A Study of Atumelnant for Children with Congenital Adrenal Hyperplasia

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What is this study about?

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.

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 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 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 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 Study?

  • 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 Study?

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

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Other Sites

Site Name City Country Status
IRCCS Istituto Giannina Gaslini Genoa Italy
Azienda Ospedaliera Universitaria Meyer IRCCS Florence Italy
Universitair Ziekenhuis Gent Gent Belgium
Instytut Centrum Zdrowia Matki Polki Lodz Poland
Uniwersyteckie Centrum Kliniczne Warszawskiego Uniwersytetu Medycznego Warsaw Poland
Universitaetsklinikum Tuebingen AöR Tuebingen Germany
Ospedale San Raffaele S.r.l. Milan Italy
Charite Universitaetsmedizin Berlin KöR Berlin Germany
Saarland University Hospital Homburg Germany
Stichting Radboud University Medical Center Nijmegen The Netherlands
Medical Center – University Of Freiburg Freiburg Im Breisgau Germany
Uniwersytecki Dzieciecy Szpital Kliniczny Im. L. Zamenhofa W Bialymstoku samodzielny publiczny zakład opieki zdrowotnej Bialystok Poland
Hopital Beaujon Clichy France
Ospedale Pediatrico Bambino Gesu’ Rome Italy
Cpwgbxeqv Uwdovqwezlgjqc Ssrxrfmsn Woluwe-Saint-Lambert Belgium
Uiwxscqftz Hwxztkbq Cbhfddl Cologne Germany
Adbgnmrjox Pfikepln Hqjvfsdr Do Mwostyyvn Marseille France
Uqilrrubmv Oe Acfbnzx Edegem Belgium
Ctwhtd Hndkgqjdicg Rseylevl Dyllcywpvbaxkt Angers France
Slhvaiueeyt Pbcmdfnbx Sgyidba Kfusqoiwc Nh 1 Ihfmfvnlvraaifhqox Szxnctu Sybfsbico Uyjbastjedoj Mrykikmqtl W Ktbmnxafkf Zabrze Poland

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Belgium Belgium
Recruiting
26.11.2025
France France
Recruiting
26.11.2025
Germany Germany
Recruiting
26.11.2025
Italy Italy
Recruiting
26.11.2025
Poland Poland
Recruiting
26.11.2025
The Netherlands The Netherlands
Not yet recruiting
26.11.2025

Trial locations

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.

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 ID:
2024-519578-38-00
Protocol code:
CRN04894-13
Trial Phase:
Therapeutic use (Phase IV)

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