A Study of Atumelnant for Children with Congenital Adrenal Hyperplasia
This study is testing a drug called atumelnant in children aged 1 to 17 years old who have classic congenital adrenal hyperplasia (CAH), a condition where the adrenal glands don't make enough of certain hormones. The main goals are to see how safe and effective atumelnant is, and how the body uses and reacts to the drug. Researchers will measure changes in a hormone called androstenedione (A4) and how much steroid medicine (glucocorticoid) participants need. Some participants will receive atumelnant, while others will receive a placebo (an inactive substance that looks like the drug). The study aims to enroll about 153 participants.
- Study design
- This is a Phase 2/3 study with an open-label extension. It includes a double-blind, randomized, placebo-controlled part, meaning some participants will receive atumelnant and others a placebo, and neither you nor your doctor will know which you are receiving.
- What's involved
- Not specified in the trial record.
- Compensation
- Not stated in the trial record.
- Follow-up
- Some participants may be followed for up to 260 weeks (about 5 years) to observe changes in serum early morning A4 over time.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH)
At a glance
Conditions
NCT07159841
Where you'd take part
This study runs at 48 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.
Alder Hey Children's NHS Foundation Trust, Alder Hey Children's Hospital
Liverpool, United Kingdomno site contact published
Recruiting
AOU Federico II
Naples, Campania, Italyno site contact published
Recruiting
APHM -Hopital La Timone Enfants
Marseille, Franceno site contact published
Recruiting
Azienda Ospedaliera Universitaria Meyer IRCCS
Florence, Tuscany, Italyno site contact published
Recruiting
Boston's Children's Hospital
Boston, Massachusettsno site contact published
Recruiting
CEDIE "Centro de Investigaciones Endocrinológicas", CONICET-FEI División de Endocrinología, Hosp de Niños Ricardo Gutiérrez
Buenos Aires, Argentinano site contact published
Recruiting
Centre Hospitalier Universitaire (CHU) d'Angers
Angers, Franceno site contact published
Recruiting
Charité - Universitätsmedizin Berlin Campus Virchow-Klinikum Klinik für pädiatrische Endokrinologie und Diabetologie
Berlin, Germanyno site contact published
Recruiting
Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.
Who to contact
Opens a ready-to-send draft in your own email app — review before sending.
What this trial measures
- Change from baseline in morning serum androstenedione (A4) (Part A)Week 8
- Percent change from baseline in glucocorticoid (GC) daily dose while serum early morning A4 ≤Upper Limit of Normal (ULN) (Part B)Week 28
- Change from baseline in serum early morning A4 over time (Part C)Up to Week 260