A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of SPR001 (Tildacerfont) in Reducing Supraphysiologic Glucocorticoid Use in Adult Subjects with Classic Congenital Adrenal Hyperplasia
This study is clinical trial in its second phase, exploring a new medication known as Tildacerfont. It's for adults who have classic congenital adrenal hyperplasia, a lifelong condition affecting the adrenal glands. The main goal is to find out if Tildacerfont can help people reduce their daily dosage of steroid medicines like hydrocortisone or prednisolone, which are often used to manage their condition. Researchers will compare Tildacerfont to a dummy pill (placebo) to see if it works. They'll also look at whether the new medication is safe to use and if it can improve other health markers, like cardiovascular risk factors, over a 24-week period. This type of research is important for finding new ways to manage congenital adrenal hyperplasia.
At a glance
What is this study about?
This research study is about finding new ways to help adults living with a condition called classic congenital adrenal hyperplasia (CAH). CAH is a genetic condition that means the body doesn't produce enough of certain important hormones. To manage CAH, most people need to take steroid medications, like hydrocortisone or prednisolone, every day. While these medicines are vital, taking them long-term can sometimes lead to side effects or make it tricky for doctors to find the right dose.
The study is investigating a new medication, currently known as Tildacerfont. It's designed to see if it can help people with CAH reduce the amount of steroid medication they need to take. Imagine it like a helper medication that might allow your own body to manage things a little better, potentially meaning you need less of your usual steroid treatment.
Researchers want to carefully check if Tildacerfont is effective and safe. They will compare it to a 'dummy' pill (called a placebo) to make sure any improvements are really due to the new medicine. They will also be looking at whether Tildacerfont can positively affect other health measures, such as reducing the risk of heart and circulation problems, which can sometimes be a concern for people with long-term conditions like CAH.
Key takeaways
- This is a study for adults with classic congenital adrenal hyperplasia.
- It's testing a new medication called Tildacerfont.
- The main aim is to see if Tildacerfont can help reduce the amount of steroid medicine people need.
- The study lasts about 6 months, with regular clinic visits.
- You might receive the new medication or a 'dummy' pill.
- Your current steroid medication will continue, potentially with dose adjustments.
Who may be eligible?
This study is looking for adult participants, meaning you must be 18 years old or older to take part. There is no upper age limit, and both men and women are welcome to participate.
To be eligible, you must have a confirmed diagnosis of classic congenital adrenal hyperplasia. The study is specifically designed for people with this type of CAH who are currently taking steroid medications to manage their condition.
Other specific medical criteria, which your doctor would be able to explain, will also need to be met to ensure the study is safe and appropriate 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 diagnosis of classic congenital adrenal hyperplasia?
- Are you currently taking steroid medication (like hydrocortisone or prednisolone) for your CAH?
- Are you able to attend regular clinic appointments for about 6 months?
What does participation involve?
If you decide to take part in this study, you would be assigned by chance to either receive the new medication, Tildacerfont, or a 'dummy' pill (placebo). Neither you nor your study doctor would know which you are receiving, which helps us to truly understand the medication's effects. You would also continue to take your existing steroid medication, potentially with adjustments guided by the study team.
Your involvement would last for about 24 weeks, which is roughly six months. During this time, you would have regular visits to the clinic. At these visits, doctors and nurses will monitor your health carefully, take blood samples to check hormone levels, and assess how your steroid medication might be adjusted. They will also ask you about your general well-being and any side effects you might experience. These assessments are important to track how the medication is working and to ensure your safety throughout the study.
Potential risks and benefits
Locations (12)
- —UnverifiedSweden
- —UnverifiedNetherlands
- —UnverifiedDenmark
- —UnverifiedRomania
- —UnverifiedIreland
- —UnverifiedLatvia
- —UnverifiedEstonia
- —UnverifiedGermany
- —UnverifiedSpain
- —UnverifiedItaly
- —UnverifiedLithuania
- —UnverifiedPoland
Common questions
What is 'classic congenital adrenal hyperplasia'?
It's a lifelong genetic condition where your body doesn't produce enough of certain hormones, requiring you to take steroid medication.
What is Tildacerfont?
It's a new medication being tested to see if it can help people with CAH reduce their steroid medication use.
What does 'double-blind' mean?
It means neither you nor your study doctor will know whether you are receiving the active medication or a dummy pill (placebo), to ensure accurate results.
How long does the study last?
Your active participation in the study would last for approximately 24 weeks, which is about six months.
Will I have to stop my current medication if I join?
No, you will continue to take your current steroid medication, though your dose might be adjusted by the study team as part of the trial.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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