A Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging Study to Evaluate the Efficacy and Safety of SPR001 (Tildacerfont) in Adult Subjects with Classic Congenital Adrenal Hyperplasia
CAH is an inherited genetic disorder that affects the adrenal glands, a pair of walnut-sized organs above the kidneys. The disease affects the production of steroid hormones by the adrenal glands, which include “glucocorticoids” such as cortisol, which regulate your body’s response to illness or stress. People with CAH often have abnormal levels of certain adrenal sex hormones, which can have negative effects on overall health. The purpose of this study is to see if tildacerfont is safe and effective in reducing the level of certain hormones. Tildacerfont will be compared to “placebo”, which is a drug that looks like the investigational drug but does not contain any tildacerfont or any other active compound.
• at least 18 years old
• childhood diagnosis of classic CAH due to 21-hydroxylase deficiency based genetic mutation in CYP21A2 and/or documented elevated 17-OHP
• currently treated with HC, HC acetate, prednisone, prednisolone, methylprednisolone (or a combination)
• on a stable dose of GC replacement of at least 15 mg/day and less than 60 mg/day in HC equivalents
• people with the salt-wasting form of CAH, must be on a stable dose of mineralocorticoid replacement for at least 1 month
• known or suspected diagnosis of any other known form of classic CAH (not due to 21 hydroxylase deficiency)
• history of bilateral adrenalectomy or hypopituitarism
• allergy or hypersensitivity to Tildacerfont, any of its forms, or any other CRF1 receptor antagonist
• current treatment with dexamethasone as GC therapy for CAH.
• clinical signs or symptoms of adrenal insufficiency
Diabetes & Endocrine, Rare Diseases
Congenital Adrenal Hyperplasia