This study is designed to evaluate the safety and efficacy of deflazacort in participants with LGMD2I. Most participants enrolled will have a screening visit and 3 additional visits (after 1, 13, and 26 weeks of treatment).
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
11
Deflazacort tablet will be administered as per the dose and schedule specified in the arm.
Rare Disease Research, LLC
Atlanta, Georgia, United States
University of Iowa Hospitals and Clinics
Iowa City, Iowa, United States
Change From Baseline in Time to Climb 4 Stairs After 26 Weeks of Treatment With Deflazacort
Time frame: Baseline, Week 26
Change From Baseline in Forced Vital Capacity (FVC) After 26 Weeks of Treatment With Deflazacort
Time frame: Baseline, Week 26
Change From Baseline in 2-Minute Walk Test After 26 Weeks of Treatment of Deflazacort
Time frame: Baseline, Week 26
Change From Baseline in Time to up and go After 26 Weeks of Treatment With Deflazacort
Time frame: Baseline, Week 26
Change From Baseline in Time to Descend 4 Stairs After 26 Weeks of Treatment With Deflazacort
Time frame: Baseline, Week 26
Change From Baseline in Time to Run/Walk 10 Meters After 26 Weeks of Treatment With Deflazacort
Time frame: Baseline, Week 26
Change From Baseline in Maximal Inspiratory Pressure (MIP) and Maximal Expiratory Pressure (MEP) After 26 Weeks of Treatment With Deflazacort
Time frame: Baseline, Week 26
Change From Baseline in Hand-Held Myometry After 26 Weeks of Treatment With Deflazacort
Time frame: Baseline, Week 26
Change From Baseline in Global T2 Relaxation Time of Selected Upper and Lower Limb Muscles After 26 Weeks of Treatment With Deflazacort
Time frame: Baseline, Week 26
Number of Participants With Adverse Events (AEs)
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The University of Kansas Medical Center
Kansas City, Kansas, United States
Hugo W Moser Research Institute at Kennedy Krieger Institute
Baltimore, Maryland, United States
University of Minnesota
Minneapolis, Minnesota, United States
Washington University School of Medicine
St Louis, Missouri, United States
University of Pennsylvania
Philadelphia, Pennsylvania, United States
University of Washington
Seattle, Washington, United States
University of Alberta
Edmonton, Alberta, Canada
Ottawa Hospital
Ottawa, Canada
...and 8 more locations
An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'. AEs were summarized separately for Stage 1 and for the overall ataluren experience.
Time frame: Baseline up to Week 52
Area Under the Concentration Curve From Time Zero to t (AUC0-t) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort
Time frame: Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13
Area Under the Concentration Curve From Time Zero to Infinity (AUC0-inf) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort
Time frame: Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13
Maximum Observed Plasma Concentration (Cmax) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort
Time frame: Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13
Time to Reach Cmax (Tmax) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort
Time frame: Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13
Half-Life (t1/2) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort
Time frame: Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13