RecruitingPHASE2, PHASE3INTERVENTIONAL

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

This study is exploring a new medication called atumelnant for young people aged 1 to 17 with a specific type of Congenital Adrenal Hyperplasia (CAH). CAH is a rare genetic condition that affects the body's ability to make certain hormones. The main goals are to check if atumelnant is safe, if it helps manage the condition, and to understand how it works in the body. The study will involve different stages, starting with smaller groups to test safety and then moving to a larger, more controlled stage where some participants will receive the study drug and others a dummy treatment (placebo). There's also an option for those who complete the main parts to continue receiving the medication if it's considered beneficial.

At a glance

Status

Recruiting

Phase

PHASE2, PHASE3

Sponsor

Crinetics Pharmaceuticals Inc.

Enrolment target

153

Start

22 Jan 2026

Estimated completion

01 Mar 2030

Congenital Adrenal Hyperplasia Classic Congenital Adrenal Hyperplasia

What is this study about?

This research study, called Balance-CAH, is designed to investigate a new medication named atumelnant for children and teenagers who have a specific type of inherited condition called Congenital Adrenal Hyperplasia (CAH). CAH affects the adrenal glands, which are small glands above your kidneys that produce important hormones. In classic CAH, the body doesn't make enough of certain hormones, like cortisol, which is vital for stress response and energy. This can lead to various health issues.

The main aim of this study is to find out if atumelnant is a safe and effective treatment option for these young people. It will look closely at how well the medication works to manage their CAH and how their bodies react to it. This kind of research is essential to discover new and better ways to help people living with long-term conditions like CAH.

The study is broken down into different parts. Initially, smaller groups of older teenagers will try the medication to assess safety. Then, a larger group of children and teenagers will take part, some receiving atumelnant and others a placebo (a dummy treatment without active medicine) to compare their effects. This allows researchers to understand the true impact of the new medication. Those who complete these main parts might be able to continue on the study drug if their doctors think it would be helpful.

Key takeaways

A study for children and teenagers (1-17 years old) with classic CAH.
Investigating a new medication called atumelnant for safety and effectiveness.
Compares atumelnant to a placebo (dummy treatment) in some stages.
Requires regular clinic visits and health check-ups.
Participation is voluntary, and you can withdraw at any time.

Who may be eligible?

This study is looking for children and teenagers aged between 1 and 17 years old who have a confirmed diagnosis of classic Congenital Adrenal Hyperplasia (CAH) due to a specific enzyme deficiency (21-hydroxylase deficiency). To be considered, their morning blood tests need to show higher-than-normal levels of a hormone called A4, and they must have been taking their current regular steroid medication consistently for at least one month before starting the study.

There are also some important reasons why someone might not be able to join. For example, if they have any other form of CAH, or if they have recently received other steroid treatments, including a 'stress dose' to manage an illness or injury. Also, if they have used growth hormones or certain other medications recently, they would not be eligible to participate.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Is the person between 1 and 17 years old?
Do they have a confirmed diagnosis of classic CAH?
Have they been taking their regular steroid medication consistently for at least one month?
Have they recently had any 'stress doses' of steroids or used growth hormones?
Are their morning blood tests showing higher-than-normal A4 levels?

Answer every question to see your result.

What does participation involve?

If you decide to take part, the study will involve several visits to the clinic over a period of time. You'll have regular check-ups, blood tests, and other assessments to monitor your health and how the study medication is affecting you. You will be given either the new study medication, atumelnant, or a placebo (dummy drug) during the main part of the study. You'll need to take the medication as instructed and keep a record. If you complete the main study parts and your doctor thinks it's beneficial, you might have the option to continue receiving the atumelnant in a longer-term period of the study. The exact total duration of your involvement will depend on which part of the study you join and whether you continue into the extension phase.

Potential risks and benefits

Taking part in a clinical trial may offer potential benefits, such as access to a new medication before it's widely available and closer monitoring of your health by medical professionals. However, there are also potential risks, including side effects from the study medication, or the possibility of receiving a placebo instead of the active drug. The study team will explain all known risks to you before you decide to participate, and they will carefully monitor you throughout the study. Remember, your participation is completely voluntary, and you are free to withdraw from the study at any time without affecting your usual medical care.

Locations (33)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Boston's Children's Hospital
Verified postcode
Boston, United States· Recruiting
University of Michigan
Verified postcode
Ann Arbor, United States· Recruiting
University of Minnesota
Verified postcode
Minneapolis, United States· Recruiting
Rutgers Robert Wood Johnson Medical School
Verified postcode
New Brunswick, United States· Recruiting
Children's Hospital of Philadelphia
Verified postcode
Philadelphia, United States· Recruiting
Cook Children's Health Care System
Verified postcode
Fort Worth, United States· Recruiting
University of Virginia Health System
Verified postcode
Charlottesville, United States· Recruiting
Instituto de Investigaciones Metabólicas
Unverified
Buenos Aires, Argentina· Recruiting
Hospital de Niños de la Santísima Trinidad
Verified postcode
Córdoba, Argentina· Recruiting
Hospital Italiano de Buenos Aires
Verified postcode
Buenos Aires, Argentina· Recruiting
Instituto Médico Especializado (IME)
Verified postcode
Buenos Aires, Argentina· Recruiting
CEDIE "Centro de Investigaciones Endocrinológicas", CONICET-FEI División de Endocrinología, Hosp de Niños Ricardo Gutiérrez
Verified postcode
Buenos Aires, Argentina· Recruiting

Common questions

What is Congenital Adrenal Hyperplasia (CAH)?

CAH is a genetic condition where your body can't make enough of certain important hormones because your adrenal glands don't work properly.

What is atumelnant?

Atumelnant is a new medication being tested to see if it can help manage classic CAH in children and teenagers.

What does 'placebo' mean?

A placebo is a dummy treatment without any active medicine. It helps researchers compare the effects of the new drug to no treatment at all.

Can my child definitely get the new medication if we join?

During parts of the study, some participants will receive the new medication and others will receive a placebo. It's not guaranteed your child will get the active drug at all stages.

What if we change our mind about participating?

You are free to stop participating in the study at any time, for any reason, without it affecting your child's medical care.

How to find out more

Crinetics Clinical Trials

clinicaltrials@crinetics.com 833-827-9741 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.