Evaluate the Safety, Efficacy, and Pharmacokinetics of CRN04894 in Participants With Congenital Adrenal Hyperplasia
Study Details
Study Description
Brief Summary
The purpose of this Phase 2, open-label, sequential dose cohort study is to evaluate the safety, efficacy, and pharmacokinetics (PK) of CRN04894 in participants with classic congenital adrenal hyperplasia (CAH) caused by 21-hydroxylase deficiency.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
| Phase 2 |
Detailed Description
This Phase 2, open-label, sequential dose cohort study will evaluate the efficacy, safety, PK, and PD of CRN04894 when administered for 12 weeks in participants with CAH caused by 21-hydroxylase deficiency. Up to approximately 30 participants will be enrolled in the study.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Sequential Dose Sequential, open-label, 12-week fixed-dose cohorts. |
Drug: CRN04894
CRN04894 is an orally active nonpeptide melanocortin 2 receptor (MC2R) or adrenocorticotropic hormone (ACTH) antagonist.
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Outcome Measures
Primary Outcome Measures
- Change from baseline in morning (before 11:00) serum androstenedione (A4) [Week 12]
- Incidence of treatment-emergent adverse events (TEAEs) throughout the study [Week 12]
Secondary Outcome Measures
- Change from baseline in morning (before 11:00) serum 17-hydroxyprogesterone (17-OHP) [Week 12]
Eligibility Criteria
Criteria
Inclusion Criteria:
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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
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Classic 21-hydroxylase deficiency
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On a stable regimen of glucocorticoid replacement (eg, hydrocortisone, prednisolone, prednisone, methylprednisolone)
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Compliance with glucocorticoid replacement and mineralocorticoid replacement (if applicable) regimen during the Screening Period
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Minimum total daily dose of ≥15 mg hydrocortisone (or equivalent)
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If on estrogen therapy (any route), dose must be stable for at least 3 months prior to Screening
Exclusion Criteria:
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Diagnosis of any other form of CAH other than classic 21-hydroxylase deficiency
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Dexamethasone use within 30 days of Screening
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History of bilateral adrenalectomy, hypopituitarism, or other condition requiring chronic glucocorticoid therapy
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Night shift workers or any other reason for abnormal sleep/wake cycles
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Clinically significant unstable medical condition or chronic disease other than CAH
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History of major surgery/surgical therapy for any cause within 4 weeks prior to Screening
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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
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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)
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Participants with hypothyroidism who are not receiving adequate hormone replacement therapy based on thyroid hormone levels measured at the time of Screening
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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
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History of cancer excluding cured/treated dermal squamous or basal cell carcinoma or cervical carcinoma in situ
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Pregnant or lactating
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Known history of illicit drug or alcohol abuse within the last year
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Use of antiandrogen therapy in the past 3 months (eg, spironolactone, finasteride, cyproterone acetate, flutamide)
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Use of testosterone, androgen-containing supplements, aromatase inhibitors, or growth hormone
Contacts and Locations
Locations
No locations specified.Sponsors and Collaborators
- Crinetics Pharmaceuticals Inc.
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- CRN04894-03
- 2023-503488-40-00