Oral nonsteroidal treatment reduces glucocorticoid dose in congenital adrenal hyperplasia
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Adults with congenital adrenal hyperplasia receiving an oral corticotropin-releasing factor type 1 receptor antagonist had a greater reduction in daily glucocorticoid dose at 24 weeks than placebo, according to top-line phase 3 trial data.
In the CAHtalyst phase 3 trial, 182 adults with classic congenital adrenal hyperplasia were randomly assigned to receive crinecerfont (Neurocrine Biosciences), an oral nonsteroidal medication designed to reduce and control excess adrenal androgens for adults with congenital adrenal hyperplasia due to 21-hydroxylase deficiency, or placebo for 24 weeks. Crinecerfont met the study’s primary endpoints, as adults receiving the agent had a greater percent reduction in daily glucocorticoid dose while maintaining androgen control compared with those receiving placebo (P < .0001). Adults receiving crinecerfont also had a larger decrease in androstenedione at 4 weeks compared with placebo (P < .0001). At 24 weeks, 63% of the crinecerfont group had a reduction in physiologic glucocorticoid dose compared with 18% of the placebo group (P < .0001).
Crinecerfont was generally well-tolerated. The most common adverse events were fatigue, headache and coronavirus infection. There were few serious adverse events and none were related to the study drug, according to a company press release.
The 24-week placebo-controlled period of the trial was followed by 1 year of open-label crinecerfont treatment and an optional open-label extension. The open-label portion of the trial is still ongoing.
"I am gratified to see the extremely positive and clinically meaningful results from this study, the largest ever interventional trial conducted in this rare disease,” Richard Auchus, MD, PhD, the trial’s principal investigator and professor of pharmacology and internal medicine in the division of metabolism, endocrinology and diabetes at the University of Michigan, said in the press release. “It required a global effort, and the top-line results confirm our confidence in crinecerfont as a potential first-in-class medication and first-ever non-glucocorticoid treatment option for patients living with congenital adrenal hyperplasia. It has been 60 years since we've seen a significant treatment advance for patients with congenital adrenal hyperplasia, and the data from this study suggest that crinecerfont might improve their outcomes and quality of life."
According to the press release, Neurocrine Biosciences will proceed with a regulatory submission for crinecerfont to the FDA in 2024. Additional data from the study will be released in a peer-reviewed medical journal or at a medical conference at a later date. Data from the phase 3 CAHtalyst pediatric study will be released in early fourth quarter of 2023.
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