Study on Crizanlizumab for Adolescents and Adults with Sickle Cell Disease Experiencing Vaso-Occlusive 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, causing them to become misshapen and leading to various health complications. The study is testing a treatment called crizanlizumab, which is also known by the code name SEG101. This treatment is a type of medication known as a monoclonal antibody, which is designed to target specific proteins in the body. The trial will compare two different doses of crizanlizumab to a placebo, with some participants also receiving standard treatments like hydroxyurea or hydroxycarbamide.

The purpose of the study is to evaluate how effective and safe crizanlizumab is in reducing the number of painful episodes, known as vaso-occlusive crises, that lead to healthcare visits in people with sickle cell disease. Participants in the study will receive either crizanlizumab or a placebo through an intravenous infusion, which means the medication is given directly into a vein. The study will last for about a year, during which time participants will have regular check-ups to monitor their health and the effects of the treatment.

Throughout the study, researchers will collect information on how often participants experience vaso-occlusive crises, how long these episodes last, and any other health changes that occur. This information will help determine if crizanlizumab is a beneficial treatment option for managing sickle cell disease. The study aims to provide valuable insights into improving care for individuals living with this condition.

1 joining the study

Upon joining the study, informed consent is required. This involves understanding the study’s purpose and agreeing to participate.

Eligibility is confirmed through a diagnosis of sickle cell disease and a history of vaso-occlusive crises (VOCs) that required medical attention.

2 initial assessment

An initial assessment is conducted to ensure health criteria are met. This includes blood tests to check levels of neutrophils, platelets, hemoglobin, and kidney function.

Performance status is evaluated to ensure the ability to participate in the study.

3 treatment administration

The treatment involves receiving crizanlizumab or a placebo through an intravenous infusion. The dosage is either 7.5 mg/kg or 5.0 mg/kg.

Infusions are scheduled regularly, with the first and fifth doses being key points for monitoring.

4 monitoring and follow-up

Throughout the study, the frequency and severity of VOCs are monitored. This includes both those requiring healthcare visits and those managed at home.

Regular visits to the clinic are necessary to assess the treatment’s impact and any side effects.

5 end of study

The study concludes after one year of treatment. Final assessments are conducted to evaluate the overall health and any changes in condition.

Data collected during the study is analyzed to determine the effectiveness and safety of the treatment.

Who Can Join the Study?

You must provide written informed consent before any screening procedures.
You must be a male or female aged 12 years or older on the day you sign the informed consent. This includes adolescents aged 12 to 17 years and adults aged 18 years and older.
You must have a confirmed diagnosis of Sickle Cell Disease (SCD), which is a blood disorder, confirmed by specific tests called Hb electrophoresis or high-performance liquid chromatography (HPLC).
You must have experienced at least 2 Vaso-Occlusive Crises (VOCs), which are painful episodes, leading to a healthcare visit within the 12 months before the screening visit. These crises must have resolved at least 7 days before the start of the study. VOCs include:
- Pain crisis, which is sudden pain with no other medical explanation.
- Visits to a medical facility or healthcare professional.
- Receiving pain relief medication like opioids or nonsteroidal anti-inflammatory drugs (NSAIDs).
- Other conditions like Acute Chest Syndrome (ACS), priapism, and hepatic or splenic sequestration are also considered VOCs.
If you are taking medications like Hydroxyurea (HU/HC) or L-glutamine, you must have been on them for at least 6 months and at a stable dose for at least 3 months before the screening visit. You should plan to continue taking them at the same dose and schedule for one year of the study. If you are not taking these medications, you must not have taken them for at least 6 months before the screening visit. You must have evidence of insufficient control of acute pain, such as at least one VOC leading to a healthcare visit while on these treatments. If you are taking an erythropoietin stimulating agent, you must have been on it for at least 6 months before the screening visit and plan to continue to maintain stable hemoglobin (Hb) levels for one year of the study.
You must meet certain laboratory values before starting the study:
- Absolute Neutrophil Count: At least 1.0 x 10⁹/L
- Platelet count: At least 75 x 10⁹/L
- Hemoglobin (Hb): At least 4.0 g/dL for adults and 5.5 g/dL for adolescents
- Glomerular filtration rate: At least 45 mL/min/1.73 m², which measures kidney function
- Direct (conjugated) bilirubin: Less than 2.0 times the upper limit of normal (ULN)
- Alanine transaminase (ALT): Less than 3.0 times the ULN, which measures liver function
Your ECOG performance status must be 2.0 or less for adults, which measures your ability to perform daily activities. For adolescents, the Karnofsky score must be 50% or more, which is another way to measure daily activity ability.

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 are shaped like a sickle or crescent, which can cause health problems.
Patients who are not within the specified age range for the study cannot participate. The study includes certain age groups, so it’s important to check if you fall within these groups.
Patients who are not part of the specified clinical trial groups cannot participate. This means the study is looking for people with specific characteristics or conditions.
Both male and female patients are eligible, so gender is not a reason for exclusion.
Patients who are considered part of a vulnerable population may not be eligible. Vulnerable populations include groups that might need special protection or care.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Université Libre de Bruxelles – Hôpital Erasme	Brussels	Belgium

Other Sites

Site Name	City	Country
Haga Hospital	Hague	The Netherlands
Hopital Beaujon	Clichy	France
Aelidxdrzh Pvwuycen Hjjcrjbc Dn Mkjxynbiv	Marseille	France
Hfzlqvrd Usenteectf Ctxqwwj Hbzuquuc	Helsinki	Finland
Ekxeclc Urrmvqizjhir Momojlr Ckswklw Rwyixcmhh (xfhcokn Mmw	Rotterdam	The Netherlands
Asjripejt Uab	Amsterdam	The Netherlands
Unsavciiqe Oh Afhwxje	Edegem	Belgium
Fpguimgnh Psgf Lj Ikllwruxpxxgq Bhyuescmi Dzy Htrikegg Umqjjlafkrhnh Lk Pdm	Madrid	Spain
Hphnndil Vepc dlrpehyb	Barcelona	Spain

Trial status

Country	Status	Recruitment Start
Belgium	Not recruiting	16.09.2019
Finland	Not recruiting	16.09.2019
France	Not recruiting	16.09.2019
Spain	Not recruiting	16.09.2019
The Netherlands	Not recruiting	16.09.2019

Trial locations

Investigated drugs:

Crizanlizumab

Crizanlizumab is a medication being studied for its effectiveness in treating sickle cell disease. It is designed to help reduce the number of painful episodes, known as vaso-occlusive crises, that patients with sickle cell disease experience. These crises occur when blood flow is blocked by sickle-shaped red blood cells. Crizanlizumab works by preventing these cells from sticking together and blocking blood vessels, which may help decrease the frequency of these painful episodes.

Hydroxyurea (also known as Hydroxycarbamide) is a medication that is often used in the treatment of sickle cell disease. It helps to reduce the frequency of painful crises and the need for blood transfusions. Hydroxyurea works by increasing the production of fetal hemoglobin, a type of hemoglobin that helps prevent the sickling of red blood cells. This medication is part of the standard care for sickle cell disease and may be used in combination with other treatments to improve patient outcomes.

Investigated diseases:

Sickle cell disease

Sickle Cell Disease – Sickle cell disease is a genetic disorder that affects the red blood cells, causing them to become rigid and shaped like a crescent or sickle. These abnormally shaped cells can block blood flow in small blood vessels, leading to pain and potential organ damage. The disease is characterized by episodes of pain, known as vaso-occlusive crises, which can vary in frequency and severity. Over time, sickle cell disease can lead to complications such as anemia, increased risk of infections, and damage to organs like the spleen and liver. The condition is inherited in an autosomal recessive pattern, meaning both parents must carry a copy of the mutated gene for a child to be affected. Regular medical care and monitoring are essential to manage symptoms and prevent complications.

Trial ID:

2023-508689-14-00

Protocol code:

CSEG101A2301

NCT ID:

NCT03814746

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on Crizanlizumab for Adolescents and Adults with Sickle Cell Disease Experiencing Vaso-Occlusive Crises

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