Health TrialsbyHealthPanel

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

ConditionsSickle Cell DiseaseAcute Chest Syndrome
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age2+
SponsorAssistance Publique - Hôpitaux de Paris

About this trial

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 criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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

Trial design

Design model

Parallel

Treatments tested in this trial

  • Tocilizumab (RoActemra®, 20 mg/mL).

    Drug

    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%)

    Drug

    One single intravenous infusion

Treatment groups

130 Participants
are divided into 2 treatment groups
Group A: Arms A : Tocilizumab (RoActemra®, 20 mg/mL)Experimental treatment 1 intervention
Group B: Arm B : Placebo (NaCl 0.9%)Placebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

Time to successful weaning from both supplemental oxygen and any respiratory support

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

Time frame
During hospitalization for ACS, from randomization until day 28 after randomization

Secondary outcomes

1

Adverse events during hospitalization and within 3 months following tocilizumab or placebo injection

Severe and not severe adverse events (including hypertension, hypersensitivity reactions, hypokalemia, neutropenia, thrombocytopenia, infections, pulmonary embolism/thrombosis, hepatic cytolysis, organ failure)

Time frame
Within 3 months after randomization
2

Time to discharge

Length of hospital stay

Time frame
From the date of randomization until the date of end of hospitalization, assessed up to 3 months
3

Mortality

Mortality

Time frame
Within 3 months after randomization
4

Need for transfusion

Need for red blood cell transfusion

Time frame
From the date of randomization until the date of end of hospitalization, assessed up to 3 months

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Assistance Publique - Hôpitaux de Paris

Lead sponsor

URC-CIC Paris Descartes Necker Cochin

Collaborator

Source ClinicalTrials.gov
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