Ireland
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Recruiting Phase 3

Efficacy and Safety of Tocilizumab for Acute Chest Syndrome Treatment in Patients With Sickle Cell Disease

NCT06818266 · tracked via the Priya Life Science France tracker
Phase
Phase 3
Started
2025-08-27
Last updated
2026-06-24

Condition(s) studied

Sickle Cell DiseaseAcute Chest Syndrome

Investigational drug(s) / intervention(s)

Tocilizumab (RoActemra®, 20 mg/mL).Placebo (NaCl 0.9%)

Tocilizumab (RoActemra®, 20 mg/mL).: One single intravenous infusion at 8 mg/kg (up to a maximum of 800 mg) for patients ≥ 30 kg and 12 mg/kg for patients \< 30 kg

Placebo (NaCl 0.9%): One single intravenous infusion

Study summary

The purpose of this study is to determine whether a single infusion of tocilizumab is effective in reducing the time to successful weaning from both supplemental oxygen and any respiratory support, in pediatric and adult patients with sickle cell disease (SCD) during acute chest syndrome (ACS).

Eligibility

Sex
ALL
Min age
2 Years
Max age
—
Healthy volunteers
No
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

Primary outcome measure(s)

  • Time to successful weaning from both supplemental oxygen and any respiratory support — During hospitalization for ACS, from randomization until day 28 after randomization
    Successful weaning from both supplemental oxygen and any respiratory support, defined as SpO2 ≥ 95% without oxygen during the next 24 hours, and spontaneous breathing without any respiratory support (non-invasive or invasive) during the next 48 hours

Trial sites (1)

FacilityCityRegionStatus
Department of General Pediatrics and Sickle Cell Center, Necker-Enfants malades Hospital Paris France Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06818266 on ClinicalTrials.gov ↗ ← All trials in France