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TOCIACS : Efficacy and safety of Tocilizumab for acute chest syndrome treatment in paediatric and adult patients with sickle cell disease
EU CTIS ID: 2023-505109-17-00
What this study is testing
To evaluate the efficacy over placebo of tocilizumab (single intravenous infusion at 8 mg/kg for patients ≥ 30 kg (up to a maximum of 800 mg) and 12 mg/kg for patients < 30 kg) on time to successful weaning from both supplemental oxygen and any respiratory support (non-invasive or invasive), in paediatric and adult Sickle Cell Disease (SCD) patients with Acute Chest Syndrome (ACS).
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- SCD patient of all genotypes (SS, SC, S/β0 and S/β+) or other major SCD syndrome
- Age ≥ 2 years old
- Hospitalised 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 (15-50 years old)
- Freely given, informed and written consent of patient or legal representatives
You likely can't join if
- Impossibility to perform tocilizumab/placebo injection within the first 48 hours of supplemental oxygen ≥2L/min or 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.
- 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)
- History of bowel diverticulitis or gastrointestinal perforation
See the full eligibility criteria
- SCD patient of all genotypes (SS, SC, S/β0 and S/β+) or other major SCD syndrome
- Age ≥ 2 years old
- Hospitalised 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 (15-50 years old)
- Freely given, informed and written consent of patient or legal representatives
- Affiliation to the social security (or health insurance)
- Effective contraception up to 6 months after treatment administration
- Impossibility to perform tocilizumab/placebo injection within the first 48 hours of supplemental oxygen ≥2L/min or 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.
- 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)
- History of bowel diverticulitis or gastrointestinal perforation
- Immunomodulatory or anti-rejection therapy within the last 3 months
- Current enrolment in another interventional research concerning a medicinal product for human use
- 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
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years, 18-64 years, 65+ years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.