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It was very gratifying to see that the patients who were in the trial, and we sort of had a hint that they were on study drug and not on placebo when patients who were previously uncontrolled for their CAH on glucocorticoids began to show reductions in their androstenedione. So once a patient in the trial, a subject was able to reduce their androstenedione level on a fixed dose of glucocorticoid, which had been maintained for a designated period of time before study trial entry, then we could ask the question, could we maintain those lower levels of androstenedione while at the same time with the crinecerfont on board, or in the placebo case, reduce the level of glucocorticoid. So that was another study objective that again proved to be a valid suspicion that this was an achievable goal of using this adjunctive therapy to treat our patients.

Video

In phase 1 of the CAHtalyst pediatric study, did investigators demonstrate a statistically significant decrease in serum androstenedione from baseline with crinecerfont at Week 4 over baseline?

In phase 1 of the CAHtalyst pediatric study, did investigators demonstrate a statistically significant decrease in serum androstenedione from baseline with crinecerfont at Week 4 over baseline?


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CMEducation Resources | iQ&A Congenital Adrenal Hyperplasia (CAH) Medical Intelligence Zone 

Presenter

Phyllis W. Speiser, MD

Phyllis W. Speiser, MD

Associate Professor, Institute of Molecular Medicine
Feinstein Institutes for Medical Research
Emerita Professor, Pediatrics Donald and Barbara Zucker School of Medicine
Hofstra/Northwell
New Hyde Park, NY