Global Safety and Efficacy Registration Study of Crinecerfont in Pediatric Participants With Classic Congenital Adrenal Hyperplasia (CAHtalyst Pediatric Study)

Neurocrine Biosciences

Status and phase: Active, not recruiting; Phase 3

Conditions: Congenital Adrenal Hyperplasia

Treatments: Drug: Placebo; Drug: Crinecerfont

Study type: Interventional

Funder types: Industry

Identifiers

NCT04806451

2023-509170-33-00 (EU Trial (CTIS) Number)

2020-004381-19 (EudraCT Number)

NBI-74788-CAH2006

Details and patient eligibility

About

This is a Phase 3 study to evaluate the efficacy, safety, and tolerability of crinecerfont versus placebo administered for 28 weeks in approximately 81 pediatric participants with classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency. The study consists of a 28-week double blind, placebo-controlled period, followed by 24 weeks of open-label treatment with crinecerfont. Subsequently, participants may elect to participate in the open-label extension (OLE) period. The duration of participation in the study is approximately 14 months for the core study and will be a variable amount of time per participant for the OLE (estimated to be approximately 3 years).

Enrollment: 103 patients

Sex: All

Ages: 2 to 17 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Be willing and able to adhere to the study procedures, including all requirements at the study center, and return for the follow-up visit.
• Have a medically confirmed diagnosis of classic CAH due to 21-hydroxylase deficiency.
• Be on a stable steroid regimen.
• Have elevated androgen levels.
• Participants of childbearing potential must be abstinent or agree to use appropriate birth control during the study.

Exclusion criteria

• Have a diagnosis of any of the other forms of classic CAH.
• Have a history of bilateral adrenalectomy, hypopituitarism, or other condition requiring chronic glucocorticoid therapy.
• Have a clinically significant unstable medical condition or chronic disease other than CAH.
• Have a history of cancer unless considered to be cured.
• Have a known history of clinically significant arrhythmia or abnormalities on electrocardiogram (ECG).
• Have a known hypersensitivity to any corticotropin-releasing hormone antagonist.
• Have received an investigational drug within 30 days before initial screening or plan to use an investigational drug (other than the study drug) during the study.
• Have current substance dependence or substance (drug) or alcohol abuse.
• Have had a significant blood loss or donated blood or blood products within 8 weeks prior to the study.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

---

103 participants in 2 patient groups, including a placebo group

Crinecerfont

Experimental group

Description:

Crinecerfont solution or capsule, administered orally, twice daily for 28 weeks during the placebo-controlled treatment period, followed by active treatment with crinecerfont for at least 24 weeks.

Treatment:

Drug: Crinecerfont

Placebo

Placebo Comparator group

Description:

Placebo solution or capsule, administered orally, twice daily for 28 weeks, followed by active treatment with crinecerfont for at least 24 weeks.

Treatment:

Drug: Crinecerfont

Drug: Placebo