Active not recruitingPHASE3INTERVENTIONAL

Global Safety and Efficacy Registration Study of Crinecerfont in Pediatric Participants With Classic Congenital Adrenal Hyperplasia (CAHtalyst Pediatric Study)

This important study, called CAHtalyst Pediatric Study, is looking into a new medicine called crinecerfont for children and teenagers (aged 2 to 17) who have a condition called classic Congenital Adrenal Hyperplasia (CAH) due to 21-hydroxylase deficiency. The main goal is to see if crinecerfont is effective and safe compared to a placebo (a dummy pill). Participants will take either crinecerfont or the placebo for 28 weeks. After this, all participants will receive crinecerfont for 24 weeks. There's also an option to continue with the crinecerfont treatment for a longer period if participants wish. The study helps researchers understand if this new medicine could be a better way to manage CAH.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Neurocrine Biosciences

Enrolment target

103

Start

25 Jun 2021

Estimated completion

01 Aug 2027

Congenital Adrenal Hyperplasia

Results

Results from this study

Posted February 2025

Results have been published for this study.

Primary outcome

Change From Baseline in Serum Androstenedione at Week 4

Blood serum samples were collected for the analysis of serum androstenedione concentrations. Least square (LS) mean and standard error (SE) were calculated using analysis of covariance (ANCOVA) model.

Full results on the registry

What is this study about?

This study is about a health condition called Congenital Adrenal Hyperplasia (CAH). CAH is a genetic condition that affects the adrenal glands, which are small glands located above your kidneys. These glands produce important hormones your body needs. In classic CAH due to 21-hydroxylase deficiency, the body doesn't make enough of certain hormones, like cortisol, and might make too much of others, like androgens. This can cause various health problems.

The purpose of this research is to test a new medicine called crinecerfont. Doctors want to find out if crinecerfont can help manage CAH symptoms more effectively and safely than current treatments. They will compare it to a placebo, which looks like the study medicine but contains no active drug, to see if the new medicine has a real effect. This is a "Phase 3" study, which means it's one of the final stages of testing before a medicine might be approved for wider use.

Over 80 children and teenagers with classic CAH will take part. By carefully studying how crinecerfont works, researchers hope to offer new and potentially better treatment options for young people living with this condition. Your participation could help improve the lives of many others with CAH in the future.

Key takeaways

Tests a new medicine, crinecerfont, for children and teenagers with classic CAH.
Compares crinecerfont to a placebo (dummy pill) to check effectiveness and safety.
Participation involves regular check-ups and taking study medicine for over a year.
Aims to find better ways to manage hormone imbalances in CAH.
Offers a chance to contribute to important research and potentially receive a new treatment.
You can stop participating at any time without affecting your medical care.

Who may be eligible?

To join this study, participants must be between 2 and 17 years old and have a confirmed diagnosis of classic Congenital Adrenal Hyperplasia (CAH) due to 21-hydroxylase deficiency. They should also be on a stable dose of their usual steroid medicine and have higher-than-normal levels of certain hormones called androgens.

There are some reasons why someone might not be able to join. For example, if they have other forms of CAH, other serious health conditions that are not under control, a history of certain cancers (unless fully cured), or specific heart problems. If a participant is of childbearing age, they must agree to use suitable birth control during the study.

Also, participants shouldn't have taken part in another drug study recently or be planning to use other experimental drugs during this study. These rules are in place to help ensure the safety of participants and the accuracy of the study results.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you between 2 and 17 years old?
Do you have a diagnosis of classic CAH due to 21-hydroxylase deficiency?
Are you currently on a stable dose of your steroid medicine?
Do you have elevated androgen hormone levels?
Are you able to attend regular study visits?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you will be in the study for about 14 months initially, with an option to continue for much longer. For the first 28 weeks, you will take either the study medicine, crinecerfont, or a placebo (a dummy pill), though you won't know which one you're receiving – this is decided randomly. After these 28 weeks, all participants will receive crinecerfont for another 24 weeks. Following this, you may choose to continue taking crinecerfont in a longer-term part of the study, which could last about 3 years or more.

Taking part will involve regular visits to a study centre for check-ups. These visits will include various assessments like blood tests to measure hormone levels, physical examinations, and possibly tests like an electrocardiogram (ECG) to check heart health. You will also need to follow specific study instructions and keep track of your medication. The research team will explain everything in detail and be available to answer any questions you have throughout the study.

Potential risks and benefits

Participating in this study might offer potential benefits, such as receiving a new medicine that could help manage your CAH, and you would be contributing to valuable research that could help others. However, like all medicines, crinecerfont might have side effects, and some tests involve minor discomfort, such as blood draws. The placebo also means there's a chance you might not be receiving the active study medicine for a period. The research team will carefully monitor your health and well-being throughout the study. Remember, your participation is completely voluntary, and you have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (46)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Neurocrine Clinical Site
Verified postcode
Birmingham, United States
Neurocrine Clinical Site
Verified postcode
Los Angeles, United States
Neurocrine Clinical Site
Verified postcode
Orange, United States
Neurocrine Clinical Site
Verified postcode
San Diego, United States
Neurocrine Clinical Site
Verified postcode
San Francisco, United States
Neurocrine Clinical Site
Verified postcode
Aurora, United States
Neurocrine Clinical Site
Verified postcode
Hartford, United States
Neurocrine Clinical Site
Verified postcode
Washington D.C., United States
Neurocrine Clinical Site
Verified postcode
Atlanta, United States
Neurocrine Clinical Site
Verified postcode
Indianapolis, United States
Neurocrine Clinical Site
Verified postcode
Boston, United States
Neurocrine Clinical Site
Verified postcode
Ann Arbor, United States

Common questions

What is Congenital Adrenal Hyperplasia (CAH)?

CAH is a genetic condition that affects the adrenal glands, leading to an imbalance in the hormones your body needs, particularly a lack of cortisol and sometimes too much of other hormones like androgens.

What is crinecerfont?

Crinecerfont is a new medicine being tested to see if it can help manage CAH more effectively by helping to balance hormone levels in the body.

What is a placebo?

A placebo is a 'dummy' treatment that looks just like the real medicine but contains no active drug. It helps researchers understand if the new medicine has a real effect beyond expectations.

How long will I be in the study?

The main part of the study lasts about 14 months, but there's an option to continue with the study medicine for a longer period, possibly around 3 years.

Can I stop participating if I want to?

Yes, your participation is completely voluntary, and you can withdraw from the study at any time without having to give a reason, and it won't affect your regular medical care.