Evaluate the Safety, Efficacy, and Pharmacokinetics of CRN04894 in Participants with Congenital Adrenal Hyperplasia
- Conditions
- CONGENITAL ADRENAL HYPERPLASIAMedDRA version: 20.0Level: LLTClassification code: 10010323Term: Congenital adrenal hyperplasia Class: 10010331Therapeutic area: Diseases [C] - Hormonal diseases [C19]Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- CTIS2023-503488-40-00
- Lead Sponsor
- Crinetics Pharmaceuticals Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 33
Male or female participants =18 to 75 years of age at the time of signing the Informed Consent Form (ICF). Participants =16 years of age may be included in sites located in the United States (US), Classic 21-hydroxylase deficiency confirmed by the Investigator and approved by the Medical Monitor., On a stable (defined as no dose change of >5 mg/day hydrocortisone equivalent (see Section 12.5) within 6 months prior to Screening) regimen of glucocorticoid replacement (eg, hydrocortisone, prednisolone, prednisone, methylprednisolone)., Compliance, as judged per investigator discretion, with glucocorticoid replacement and mineralocorticoid replacement (if applicable) regimen documented during the Screening Period, Minimum total daily dose of =15 mg hydrocortisone (or equivalent), If on estrogen therapy (any route), dose must be stable for at least 3 months prior to Screening.
Diagnosis of any other form of CAH other than classic 21-hydroxylase deficiency, History of unstable angina or acute myocardial infarction within 12 weeks prior to Screening or other clinically significant cardiac disease at the time of Screening as judged by the Investigator, 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, History of cancer excluding cured/treated dermal squamous or basal cell carcinoma or 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 illicit drug or alcohol abuse within the last year, Use of antiandrogen therapy in the past 3 months (eg, spironolactone, finasteride, cyproterone acetate, flutamide), Use of testosterone, androgen-containing supplements, aromatase inhibitors, or growth hormone, Dexamethasone or oral betamethasone use within 30 days of Screening, History of bilateral adrenalectomy, hypopituitarism, or other condition requiring chronic glucocorticoid therapy, Night shift workers or any other reason for abnormal sleep/wake cycles, Clinically significant medical condition or abnormal laboratory tests, as judged by the Investigator, other than CAH, History of major surgery/surgical therapy for any cause within 4 weeks prior to Screening, Diabetes mellitus treated with insulin for less than 6 weeks prior to Screening, or with change in total daily insulin dose by >15% within 6 weeks prior to Screening, Poorly controlled diabetes mellitus defined as having a hemoglobin A1c (HbA1c) =8.5%(=69 mmol/mL), or estimated HbA1c based on fructosamine if HbA1c is not evaluable (eg, due to hemoglobinopathies), Participants with hypothyroidism who are not receiving adequate hormone replacement therapy based on thyroid hormone levels measured at the time of Screening, as determined by the Investigator
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: Safety Objective: To evaluate the safety and tolerability of CRN04894<br>Primary Efficacy Objective: To evaluate efficacy of CRN04894, measured by change from Baseline in serum androstenedione (A4);Secondary Objective: Secondary Efficacy Objective: To evaluate efficacy of CRN04894, measured by change from Baseline in serum 17-hydroxyprogesterone (17-OHP);Primary end point(s): Primary Safety Endpoints: Incidence of treatment-emergent adverse events (TEAEs), including treatment-emergent serious adverse events (SAEs) and any adverse events (AEs) leading to discontinuation, Primary Efficacy Endpoint: Change from Baseline in morning (before 11:00) serum A4 at Week 12
- Secondary Outcome Measures
Name Time Method Secondary end point(s):Secondary Efficacy Endpoint: Change from Baseline in morning (before 11:00) serum 17-OHP at Week 12