Title: 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

Protocol SPR001-203

RPN: IRB00240449

PI: Amir H. Hamrahian, MD

Congenital adrenal hyperplasia (CAH) is an inherited genetic disorder that affects the adrenal glands, a pair of walnut-sized organs above the kidneys. The adrenal glands control the production of androgens (sex hormones). People with CAH often have abnormal levels of certain adrenal sex hormones, which can have negative effects on overall health. The current standard of care for CAH is the use of glucocorticoids (GCs).

The purpose of this study is to see if an investigational drug, tildacerfont (SPR001) is safe and effective in reducing the level of certain hormones in the body in people with congenital adrenal hyperplasia. Because this is a scientific research study, it is also important to collect information about patients with CAH who are not receiving tildacerfont. Therefore, tildacerfont will be compared to “placebo”, which is an inactive material that does not contain any active study drug.

Male and female subjects who are 18 to 55 years old and had a documented historical diagnosis of classic CAH due to 21-hydroxylase deficiency based on genetic testing may be eligible for this study.

The use of tildacerfont in this research study is investigational. The word “investigational” means that tildacerfont is not approved for marketing by the Food and Drug Administration (FDA). The FDA is allowing the use of tildacerfont in this study.

Participation in this study lasts about 66 weeks and includes 12 visits to the study site and about 15 phone calls. Participants will have some routine medical tests (physical exam, blood draw, and an electrocardiogram [ECG]) and will have to come to some visits fasting.

Any research has some risks. There may be adverse effects related to the study drug while taking part in the study, so everyone taking part in the study will be watched carefully. Information about risks is discussed in greater detail in the consent form.

Participants may or may not benefit as a result of participation in this study. Results from this study may benefit others in the future.

It will not cost anything to be in this study. Participants will be paid for the study visits completed to compensate for the time and inconvenience related to participation in this study.

Some of eligibility (inclusion) criteria for patients who may be interested in the study:

1. Male and female subjects 18 to 55 years old
2. Has a documented historical diagnosis of classic CAH due to 21-hydroxylase deficiency based on genetic testing.

For more information, contact the study coordinator at 667-217-5528

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