official Title:||A Multi-center Study to Evaluate the Pharmacokinetics of 21-Desacetyldeflazacort and the Safety of Deflazacort After Oral Administration of Deflazacort Tablets to Children and Adolescent Subjects With Duchenne Muscular Dystrophy|
- The area under the plasma concentration time curve, from time 0 to the last measurable concentration non-zero, for single-state pharmacokinetics on Day 1 of deflazacort and 21-desacetyl-DFZ, the active metabolite [ Time Frame: Day 1, Day 8 ]The area under the plasma concentration time curve, from time 0 to the last measurable concentration non-zero, for single-state pharmacokinetics on Day 1 of deflazacort and 21-desacetyl-DFZ, the active metabolite
- The area under the plasma concentration versus time curve over the final dosing interval for steady state pharmacokinetics on Day 8 of deflazacort and 21-desacetyl-DFZ, the active metabolite [ Time Frame: Day 8 ]The area under the plasma concentration versus time curve over the final dosing interval for steady state pharmacokinetics on Day 8 of deflazacort and 21-desacetyl-DFZ, the active
- Number of participants with adverse events as a measure of safety and tolerability [ Time Frame: Day 1-8 ]To assess the safety and tolerability of single-dose and steady-state deflazacort in DMD subjects.
|Study Start Date:||December 2014|
|Study Completion Date:||October 2015|
|Primary Completion Date:||October 2015 (Final data collection date for primary outcome measure)|
This is an open label and single period study , dosed with 0.9mg/kg Deflazacort.
Oral deflazacort administered once daily at 0.9 mg/kg for eight days
Other Name: DFZ
This is an open label, single period study in 24 male DMD subjects consisting of children (ages 4-12, inclusive) and adolescents (ages 13-16, inclusive) with at least 12 subjects between the ages of 4-12 (children). Subjects taking maintenance corticosteroid therapy will be required to take their dose of corticosteroid 24 hours (± 2 hours) prior to the first dose of deflazacort on Day 1. Subjects receiving deflazacort as maintenance corticosteroid therapy must take their last dose at least 30 days prior to the first dose of deflazacort on Day 1. Concomitant corticosteroid therapy will be prohibited during the study while the subject is taking deflazacort. On Day 1, a single oral dose of deflazacort under fasting conditions will be administered in the CRU followed by blood sampling for plasma analysis of DFZ and 21-desacetyl-DFZ for 8 hours. Following the 8 hour PK sample on Day 1, subjects will be given medication to take at home for once-daily, morning dosing on Days 2 through 7 (+2 days). On Day 8 (+2 days), subjects will return for a PK sample predose and an 8th oral dose of deflazacort under fasting conditions which will be administered in the CRU followed by PK sampling for 8 hours.
Safety will be monitored throughout the study by repeated clinical and laboratory evaluations on Days 1 and 8 (+2 days). Subjects will be contacted via telephone approximately 7 days (± 1 day) following study drug administration on Day 8 (+2 days) for a follow-up assessment to determine if any adverse event (AE) has occurred since the last study visit. Subjects who terminate the study early will be contacted if the Principal Investigator (PI) deems necessary.
Subjects that complete this study or receive at least one dose of study medication will be eligible for an open-label extension study with deflazacort treatment conducted under a separate protocol.
Please refer to this study by its ClinicalTrials.gov identifier: NCT02251600
|United States, California|
|Los Angeles, California, United States, 90095|
|United States, Illinois|
|Lurie Children's Hospital|
|Chicago, Illinois, United States, 60611|
|United States, New York|
|University of Rochester Medical Center|
|Rochester, New York, United States, 14642|
|United States, Utah|
|University of Utah|
|Salt Lake City, Utah, United States, 84112|
|Study Chair:||Katherine Smith, MD||Drug Safety Solutions/Celerion|