An investigation of the efficacy and safety of up to 70 weeks of treatment with Tildacerfont in subjects with classic CAH who have elevated biomarkers at baseline on their current GC regimen. Optional open label treatment extension period up to 240 weeks with 200mg Tildacerfont QD.
This is a study that will test the efficacy and safety of Tildacerfont. The first 12-weeks will be a double-blind, placebo controlled, dose ranging study. The following 58-weeks will assess the long term safety of Tildacerfont. Optional open label treatment extension period up to 240 weeks with 200mg Tildacerfont QD.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
96
Tablet, administered daily
To Evaluate the Effect of Tildacerfont in Reducing A4 in Participants With Classic Congenital Adrenal Hyperplasia (CAH) Over 12 Weeks
Assessment of dose response for change from baseline in log A4 after 12 weeks on double-blind placebo-controlled treatment (Week 18)
Time frame: Baseline and 12 weeks of treatment (Week 18)
To Evaluate the Effect of Tildacerfont in Reducing A4 in Participants With Classic CAH Over 12 Weeks
Absolute change from baseline in A4 as determined via application of the delta theorem to the log scale analysis results after 12 weeks on double-blind, placebo-controlled treatment (Week 18). Results show mean Treatment Effect of Dose vs Placebo. A negative value represents a reduction in absolute concentration compared to baseline.
Time frame: 12 weeks
To Evaluate the Effect of Tildacerfont in Reducing A4 in Participants With Classic CAH Over 12 Weeks
Change from baseline in A4 as assessed on the log scale after 12 weeks on double-blind, placebo-controlled treatment (Week 18). Results show mean % of Treatment Effect of Dose vs Placebo. A negative value represents a % reduction from baseline.
Time frame: 12 weeks
To Evaluate the Effect of Tildacerfont in Reducing 17-OHP in Participants With Classic CAH Over 12 Weeks
Change from baseline in 17-OHP as assessed on the log scale after 12 weeks on double-blind, placebo-controlled treatment (Week 18). A negative value represents a % reduction from baseline.
Time frame: 12 weeks
To Evaluate the Effect of Tildacerfont in Reducing 17-OHP in Participants With Classic CAH Over 12 Weeks
Absolute change from baseline in 17-OHP as determined via application of the delta theorem to the log scale analysis results after 12 weeks on double-blind, placebo-controlled treatment (Week 18). Results show mean Treatment Effect of Dose vs Placebo. A negative value represents a reduction in absolute concentration compared to baseline.
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Spruce Study Site
Birmingham, Alabama, United States
Spruce Study Site
Los Angeles, California, United States
Spruce Clinical Site
Orange, California, United States
Spruce Study Site
Sacramento, California, United States
Spruce Study Site
Englewood, Colorado, United States
Spruce Clinical Site
Tampa, Florida, United States
Spruce Study Site
West Palm Beach, Florida, United States
Spruce Study Site
Chicago, Illinois, United States
Spruce Clinical Site
Indianapolis, Indiana, United States
Spruce Study Site
Baltimore, Maryland, United States
...and 55 more locations
Time frame: 12 weeks