Skip to content
Clinical Trials – home

Rare diseases

Investigational molecules

Locations

Our offerNewsroom

RecruitingRare disease

A Study of Atumelnant for Children with Congenital Adrenal Hyperplasia

Fast replyInvestigationalNo placebo
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

What is this trial about?

A plain-language summary of the goals, design and what participants do

This study involves children and teenagers with classic congenital adrenal hyperplasia, which is a condition where the adrenal glands do not produce certain hormones properly. Because of this condition, the body makes too much of some hormones called androgens and not enough of a hormone called cortisol. People with this condition usually need to take replacement hormones called glucocorticoids, which are medicines that replace the missing cortisol. However, sometimes these medicines need to be given in higher doses than the body would normally make, which can cause unwanted effects. The study will test a medicine called atumelnant, which is also known by its code name CRN04894. This medicine comes in tablet form and is taken by mouth. It works by blocking certain receptors in the body to help control the production of androgens.

The purpose of this study is to see if atumelnant is safe and works well in children with congenital adrenal hyperplasia, and whether it can help reduce the amount of glucocorticoid medicine they need to take while keeping their hormone levels normal. The study is divided into three parts. In Part A, participants will receive atumelnant for eight weeks to check how safe it is and to measure changes in a hormone called androstenedione, which is one of the androgens that becomes too high in people with this condition. In Part B, which lasts twenty-eight weeks, the study will look at whether atumelnant allows doctors to lower the daily dose of glucocorticoid medicine while keeping androstenedione levels normal. Some participants may receive placebo. In Part C, which is a long-term extension, participants who completed Part A or Part B can continue treatment to see how atumelnant works over a longer period of time.

During the study, participants will have regular visits where doctors will check their hormone levels through blood tests, monitor their safety, and measure how much medicine is in their blood. The study will also look at changes in another hormone called 17-hydroxyprogesterone, which is also elevated in people with congenital adrenal hyperplasia. The doctors will carefully watch for any side effects and see how well participants tolerate the medicine. Throughout the study, participants will continue to take their regular replacement hormone medicines, and doctors will adjust the doses as needed based on how the participant responds to atumelnant.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Starting treatment and initial evaluation

    Upon joining the study, your current glucocorticoid replacement therapy (hormone replacement medication such as hydrocortisone, prednisolone, prednisone, methylprednisolone, or dexamethasone) will continue as prescribed by your doctor.

    Blood samples will be taken in the morning (before 11:00) to measure hormone levels, specifically androstenedione (a hormone called A4) and 17-hydroxyprogesterone (17-OHP). These are hormones that are elevated in congenital adrenal hyperplasia.

    The blood sample will be collected before taking the morning dose of glucocorticoid medication.

  2. Step 2

    Part A: Treatment with atumelnant for 8 weeks

    Treatment with atumelnant tablets will begin. This medication is taken by mouth.

    The dose of atumelnant may be 20 mg, 40 mg, or 80 mg depending on individual needs as determined by the study doctor.

    This treatment period lasts for 8 weeks.

    During this period, safety and how well the body tolerates the medication will be monitored.

    Blood samples will be taken to measure hormone levels (A4 and 17-OHP) and to check the amount of atumelnant in the blood.

    At the end of 8 weeks, changes in morning A4 hormone levels compared to the start of treatment will be assessed.

  3. Step 3

    Part B: Adjusting glucocorticoid dose for 28 weeks

    If eligible to continue, the next phase lasts for 28 weeks.

    The goal during this period is to reduce the daily dose of glucocorticoid medication while keeping hormone levels (A4) within normal range.

    Treatment with atumelnant tablets continues during this period.

    Blood samples will be taken in the morning to monitor A4 and 17-OHP hormone levels.

    Blood samples will be collected at Week 4 and at Week 28 to measure changes in hormone levels.

    The study doctor will adjust the glucocorticoid dose as appropriate based on hormone level results.

  4. Step 4

    Part C: Long-term extension phase

    After completing Part A or Part B, there is an option to continue in a long-term extension phase if the study doctor determines it would be beneficial.

    This phase allows continued treatment and monitoring over an extended period.

    Treatment with atumelnant tablets continues.

    Regular blood samples will be taken over time to monitor morning A4 and 17-OHP hormone levels.

    The glucocorticoid dose may continue to be adjusted with the goal of reaching a normal physiologic dose while maintaining hormone levels within normal range.

    The duration of this phase will be determined based on individual progress and study requirements.

Who can join the trial?

12 criteria

  • The participant must be male or female at birth, between 1 and less than 18 years old at the time of signing the consent form
  • The participant must have a confirmed diagnosis of classic congenital adrenal hyperplasia, which is a condition where the adrenal glands do not produce certain hormones properly, caused by a specific enzyme problem called 21-OHD
  • The diagnosis must be confirmed by one of the following: elevated levels of a hormone called 17-OHP, genetic testing showing changes in the CYP21A2 gene, positive newborn screening with additional testing, or a stimulation test using a substance called cosyntropin
  • The participant must have high levels of a hormone called A4 in the blood, measured in the morning before 11:00 AM and before taking their morning medication
  • The participant must be taking stable doses of glucocorticoid replacement therapy, which are medications that replace missing hormones, for at least one month before entering the study
  • The participant must be following their prescribed medication schedule for both glucocorticoid replacement and mineralocorticoid replacement if needed, as determined by the doctor
  • The participant must have normal levels of thyroid hormones called TSH and T4 within three months before entering the study
  • Female participants who have started their menstrual periods and are sexually active must either be surgically unable to have children or agree to use highly effective birth control methods from the beginning of the study until at least 2 weeks after the last dose of study medication
  • Male participants who are sexually active must agree to use condoms when having sex with female partners who can become pregnant, or remain abstinent, from the beginning of the study until at least 2 weeks after the last dose of study medication
  • Male participants must agree not to donate sperm during the study and until at least 2 weeks after the last dose of study medication
  • The participant's parent or legal guardian must be willing and able to provide signed consent for the participant to join the study
  • The participant and their family must be willing and able to follow all study procedures and take the study medication as directed

Who cannot join the trial?

1 criterion

  • The clinical trial data provided does not contain specific exclusion criteria information at this time.
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

We usually reply within a few days

Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Trial locations

Where you can join this trial

Countries are shaded by recruitment status. Click a recruiting country to ask about joining there.

Recruiting
Not finding your country?

Not sure what to do next?

Joining a clinical trial can seem overwhelming. We guide you step by step, so you know exactly what to expect and how we support you through the process.

See the full process and FAQ

Investigated drugs

  • Atumelnant

    is an investigational medication being tested in this clinical trial for children with congenital adrenal hyperplasia, a condition that affects hormone production. This medication is being studied to see if it can help control androgen levels (certain hormones) in the blood and potentially reduce the amount of glucocorticoid medication that patients need to take daily.

  • Glucocorticoids

    are steroid medications that are commonly used to treat congenital adrenal hyperplasia. In this study, researchers want to see if atumelnant can help lower the daily dose of these medications while still keeping hormone levels under control.

What is already known about the treatment

Atumelnant – Atumelnant is an investigational medication currently being studied in clinical trials for the treatment of classic congenital adrenal hyperplasia, a genetic condition that affects hormone production in the adrenal glands. The drug is being tested in pediatric patients to evaluate its safety, effectiveness, and how the body processes it, with the goal of reducing excess male hormones (androgens) in the blood and potentially lowering the need for steroid medications that these patients typically require. Atumelnant works at the molecular level by targeting specific pathways involved in hormone regulation, though it is still in the research phase and not yet approved for general medical use. The medication belongs to a class of drugs designed to help normalize hormone levels in patients whose adrenal glands produce too much androgen due to enzyme deficiencies.

Investigated diseases

Classic Congenital Adrenal Hyperplasia – Classic congenital adrenal hyperplasia is a genetic disorder that affects the adrenal glands, which are small organs located above the kidneys. In this condition, the adrenal glands cannot produce enough cortisol, a hormone that helps the body respond to stress and maintain blood pressure and blood sugar levels. The body tries to compensate by producing excess amounts of male hormones called androgens. This leads to various symptoms including abnormal development of external genitalia in females, early appearance of pubic hair, rapid growth in childhood followed by shorter than average adult height, and irregular menstrual periods. The condition is present from birth and requires lifelong management. Without proper care, individuals may experience problems with growth, development, and hormone balance throughout their lives.
Trial detailsLast updated 2 Oct 2026
Age0-17PhasePhase IVTrial ID2024-519578-38-00Protocol codeCRN04894-13Estimated enrolment156 patientsSponsorCrinetics Pharmaceuticals Inc.

sourced from the EU Clinical Trials Register and site verification

Want to learn more about this trial or check if you can participate?

Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

ClinicalTrials.eu

Every clinical trial in Europe, in plain language.

Explore the full ecosystem of clinical research in Europe.

Search trials

Legal notice · Published by CTIN POLAND sp. z o.o., ul. rtm. Witolda Pileckiego 67/109, 02-781 Warsaw, Poland · KRS 0001111334 · REGON 528919042 · NIP 9512598637

© 2026 Clinical Trials EU – European Clinical Trials Information Network

GDPR compliance, ISO 9001 and ISO 27001 certified (LL-C Certification)

On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).