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Study on Tocilizumab for Treating Acute Chest Syndrome in Children and Adults with Sickle Cell Disease

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying the treatment of Sickle Cell Disease, a genetic condition that affects the red blood cells, leading to various health complications. The study is specifically looking at a condition called Acute Chest Syndrome, which can occur in people with Sickle Cell Disease and involves symptoms like fever and breathing difficulties. The treatment being tested is a medication called Tocilizumab, which is given as an intravenous infusion, meaning it is administered directly into the bloodstream through a vein. Tocilizumab is being compared to a placebo to see if it can help patients recover more quickly from the need for extra oxygen and breathing support.

The purpose of the study is to evaluate how effective Tocilizumab is in helping patients with Acute Chest Syndrome due to Sickle Cell Disease. Participants in the study will receive a single dose of Tocilizumab, with the amount based on their weight. The study will monitor how quickly patients can stop using supplemental oxygen and other forms of breathing assistance. This will help determine if Tocilizumab can improve recovery times for these patients.

Throughout the study, participants will be closely observed to ensure their safety and to gather information on how well the treatment works. The study aims to provide valuable insights into the potential benefits of Tocilizumab for people with Sickle Cell Disease experiencing Acute Chest Syndrome, ultimately contributing to better treatment options for this serious condition.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the trial

    Upon joining the clinical trial, you will be required to provide freely given, informed, and written consent. This consent can be given by you or your legal representatives.

    If you are a girl or woman of childbearing age (15-50 years old), a negative pregnancy test is required before participation.

    You must be affiliated with social security or have health insurance.

  2. Step 2

    Eligibility confirmation

    To participate, you must be a patient with sickle cell disease of any genotype (SS, SC, S/β0, and S/β+).

    You must be at least 2 years old and hospitalized for acute chest syndrome, which is defined by the World Health Organization as having a fever and/or acute respiratory symptoms with a new lung issue visible on a chest image (X-ray, lung ultrasound, or CT scan).

    You must require supplemental oxygen of at least 2 liters per minute to maintain oxygen saturation (SpO2) of 95% or higher, or need non-invasive or invasive respiratory support for less than 48 hours.

  3. Step 3

    Treatment administration

    You will receive a single intravenous infusion of tocilizumab. The dosage is 8 mg per kilogram of body weight for patients weighing 30 kg or more, up to a maximum of 800 mg. For patients weighing less than 30 kg, the dosage is 12 mg per kilogram.

    The infusion will be administered through a vein, which is known as an intravenous infusion.

  4. Step 4

    Monitoring and evaluation

    The primary goal is to evaluate the time it takes for you to successfully stop using both supplemental oxygen and any respiratory support. This is defined as maintaining an oxygen saturation (SpO2) of 95% or higher without oxygen for the next 24 hours, and breathing on your own without any respiratory support for the next 48 hours.

    Your progress will be closely monitored to ensure safety and effectiveness of the treatment.

  5. Step 5

    Post-treatment requirements

    If you are a girl or woman of childbearing age, effective contraception is required for up to 6 months after receiving the treatment.

Who can join the trial?

8 criteria

  • Must have Sickle Cell Disease of any type (SS, SC, S/β0, and S/β+).
  • Must be 2 years old or older.
  • Must be hospitalized for Acute Chest Syndrome (ACS). ACS is when you have a fever and/or sudden breathing problems with a new lung issue seen on a chest X-ray, lung ultrasound, or CT scan.
  • Must need extra oxygen (at least 2 liters per minute to keep oxygen levels at 95% or higher) or need help with breathing using machines like high flow nasal oxygen, continuous positive airway pressure, bilevel non-invasive ventilation, or invasive mechanical ventilation or ECMO, for less than 48 hours.
  • Girls or women who can have children (ages 15-50) must have a negative pregnancy test.
  • Must have freely given, informed, and written consent from the patient or their legal representatives.
  • Must have health insurance or social security coverage.
  • Must use effective birth control for up to 6 months after receiving the treatment.

Who cannot join the trial?

3 criteria

  • Patients who do not have Sickle Cell Disease cannot participate.
  • Patients who are not experiencing Acute Chest Syndrome cannot participate. Acute Chest Syndrome is a condition where there is sudden chest pain and difficulty breathing.
  • Patients who are not using supplemental oxygen or any form of respiratory support cannot participate. Supplemental oxygen is extra oxygen given to help with breathing, and respiratory support includes machines that help you breathe.
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Investigated drugs

Tocilizumab is a medication used in this clinical trial to help treat a condition called Acute Chest Syndrome in people with Sickle Cell Disease. It is given as an infusion, which means it is delivered directly into the bloodstream through a vein. The goal of using Tocilizumab in this trial is to see if it can help patients recover faster by reducing the need for extra oxygen and breathing support. This medication works by targeting specific proteins in the body that can cause inflammation, which is a part of the body's immune response. By reducing inflammation, Tocilizumab may help improve breathing and overall lung function in patients experiencing this serious complication of Sickle Cell Disease.

What is already known about the treatment

Tocilizumab – Tocilizumab is administered as a single intravenous infusion, meaning it is given directly into a vein. It is currently being studied for its effectiveness in treating acute chest syndrome in both children and adults with sickle cell disease. The main goal is to help patients reduce their need for extra oxygen and breathing support. Tocilizumab works by blocking a specific protein in the body that can cause inflammation, which is part of the immune response. It is classified as an immunosuppressive drug, which means it helps to reduce the activity of the immune system.

Investigated diseases

Sickle Cell Disease – Sickle Cell Disease is a genetic blood disorder characterized by the production of abnormal hemoglobin, known as hemoglobin S. This causes red blood cells to become rigid, sticky, and shaped like a crescent or sickle. These sickle-shaped cells can block blood flow in small vessels, leading to pain and potential organ damage. The disease progresses with episodes of pain, known as pain crises, and can lead to complications such as anemia, infections, and acute chest syndrome. Over time, repeated blockages and lack of oxygen can damage tissues and organs. The severity and frequency of symptoms can vary widely among individuals.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-505109-17-00Protocol codeAPHP220797Estimated enrolment130 patientsSponsorAssistance Publique Hopitaux De Paris

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