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 study about?

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.

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)

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

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

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

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 Study?

  • 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 Study?

  • 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

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Universitaetsklinikum Heidelberg AöR Heidelberg Germany

Other Sites

Site Name City Country Status
Charite Universitaetsmedizin Berlin KöR Berlin Germany
Upoxyfvedj Hlclaolz Ceentcu Cologne Germany
Uvvgjxulsfkpqaovatfdd Dhddixrvvvq Ahq Duesseldorf Germany

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Germany Germany
Recruiting
26.06.2025

Trial locations

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.

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.

Trial ID:
2024-514127-42-00
Protocol code:
NBI-74788-CAH2011
Trial Phase:
Therapeutic exploratory (Phase II)

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