Pharmacokinetics, Safety and Tolerability of Crinecerfont in Participants With Congenital Adrenal Hyperplasia Who Are Less Than 2 Years Old
This research study is for very young children, from birth up to two years old, who have a condition called Congenital Adrenal Hyperplasia (CAH). Doctors are testing a new medicine called crinecerfont. The main aim is to understand how this medicine moves through such young children's bodies and how long it stays there. They also want to make sure it's safe and that children can take it without too many problems. CAH is a condition where the body doesn't make enough of certain hormones, and current treatments often involve daily steroids. This new medicine hopes to offer a different approach. By studying it, researchers hope to improve treatment options for children with CAH in the future.
At a glance
What is this study about?
This study is focused on children under two years old who have Congenital Adrenal Hyperplasia (CAH). CAH is a condition that babies are born with where their bodies don't produce enough of important hormones, specifically affecting their adrenal glands. This can lead to various health problems, and children with CAH usually need to take medication, often steroids like hydrocortisone, for their whole lives.
The new medicine being tested in this study is called crinecerfont. The main goal is to understand how this medicine behaves inside the bodies of very young children. This includes how much of the medicine is absorbed, how it's processed, and how quickly it leaves the body. This information is really important because children's bodies are different from adults', and medicines can work differently in them.
Researchers also want to check how safe crinecerfont is for these young children and see if they can take it without experiencing too many side effects. By carefully studying this, they hope to gather important information that could lead to new or improved treatments for CAH in the future, potentially making life better for children with this condition. This is a 'Phase 2' study, which means it's an early stage of testing in a small group of people to see if the medicine is safe and effective.
Key takeaways
- New medicine called crinecerfont is being tested for CAH.
- Study is for babies and toddlers under two years old.
- Aims to check safety and how the medicine works in young bodies.
- Could help improve future treatments for CAH.
- Participation involves taking the study drug and regular check-ups.
Who may be eligible?
This study is looking for babies and toddlers who are less than two years old. They can be either boys or girls. To join, they must have a confirmed diagnosis of a specific type of Congenital Adrenal Hyperplasia (CAH) called 'classic CAH' due to a problem with an enzyme called 21-hydroxylase. This diagnosis needs to be officially confirmed by a doctor.
Your child also needs to be on a steady and stable treatment plan with hydrocortisone (and possibly fludrocortisone) for their CAH. This means their current medication dose shouldn't be changing often and should be working well for them already.
Children cannot join if they have another type of CAH, or if they need to take daily steroid medicine for any other health problem besides CAH. They also can't take part if they have any other serious ongoing health issues or illnesses.
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.
- Is my child under 2 years old?
- Does my child have a confirmed diagnosis of classic CAH due to 21-hydroxylase deficiency?
- Is my child currently on a stable treatment of hydrocortisone (and possibly fludrocortisone) for CAH?
- Does my child NOT have any other serious ongoing health conditions requiring daily medicine?
What does participation involve?
Taking part in this study would involve your child receiving the new medicine, crinecerfont. The study team will need to take blood samples from your child at different times to see how the medicine is working in their body. You would also have regular visits to the clinic so the doctors can monitor your child's health closely and check for any effects of the medicine. The full duration of how long your child would be involved in the study is not specified in the brief summary, but clinical trials typically involve several visits over a period of weeks or months, followed by follow-up checks.
Potential risks and benefits
Locations (3)
- Neurocrine Clinical SiteVerified postcodeBerlin, Germany· Recruiting
- Neurocrine Clinical SiteVerified postcodeDüsseldorf, Germany· Recruiting
- Neurocrine Clinical SiteVerified postcodeHeidelberg, Germany· Recruiting
Common questions
What is Congenital Adrenal Hyperplasia (CAH)?
It's a condition babies are born with where their bodies don't make enough of certain important hormones because of a problem with their adrenal glands.
What is crinecerfont?
It's a new medicine being tested to see if it can help treat Congenital Adrenal Hyperplasia in young children.
Why are they studying children under two?
Researchers want to understand how the medicine works in very young children's bodies, as their systems are different from adults.
Will my child still take their usual CAH medicine?
The study mentions children need to be on a stable dose of hydrocortisone (and possibly fludrocortisone) already, so they would likely continue this alongside the study medicine, but this would be discussed in detail.
Is this a new treatment for CAH?
Yes, crinecerfont is a new potential treatment being investigated. This study is an early step in understanding if it's safe and effective.
How to find out more
Neurocrine Medical Information Call Center
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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