RecruitingPHASE2INTERVENTIONAL

Safety, Efficacy, and Pharmacokinetics of CSL889 in Adults and Adolescents With Sickle Cell Disease During Vaso-Occlusive Crisis

This study is looking at a new potential medicine, CSL889, for adults and teenagers with sickle cell disease when they are experiencing a painful crisis (called a vaso-occlusive crisis or VOC). The main goals are to check if CSL889 is safe to use and how well people tolerate it. Researchers will also see if it helps reduce the time it takes for these painful crises to get better. Participants will be randomly assigned to receive either CSL889 or a placebo (a treatment that looks the same but contains no active medicine). This is a Phase 2 study, meaning it's an early stage where researchers are primarily gathering information on safety and initial effectiveness.

At a glance

Status

Recruiting

Phase

PHASE2

Sponsor

CSL Behring

Enrolment target

Start

07 Aug 2025

Estimated completion

22 Oct 2027

Sickle Cell Disease Vaso-occlusive Crisis

What is this study about?

Sickle cell disease is a lifelong condition that can cause severe pain crises, known as vaso-occlusive crises (VOCs). These crises happen when sickle-shaped red blood cells block small blood vessels, stopping blood flow and causing pain. When these crises are severe, people often need to go to the hospital for strong pain relief.

This research study is looking at a new potential medicine called CSL889. We want to understand if CSL889 can help people with sickle cell disease manage their pain crises more effectively. The study aims to see if this new medicine is safe and if it can help shorten the time a pain crisis lasts.

By taking part, you would be helping doctors learn more about CSL889. This information is crucial for developing new treatments that could improve the lives of people living with sickle cell disease. Your participation could help future patients facing similar challenges.

Key takeaways

This study tests a new medicine (CSL889) for sickle cell pain crises.
It aims to check safety and if pain crises resolve faster.
Participants will receive CSL889 or a placebo by chance.
It's for adults and teenagers (12+) with sickle cell disease experiencing a crisis.
Participation involves IV medicine, regular checks, and blood tests.
You can withdraw at any time without affecting your care.

Who may be eligible?

To join this study, you must be either an adult (18 years or older) or a teenager between 12 and 18 years old. You must have a diagnosis of sickle cell disease and be currently experiencing a new, acute pain crisis (VOC) that requires strong pain medicine given through a drip (intravenously).

There are also some reasons why you might not be able to join. For example, if your current pain crisis started more than three days ago before you received your first strong pain medicine, or if you've had more than five hospital admissions for pain crises in the last six months. You also can't join if you have signs of a serious lung problem (acute chest syndrome), new symptoms of a stroke, specific kidney problems, or if you were recently discharged from the hospital for a pain crisis within the last 14 days.

Certain health conditions or medications might also mean you can't take part. This includes having very low blood iron levels (hemoglobin below 6 g/dL), very high iron stores (ferritin of 2000 ng/mL or more), not being on a stable treatment for sickle cell disease, or if you are currently taking certain pain medicines like methadone or buprenorphine.

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 12 years old or older?
Do you have sickle cell disease?
Are you currently having a new, acute pain crisis that needs strong pain relief through a drip?
Did your current pain crisis start less than 3 days ago before you received your first strong pain relief?
Have you had 5 or fewer hospital stays for pain crises in the last 6 months?
Are you NOT currently taking methadone or buprenorphine?
Do you NOT have new stroke signs, serious lung issues (acute chest syndrome), or certain kidney problems?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you'll first be checked to see if you're suitable for the study. If you are, you'll be randomly assigned to receive either the study medicine (CSL889) or a placebo (a dummy medicine with no active ingredients). Both you and your doctors won't know which one you are receiving during the study.

The study medicine will be given through a drip into your vein. You'll have regular checks by the study team, which will include blood tests, physical examinations, and questions about your pain and how you're feeling. These visits will help the doctors understand how you're responding to the treatment and if there are any side effects. The total length of your participation, including the treatment period and follow-up checks, will be explained by the study team.

Potential risks and benefits

Taking part in a clinical trial offers the chance to try a new medicine that might help your pain crisis get better faster. However, there's also a chance you could receive the placebo, or that CSL889 might not work for you. Like all medicines, CSL889 could have side effects, some of which might be unknown. The research team will monitor you closely for any issues and explain all known risks. Your safety and well-being are the top priority, and you are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (26)

Univ. of California, San Francisco Health Care
Verified postcode
Oakland, United States· Recruiting
University of California Irvine
Verified postcode
Orange, United States· Not yet recruiting
Golisano Children's Hospital
Verified postcode
Fort Myers, United States· Recruiting
The Foundation for Sickle Cell Disease
Verified postcode
Hollywood, United States· Recruiting
Arthur M. Blank Hospital-Children's Healthcare of Atlanta
Verified postcode
Atlanta, United States· Recruiting
University of Illinois at Chicago
Verified postcode
Chicago, United States· Recruiting
University of Louisville Hospital
Verified postcode
Louisville, United States· Recruiting
University of Maryland
Verified postcode
Baltimore, United States· Recruiting
Detroit Medical Center
Verified postcode
Detroit, United States· Recruiting
Henry Ford Health System
Verified postcode
Detroit, United States· Recruiting
St. Louis Children's Hospital
Verified postcode
St Louis, United States· Recruiting
Mount Sinai Medical Center
Verified postcode
New York, United States· Recruiting

Common questions

What is a 'placebo'?

A placebo is a treatment that looks exactly like the study medicine but doesn't contain any active ingredients. It helps researchers fairly compare the new medicine's effects.

What does 'randomized' mean?

It means you'll be assigned to either the CSL889 group or the placebo group by chance, like flipping a coin. This helps make the study fair and reliable.

What is a 'Phase 2' study?

Phase 2 means it's an early stage of testing where doctors are mostly checking if the new medicine is safe and starting to see if it works, before large-scale trials.

Will I know if I'm getting the real medicine or the placebo?

No, neither you nor the study doctors will know which treatment you are receiving until after the study is over. This is called 'blinding' and helps keep the study fair.

Can I leave the study at any time?

Yes, you are free to leave the study at any point, for any reason. Your decision will not affect your ongoing medical care.

How to find out more

Trial Registration Coordinator

clinicaltrials@cslbehring.com +1 610-878-4697 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.