Tocilizumab for Acute Chest Syndrome
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age12+
SponsorUniversity of Chicago
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Adults ≥ 12 years of age
Prior diagnosis of sickle cell disease (Hb SS, Hb SC, Hb Sb+, and Hb Sb0)
Disqualifiers
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)
Trial design
Design model
Parallel
Treatments tested in this trial
Tocilizumab
DrugTocilizumab 80 mg IV dose (one time per patient)
Treatment groups
200 Participants
are divided into 2 treatment groupsGroup A: Early TocilizumabExperimental treatment 1 intervention
Group B: Delayed TocilizumabActive comparator 1 intervention
Trial outcomes
Primary outcomes
1
Time-weighted SaO2/FiO2 ratio
Oxygenation data will be obtained as part of routine clinical care. All changes in pulse oximetry measurement that are documented in the chart will be recorded in an oxygen saturation case report form with the date and time from Day 0 to Day 4. These peripheral oxygen saturation (SpO2) measurements will serve as surrogates for SaO2. Additionally, all changes in the route of supplemental oxygen delivery, rate of supplemental oxygen delivery, and fraction of inspired oxygen (FiO2) will be recorded in a corresponding case report form with the date and time from Day 0 to Day 4. The time-weighted SaO2/FiO2 ratio, our primary endpoint, will be calculated based on these two case report forms.
Time frame
Total of 4 days (Day 0 to Day 4)Secondary outcomes
1
Red cell exchange transfusion rate
As part of routine clinical care by the inpatient team, patients may receive a red cell exchange transfusion. The study team will assess if participants received any exchange transfusions from Day 0 to Day 8, and if so, they will record the date of the first exchange transfusion and the total number of units transfused during that time period.
Time frame
Total of 9 days (Day 0 to Day 8)2
Intensive Care Unit (ICU) transfer rate
Patients will be assessed for if they were admitted directly to the intensive care unit (ICU) or if they were transferred from the general medicine floor to the ICU between Day 0 and Day 8. The date of transfer to the ICU will be recorded if applicable.
Time frame
Total of 9 days (Day 0 to Day 8)3
Length of stay
Patients will be assessed for their admission and discharge dates. Length of stay will be calculated based on those two dates.
Time frame
Up to 3 months (Admission Date to Discharge Date)4
Readmission rate
Patients will be assessed for readmission for 28 days from discharge. Readmission will be assessed at the University of Chicago as well as through any linked hospitals through Care Everywhere within the electronic medical record system. The date of readmission will be recorded if applicable.
Time frame
Total of 29 days (Discharge Date to 28 days after discharge)Sponsors and contacts
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Source ClinicalTrials.gov