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

Recruiting now · Phase 3 · Has a placebo group

Conditions studied: Sickle Cell Disease, Acute Chest Syndrome

In brief

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).

Key facts

Study ID: NCT06818266
Run by: Assistance Publique - Hôpitaux de Paris
People needed: 130
Starts: 2025-08-27
Expected to finish: 2027-07-01
Last updated by the study team: 2026-06-24

Who can join

Age: 2 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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)

You may not qualify if…

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.
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)
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
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
Pregnancy or breastfeeding
Imminent and inevitable progression towards death in the opinion of the investigator
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
Current enrolment in another interventional research concerning a medicinal product for human use

Where it is running

Department of General Pediatrics and Sickle Cell Center, Necker-Enfants malades Hospital — Paris, France (enrolling)

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.