Congenital Adrenal Hyperplasia (CAH)
Congenital adrenal hyperplasia (CAH) is a rare genetic disorder affecting the adrenal glands. People with CAH have an impaired ability to produce cortisol, also known as “the stress hormone,” which has an effect on energy, blood pressure, and the body’s response to stress, illness and injury.
About 75% of CAH patients are unable to produce sufficient aldosterone, which helps the body maintain blood pressure and adequate levels of sodium.
In some cases, those affected with CAH produce high levels of androgens. While both sexes need androgens for proper growth and development, an excess can cause problems including increased risk of testicular adrenal rest tumors (TART) in men, precocious puberty, short stature, hirsutism, virilization, and impairments to fertility.
Tildacerfont is an investigational small molecule currently being tested for the treatment of CAH. Tildacerfont works by blocking CRF type-1 receptors on the pituitary gland to attenuate overproduction of ACTH, 17-OHP, and adrenal androgens.
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+.
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.Learn More