NCT07159841

A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH)

A Phase 2/3 Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Atumelnant Treatment in Pediatric Participants With Congenital Adrenal Hyperplasia Including a Long-Term Extension

• Status: Recruiting
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 153 (estimated)
• Sponsor: Crinetics Pharmaceuticals Inc. · Industry
• Sex: All
• Age: 1 Year – 17 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to evaluate the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of atumelnant treatment in pediatric participants with classic congenital adrenal hyperplasia (CAH).

Detailed description

This Phase 2/3 plus open-label extension study is designed to evaluate the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of atumelnant treatment in pediatric participants with classic CAH. Part A is a Phase 2, open-label, semi-sequential cohorts portion of the study. Part B is the Phase 3, double-blind, randomized, placebo controlled confirmatory portion of the study. Part C is the open-label extension (OLE) portion of the study. Participants in Part A and B are eligible to enroll in Part C (OLE). A total of approximately 153 participants may be enrolled in the study (planned and optional cohorts) ages 1 to \< 18 years old. The first 3 cohorts in Part A are for ages 12 to \<18 years and will be sequential, and Safety Review Committee (SRC) review of data and approval to proceed is required prior to enrolling each subsequent cohort. The fourth cohort in Part A is for ages 1 to 11 years old and will begin after Cohorts 1 and 2 have been completed, additional requirements are fulfilled, and following SRC review of Cohorts 1 and 2 data.

Conditions

• Congenital Adrenal Hyperplasia
• Classic Congenital Adrenal Hyperplasia

Interventions

Type	Name	Description
DRUG	Atumelnant	Atumelnant, tablets, once daily by mouth, weight-based dosing
DRUG	Placebo	Placebo, tablets, once daily by mouth, weight-based dosing

Timeline

• Start date: 2026-01-22
• Primary completion: 2030-03-01
• Completion: 2030-03-01
• First posted: 2025-09-08
• Last updated: 2026-04-08

Locations

24 sites across 9 countries: United States, Argentina, Australia, Belgium, Brazil, France, Italy, Poland, United Kingdom

Regulatory

• FDA-regulated drug study