Tocilizumab for Acute Chest Syndrome
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Tocilizumab.
- Who it may be relevant to
- Registry conditions: Sickle Cell Disease, Acute Chest Syndrome. Basic parameters: from 12 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
Low-Dose Tocilizumab for Acute Chest Syndrome in Sickle Cell Disease
Overview
The investigators are evaluating the role of a low dose of tocilizumab in treating acute chest syndrome in patients with sickle cell disease. Tocilizumab inhibits interleukin-6 (IL-6) receptors and is used to treat rheumatoid arthritis and severe cytokine release syndrome, which can be seen with chimeric antigen receptor T-cell (CAR-T) therapy, and it is also authorized for treatment of COVID-19. Since IL-6 levels are elevated in the sputum of patients with acute chest syndrome, the investigators are hopeful that this will be an effective strategy. The investigators will be looking at how a low dose of tocilizumab affects oxygen status, clinical outcomes, and laboratory markers in patients admitted to the hospital with acute chest syndrome.
Detailed description
In this randomized, placebo-controlled, double-blinded phase II study, enrolled patients admitted to the University of Chicago who are diagnosed with acute chest syndrome will receive one dose of tocilizumab 80 mg IV and one normal saline placebo dose. The order of these doses will be randomized at a 1:1 ratio. After collecting oxygenation data as a baseline for 8 hours, patients will then receive tocilizumab versus placebo as their early dose and then the opposite (placebo versus tocilizumab) 48 hours later. Clinical, laboratory, and patient-reported outcome data will be collected during their admission and compared between arms.
Interventions
- Drug Tocilizumab
Tocilizumab 80 mg IV dose (one time per patient)
Primary outcome measures
- Time-weighted SaO2/FiO2 ratio [Time frame: Total of 4 days (Day 0 to Day 4)]
Secondary outcome measures (5)
- Red cell exchange transfusion rate [Time frame: Total of 9 days (Day 0 to Day 8)]
- Intensive Care Unit (ICU) transfer rate [Time frame: Total of 9 days (Day 0 to Day 8)]
- Length of stay [Time frame: Up to 3 months (Admission Date to Discharge Date)]
- Readmission rate [Time frame: Total of 29 days (Discharge Date to 28 days after discharge)]
- Mortality rate [Time frame: Total of 29 days (Day 0 to Day 28)]
Eligibility criteria
Inclusion criteria
- Adults ≥ 12 years of age
- Prior diagnosis of sickle cell disease (Hb SS, Hb SC, Hb Sb+, and Hb Sb0)
Exclusion criteria
- Pregnant patients or breastfeeding mothers.
- Prior treatment with gene therapy or a stem cell transplant.
- Current enrollment in a clinical trial involving an FDA-regulated drug or biologic.
- Current neutropenia (absolute neutrophil count \< 1000/mm\^3)
- Current thrombocytopenia (platelet count \< 50,000 mm\^3)
- Aspartate aminotransferase (AST) or alanine transaminase (ALT) \> 10 times the upper limit of normal (ULN)
- History of tuberculosis (TB).
- Positive purified protein derivative (PPD) TB screening test.
- On active therapy with a Bruton's tyrosine kinase-targeted agent, which include the following: Acalabrutinib, Ibrutinib, Zanubrutinib
- On active therapy with a JAK2-targeted agent, which include the following: Baricitinib, Ruxolitinib, Tofacitinib, Upadacitinib
- Any of the following biologic immunosuppressive agent (and any biosimilar versions thereof) administered in the past 6 months:
Abatacept, Adalimumab, Alemtuzumab, Atezolizumab, Belimumab, Blinatumomab, Brentuximab, Certolizumab, Daratumumab, Durvalumab, Eculizumab, Elotuzumab, Etanercept, Gemtuzumab, Golimumab, Ibritumomab, Infliximab, Inotuzumab, Ipilimumab, Ixekizumab, Moxetumomab, Nivolumab, Obinutuzumab, Ocrelizumab, Ofatumumab, Pembrolizumab, Polatuzumab, Rituximab, Sarilumab, Secukinumab, Tocilizumab, Tositumumab, Tremelimumab, Urelumab, Ustekinumab
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Quadruple blind
- Primary purpose
- Treatment
Study locations
United States · 1 center
- University of Chicago — Chicago
Identifiers
NCT: NCT05640271 · IRB22-0277