An investigation of the ability of Tildacerfont to reduce supraphysiologic glucocorticoid dosing in classic Congenital adrenal hyperplasia (CAH) subjects up to 76 weeks of treatment. Optional open label extension up to 240 weeks.
This is a study that evaluated the ability of tildacerfont to reduce the glucocorticoid steroid dose used by adult subjects with CAH. The first 24-weeks were a double-blind, placebo controlled, comparison of tildacerfont vs placebo. The following 52-weeks allowed all subjects to move to open label tildacerfont to continue to reduce steroid dose where appropriate, and observe long term safety. Subjects were offered a long term open label extension up to 240 weeks.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
100
Tablet, administered daily
Change in Total Daily GC Dose in Subjects With Classic CAH Over the 24-week, Double Blind, Placebo-Controlled Treatment Period
Absolute change from baseline (Day 1) in GC dose in HCe at Week 24. The analysis of absolute change in total daily GC dose in HCe will include data from Weeks 3, 6, 12, 18 and 24 in a mixed model
Time frame: 24 Weeks
Effect of Tildacrfont in Reducing GC Use to Near-physiologic Levels While Maintaining Androgen Control in Subjects With CAH
Proportion of subjects with GC dose ≤11mg/m2/day in HCe and A4 ≤1.2x baseline or A4 ≤ ULN at Week 24
Time frame: 24 weeks
Effect of Tildacrfont in Reducing GC Use to Near-physiologic Levels While Maintaining Androgen Control in Subjects With CAH
Proportion of subjects with baseline GC dose ≤ 35mg HCe who achieve GC dose ≤11 mg/m2/day in HCe and A4 ≤ 1.2x baseline or ≤ ULN at Week 24
Time frame: 24 Weeks
Effectiveness in Reducing Cardiovascular Risk in Subjects With CAH
Proportion of subjects with improvement in at least one cardiovascular risk factor at Week 24
Time frame: 24 Weeks
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Birmingham, Alabama, United States
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Indianapolis, Indiana, United States
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Baltimore, Maryland, United States
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New Brunswick, New Jersey, United States
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