A clinical trial to evaluate the safety and effectiveness of crovalimab in reducing pain crises, also called vaso-occlusive episodes (VOE), in people with sickle cell disease (SCD)

Background and study aims:

1. HOW DOES THE BO42451 CLINICAL TRIAL WORK?

This clinical trial is recruiting people who have sickle cell disease (SCD). 

The purpose of this clinical trial is to compare the effects, good or bad, of crovalimab versus placebo on patients with SCD. In this clinical trial, you will get either crovalimab or placebo. 

2. HOW DO I TAKE PART IN THIS CLINICAL TRIAL?

You will be able to take part in this clinical trial, if you: 

• Have been diagnosed with a certain type of SCD (sickle cell anaemia or sickle cell beta zero thalassaemia)
• Are between 12‒55 years of age
• Have had at least two (but no more than 10) pain attacks (crises) requiring a medical visit in the previous year 
• Are up to date with certain vaccinations

You will not be eligible to take part in this clinical trial if you: 

• Have previously received a stem cell transplant
• Are participating in a chronic transfusion program
• Are pregnant, breastfeeding or intending to become pregnant during or within 10.5 months after the last dose of study treatment
• Have had a recent infection

You may also not be able to take part in the clinical trial if you have certain other medical conditions or if you have previously received certain medications.

If you think this clinical trial may be suitable for you and would like to take part, please talk to your doctor. If your doctor thinks that you might be able to take part in this clinical trial, he/she may refer you to the closest clinical trial doctor. They will give you all the information you need to make your decision about taking part in the clinical trial. You can also find the clinical trial locations on this page.

You will have some further tests to make sure you will be able to take the treatments given in this clinical trial. Some of these tests or procedures may be part of your regular medical care. They may be done even if you do not take part in the clinical trial. If you have had some of the tests recently, they may not need to be done again.

Before starting the clinical trial, you will be told about any risks and benefits of taking part in the trial. You will also be told what other treatments are available so that you may decide if you still want to take part. 

For safety reasons while taking part in the clinical trial, women who are not currently pregnant but can become pregnant will need to agree to either use a reliable birth control method or not have heterosexual intercourse.

3. WHAT TREATMENT WILL I BE GIVEN IF I JOIN THIS CLINICAL TRIAL? 

Everyone who joins this clinical trial will be split into two groups randomly (like flipping a coin) and will receive either:

• Crovalimab as an infusion into the vein on Day 1, and then as an injection under the skin (subcutaneous) on Day 2. After this, crovalimab will be given as a subcutaneous injection once a week in Weeks 2, 3, 4, and 5, and then once every four weeks until study completion (roughly 48 weeks after starting treatment)

OR 

• Placebo as an infusion into the vein on Day 1, and then as an injection under the skin (subcutaneous) on Day 2. After this, placebo will be given as a subcutaneous injection once a week in Weeks 2, 3, 4, and 5, and then once every four weeks until study completion (roughly 48 weeks after starting treatment)

You will have an equal chance of being placed in either group. 

During the clinical trial, you can continue to have standard treatment for SCD as recommended by your clinical trial doctor.

This is a ‘placebo-controlled’ clinical trial, which means that one of the groups will be given a saline (salt water) infusion with no active ingredients (also known as a ‘placebo’). A placebo is used as a control, to make sure any health effects are from the clinical trial treatment rather than other factors.

Neither you nor your clinical trial doctor can choose or know the group you are in. An exception is made if your clinical trial doctor needs to know which group you are in for safety reasons.

4. HOW OFTEN WILL I BE SEEN IN FOLLOW-UP APPOINTMENTS AND FOR HOW LONG?

You will be given the clinical trial treatment crovalimab or placebo for 48 weeks. You are free to stop this treatment at any time. During the study, you will be seen regularly by the clinical trial doctor, and you will also have some telephone check-ups. These appointments will include checks to see how you are responding to the treatment and any side effects that you may be having. Once you have received your final dose of treatment, you will have 2 additional safety follow up visits: one clinic visit 24 weeks after the final dose and one telephone call with your clinical trial doctor 46 weeks after the final dose to check on any side effects you may have had.

The clinical trial will last for a total of 91 weeks (about 2 years), including the additional safety follow-ups.

5. WHAT HAPPENS IF I AM UNABLE TO TAKE PART IN THIS CLINICAL TRIAL?

If this clinical trial is not suitable for you, you will not be able to take part. Your doctor will suggest other clinical trials that you may be able to take part in or other treatments that you can be given. You will not lose access to any of your regular care.

For more information about this clinical trial see the For Expert tab on the specific ForPatient page or follow this link to ClinicalTrials.gov

Trial-identifier: NCT05075824