Study to Evaluate the Safety and Efficacy of Crinecerfont in Pediatric Patients With Classic Congenital Adrenal Hyperplasia
- Conditions
- Classic Congenital Adrenal Hyperplasia (CAH)MedDRA version: 20.0Level: LLTClassification code 10010323Term: Congenital adrenal hyperplasiaSystem Organ Class: 100000004850Therapeutic area: Diseases [C] - Hormonal diseases [C19]
- Registration Number
- EUCTR2020-004381-19-PL
- Lead Sponsor
- eurocrine Biosciences, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 81
- Be willing and able to adhere to the study procedures, including all requirements at the study center and return for the follow-up visit.
- Be a female or male 2 to 17 years of age with a body weight of at least 10 kg.
- Have a medically confirmed diagnosis of classic CAH due to 21- hydroxylase deficiency.
- Be on a stable regimen of glucocorticoid treatment for CAH.
- Have elevated adrenal androgens.
- If treated with fludrocortisone, dose should be stable for at least 1 month prior to screening. Regardless of fludrocortisone treatment, upright plasma renin activity (PRA) (in the absence of medications that confound interpretation of PRA) during screening should be <3× ULN and >lower limit of normal (LLN) on the subject’s usual sodium intake (if PRA >2 × ULN and <3 × ULN, subject must have normal age-specific systolic blood pressure and heart rate and serum potassium Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
- Have a diagnosis of any of the other forms of classic CAH.
- Have a history of bilateral adrenalectomy, hypopituitarism, or other condition requiring chronic daily therapy with oral glucocorticoids.
- Have a clinically significant unstable medical condition or chronic disease other than CAH
- Have a history of malignancy, unless successfully treated with curative intent and considered to be cured.
- Have a known history of clinically significant arrhythmia or abnormalities on screening ECG.
- Have a known hypersensitivity or allergy to any corticotropin-releasing hormone (CRH) receptor antagonist or any component of the study drug.
- Females who are pregnant or lactating.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: • To evaluate the efficacy of crinecerfont, compared with placebo, in reducing adrenal steroid levels during a glucocorticoid-stable period.<br><br>;Secondary Objective: • To evaluate the efficacy of crinecerfont, compared with placebo, in reducing daily glucocorticoid dosage while maintaining adrenal androgen control.<br>• To evaluate the effect of crinecerfont, compared with placebo, on clinical endpoints associated with supraphysiologic glucocorticoid dosing and androgen excess.<br>• To evaluate plasma concentrations of crinecerfont and metabolites.<br>• To assess the safety and tolerability of crinecerfont.;Primary end point(s): Change from baseline in serum androstenedione at Week 4.;Timepoint(s) of evaluation of this end point: Week 4
- Secondary Outcome Measures
Name Time Method Secondary end point(s): - Percent change from baseline in glucocorticoid dose at Week 28<br>- Change from baseline in serum 17-hydroxyprogesterone at Week 4<br>;Timepoint(s) of evaluation of this end point: Week 4 and Week 28