Tocilizumab for Acute Chest Syndrome in Sickle Cell Disease

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This phase 3 trial evaluates a single dose of tocilizumab versus placebo in pediatric and adult patients with sickle cell disease hospitalized for acute chest syndrome. Participants must require some level of oxygen or respiratory support and be within 48 hours of the need. The primary goal is to see if the treatment shortens the time to wean off supplemental oxygen and any form of respiratory support. The rationale is based on elevated IL-6 levels observed in ACS and prior rapid responses to tocilizumab in similar settings. Safety and efficacy will be monitored across multiple clinical centers in Paris.

Why it matters

Who can join: the full rules

Inclusion Criteria:

1. SCD patient of all genotypes (SS, SC, S/β0 and S/β+ or other major SCD syndrome)
2. Age ≥ 2 years old
3. Hospitalized for ACS, defined by the WHO as the association of fever and/or acute respiratory symptoms with a new pulmonary infiltrate on chest imaging, (X-ray, lung ultrasound, or CT scan)
4. Requiring supplemental oxygen ≥ 2 L/min for SpO2 ≥ 95% or non-invasive respiratory support (high flow nasal oxygen or continuous positive airway pressure or bilevel non-invasive ventilation) or invasive mechanical ventilation or ECMO, for less than 48 hours
5. Negative pregnancy test for girls or women of childbearing age
6. Freely given, informed and written consent of patient or legal representatives
7. Affiliation to the social security (or health insurance)
8. Effective contraception up to 3 months after the administration of treatment (tocilizumab or placebo)

Exclusion Criteria:

1. Impossibility to perform tocilizumab/placebo injection within the first 48 hours of supplemental oxygen ≥2L/min for SpO2≥95% and/or respiratory support (as defined in inclusion criteria n°4). If exchange transfusion is indicated at inclusion, it has to be performed before the injection of tocilizumab/placebo.
2. Known hypersensitivity to tocilizumab or its excipients
3. Known active current severe bacterial, viral, fungal, mycobacterial, or other infections (including but not limited to tuberculosis and atypical mycobacterial disease, hepatitis B and C, and herpes zoster)
4. Immunization with a live/attenuated vaccine within the last 4 weeks
5. Immunomodulatory therapy, anti-rejection therapy, cell depleting therapies and investigational agents within the last 3 months
6. History of severe allergic or anaphylactic reactions to human, humanized, or murine monoclonal antibodies
7. History of diverticulitis, diverticulosis requiring antibiotic treatment, or chronic ulcerative lower gastrointestinal disease such as Crohn's disease, ulcerative colitis, or other symptomatic lower gastrointestinal conditions that might predispose a patient to perforations
8. Evidence of malignant disease or malignancies diagnosed within the last 3 years
9. Pregnancy or breastfeeding
10. Imminent and inevitable progression towards death in the opinion of the investigator
11. Absolute neutrophil count \< 1.0 G/L or platelets \< 50 G/L
12. ALT or AST \> 5-fold the upper limit of normal
13. Glomerular Filtration rate (GFR) \< 60 mL/min/1,73 m²
14. Current enrolment in another interventional research concerning a medicinal product for human use

Where it's running

• Department of General Pediatrics and Sickle Cell Center, Necker-Enfants malades Hospital
  Paris, France Recruiting

Study contacts

• Principal investigator: Slimane ALLALI, MD, PhD — Department of General Pediatrics and Sickle Cell Center, Necker-Enfants malades Hospital, Paris, France
• Study coordinator: Slimane ALLALI, MD, PhD
• Email: slimane.allali@aphp.fr
• Phone: 01 44 49 48 96

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Anticoagulation Plus Tocilizumab for Acute Cerebral Venous Thrombosis

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• Cerebrovascular Reactivity Testing with HD-DOT in Sickle Cell Patients

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• Newborn Sickle Cell Screening in Mozambique

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.