This is a randomized, double-blind, placebo-controlled study evaluating the potential of Tildacerfont to reduce the use of glucocorticoids (GCs) in adult subjects with congenital adrenal hyperplasia (CAH) receiving GC therapy at supraphysiological doses.

Zusammenfassung der Studie

Congenital adrenal hyperplasia (CAH) is a severe, chronically debilitating, and life-threatening genetic disorder characterized by impaired adrenal synthesis of cortisol and a subsequent overproduction of adrenal androgens. Cortisol deficiency leads to hypersecretion or overproduction of certain hormones, ultimately resulting in an excess of androgens. An excess of androgens can lead to irregular menstruation, absence of menstrual periods (amenorrhea), hirsutism, and virilization in women; testicular adrenal rest tumors (TARTs) in men; and increased sebum production, acne, altered blood pressure profiles in the afternoon, and impaired fertility in both sexes. The current standard of care for CAH is long-term use of glucocorticoids (GCs) at supraphysiological doses to replace the missing cortisol and suppress androgen overproduction. This is a problematic therapy with significant side effects, and a non-steroidal treatment option would be preferable. Tildacerfont may allow a CAH patient to have normal androgen levels while taking GC in the normal replacement range. Currently, Tildacerfont has shown an acceptable safety profile at effective doses in non-clinical toxicology studies, phase 1 clinical studies in healthy volunteers, and phase 2 studies in adult patients with classic CAH. The SPR001-204 study will be the first study of Tildacerfont evaluating its ability to reduce the necessary GC dose. Neither the patients nor their investigator physician know which treatment the patients are receiving, and patients will be selected, e.g., by coin toss, to receive either the study drug or the placebo. An optional open-label extension period provides additional treatment with Tildacerfont at 200 mg QD for up to 240 weeks.

(BASEC)

Untersuchte Intervention

The main purpose of this study is to investigate the drug SPR001 (Tildacerfont) for patients with classic congenital adrenal hyperplasia (CAH). SPR001 is an investigational drug, i.e., a drug being tested. The efficacy and safety of SPR001 (Tildacerfont) in reducing the supraphysiological (higher or more effective than naturally occurring dose) use of glucocorticoids in adult patients with classic CAH will be evaluated. The study consists of a two-part treatment phase. During the first 24 weeks of the treatment period, patients will be randomized in a 1:1 ratio, e.g., by coin toss. They will receive either placebo or Tildacerfont at 200 mg once daily. During the remaining 52 weeks of the treatment period, all subjects will receive 200 mg of Tildacerfont once daily.

(BASEC)

Untersuchte Krankheit(en)

Congenital adrenal hyperplasia (CAH) is a group of rare disorders. There is a deficiency of one of the enzymes needed to produce certain hormones. CAH affects the adrenal glands, which are located on top of each kidney.

(BASEC)

Sponsor

Medpace Switzerland AG

(BASEC)

Name der bewilligenden Ethikkommission (bei multizentrischen Studien nur die Leitkommission)

Ethikkommission Zürich

(BASEC)

Datum der Bewilligung durch die Ethikkommission

19.10.2022

(BASEC)

Ergebnisse der Studie