A Phase IB, Randomized, Placebo-Controlled Study Evaluating the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Crovalimab for the Management of Acute Uncomplicated Vaso‑Occlusive Episodes (VOE) in Patients With Sickle Cell Disease (SCD)
This study is looking into a new medicine called crovalimab for adults with sickle cell disease who are experiencing a sudden pain crisis, also known as a vaso-occlusive episode (VOE). We want to find out how safe the medicine is and if it causes any side effects. We'll also be checking how crovalimab moves through the body and if it has a positive effect on the pain crises. Participants will either receive the new medicine or a placebo (a dummy medicine with no active ingredient) to help us compare the results. The main goal is to understand if crovalimab can safely help manage these painful episodes, making them less severe or shorter in duration.
At a glance
What is this study about?
This study, called a Phase 1B trial, is testing a new medicine named crovalimab for adults who have sickle cell disease. People with sickle cell disease sometimes have very painful episodes called vaso-occlusive episodes (VOEs), often referred to as pain crises. These happen when the sickle-shaped red blood cells block small blood vessels, stopping blood flow and causing pain.
The main purpose of this study is to find out if crovalimab is safe for people to take and to see what side effects it might cause. We'll also be looking at how the medicine works in the body and if it helps to make these painful crises better. Some people in the study will receive crovalimab, while others will receive a placebo, which looks like the medicine but doesn't contain any active drug. This helps us to see if the new medicine is truly making a difference compared to no treatment.
By carefully studying how crovalimab affects those with sickle cell disease and painful episodes, we hope to learn if it could be a helpful new treatment option in the future. The information we gather will help us understand if this medicine is both safe and effective at managing these difficult pain crises.
Key takeaways
- This study is testing a new medicine called crovalimab for adults with sickle cell disease experiencing a pain crisis.
- It aims to check the medicine's safety, side effects, and how it works in the body.
- Some participants will receive the new medicine, while others will get a placebo (dummy medicine).
- You'll have regular hospital visits for check-ups, blood tests, and to monitor your health.
- Participation is voluntary, and you can withdraw at any time without affecting your medical care.
Who may be eligible?
This study is looking for adults aged 18 and over who have sickle cell disease. Both men and women can take part.
You might be able to join if you are experiencing a current, uncomplicated pain crisis (vaso-occlusive episode) from your sickle cell disease. The study team will check your medical history to make sure taking part is safe for you and that you meet all the specific requirements.
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 sickle cell disease?
- Are you currently having an uncomplicated sickle cell pain crisis (VOE)?
- Are you able to attend regular study visits and follow all study procedures?
- Are you willing to potentially receive either the new medicine or a placebo?
What does participation involve?
If you join this study, you will either receive the new medicine (crovalimab) or a placebo (a dummy medicine). You will have regular visits to the clinic so the study team can monitor your health, check for any side effects, and see how the medicine is affecting you. These visits will likely involve physical examinations, having your vital signs (like blood pressure and heart rate) checked, and giving blood and urine samples.
You will be closely monitored during and after receiving any study medication. The total duration of your participation in the study will be explained in detail by the study team, including how many visits you'll need to make and for how long. There will also be follow-up appointments after you finish receiving the study medicine to ensure your well-being.
Potential risks and benefits
Locations (4)
- —UnverifiedFrance
- —UnverifiedNetherlands
- —UnverifiedSpain
- —UnverifiedItaly
Common questions
What is a 'pain crisis' (vaso-occlusive episode)?
It's a very painful event in sickle cell disease when sickle-shaped red blood cells block small blood vessels, stopping blood flow and causing severe pain.
What does 'placebo-controlled' mean?
It means some people will get the new medicine, and others will get a 'dummy' medicine (placebo) that looks the same but has no drug in it. This helps us see if the new medicine actually works.
Will I know if I'm getting the real medicine or the placebo?
No, usually in these types of studies, neither you nor the study team will know who is getting which. This helps make the study results fair and unbiased.
What is the new medicine, crovalimab?
Crovalimab is a new medicine being tested to see if it can help manage acute pain crises in people with sickle cell disease. We are trying to understand how it works and if it is safe and effective.
How long will the study last?
The full duration of the study and how long you might be involved will be explained by the study team. It will include treatment periods and follow-up checks.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
Community discussion
Powered by our forum at community.patient.info. Please be respectful — this is not medical advice.