Clinical trial NCT05907291: Evaluate the Safety, Efficacy, and Pharmacokinetics of CRN04894 in Participants With Congenital Adrenal Hyperplasia (TouCAHn)
Official title: A 12-week, Phase 2 Open-label, Sequential Dose Cohort Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of CRN04894 Treatment in Participants With Congenital Adrenal Hyperplasia (TouCAHn)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Congenital Adrenal Hyperplasia
Classic Congenital Adrenal Hyperplasia
Keywords: Congenital Adrenal Hyperplasia
CAH
TouCAHn
CRN04894
Lead sponsor: Crinetics Pharmaceuticals Inc.
Sponsor class: INDUSTRY
First posted: 2023-06-18
Enrollment: 30
Sex: ALL
Minimum age (years): 16
Maximum age (years): 75
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

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.

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.

Eligibility criteria: Inclusion Criteria:

1. 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
2. Classic 21-hydroxylase deficiency
3. On a stable regimen of glucocorticoid replacement (eg, hydrocortisone, prednisolone, prednisone, methylprednisolone)
4. Compliance with glucocorticoid replacement and mineralocorticoid replacement (if applicable) regimen during the Screening Period
5. Minimum total daily dose of ≥15 mg hydrocortisone (or equivalent). For Cohort 4, a mean daily dose of ≥11 mg/m²/day of hydrocortisone or hydrocortisone equivalents will be used for inclusion
6. If on estrogen therapy (any route), dose must be stable for at least 3 months prior to Screening

Exclusion Criteria:

1. Diagnosis of any other form of CAH other than classic 21-hydroxylase deficiency
2. Dexamethasone use within 30 days of Screening for Cohorts 1-3. In Cohort 4, dexamethasone is permitted
3. History of bilateral adrenalectomy, hypopituitarism, or other condition requiring chronic glucocorticoid therapy
4. Night shift workers or any other reason for abnormal sleep/wake cycles
5. Clinically significant unstable medical condition or chronic disease other than CAH
6. History of major surgery/surgical therapy for any cause within 4 weeks prior to Screening
7. 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
8. 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)
9. Participants with hypothyroidism who are not receiving adequate hormone replacement therapy based on thyroid hormone levels measured at the time of Screening
10. 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
11. History of cancer excluding cured/treated dermal squamous or basal cell carcinoma or cervical carcinoma in situ
12. Pregnant or lactating
13. Known history of illicit drug or alcohol abuse within the last year
14. Use of antiandrogen therapy in the past 3 months (eg, spironolactone, finasteride, cyproterone acetate, flutamide)
15. Use of testosterone, androgen-containing supplements, aromatase inhibitors, or growth hormone

Primary outcomes:
measure: Change from baseline in morning (before 11:00) serum androstenedione (A4)
timeFrame: Week 12
measure: Incidence of treatment-emergent adverse events (TEAEs) throughout the study
timeFrame: Week 12
Secondary outcomes:
measure: Change from baseline in morning (before 11:00) serum 17-hydroxyprogesterone (17-OHP)
timeFrame: Week 12
Other outcomes:
Locations:
city: Pasadena
country: United States
facility: Crinetics Study Site
state: California
city: Ann Arbor
country: United States
facility: Crinetics Study Site
state: Michigan
city: Minneapolis
country: United States
facility: Crinetics Study Site
state: Minnesota
city: Saint Louis
country: United States
facility: Crinetics Study Site
state: Missouri
city: Morehead City
country: United States
facility: Crinetics Study Site
state: North Carolina
city: Cleveland
country: United States
facility: Crinetics Study Site
state: Ohio
city: Philadelphia
country: United States
facility: Crinetics Study Site
state: Pennsylvania
city: East Providence
country: United States
facility: Crinetics Study Site
state: Rhode Island
city: Córdoba
country: Argentina
facility: Crinetics Study Site
state: Córdoba Province
city: Buenos Aires
country: Argentina
facility: Crinetics Study Site
city: Curitiba
country: Brazil
facility: Crinetics Study Site
state: Paraná
city: Porto Alegre
country: Brazil
facility: Crinetics Study Site
state: Rio Grande Do Sul
city: Botucatu
country: Brazil
facility: Crinetics Study Site
state: São Paulo
city: Ribeirão Preto
country: Brazil
facility: Crinetics Study Site
state: São Paulo
city: Rio De Janeiro
country: Brazil
facility: Crinetics Study Site
city: Rio De Janeiro
country: Brazil
facility: Crinetics Study Site
city: São Paulo
country: Brazil
facility: Crinetics Study Site
city: São Paulo
country: Brazil
facility: Crinetics Study Site
city: Munich
country: Germany
facility: Crinetics Study Site
state: Bavaria
city: Bangalore
country: India
facility: Crinetics Study Site
state: Karnataka
city: Vellore
country: India
facility: Crinetics Study Site
state: Tamil Nadu
city: Napoli
country: Italy
facility: Crinetics Study Site
city: Roma
country: Italy
facility: Crinetics Study Site
city: Rozzano
country: Italy
facility: Crinetics Study Site
city: Sheffield
country: United Kingdom
facility: Crinetics Study Site
state: South Yorkshire
city: London
country: United Kingdom
facility: Crinetics Study Site
Study officials:
References: