Efficacy and Safety of Tocilizumab for Acute Chest Syndrome Treatment in Patients With Sickle Cell Disease
About this study
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).
- Condition
- Sickle Cell Disease, Acute Chest Syndrome
- Tested
- Tocilizumab (RoActemra®, 20 mg/mL)., Placebo (NaCl 0.9%)
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Who can join
- Age
- 2 years and older
- Sex
- All sexes
- Healthy volunteers
- Not accepted
Inclusion 10
- Minimum age: 2 years
- Study condition: Sickle Cell Disease, Acute Chest Syndrome
- 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 14
- 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
1 site, 1 recruiting
Department of General Pediatrics and Sickle Cell Center, Necker-Enfants malades Hospital
Paris, France
Contact
-
Slimane ALLALI, MD, PhD
01 44 49 48 96 slimane.allali@aphp.fr
-
Aminata TRAORE, Project advisor
Potential match only. Final eligibility is determined by the study team.