A Phase 2 / Phase 3, Multicenter, Randomized, Multiple-Dose, Double-Blind, Placebo-Controlled Adaptive Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of CSL889 in Adults and Adolescents with Sickle Cell Disease during Vaso-Occlusive Crisis
This research study is about a new medicine, CSL889, for adults and teenagers experiencing a sickle cell pain crisis, also known as a vaso-occlusive crisis. We want to find out if CSL889 is safe, if it helps to ease the pain more quickly, and how the body processes the medication. Some participants will receive CSL889, while others will get a placebo (a treatment with no active medicine) for comparison. The study will look at how long it takes for pain to go away and if there are any side effects. Our goal is to see if CSL889 could be a helpful new treatment option for people suffering during these painful episodes.
At a glance
What is this study about?
This study is investigating a new medicine called CSL889 for a specific problem that people with sickle cell disease can experience: a vaso-occlusive crisis, often called a sickle cell pain crisis. These crises happen when sickle-shaped red blood cells block small blood vessels, causing severe pain and sometimes leading to serious health issues. Currently, treatments for these painful episodes mostly focus on managing the pain.
Researchers want to see if CSL889 can help stop these painful crises more quickly and effectively. To do this, they will compare CSL889 to a 'placebo.' A placebo looks just like the real medicine but contains no active ingredients. This helps researchers understand if any improvements are truly due to CSL889 or if they might happen naturally or from other care.
The main things the study will look at are how quickly pain goes away (specifically, how long until someone no longer needs strong pain relief given by injection or drip) and if participants experience any side effects. They will also check how the body handles CSL889. By doing this research, scientists hope to learn if CSL889 could become a new, better way to treat sickle cell pain crises.
Key takeaways
- This study is for adults with a sickle cell pain crisis (VOC).
- It tests a new medicine, CSL889, against a dummy treatment (placebo).
- It aims to see if CSL889 is safe and helps reduce pain faster.
- Participants will receive the medicine as a drip until their pain crisis improves.
- Close monitoring for side effects and how the body uses the medicine is part of the study.
- Your decision to join or leave the study won't affect your regular medical care.
Who may be eligible?
This study is looking for adults (age 18 and over) who are currently experiencing a painful sickle cell crisis, also known as a vaso-occlusive crisis. Both men and women are welcome to take part in this research.
There will be other specific health requirements that you would need to meet to ensure the study is safe for you and that the results are clear. These details would be discussed with you by the study team.
It's important that you or your main doctor discuss your full medical history with the research team to make sure this study is the right fit 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?
- Are you currently having a sickle cell pain crisis (vaso-occlusive crisis)?
- Are you able to attend clinic visits and assessments as required?
- Are you willing to potentially receive a placebo (dummy treatment)?
What does participation involve?
If you decide to take part, you would receive either the new medicine, CSL889, or a placebo, given as a drip into your arm (infusion). You wouldn't know which one you are getting, and neither would your study doctor. This is called a 'double-blind' study.
The study would involve regular check-ups and assessments while you are experiencing the crisis and afterward. These would include pain assessments, monitoring for any side effects, and blood tests to see how the study medicine is working in your body. We would keep a close eye on your hospital stays, any follow-up visits, and your general health. The total time you'd be involved in the study would depend on how your crisis develops and your recovery.
Potential risks and benefits
Locations (7)
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- —UnverifiedGreece
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- —UnverifiedBelgium
- —UnverifiedGermany
- —UnverifiedFrance
Common questions
What is a 'vaso-occlusive crisis'?
It's a very painful episode in people with sickle cell disease. It happens when sickle-shaped red blood cells block blood vessels, causing severe pain.
What is a 'placebo'?
A placebo is a treatment that looks like the study medicine but doesn't contain any active ingredients. It's used for comparison to see if the new medicine really works.
Will I know if I'm getting the new medicine or the placebo?
No, neither you nor your study doctor will know. This is to make sure the study results are as fair and accurate as possible.
What happens if I get side effects?
The study team will closely monitor you for any side effects, and they will be there to provide care and support if you experience any.
Can I leave the study at any time?
Yes, absolutely. You can choose to leave the study at any point, and your decision will not affect your future medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
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