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

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What is this trial about?

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

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.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Joining the study
Participation begins after providing written informed consent. This consent is required from both the pregnant mother and the legal guardians of the unborn child.
Eligibility is determined by the presence of genetic mutations in both parents, indicating a risk of classic congenital adrenal hyperplasia (CAH) in the fetus. The gestational age must be less than 8 weeks post menstruation.
Step 2
Medication administration
The medication used in this trial is dexamethasone, administered orally in the form of hard capsules.
The available dosages are 0.5 mg, 0.21 mg, 0.41 mg, and 0.15 mg. The specific dosage and frequency will be determined by the study protocol.
Step 3
Monitoring and assessments
The primary focus is on preventing prenatal virilization in female fetuses with classic CAH and monitoring maternal weight gain during pregnancy.
Regular assessments will be conducted to evaluate the closure of the urogenital sinus (Prader stage) and gestational weight gain.
Step 4
Safety and outcome evaluations
Secondary evaluations include monitoring treatment safety for both mothers and children. This involves tracking any stillbirths, miscarriages, maternal side effects, and adverse events.
Additional assessments will consider maternal quality of life, mental health, sleep, and the child's birth weight, height, gestational age at birth, nervous system development, and any malformations.
Step 5
Completion of the trial
The trial is expected to conclude by January 1, 2035. Participants will be informed of the outcomes and any relevant findings related to the study objectives.

Who can join the trial?

3 criteria

Adult pregnant women who have a risk of their unborn child having **classic congenital adrenal hyperplasia (CAH)**. This means there are specific genetic changes in both parents.
The pregnant woman and the legal guardians of the unborn child must provide **written informed consent**. This means they agree to participate in the study after understanding all the details.
The pregnancy must be less than **8 weeks** from the last menstrual period.

Who cannot join the trial?

4 criteria

Individuals who do not have congenital adrenal hyperplasia. This is a genetic condition affecting the adrenal glands, which are small glands located on top of the kidneys.
Individuals who are not female. The study is only for females.
Individuals who are not part of the specified age range for the study. The study has specific age requirements.
Individuals who are considered part of a vulnerable population that the study does not include. This means people who might need special protection or care.

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Verified sites

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Charite Universitaetsmedizin Berlin KöR
Berlin, Germany
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Klinikum der Universitaet Muenchen AöR
Munich, Germany

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Investigated drugs

Dexamethasone is a medication being studied in this clinical trial. It is used to prevent prenatal virilization in females with classic congenital adrenal hyperplasia (CAH). The trial is investigating whether a reduced dose of dexamethasone is as effective as the higher dose that is currently used experimentally. The study also looks at how the medication affects maternal weight gain during pregnancy.

Dexamethasone

What is already known about the treatment

Dexamethasone – Dexamethasone is administered orally or via injection and is currently being studied in clinical trials for its effectiveness in treating congenital adrenal hyperplasia (CAH). It is a well-established medication in medical literature, primarily used for its anti-inflammatory and immunosuppressant properties. The main therapeutic indication in this trial is to prevent prenatal virilization in females with classic CAH. At the molecular level, dexamethasone works by binding to glucocorticoid receptors, influencing gene expression to reduce inflammation and suppress the immune response. It is classified pharmacologically as a corticosteroid.

Investigated diseases

Congenital adrenal hyperplasia – This is a genetic disorder affecting the adrenal glands, which are responsible for producing certain hormones. In this condition, the body lacks an enzyme needed to make hormones like cortisol and aldosterone, leading to an overproduction of male sex hormones. This hormonal imbalance can cause early or inappropriate development of male characteristics in both males and females. In females, it may lead to prenatal virilization, where the external genitalia develop in a more male-typical pattern. The condition can also result in issues with salt balance, blood pressure, and energy levels. Symptoms and severity can vary widely, depending on the specific genetic mutation and enzyme deficiency.