A study of safety and efficacy of NBI-74788 in Classic Congenital Adrenal Hyperplasia

Phase 1

• Conditions: Classic Congenital Adrenal Hyperplasia (CAH); MedDRA version: 20.0Level: LLTClassification code: 10010323Term: Congenital adrenal hyperplasia Class: 10010331; Therapeutic area: Diseases [C] - Hormonal diseases [C19]

• Registration Number: CTIS2023-509171-16-00
• Lead Sponsor: eurocrine Biosciences Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2024-06-07
• Last Update Posted: 21 August 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 176

Inclusion Criteria

Subjects must provide written informed consent., Be a female or male at least 18 years of age., Have a medically confirmed diagnosis of classic 21-hydroxylase deficiency., Be on a stable, supraphysiologic glucocorticoid dose regimen that has been stable for at least 1 month prior to screening., If treated with fludrocortisone, dose should be stable for at least 1 month prior to screening with an upright plasma renin activity (PRA) during screening that is not greater than ULN on the subject's usual sodium intake. If PRA is >ULN, the subject must have systolic blood pressure >100 mmHg, without orthostatic hypotension, and with serum sodium and potassium in the normal range., Female subjects of childbearing potential with fertile male partners must agree to use contraception consistently from screening until the final study visit or 30 days after the last dose of study drug, whichever is longer. A female who is not of childbearing potential must meet one of the following:

Exclusion Criteria

Have a known or suspected diagnosis of any of the other forms of classic CAH including 11-ß-hydroxylase deficiency, 17-a-hydroxylase deficiency, 3-ß-hydroxysteroid dehydrogenase deficiency, P450 sidechain cleavage deficiency, or P450 oxidoreductase deficiency., Have a history of bilateral adrenalectomy, hypopituitarism, or other condition requiring chronic therapy with oral glucocorticoids, or requiring chronic therapy with inhaled glucocorticoids that based on dose and hormone profile the investigator deems would yield significant systemic exposure interfering with study endpoints., Have a clinically significant medical condition or chronic disease (including history of neurological, hepatic, renal, cardiovascular, gastrointestinal, significant malabsorption, hematologic, pulmonary, psychiatric, or endocrine disease [excluding CAH]) that in the opinion of the investigator would preclude the subject from participating in and completing the study or that could confound interpretation of study outcome., History of malignancy, unless successfully treated with curative intent and considered to be cured., Have a known history of clinically concerning cardiac arrhythmia (including long QT syndrome) or prolongation of screening (pretreatment) QT interval corrected for heart rate using Fridericia's correction (QTcF) of >450 msec (males) or >470 msec (females)., Known sensitivity (ie, hypersensitivity) or allergy to any corticotropinreleasing hormone (CRH) receptor antagonist., Have evidence of chronic renal or liver disease Used any active investigational drug within 30 days or 5 half-lives (whichever is longer) before screening, or plans to use an investigational drug (other than the study drug) during the study., Females who are pregnant or lactating., Are using any excluded concomitant medication and cannot discontinue use of these medications for the duration of the study (also refer to Section 9.9.1): • Orally administered glucocorticoids for indications other than CAH. • Strong inducers of CYP3A4 or CYP2B6 except topically administered medications. • Medications that affect cortisol or glucocorticoid metabolism (eg, phenytoin, mitotane, phenobarbital, strong CYP3A4 inhibitors such as ketoconazole, clarithromycin, cholestyramine, certain antivirals) except topically administered medications. • Aromatase inhibitors (eg, anastrozole, letrozole, testolactone).

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified