A Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging Study to Evaluate the Efficacy and Safety of SPR001 (Tildacerfont) in Adult Subjects with Classic Congenital Adrenal Hyperplasia
This study is looking at a new medicine called Tildacerfont for adults with Classic Congenital Adrenal Hyperplasia (CAH). CAH is a lifelong condition that affects how the body makes important hormones. Researchers want to find out how well Tildacerfont works to lower levels of a specific hormone called androstenedione after 12 weeks of treatment. They also want to check if it's safe and if it can help with other hormone levels and a related issue called testicular adrenal rest tumors. This is an early-stage study, meaning it's exploring whether the new medicine has promise.
At a glance
What is this study about?
This research study is about a new medicine, Tildacerfont, for adults who have a medical condition called Classic Congenital Adrenal Hyperplasia (CAH). CAH is a condition you're born with that affects the adrenal glands, which are small glands above your kidneys. These glands normally make important hormones that help your body work properly. In CAH, these glands don't make enough of some hormones and might make too much of others.
The main goal of this study is to see if Tildacerfont can help reduce the levels of a hormone called androstenedione in people with CAH. High levels of this hormone can be a problem in CAH. The researchers will be comparing Tildacerfont to a 'dummy' medicine (placebo) and also to hydrocortisone, which is a standard treatment for CAH, to see which works best and if Tildacerfont offers any improvements.
Over 12 weeks of treatment, the study will carefully check how much the androstenedione levels change. They will also look at how many people's hormone levels become normal and if Tildacerfont affects certain growths in the testes that can sometimes happen with CAH. All of this information will help doctors understand if Tildacerfont could be a useful new treatment option for people living with Classic CAH.
Key takeaways
- This study is testing a new medicine (Tildacerfont) for adults with Classic CAH.
- It aims to see if Tildacerfont can lower certain hormone levels (androstenedione).
- Comparisons will be made against standard treatment (hydrocortisone) and a dummy pill.
- Both effectiveness and safety of Tildacerfont will be carefully checked.
- Participation involves regular visits and blood tests over approximately 18 weeks.
Who may be eligible?
To join this study, you need to be an adult, at least 18 years old. Both men and women can take part.
Because this is a specific study for Classic Congenital Adrenal Hyperplasia, you would need to have this diagnosed condition to be considered. The study will have other specific medical requirements, which the study doctors will check to make sure the treatment is safe and right for you.
It's important to talk to the study team or your doctor to find out if your individual health situation matches all the requirements for this particular research.
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?
- Have you been diagnosed with Classic Congenital Adrenal Hyperplasia?
- Are you able to attend regular clinic appointments?
- Are you willing to take study medication as instructed?
- Are you willing to have blood tests?
- Are you comfortable not knowing if you are receiving the active medicine or a dummy pill?
What does participation involve?
If you decide to take part in this study, you would be given either the new medicine (Tildacerfont), a standard treatment (Hydrocortisone), or a dummy pill (placebo) that looks just like the study medicine but has no active ingredients. You wouldn't know which one you are taking, and neither would your study doctor, to make sure the results are fair.
You would have regular visits to the clinic over a period of 18 weeks. These visits will involve blood tests to measure your hormone levels, particularly androstenedione, and possibly other checks depending on your individual health profile. The main treatment period is for 12 weeks, with follow-up appointments after that. The total duration including initial screening and follow-up would be explained to you by the study team.
Potential risks and benefits
Locations (12)
- —UnverifiedSweden
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- —UnverifiedIreland
- —UnverifiedItaly
- —UnverifiedRomania
- —UnverifiedEstonia
- —UnverifiedDenmark
- —UnverifiedLatvia
- —UnverifiedLithuania
- —UnverifiedSpain
- —UnverifiedGermany
- —UnverifiedPoland
Common questions
What is Classic Congenital Adrenal Hyperplasia (CAH)?
It's a condition you're born with that affects your adrenal glands, which don't make hormones correctly.
What is Tildacerfont?
It's a new medicine being tested to see if it can help manage hormone levels in adults with CAH.
Will I know if I'm getting the new medicine?
No, neither you nor the doctors will know if you're getting Tildacerfont, hydrocortisone, or a dummy pill. This helps keep the results accurate.
What does a 'Phase II' study mean?
It means this is an early study to explore if the new medicine works and is safe before it's tested more widely.
How long will I be involved in the study?
The main treatment lasts 12 weeks, and you'll have appointments over a total of about 18 weeks, which includes check-ups.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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