Active not recruitingPHASE3INTERVENTIONAL

Global Safety and Efficacy Registration Study of Crinecerfont for Congenital Adrenal Hyperplasia

This study is looking into a new medicine called crinecerfont for adults living with Congenital Adrenal Hyperplasia (CAH). CAH is a condition that affects the adrenal glands. Currently, people with CAH often take steroids, and this new medicine aims to help manage the condition differently. The study involves comparing crinecerfont with a 'placebo' – a dummy medicine that looks the same but contains no active drug. Participants won't know if they're receiving the new medicine or the placebo for the first 24 weeks. After this, everyone will receive crinecerfont for a year. Some people might then choose to continue on crinecerfont for even longer. The main goals are to check if crinecerfont is effective (works well) and safe to use. The study will last about 20 months for most people, with an option to continue for longer if participants wish.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Neurocrine Biosciences

Enrolment target

182

Start

16 Dec 2020

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

Percent Change From Baseline in Glucocorticoid Daily Dose at Week 24

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 focused on a health condition called Congenital Adrenal Hyperplasia, or CAH for short. CAH is a lifelong condition that affects the adrenal glands, which are small glands located on top of your kidneys. These glands usually make important hormones, but in CAH, they don't produce enough of certain hormones, and sometimes make too much of others. This can lead to a range of health issues.

Currently, people with CAH often need to take steroid medicines to manage their condition. While these can be very helpful, they can also have side effects over time. This new study is investigating a different type of medicine called crinecerfont. The hope is that crinecerfont could offer a new way to help people with CAH, potentially by addressing some of the underlying hormone imbalances in a different way than current treatments.

By carefully comparing crinecerfont to a dummy medicine (placebo), and then offering crinecerfont to all participants, researchers want to clearly understand if this new medicine is safe and effective in helping adults manage their CAH. This is a very important step in developing new treatments that could improve the health and quality of life for people living with CAH.

Key takeaways

The study is testing a new medicine, crinecerfont, for adults with Congenital Adrenal Hyperplasia (CAH).
It aims to see if crinecerfont is safe and effective compared to a dummy medicine.
Participation involves regular clinic visits, blood tests, and taking the study medicine.
The main study period is around 20 months, with an option for longer participation.
You can discuss any concerns or withdraw from the study at any time.
This study is for adults aged 18 and over.

Who may be eligible?

To join this study, you must be an adult aged 18 or older and have a confirmed diagnosis of a specific type of CAH called 'classic CAH due to 21-hydroxylase deficiency'. It's also important that your current steroid medicine dose has been stable for a while.

However, there are certain reasons why you might not be able to take part. For example, if you have other serious medical conditions like certain heart problems, another type of CAH, or a history of cancer that isn't completely cured. You also can't be pregnant or planning to become pregnant during the study, and if you could become pregnant, you'd need to use reliable contraception.

Other reasons for not being able to join include having recently had a lot of blood loss, a history of drug or alcohol abuse, or if you've recently taken part in another clinical trial with a different experimental medicine. The research team will carefully check all these points with you to make sure the study is right for you.

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 18 years old or older?
Do you have a medically confirmed diagnosis of 'classic CAH due to 21-hydroxylase deficiency'?
Has your current steroid medicine dose been stable recently?
Are you able to attend regular clinic appointments and follow instructions?
Are you not pregnant or planning to become pregnant during the study period?

Answer every question to see your result.

What does participation involve?

If you decide to take part, the study will last about 20 months, with an option to continue for approximately three more years if you and the study team agree. For the first 24 weeks (about 6 months), you will receive either crinecerfont or a placebo (dummy medicine) – you won't know which one you're getting. After this initial period, all participants will receive crinecerfont for one year.

Throughout the study, you will have regular visits to the clinic for check-ups, blood tests, and other assessments to see how you're responding to the treatment and to monitor your health. You'll need to follow all study instructions carefully, including how and when to take your study medicine. There will also be a follow-up visit after you stop taking the study medicine.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. You might benefit from receiving a new medicine that could help manage your CAH, and you'll receive careful medical monitoring throughout the study. However, there's also a chance you could receive the placebo at first, which means you wouldn't be getting the active medicine. There might be side effects from crinecerfont that we don't fully know about yet, or your condition might not improve. Taking part will also involve several clinic visits and tests. It's very important to remember that you have the right to leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (70)

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

Neurocrine Clinical Site
Verified postcode
Los Angeles, United States
Neurocrine Clinical Site
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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
Atlanta, United States
Neurocrine Clinical Site
Verified postcode
Indianapolis, United States
Neurocrine Clinical Site
Verified postcode
Bethesda, United States
Neurocrine Clinical Site
Verified postcode
Boston, United States
Neurocrine Clinical Site
Verified postcode
Ann Arbor, United States
Neurocrine Clinical Site
Verified postcode
Minneapolis, United States
Neurocrine Clinical Site
Verified postcode
Rochester, United States
Neurocrine Clinical Site
Verified postcode
St Louis, United States

Common questions

What is Congenital Adrenal Hyperplasia (CAH)?

CAH is a lifelong condition affecting the adrenal glands, which don't produce enough of certain important hormones.

What is crinecerfont?

Crinecerfont is a new, experimental medicine being tested to see if it can help manage CAH.

What is a 'placebo'?

A placebo is a dummy medicine that looks like the real thing but doesn't contain any active drug. It helps researchers compare the new medicine's effects accurately.

How long will I be in the study?

The main part of the study is about 20 months, with an option to continue for approximately three more years if you choose.

Do I have to stop my current CAH medicine?

No, you will stay on your stable steroid regimen. The study medicine is being tested alongside your current treatment.