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

Fast replyInvestigationalNo placebo
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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 a condition known as Classic Congenital Adrenal Hyperplasia (CAH), which is a genetic disorder affecting the adrenal glands. The study is evaluating a treatment called Crinecerfont, also known by its code name NBI-74788. Crinecerfont is being tested in two forms: an oral solution and a capsule. The purpose of the study is to assess the safety and effectiveness of Crinecerfont in reducing certain hormone levels in children with CAH.

Participants in the study will be randomly assigned to receive either Crinecerfont or a placebo, which is a substance with no active medication. The study will begin with a period where neither the participants nor the researchers know who is receiving the actual medication or the placebo. This is known as a double-blind study. After this initial phase, all participants will have the opportunity to receive Crinecerfont in an open-label phase, where everyone knows they are receiving the active treatment.

The study will last for up to 52 weeks, during which time participants will have regular visits to the study center to monitor their health and hormone levels. The main goal is to see if Crinecerfont can safely lower adrenal steroid levels while participants continue their usual treatment for CAH. This study is important for understanding how Crinecerfont can help manage CAH in children and improve their quality of life.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, the patient will be assessed to ensure they meet the necessary criteria, such as age, weight, and medical history related to classic congenital adrenal hyperplasia (CAH).

    The patient must be on a stable glucocorticoid treatment regimen and have elevated adrenal androgens.

  2. Step 2

    Randomized treatment phase

    The patient will be randomly assigned to receive either crinecerfont (NBI-74788) or a placebo. The medication will be administered orally, either as a solution or in capsule form.

    The primary goal during this phase is to evaluate the reduction in adrenal steroid levels while maintaining a stable glucocorticoid regimen.

  3. Step 3

    Evaluation at week 4

    At week 4, the patient's serum androstenedione levels will be measured to assess changes from the baseline.

    Additionally, serum 17-hydroxyprogesterone levels will be evaluated to determine any changes from the baseline.

  4. Step 4

    Open-label treatment phase

    Following the initial randomized phase, the patient will enter an open-label treatment phase where they will receive crinecerfont.

    This phase allows for continued monitoring and assessment of the medication's efficacy and safety.

  5. Step 5

    Evaluation at week 28

    At week 28, the patient's glucocorticoid dose will be reviewed to determine any percentage change from the baseline.

    This evaluation helps in understanding the long-term impact of crinecerfont on glucocorticoid requirements.

  6. Step 6

    Follow-up

    After completing the treatment phases, the patient will return for a follow-up visit to ensure their well-being and to gather additional data on the treatment's effects.

Who can join the trial?

8 criteria

  • Be willing and able to follow the study procedures, including all requirements at the study center and return for the follow-up visit.
  • Be a female or male between 2 to 17 years old with a body weight of at least 10 kg (about 22 pounds).
  • Have a medically confirmed diagnosis of classic Congenital Adrenal Hyperplasia (CAH) due to 21-hydroxylase deficiency. This means a specific genetic condition affecting the adrenal glands.
  • Be on a stable treatment plan with glucocorticoids for CAH. Glucocorticoids are a type of medication used to manage CAH.
  • Have high levels of adrenal androgens. These are hormones produced by the adrenal glands.
  • If taking fludrocortisone, the dose should be stable for at least 1 month before the screening. Fludrocortisone is a medication that helps manage certain hormone levels.
  • During screening, the plasma renin activity (PRA) should be less than 3 times the upper limit of normal (ULN) and more than the lower limit of normal (LLN) on the usual sodium intake. PRA is a measure of hormone activity related to blood pressure and fluid balance.
  • If PRA is more than 2 times ULN but less than 3 times ULN, the subject must have normal age-specific blood pressure, heart rate, and serum potassium levels.
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Investigated drugs

Crinecerfont (NBI-74788) is a medication being studied for its ability to help manage classic congenital adrenal hyperplasia in children. This condition affects the adrenal glands, which are responsible for producing important hormones. Crinecerfont is being tested to see if it can effectively reduce the levels of certain adrenal steroids, which are hormones produced by the adrenal glands. The study aims to determine if Crinecerfont can help maintain stable hormone levels when used alongside other treatments.

What is already known about the treatment

Crinecerfont – Crinecerfont is administered orally and is currently being studied in clinical trials for its potential use in treating Classic Congenital Adrenal Hyperplasia (CAH). It is not yet widely available in medical practice, as it is still under investigation to determine its safety and effectiveness. The main therapeutic indication for Crinecerfont is to help manage CAH by reducing adrenal steroid levels. At the molecular level, Crinecerfont works by modulating the activity of certain receptors involved in steroid production, aiming to balance hormone levels. It is classified pharmacologically as a corticotropin-releasing factor receptor antagonist.

Investigated diseases

Classic Congenital Adrenal Hyperplasia (CAH) – This is a genetic disorder affecting the adrenal glands, which are responsible for producing certain hormones. In CAH, the body lacks an enzyme needed to make cortisol, a hormone crucial for stress response and metabolism. As a result, the adrenal glands produce excess androgens, which are male sex hormones. This hormonal imbalance can lead to early signs of puberty in children and affect growth and development. The condition is present from birth and can vary in severity. It often requires lifelong management to maintain hormone levels within a normal range.
Trial detailsLast updated 2 Oct 2026
Age0-17PhasePhase IIITrial ID2023-509170-33-00Protocol codeNBI-74788-CAH2006Estimated enrolment93 patientsSponsorNeurocrine Biosciences Inc.

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