Evaluate the Safety, Efficacy, and Pharmacokinetics of CRN04894 in Participants With Congenital Adrenal Hyperplasia (TouCAHn)

NCT05907291 | Completed Phase 2 FDA Drug

• 2 other identifiers: CRN04894-032023-503488-40-00
• Study Type: interventional
• Target: 38
• Locations: 7 countries
• Sites: 27

Brief Summary

The purpose of this Phase 2, open-label, sequential dose cohort study is to evaluate the safety, efficacy, and pharmacokinetics (PK) of atumelnant (CRN04894) in participants with classic congenital adrenal hyperplasia (CAH) caused by 21-hydroxylase deficiency.

Conditions

Congenital Adrenal Hyperplasia; Classic Congenital Adrenal Hyperplasia

Keywords

Congenital Adrenal Hyperplasia; CAH; TouCAHn; CRN04894; atumelnant

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (1)

• Change from baseline in morning (before 11:00) serum 17-hydroxyprogesterone (17-OHP)

  Week 12

Study Arms (1)

Sequential Dose

EXPERIMENTAL

Sequential, open-label, 12-week fixed-dose cohorts.

Drug: atumelnant (CRN04894)

Interventions

atumelnant (CRN04894) DRUG

Atumelnant is an orally active nonpeptide melanocortin 2 receptor (MC2R) or adrenocorticotropic hormone (ACTH) antagonist.

Sequential Dose

Eligibility Criteria

• Age: 16 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• Classic 21-hydroxylase deficiency
• On a stable regimen of glucocorticoid replacement (eg, hydrocortisone, prednisolone, prednisone, methylprednisolone)
• Compliance with glucocorticoid replacement and mineralocorticoid replacement (if applicable) regimen during the Screening Period
• If on estrogen therapy (any route), dose must be stable for at least 3 months prior to Screening

You may not qualify if:

• Diagnosis of any other form of CAH other than classic 21-hydroxylase deficiency
• Dexamethasone use within 30 days of Screening for Cohorts 1-3. In Cohort 4, dexamethasone is permitted
• 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 unstable medical condition or chronic disease 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
• 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
• History of cancer excluding cured/treated dermal squamous or basal cell carcinoma or cervical carcinoma in situ
• Pregnant or lactating
• 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

Sponsors & Collaborators

• Crinetics Pharmaceuticals Inc.: lead

Study Sites (27)

Crinetics Study Site

Pasadena, California, 91105, United States

Location

Crinetics Study Site

Ann Arbor, Michigan, 48109, United States

Location

Crinetics Study Site

Minneapolis, Minnesota, 55454, United States

Location

Crinetics Study Site

St Louis, Missouri, 63110, United States

Location

Crinetics Study Site

Morehead City, North Carolina, 28557, United States

Location

Crinetics Study Site

Cleveland, Ohio, 44195, United States

Location

Crinetics Study Site

Philadelphia, Pennsylvania, 19104, United States

Location

Crinetics Study Site

East Providence, Rhode Island, 02915, United States

Location

Crinetics Study Site

Córdoba, Córdoba Province, 5000, Argentina

Location

Crinetics Study Site

Buenos Aires, C1180, Argentina

Location

Crinetics Study Site

Curitiba, Paraná, 80030-110, Brazil

Location

Crinetics Study Site

Rio de Janeiro, Rio de Janeiro, 20231-092, Brazil

Location

Crinetics Study Site

Rio de Janeiro, Rio de Janeiro, 20551-030, Brazil

Location

Crinetics Study Site

Porto Alegre, Rio Grande do Sul, 90410-000, Brazil

Location

Crinetics Study Site

Botucatu, São Paulo, 18618-686, Brazil

Location

Crinetics Study Site

Ribeirão Preto, São Paulo, 14051-140, Brazil

Location

Crinetics Study Site

São Paulo, São Paulo, 04024-002, Brazil

Location

Crinetics Study Site

São Paulo, 05403-000, Brazil

Location

Crinetics Study Site

Munich, Bavaria, 80336, Germany

Location

Crinetics Study Site

Bangalore, Karnataka, 560054, India

Location

Crinetics Study Site

Vellore, Tamil Nadu, 632004, India

Location

Crinetics Study Site

Napoli, 80131, Italy

Location

Crinetics Study Site

Roma, 00161, Italy

Location

Crinetics Study Site

Rozzano, 20089, Italy

Location

Crinetics Study Site

Sheffield, South Yorkshire, S10 2RX, United Kingdom

Location

Crinetics Study Site

Coventry, West Midlands, CV22DX, United Kingdom

Location

Crinetics Study Site

London, NW1 2PG, United Kingdom

Location

MeSH Terms

Conditions

Adrenal Hyperplasia, Congenital

Condition Hierarchy (Ancestors)

Adrenogenital Syndrome; Disorders of Sex Development; Urogenital Abnormalities; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Steroid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases; Adrenal Gland Diseases; Endocrine System Diseases; Gonadal Disorders

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 8, 2023
• First Posted: June 18, 2023
• Study Start: July 3, 2023
• Primary Completion: August 22, 2025
• Study Completion: August 22, 2025
• Last Updated: September 23, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

AR (2); US (8); BR (8); GB (3); DE (1); IT (3); IN (2)