Study on the Safety and Effectiveness of CSL889 for Adults and Adolescents with Sickle Cell Disease During Painful Crises

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What is this study about?

This clinical trial is focused on studying sickle cell disease, a genetic condition that affects the red blood cells, leading to episodes of severe pain known as vaso-occlusive crises. The study will evaluate a treatment called CSL889, which is a form of hemopexin, human, a protein that may help manage these painful episodes. The treatment is given as a solution through an intravenous infusion, meaning it is delivered directly into the bloodstream through a vein.

The purpose of the study is to assess the safety and effectiveness of CSL889 in helping to resolve vaso-occlusive crises in people with sickle cell disease. Participants in the study will receive either the CSL889 treatment or a placebo, which is a solution that does not contain the active substance. The study will monitor how quickly the painful episodes resolve and will also track any side effects that may occur.

Participants will be involved in the study for a period of time during which they will receive multiple doses of the treatment. The study will also collect information on how the body processes the treatment, known as pharmacokinetics. This includes measuring the concentration of CSL889 in the blood at different times. The study aims to provide valuable information on whether CSL889 can be a safe and effective treatment option for managing vaso-occlusive crises in sickle cell disease.

1 joining the study

Upon joining the study, you will be informed about the trial’s purpose, procedures, and potential risks and benefits. You will be asked to provide informed consent, confirming your understanding and willingness to participate.

2 initial assessment

An initial assessment will be conducted to confirm your eligibility. This includes verifying your age, diagnosis of sickle cell disease, and the presence of a new acute vaso-occlusive crisis requiring treatment with opioids.

3 randomization

You will be randomly assigned to receive either the study medication, CSL889 (human hemopexin), or a placebo (a solution without active substance). This process is double-blind, meaning neither you nor the study team will know which treatment you receive.

4 treatment administration

The treatment will be administered through an intravenous infusion. The frequency and dosage will be determined by the study protocol, and you will receive multiple doses over the course of the study.

5 monitoring and follow-up

Throughout the study, you will be closely monitored for any side effects or changes in your condition. Regular assessments will be conducted to evaluate the safety and effectiveness of the treatment, including measuring the time to resolution of the vaso-occlusive crisis and any adverse events.

6 completion of the study

Upon completion of the study, a final assessment will be conducted. This will include a review of your overall health and any changes observed during the trial. You will be informed about the next steps and any additional follow-up that may be required.

Who Can Join the Study?

  • Must be at least 18 years old or between 12 and 17 years old if approved for adolescents.
  • Must have been diagnosed with sickle cell disease (SCD). This is a group of inherited red blood cell disorders.
  • Must be experiencing a new acute vaso-occlusive crisis (VOC). This is a sudden episode of pain that occurs when blood flow is blocked in small blood vessels.
  • Must need treatment with parenteral opioids. These are strong pain medications given by injection.

Who Cannot Join the Study?

  • Patients who do not have sickle cell disease cannot participate. Sickle cell disease is a condition where red blood cells, which carry oxygen around the body, are shaped like a sickle or crescent, which can block blood flow.
  • Patients who are not experiencing a vaso-occlusive crisis cannot participate. A vaso-occlusive crisis is a painful episode that occurs when blood flow is blocked by sickle-shaped cells.
  • Patients who are not within the specified age range for the study cannot participate. The study includes specific age groups, but the exact ages are not provided here.
  • Patients who are not willing or able to receive medication through an intravenous (IV) infusion cannot participate. An IV infusion is a way to deliver medication directly into the bloodstream through a vein.
  • Patients who are not able to follow the study procedures or attend all required visits cannot participate.
  • Patients who have any other medical conditions or are taking medications that might interfere with the study cannot participate.
  • Patients who are pregnant or breastfeeding cannot participate.
  • Patients who have participated in another clinical trial recently may not be eligible to participate.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Other Sites

Site Name City Country Status
Centre Hospitalier Universitaire Rouen Rouen France
Centro Ricerche Cliniche Di Verona S.r.l. Verona Italy
IRCCS Istituto Giannina Gaslini Genoa Italy
Azienda Ospedaliero Universitaria Di Modena Modena Italy
Hospital Sant Joan De Deu Barcelona Esplugues De Llobregat Spain
Centre Hospitalier Regional De La Citadelle Liege Belgium
Hospital General Universitario Gregorio Maranon Madrid Spain
Pellegrin Hospital Bordeaux France
Azienda Ospedaliera di Padova Padua Italy
Virgen del Rocío University Hospital Sevilla Spain
Azienda Ospedaliero-Universitaria San Luigi Gonzaga Orbassano Italy
Universitair Medisch Centrum Utrecht Utrecht The Netherlands
Medical Center – University Of Freiburg Freiburg Im Breisgau Germany
Centre Hospitalier Universitaire De Nantes Nantes France
Centre Hospitalier Lyon Sud Pierre Benite France
Hopital Beaujon Clichy France
Ospedale Pediatrico Bambino Gesu’ Rome Italy
Lcvbw Gupghlt Hjtbfxsg Oe Ajcusp Athens Greece
Armklvc Oblolnqeuex Ojxxyapm Rijscxf Vtacq Sseut Cpgdplhd Palermo Italy
Acfmemgns Una Amsterdam The Netherlands
Efdnqnx Ugfcthyhiqmr Mkvsvqy Cprswsh Rhkiudzez (rmqsfcp Mnb Rotterdam The Netherlands
Uswymdnvux Ob Azippdv Edegem Belgium
Uxepirooxg Dqyoh Sqxds Dd Rrde Lk Skipeoli Rome Italy
Hjyxiobq Vmdf dworflwe Barcelona Spain

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Belgium Belgium
Not recruiting
07.07.2025
France France
Not recruiting
07.07.2025
Germany Germany
Not recruiting
07.07.2025
Greece Greece
Not recruiting
07.07.2025
Italy Italy
Not recruiting
07.07.2025
Spain Spain
Not recruiting
07.07.2025
The Netherlands The Netherlands
Not recruiting
07.07.2025

Trial locations

Investigated drugs:

CSL889 is a medication being tested in this clinical trial. It is a form of human hemopexin, which is a protein that naturally occurs in the body. In this study, CSL889 is given to participants through an intravenous (IV) infusion. The main goal of using CSL889 is to see if it can help people with sickle cell disease recover faster from a painful episode known as a vaso-occlusive crisis. This crisis happens when the blood flow is blocked by sickle-shaped cells, causing pain and other complications. The study aims to find out if CSL889 can safely and effectively reduce the time it takes for these crises to resolve.

Sickle cell disease vaso-occlusive crisis – This condition occurs in individuals with sickle cell disease, a genetic disorder affecting hemoglobin in red blood cells. During a vaso-occlusive crisis, the sickle-shaped red blood cells block small blood vessels, leading to reduced blood flow and oxygen delivery to tissues. This blockage causes severe pain and can affect various parts of the body, including the bones, lungs, and abdomen. The crisis can be triggered by factors such as dehydration, infection, or stress. It typically progresses with episodes of acute pain that can last from hours to days. Repeated episodes can lead to complications affecting organs and overall health.

Trial ID:
2024-513440-29-00
Protocol code:
CSL889_2001
Trial Phase:
Therapeutic use (Phase IV)

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