RecruitingRare disease

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

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What is this trial about?

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

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.

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
Initial evaluation
Your medical condition (congenital adrenal hyperplasia or CAH) will be confirmed through medical records and tests
Your current treatment with hydrocortisone (and fludrocortisone if applicable) will be reviewed to ensure it's stable
Blood tests will be performed to check hormone levels, particularly 17-OHP (a hormone that is elevated in CAH)
Step 2
Treatment period start
You will begin taking crinecerfont (also known as NBI-74788) as an oral solution
The treatment period will last for 14 days
You will continue your regular hydrocortisone treatment during this period
Step 3
Day 7 evaluation
Blood samples will be collected to measure the amount of crinecerfont in your blood
Your health status will be monitored for any changes or side effects
Step 4
Day 15 evaluation
Additional blood samples will be collected to measure crinecerfont levels
The medical team will assess any side effects that may have occurred during the treatment
Final health evaluation will be performed
Step 5
Safety monitoring
Throughout the study, you will be monitored for any side effects or unexpected reactions
Any changes in your health status will be recorded and evaluated

Who can join the trial?

9 criteria

Parent(s) or legal guardian must provide informed consent for the child to participate in the study
Child must be between 0 and 2 years of age at screening
Child must weigh at least 3.0 kilograms at screening
Child must have a confirmed diagnosis of classic CAH (a genetic condition affecting hormone production in the adrenal glands) with elevated 17-OHP (a hormone level)
Child must have a 17-OHP level that is more than twice the normal upper limit, measured before morning medication
Child must have had a normal newborn screening test, except for elevated 17-OHP, or any other abnormality that was cleared by a pediatric specialist
Child must be on a stable treatment plan with hydrocortisone (a steroid medication) and if applicable, fludrocortisone (a medication that helps regulate salt balance)
Child must have normal levels of plasma renin activity (PRA) - a measure of hormone activity that affects blood pressure
Parents or legal guardians must be willing to follow all study procedures

Who cannot join the trial?

10 criteria

Age above 2 years old
Not diagnosed with Congenital Adrenal Hyperplasia (CAH) - a genetic condition affecting hormone production in the adrenal glands
Known allergies or hypersensitivity to crinecerfont or similar medications
Participation in another clinical trial within the past 30 days
Any serious medical condition that could interfere with the study procedures
Unable to comply with study procedures or follow-up visits
Currently taking medications that could interact with the study drug
History of significant kidney or liver problems
Unable to provide informed consent (for parents/guardians)
Any condition that would make it unsafe to participate in the study according to the study doctor's assessment

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

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Charite Universitaetsmedizin Berlin KöR
Berlin, Germany
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Universitaetsklinikum Duesseldorf AöR
Duesseldorf, Germany
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Universitaetsklinikum Heidelberg AöR
Heidelberg, Germany

Trial locations

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Investigated drugs

Crinecerfont is an investigational medication being studied for the treatment of Congenital Adrenal Hyperplasia (CAH) in very young children. It is designed to help regulate hormone production in children with CAH. This medication aims to provide a new treatment option for managing this inherited condition that affects the body's ability to produce certain hormones. The medication is being tested to understand how it works in the bodies of infants and toddlers under 2 years of age, how safe it is, and how well these young patients tolerate it.

Crinecerfont

What is already known about the treatment

Crinecerfont - An investigational drug administered orally that is currently undergoing Phase 2 clinical trials for the treatment of Congenital Adrenal Hyperplasia (CAH) in pediatric patients aged 0 to 2 years. The medication belongs to the class of corticotropin-releasing factor type 1 (CRF1) receptor antagonists, working by reducing the excessive production of adrenocorticotropic hormone (ACTH) in patients with CAH. This novel therapeutic approach aims to address the underlying hormonal imbalances characteristic of CAH, potentially offering a new treatment option for very young patients with this rare genetic condition.

Investigated diseases

Congenital Adrenal Hyperplasia (CAH) - A genetic condition that affects the body's ability to produce essential hormones in the adrenal glands. The condition is present from birth and causes the body to produce insufficient amounts of cortisol and aldosterone while producing excess androgens (male hormones). CAH affects various body processes, including how the body regulates blood sugar, responds to stress, and maintains proper salt and water balance. The condition can affect both males and females, impacting growth and development. In its most common form, it causes abnormal development of reproductive organs and early puberty signs.