The First Potential Therapy for Congenital Adrenal Hyperplasia
Congenital adrenal hyperplasia (CAH) is a rare endocrine disorder that results in the inability to produce the critical ‘stress’ hormone cortisol. Although CAH testing is a part of the newborn screening program, there are currently no FDA-approved therapies for CAH. CAH is typically treated with chronic, high-dose steroids often resulting in significant side effects. Spruce’s lead product candidate for CAH, Tildacerfont (SPR001), is intended to correct hormone imbalance, giving patients a therapeutic option that treats their underlying disease and reduces their steroid burden.
Congenital Adrenal Hyperplasia Clinical Trials 2019
Spruce also has ongoing collaborations with the National Institutes of Health (NIH) for longer duration study of the safety and efficacy of tildacerfont in adults 18+.
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If you are age 12+ and diagnosed with Congenital Adrenal Hyperplasia, we invite you to join our Natural History Study and Registry.
The CAH Natural History Study's mission is to learn more about CAH so researchers can work to improve the quality of life and the health of individuals with CAH, and to provide hope through the promise of new treatments.
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