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Ongoing Clinical Trials for Adrenogenital Syndrome

There are currently 8 clinical trials investigating new treatments for Adrenogenital Syndrome, a genetic disorder affecting the adrenal glands’ hormone production. These studies are testing medications aimed at reducing the need for traditional steroid treatments and better managing hormone imbalances. (Also known as: Congenital Adrenal Hyperplasia, Classic Congenital Adrenal Hyperplasia, CAH)

Clinical trial locations

Study on the Effectiveness of Dexamethasone in Preventing Prenatal Virilization in Female Patients with Congenital Adrenal Hyperplasia

This study is focused on pregnant women who are at risk of having a baby with the condition. It is testing whether a lower dose of a medication called dexamethasone can work as well as the higher dose currently used to prevent prenatal virilization in female babies. Virilization refers to the development of male physical characteristics before birth.

Main inclusion criteria: Adult pregnant women whose unborn child is at risk of classic congenital adrenal hyperplasia, confirmed by genetic changes in both parents. The pregnancy must be less than 8 weeks from the last menstrual period. Both the pregnant woman and the legal guardians of the unborn child must provide written informed consent.

Main exclusion criteria: The study excludes individuals who do not have the condition, those who are not female, people outside the specified age range, and vulnerable populations not included in the study design.

Main focus: The trial aims to determine if a reduced dose of dexamethasone is as effective as higher doses in preventing prenatal virilization in female babies with the condition. The study will also monitor maternal weight gain during pregnancy and assess safety outcomes such as miscarriages, stillbirths, and any side effects in both mothers and children.

Investigational drug: Dexamethasone, a glucocorticoid medication, is administered orally in hard capsules. The trial compares different doses including 0.5mg, 0.21mg, 0.41mg, and 0.15mg over a period of up to 40 days.

Study on the Safety and Effectiveness of CRN04894 for Patients with Congenital Adrenal Hyperplasia

This study is designed for people who have already participated in previous studies involving the medication CRN04894. It aims to evaluate the long-term safety and effectiveness of this treatment in managing the condition.

Main inclusion criteria: Participants must have completed a previous study involving CRN04894 and been assessed as having an acceptable benefit-risk profile. They must be on a stable regimen of glucocorticoid replacement therapy such as hydrocortisone. Female participants of childbearing potential must agree to use highly effective birth control, and male participants must use condoms during the study and for 2 weeks afterward.

Main exclusion criteria: The study excludes people with other serious health conditions, those who are pregnant or breastfeeding, people who have participated in another clinical trial within 30 days, those with a history of allergic reactions to similar medications, and people unable to comply with study procedures.

Main focus: The study monitors the long-term safety and tolerability of CRN04894 while measuring its effectiveness in reducing levels of androstenedione, a hormone often elevated in people with this condition.

Investigational drug: CRN04894 is taken as an oral tablet. The dosage will not exceed the maximum dose used in the participant’s previous study. The exact dosage and frequency are determined by the study protocol.

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 trial is testing a medication called crinecerfont in very young children under 2 years of age with the condition. The purpose is to understand how the medication works in their bodies and how safe it is for this age group.

Main inclusion criteria: Children between 0 and 2 years of age weighing at least 3.0 kilograms, with a confirmed diagnosis of classic congenital adrenal hyperplasia and elevated hormone levels. The child must be on a stable treatment plan with hydrocortisone and, if applicable, fludrocortisone. Normal plasma renin activity levels are required. Parents or legal guardians must provide informed consent and be willing to follow all study procedures.

Main exclusion criteria: Children above 2 years old, those not diagnosed with the condition, children with known allergies to crinecerfont or similar medications, those who have participated in another clinical trial within 30 days, children with serious medical conditions that could interfere with the study, and those with significant kidney or liver problems.

Main focus: The trial evaluates the safety and tolerability of crinecerfont in infants and toddlers by measuring how the medication moves through their bodies. Researchers will monitor hormone levels and any side effects that occur during the 14-day treatment period.

Investigational drug: Crinecerfont is given as an oral solution (liquid medicine taken by mouth). Children will receive the medication for 14 days while continuing their regular hormone replacement treatment.

Study on the Effects of Tildacerfont and Hydrocortisone in Adults with Classic Congenital Adrenal Hyperplasia

This double-blind trial evaluates a new treatment called tildacerfont for adults with the condition who have high levels of certain hormones in their blood. The study aims to determine if this medication can reduce hormone levels and provide a beneficial treatment option.

Main inclusion criteria: Participants must be 18 years or older with a known childhood diagnosis of classic congenital adrenal hyperplasia confirmed by genetic testing or past medical records. They must be on a stable dose of hydrocortisone or similar medications for at least one month before joining the study. Hormone levels of androstenedione must be higher than normal. If participants have the salt-wasting form of the condition, they must be on a stable dose of specific medication for at least one month.

Main exclusion criteria: Patients without the condition, those outside the specified age range, people not part of the specified clinical trial groups, and those not meeting gender criteria.

Main focus: The study assesses how effective and safe tildacerfont is for adults with the condition over a period of up to 70 weeks. The main goal is to see if the medication can reduce levels of androstenedione over 12 weeks. The trial also monitors changes in testicular adrenal rest tumors, if applicable, and evaluates the proportion of participants who achieve normal hormone levels.

Investigational drug: Tildacerfont is administered orally in tablet form. Some participants may continue their current treatment with hydrocortisone alongside the study medication. The dosage and frequency are determined by the study protocol.

Study on the Safety and Effectiveness of Crinecerfont for Adults with Classic Congenital Adrenal Hyperplasia

This trial tests a new treatment called crinecerfont compared to a placebo in adults with classic congenital adrenal hyperplasia. The study examines whether this medication can reduce the need for glucocorticoids, which are commonly used steroid medications for managing this condition.

Main inclusion criteria: Female or male participants at least 18 years of age with a medically confirmed diagnosis of classic 21-hydroxylase deficiency. They must be on a stable, higher-than-normal dose of glucocorticoid medication that has been stable for at least one month before screening. If treated with fludrocortisone, the dose must also be stable for at least one month. Participants must agree to use contraception consistently throughout the study.

Main exclusion criteria: Patients not diagnosed with the condition, those unable to reduce their daily glucocorticoid dosage while maintaining control of adrenal androgens, patients who cannot reduce adrenal steroid levels after an initial 4-week treatment period, and those who have not reduced their glucocorticoid dose by Month 12.

Main focus: The study evaluates how well crinecerfont works in reducing the need for glucocorticoid medications over time. It monitors changes in hormone levels, particularly androstenedione, and assesses overall safety. The primary endpoint is the percent change from baseline in glucocorticoid daily dose at Week 24. Secondary endpoints include changes in weight and fat mass.

Investigational drug: Crinecerfont (NBI-74788) is taken in capsule form, 100mg twice daily. The study begins with a randomized, double-blind phase followed by an open-label phase where all participants receive the medication.

Study on the Safety and Effectiveness of Crinecerfont for Children with Classic Congenital Adrenal Hyperplasia

This trial is testing crinecerfont in children aged 2 to 17 years with classic congenital adrenal hyperplasia. The medication is available in two forms: an oral solution and a capsule. The study aims to assess the safety and effectiveness of reducing certain hormone levels in children with this condition.

Main inclusion criteria: Children between 2 to 17 years old with a body weight of at least 10 kg (about 22 pounds), with a medically confirmed diagnosis of classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency. They must be on a stable treatment plan with glucocorticoids and have high levels of adrenal androgens. If taking fludrocortisone, the dose must be stable for at least one month. Plasma renin activity must be within specified ranges during screening.

Main exclusion criteria: The trial excludes children outside the specified age range or who do not meet the weight requirements.

Main focus: The study evaluates whether crinecerfont can safely lower adrenal steroid levels while participants continue their usual treatment. The trial lasts up to 52 weeks, during which participants have regular visits to monitor health and hormone levels. At week 4, serum androstenedione and 17-hydroxyprogesterone levels are measured. At week 28, glucocorticoid dose is reviewed to determine percentage change from baseline.

Investigational drug: Crinecerfont (NBI-74788) is administered orally, either as a solution or in capsule form. Participants are randomly assigned to receive either crinecerfont or a placebo during an initial phase, followed by an open-label phase where everyone receives the active medication.

Study on the Safety and Effectiveness of CRN04894 for Patients with Congenital Adrenal Hyperplasia

This trial evaluates a new medication called CRN04894 for adults with congenital adrenal hyperplasia. The medication works by blocking a specific receptor involved in hormone production, and the study aims to assess its safety and effectiveness.

Main inclusion criteria: Participants must be male or female between 18 to 75 years old (16 years old in the United States) with classic 21-hydroxylase deficiency confirmed by a doctor and approved by a medical expert. They must be on a stable treatment plan for replacing glucocorticoids with no dose changes of more than 5mg per day of hydrocortisone in the last 6 months. Participants must follow their hormone replacement treatment plan and take at least 15mg of hydrocortisone daily. If on estrogen therapy, the dose must not have changed for at least 3 months.

Main exclusion criteria: Individuals not diagnosed with the condition, those outside the specified age range, people not part of the specified clinical trial groups, and those not considered part of the vulnerable population selected for the study.

Main focus: The study monitors safety, tolerability, and efficacy of CRN04894 over 12 weeks. The treatment is given in a sequential dose manner, with the dose potentially adjusted as the study progresses. The trial monitors levels of certain hormones in the blood, such as androstenedione, to determine treatment effectiveness.

Investigational drug: CRN04894 is administered orally for 12 weeks. Some participants may receive a placebo to compare the effects of the treatment. Other medications used in the study may include hydrocortisone and related glucocorticoids.

Study on Tildacerfont to Reduce Glucocorticoid Use in Adults with Classic Congenital Adrenal Hyperplasia

This double-blind trial evaluates the effectiveness and safety of tildacerfont (SPR001) in reducing the use of glucocorticoids in adults with classic congenital adrenal hyperplasia. Participants are currently taking higher-than-normal doses of glucocorticoids to manage their symptoms.

Main inclusion criteria: Participants must be 18 years or older with a documented diagnosis of classic congenital adrenal hyperplasia due to a specific enzyme deficiency, confirmed by genetic testing or specific hormone levels. They must be currently treated with medications like hydrocortisone or prednisone. Hormone levels, specifically androstenedione, must be within a certain range at the start of the study. Participants must have been on a stable dose of glucocorticoid replacement for at least one month. If they have the salt-wasting form, they must have been on a stable dose of mineralocorticoid replacement for at least one month.

Main exclusion criteria: Patients without the condition, those outside the specified age range, people not willing to follow study procedures, pregnant or breastfeeding women, those with other serious health conditions, people taking medications that might interfere with the study, recent participants in another clinical trial, those with a history of drug or alcohol abuse, and people with allergies to the study medication.

Main focus: The trial monitors changes in the amount of glucocorticoids needed by participants over 24 weeks. The goal is to see if tildacerfont can help reduce the need for these medications, potentially leading to better management of the condition. The study also assesses improvements in cardiovascular risk factors and other health indicators.

Investigational drug: Tildacerfont is taken orally in tablet form. The study compares it to a placebo to ensure unbiased results. Participants may also continue taking prednisolone and hydrocortisone as part of their regular treatment regimen.

Summary

The 8 ongoing clinical trials for Adrenogenital Syndrome demonstrate a concentrated research effort across multiple European countries, with Germany hosting the most trials. The studies focus primarily on three main investigational drugs: crinecerfont, tildacerfont, and CRN04894, all designed to reduce the need for traditional glucocorticoid therapy while better managing hormone imbalances.

A notable pattern emerges in the age-specific approaches, with separate trials targeting different populations: adults, children aged 2-17, and very young children under 2 years old. One unique trial focuses on prenatal treatment to prevent virilization in female babies at risk. Most trials are double-blind and placebo-controlled, lasting between 4 weeks and 70 weeks, with primary endpoints focusing on hormone level reduction and decreased glucocorticoid requirements.

The geographic distribution shows strong participation from Germany, Italy, Spain, and Poland, with trials also available in Belgium, France, Netherlands, Sweden, and several Eastern European countries. This broad distribution provides better access for patients across Europe seeking alternative treatment options for managing this genetic condition.

Ongoing Clinical Trials on Adrenogenital syndrome

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