Tests tocilizumab for acute chest syndrome in people with sickle cell disease

Trial details

Who runs it

Assistance Publique - Hôpitaux de Paris

Trial code

NCT06818266EU 2023-505109-17-00

Record updated

24 June 2026

Original description

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).Read full original descriptionShow less

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

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).

Inclusion: SCD patient of all genotypes (SS, SC, S/β0 and S/β+ or other major SCD syndrome); Age ≥ 2 years old; 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); 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; Negative pregnancy test for girls or women of childbearing age; Freely given, informed and written consent of patient or legal representatives; Affiliation to the social security (or health insurance); Effective contraception up to 3 months after the administration of treatment (tocilizumab or placebo).

Exclusion: Immunization with a live/attenuated vaccine within the last 4 weeks; Immunomodulatory therapy, anti-rejection therapy, cell depleting therapies and investigational agents within the last 3 months; Known hypersensitivity to tocilizumab or its excipients; 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); History of severe allergic or anaphylactic reactions to human, humanized, or murine monoclonal antibodies; 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; Evidence of malignant disease or malignancies diagnosed within the last 3 years; Absolute neutrophil count < 1.0 G/L or platelets < 50 G/L; ALT or AST > 5-fold the upper limit of normal; Glomerular Filtration rate (GFR) < 60 mL/min/1,73 m²; 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; Pregnancy or breastfeeding; Imminent and inevitable progression towards death in the opinion of the investigator; Current enrolment in another interventional research concerning a medicinal product for human use.

ClinicalTrials.gov NCT06818266 · EU CTIS 2023-505109-17-00