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

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What is this study about?

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.

1 joining the study

Upon joining the study, participants will be required to provide signed informed consent. This consent confirms understanding and agreement to comply with the study’s requirements and restrictions.

Participants must have completed a previous study involving the medication CRN04894 and be assessed by the investigator as having an acceptable benefit-risk profile.

2 medication administration

Participants will receive the medication CRN04894 in the form of a tablet. The medication is taken orally.

The dosage will not exceed the maximum dose used in the previous study. The exact dosage and frequency will be determined by the study protocol and the investigator.

3 ongoing treatment

Participants must maintain a stable regimen of glucocorticoid replacement therapy, such as hydrocortisone or its equivalents, as determined by the investigator.

The minimum total daily dose of glucocorticoid must be equivalent to at least a specified amount of hydrocortisone.

4 safety and efficacy assessments

The study will evaluate the safety and tolerability of CRN04894 by monitoring any adverse events or side effects that occur during the trial.

Efficacy will be assessed by measuring changes in specific hormone levels, such as serum androstenedione, from the baseline over time.

5 completion of the study

The study is expected to continue until November 30, 2027. Participants will be required to follow the study protocol until the end of the study or until they are advised otherwise by the investigator.

Participants will be monitored for any changes in their condition and any necessary adjustments to their treatment will be made by the study team.

Who Can Join the Study?

Participants must have Congenital Adrenal Hyperplasia (CAH) and have completed a previous study with the study drug CRN04894, with a positive assessment from the study doctor.
Participants must be following a stable treatment plan with glucocorticoid replacement (a type of medication that replaces hormones) and taking a minimum daily dose of hydrocortisone or its equivalent.
Female participants who have heterosexual intercourse must either be unable to have children (due to surgery or menopause) or agree to use a highly effective method of birth control during the study and for at least 2 weeks after the last dose of the study drug.
Male participants must agree to use a condom during sexual activity with a female partner who can have children, or be surgically sterile, or agree to remain abstinent. They should also agree not to donate sperm during the study and for at least 2 weeks after the last dose of the study drug.
Participants must be willing and able to provide signed consent to participate in the study, understanding all the requirements and restrictions.
Participants must be willing and able to follow all study procedures and take the study treatment as directed.

Who Cannot Join the Study?

Patients with any other serious health conditions that might affect their safety or the results of the study.
Patients who are pregnant or breastfeeding.
Patients who have participated in another clinical trial within the last 30 days.
Patients who have a history of allergic reactions to similar medications.
Patients who are unable to comply with the study procedures.
Patients with a history of drug or alcohol abuse.
Patients who are taking medications that might interfere with the study drug.
Patients with uncontrolled high blood pressure.
Patients with severe liver or kidney disease.
Patients with a history of certain heart conditions.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Azienda Ospedaliera Universitaria Federico II Di Napoli	Naples	Italy

Other Sites

Site Name	City	Country
Kwkzclys dft Uworkssogcpm Mwkyfwqw Alv	Munich	Germany
Ulabwmreix Dimns Scdvh Df Riwc Lk Sffrisyx	Rome	Italy
Iunhm Oczgeoxr Asdnopsrdv Sbg Lkmu	Milan	Italy

Trial status

Country	Status	Recruitment Start
Germany	Recruiting	28.02.2025
Italy	Recruiting	28.02.2025

Trial locations

Investigated drugs:

ATUMELNANT

CRN04894 is a medication being studied to see how safe and effective it is for people who are taking it over a long period. The main goal of this study is to check if CRN04894 can help reduce levels of a hormone called androstenedione in the blood. Androstenedione is a hormone that can affect various body functions, and the study aims to see if CRN04894 can help manage its levels safely. Participants in the study will be monitored to ensure that the medication is safe to use and to see how well it works in achieving its intended effects.

Congenital Adrenal Hyperplasia – Congenital Adrenal Hyperplasia is a group of genetic disorders affecting the adrenal glands, which are responsible for producing hormones like cortisol and aldosterone. In this condition, the adrenal glands produce an excess of androgen, a type of male sex hormone, and insufficient amounts of cortisol and sometimes aldosterone. This hormonal imbalance can lead to early signs of puberty in children and can affect growth and development. The condition is present from birth and can vary in severity. Over time, individuals may experience symptoms such as abnormal growth patterns, changes in skin pigmentation, and electrolyte imbalances. The progression of the disease can lead to complications if not managed properly.

Trial ID:

2024-514846-35-00

Protocol code:

CRN04894-09

Trial Phase:

Therapeutic exploratory (Phase II)

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

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?