A Randomized, Double-Blind, Multicenter, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Atumelnant in Adult Participants with Classic Congenital Adrenal Hyperplasia

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适应症

试验速览

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•Male or female, between >=18 to <75 years of age at the time of signing the ICF.
•Willing and able to understand and adhere to the study procedures as specified in the protocol and comply with the study treatment.
•Have classic CAH due to 21-OHD confirmed by the Investigator.
•Participants Visit2 levels of morning serum A4 as follows:
•A4 >ULN and treated with <11 mg/m2/day (physiologic) GC doses
•normal A4 (>=0.5 x ULN to 1 x ULN) and treated with >=14 mg/m2/day GC doses
•A4 >ULN and treated with >=11 mg/m2/day GC doses.
•On a stable (defined as no dose change of >5 mg/day hydrocortisone equivalent within 3 months prior to Screening) regimen of GC replacement (e.g., hydrocortisone, prednisolone, prednisone, methylprednisolone, meprednisone, dexamethasone, cortisone acetate) at the time of informed consent.
•If treated with mineralocorticoids (fludrocortisone), the dose should be stable for at least 1 month prior to Screening with a plasma renin concentration during Screening that is not greater than the ULN on the participant's usual sodium intake. If plasma renin concentration is greater than the ULN, the participant must have systolic blood pressure >100 mm Hg, without orthostatic hypotension and with serum sodium and potassium in the normal range.
•If on estrogen therapy (any route), the dose must be stable for at least 3 months prior to Screening.

•Diagnosis of any form of CAH other than classic 21-OHD.
•History of bilateral adrenalectomy, hypopituitarism, or other conditions requiring chronic GC therapy.
•Clinically significant medical condition or abnormal laboratory tests, as judged by the Investigator, other than CAH.
•Concomitant mental condition rendering him/her unable to understand the nature, scope, and possible consequences of the study, and/or evidence of poor compliance with medical instructions.
•Active malignant disease within the 5 years prior to Screening excluding dermal squamous or basal cell carcinoma of the skin with complete local excision or resected cervical carcinoma in situ.
•Women who are pregnant or lactating or, if of childbearing potential, who are unwilling to use highly effective contraception as described in this study. Male participants who are unwilling to use highly effective contraception as described in this study.
•Known history of, or concern for, risk of hypersensitivity reaction to atumelnant or any of its excipients.
•Participants with an increased risk of developing adrenal insufficiency as judged by the Investigator.
•Severe erythrocytosis as judged by the Investigator.
•Use of atumelnant prior to screening.

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