Adrenogenital syndrome – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Fri, 12 Jun 2026 11:23:17 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Adrenogenital syndrome – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 A Study of Atumelnant for Children with Congenital Adrenal Hyperplasia https://clinicaltrials.eu/trial/a-study-of-atumelnant-for-children-with-congenital-adrenal-hyperplasia/ Wed, 29 Apr 2026 15:08:45 +0000 https://clinicaltrials.eu/trial/a-study-of-atumelnant-for-children-with-congenital-adrenal-hyperplasia/ 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.

]]> A Study Testing Atumelnant Compared to Placebo in Adults with Classic Congenital Adrenal Hyperplasia to Reduce Steroid Dose While Controlling Hormones https://clinicaltrials.eu/trial/a-study-testing-atumelnant-compared-to-placebo-in-adults-with-classic-congenital-adrenal-hyperplasia-to-reduce-steroid-dose-while-controlling-hormones/ Wed, 29 Apr 2026 15:08:45 +0000 https://clinicaltrials.eu/trial/a-study-testing-atumelnant-compared-to-placebo-in-adults-with-classic-congenital-adrenal-hyperplasia-to-reduce-steroid-dose-while-controlling-hormones/ This study is looking at a condition called classic congenital adrenal hyperplasia, which is a disorder where the adrenal glands do not produce certain hormones properly. People with this condition need to take replacement hormones called glucocorticoids to help their body function normally. The treatment being tested in this study is called atumelnant, which comes in tablet form and is taken by mouth. The study will compare atumelnant with placebo to see which works better. Atumelnant is also known by its code name CRN04894.

The purpose of this study is to find out if atumelnant can help reduce the daily dose of glucocorticoid medication that people with this condition need to take, while still keeping the levels of certain hormones called adrenal androgens under control. The study will measure hormone levels in the blood, including a hormone called androstenedione and another one called 17-hydroxyprogesterone, to see if the treatment is working. The study will also look at how safe atumelnant is and what side effects it might cause.

The study will last for 32 weeks of treatment, and participants will be randomly assigned to receive either atumelnant tablets or placebo tablets. During the study, doctors will regularly check hormone levels in the blood and may adjust the dose of glucocorticoid medication based on these results. Participants will need to continue taking their regular glucocorticoid replacement medication throughout the study, and if they take a medication called fludrocortisone for mineral balance, they will continue that as well. The study will involve multiple visits to the study site for blood tests and health checks to monitor how well the treatment is working and to watch for any problems.

]]> Study of crinecerfont oral solution in children under 2 years old with congenital adrenal hyperplasia to evaluate safety and how the drug works in the body https://clinicaltrials.eu/trial/study-of-crinecerfont-oral-solution-in-children-under-2-years-old-with-congenital-adrenal-hyperplasia-to-evaluate-safety-and-how-the-drug-works-in-the-body/ Wed, 29 Apr 2026 15:07:25 +0000 https://clinicaltrials.eu/trial/study-of-crinecerfont-oral-solution-in-children-under-2-years-old-with-congenital-adrenal-hyperplasia-to-evaluate-safety-and-how-the-drug-works-in-the-body/ This study focuses on Congenital Adrenal Hyperplasia (CAH), a genetic condition that affects the body’s ability to produce certain hormones in the adrenal glands. The study will test a new medication called crinecerfont, which is given as an oral solution (liquid medicine taken by mouth) to children under 2 years of age who have this condition.

The purpose of this research is to understand how crinecerfont works in the bodies of young children with CAH, including how safe it is and how well they tolerate it. During the study, children will receive the medication for 14 days while continuing their regular hormone replacement treatment. The study will monitor how the medicine moves through their bodies and collect information about any effects they experience.

Throughout the study, researchers will take blood samples to measure the amount of medicine in the children’s bodies. They will also watch for any changes in the children’s health and record any side effects that may occur. This information will help determine if the medication might be suitable for treating young children with CAH in the future.

]]> Study on the Safety and Effectiveness of CRN04894 for Patients with Congenital Adrenal Hyperplasia https://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-crn04894-for-patients-with-congenital-adrenal-hyperplasia-2/ Wed, 29 Apr 2026 15:04:10 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-crn04894-for-patients-with-congenital-adrenal-hyperplasia-2/ This clinical trial is focused on studying a condition known as (CAH). CAH is a genetic disorder that affects the adrenal glands, which are small organs located on top of the kidneys. These glands produce important hormones that help regulate various functions in the body. The study is investigating a treatment called , which is a chemical product designed to work as an . This means it helps block certain signals in the body that can lead to hormone imbalances in people with CAH.

The purpose of this study is to learn more about the safety and effectiveness of in people who have already participated in previous studies involving this treatment. Participants will take the medication in the form of a tablet, which is taken orally, meaning it is swallowed. The study will last for a long period, allowing researchers to gather information on how well the treatment works and how safe it is over time. The main focus will be on monitoring any changes in hormone levels, specifically a hormone called , which is often elevated in people with CAH.

Throughout the study, participants will be closely monitored for any side effects or health changes. The goal is to ensure that is both safe and effective for long-term use in managing . This research is important for improving treatment options and quality of life for individuals living with this condition.

]]> Study on the Effectiveness of Dexamethasone in Preventing Prenatal Virilization in Female Patients with Congenital Adrenal Hyperplasia https://clinicaltrials.eu/trial/study-on-the-effectiveness-of-dexamethasone-in-preventing-prenatal-virilization-in-female-patients-with-congenital-adrenal-hyperplasia/ Wed, 29 Apr 2026 14:57:37 +0000 https://clinicaltrials.eu/trial/study-on-the-effectiveness-of-dexamethasone-in-preventing-prenatal-virilization-in-female-patients-with-congenital-adrenal-hyperplasia/ This clinical trial is focused on studying the effects of the medication dexamethasone in treating a condition known as Congenital Adrenal Hyperplasia (CAH). CAH is a genetic disorder that affects the adrenal glands, which are small glands located on top of the kidneys. These glands produce important hormones, and in CAH, there is a problem with hormone production that can lead to various health issues. The trial will use different doses of dexamethasone, a type of medication known as a glucocorticoid, which is often used to reduce inflammation and suppress the immune system.

The purpose of this study is to determine if a lower dose of dexamethasone is as effective as the higher dose currently used in preventing certain effects of CAH in unborn female babies. Specifically, the study aims to see if the lower dose can prevent prenatal virilization, which is the development of male physical characteristics in female babies before birth. Additionally, the study will look at how the medication affects the weight gain of the mother during pregnancy.

Participants in the study will take dexamethasone in the form of a hard capsule, which is taken orally. The study will compare different doses of the medication, including 0.5mg, 0.21mg, 0.41mg, and 0.15mg, over a period of up to 40 days. Some participants may receive a placebo. The trial will monitor various health outcomes for both the mother and the child, including the closure of the urogenital sinus in the baby and the mother’s weight gain during pregnancy. Safety aspects such as the occurrence of stillbirths, miscarriages, and any side effects in both mothers and children will also be closely observed.

]]> Study on the Safety and Effectiveness of Crinecerfont for Children with Classic Congenital Adrenal Hyperplasia https://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-crinecerfont-for-children-with-classic-congenital-adrenal-hyperplasia/ Wed, 29 Apr 2026 14:29:03 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-crinecerfont-for-children-with-classic-congenital-adrenal-hyperplasia/ This clinical trial is focused on studying a condition known as Classic Congenital Adrenal Hyperplasia (CAH), which is a genetic disorder affecting the adrenal glands. The study is evaluating a treatment called Crinecerfont, also known by its code name NBI-74788. Crinecerfont is being tested in two forms: an oral solution and a capsule. The purpose of the study is to assess the safety and effectiveness of Crinecerfont in reducing certain hormone levels in children with CAH.

Participants in the study will be randomly assigned to receive either Crinecerfont or a placebo, which is a substance with no active medication. The study will begin with a period where neither the participants nor the researchers know who is receiving the actual medication or the placebo. This is known as a double-blind study. After this initial phase, all participants will have the opportunity to receive Crinecerfont in an open-label phase, where everyone knows they are receiving the active treatment.

The study will last for up to 52 weeks, during which time participants will have regular visits to the study center to monitor their health and hormone levels. The main goal is to see if Crinecerfont can safely lower adrenal steroid levels while participants continue their usual treatment for CAH. This study is important for understanding how Crinecerfont can help manage CAH in children and improve their quality of life.

]]> Study on the Safety and Effectiveness of Crinecerfont for Adults with Classic Congenital Adrenal Hyperplasia https://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-crinecerfont-for-adults-with-classic-congenital-adrenal-hyperplasia/ Wed, 29 Apr 2026 14:27:24 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-crinecerfont-for-adults-with-classic-congenital-adrenal-hyperplasia/ This clinical trial is focused on studying a condition known as Classic Congenital Adrenal Hyperplasia (CAH). This is a genetic disorder that affects the adrenal glands, which are responsible for producing important hormones. The study is testing a new treatment called Crinecerfont, also known by its code name NBI-74788. This medication is being compared to a placebo to see how effective it is in managing the symptoms of CAH.

The purpose of the study is to evaluate how well Crinecerfont works in reducing the need for other medications called glucocorticoids, which are often used to treat CAH. The study will also look at how Crinecerfont affects hormone levels in the body and its overall safety. Participants will take the medication in capsule form, twice a day, for a period of time. The study will start with a four-week treatment period, followed by additional assessments to monitor changes in hormone levels and any side effects.

Throughout the study, researchers will collect information on how Crinecerfont affects the body, including its impact on hormone levels and any potential side effects. The study aims to provide a better understanding of how this new treatment can help manage Classic Congenital Adrenal Hyperplasia and improve the quality of life for those affected by this condition.

]]> Study on the Safety and Effectiveness of CRN04894 for Patients with Congenital Adrenal Hyperplasia https://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-crn04894-for-patients-with-congenital-adrenal-hyperplasia/ Wed, 29 Apr 2026 14:26:47 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-crn04894-for-patients-with-congenital-adrenal-hyperplasia/ This clinical trial is focused on studying a condition called , a genetic disorder affecting the adrenal glands, which are responsible for producing important hormones. The trial will evaluate a new treatment called , which is an . This means it works by blocking a specific receptor involved in hormone production. The study aims to assess the safety and effectiveness of in managing this condition.

Participants in the study will receive over a period of 12 weeks. The treatment will be given in a sequential dose manner, meaning the dose may be adjusted as the study progresses. The study will also involve monitoring the levels of certain hormones in the blood, such as androstenedione, to see how they change with the treatment. This will help determine how well the treatment is working.

In addition to , other medications may be used in the study, including , , , , and . These medications are commonly used to manage symptoms and hormone levels in people with . Some participants may receive a placebo, which is a substance with no active medication, to compare the effects of the treatment. The study will help gather important information about the potential benefits and any side effects of for people with this condition.

]]> Study on Tildacerfont to Reduce Glucocorticoid Use in Adults with Classic Congenital Adrenal Hyperplasia https://clinicaltrials.eu/trial/study-on-tildacerfont-to-reduce-glucocorticoid-use-in-adults-with-classic-congenital-adrenal-hyperplasia/ Wed, 29 Apr 2026 14:24:30 +0000 https://clinicaltrials.eu/trial/study-on-tildacerfont-to-reduce-glucocorticoid-use-in-adults-with-classic-congenital-adrenal-hyperplasia/ This clinical trial is focused on studying a condition known as Classic Congenital Adrenal Hyperplasia, a genetic disorder affecting the adrenal glands. The study aims to evaluate the effectiveness and safety of a medication called Tildacerfont (also known by its code name SPR001) in reducing the use of glucocorticoids, which are a type of steroid hormone, in adults with this condition. Participants in the study are currently taking higher-than-normal doses of glucocorticoids to manage their symptoms.

The trial involves taking Tildacerfont in tablet form, and it will be compared to a placebo to assess its potential benefits. Other medications involved in the study include Prednisolone and Hydrocortisone, which are also types of glucocorticoids. The study is designed to be double-blind, meaning neither the participants nor the researchers know who is receiving the actual medication or the placebo, to ensure unbiased results.

Over a period of 24 weeks, the study will monitor changes in the amount of glucocorticoids needed by participants. The goal is to see if Tildacerfont can help reduce the need for these medications, potentially leading to better management of Classic Congenital Adrenal Hyperplasia. Participants will take the medication orally, and their progress will be closely observed throughout the study period.

]]> Study on the Effects of Tildacerfont and Hydrocortisone in Adults with Classic Congenital Adrenal Hyperplasia https://clinicaltrials.eu/trial/study-on-the-effects-of-tildacerfont-and-hydrocortisone-in-adults-with-classic-congenital-adrenal-hyperplasia/ Wed, 29 Apr 2026 14:24:30 +0000 https://clinicaltrials.eu/trial/study-on-the-effects-of-tildacerfont-and-hydrocortisone-in-adults-with-classic-congenital-adrenal-hyperplasia/ This clinical trial is focused on studying a condition called (CAH), which is a genetic disorder affecting the adrenal glands. The trial will evaluate a new treatment using a medication called , also known by its code name . The purpose of the study is to assess how effective and safe Tildacerfont is for adults with CAH who have high levels of certain hormones in their blood.

Participants in the study will receive treatment over a period of up to 70 weeks. The study is designed to be “double-blind,” meaning neither the participants nor the researchers will know who is receiving Tildacerfont and who is receiving a placebo. This helps ensure the results are unbiased. The study will be conducted in three parts, with the main goal being to see if Tildacerfont can reduce levels of a hormone called androstenedione over 12 weeks.

In addition to Tildacerfont, some participants may continue their current treatment with , a medication commonly used to manage CAH. The trial will monitor changes in hormone levels and any side effects experienced by participants. The results will help determine if Tildacerfont is a beneficial treatment option for people with CAH.

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