Sickle Cell Disease Clinical Trial
Official title:
Low-Dose Tocilizumab for Acute Chest Syndrome in Sickle Cell Disease
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.
Status | Recruiting |
Enrollment | 200 |
Est. completion date | January 2026 |
Est. primary completion date | April 2025 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years and older |
Eligibility | Inclusion Criteria: - Adults = 18 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. - 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 |
Country | Name | City | State |
---|---|---|---|
United States | University of Chicago | Chicago | Illinois |
Lead Sponsor | Collaborator |
---|---|
University of Chicago |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | 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. | Total of 4 days (Day 0 to Day 4) | |
Secondary | 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. | Total of 9 days (Day 0 to Day 8) | |
Secondary | 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. | Total of 9 days (Day 0 to Day 8) | |
Secondary | Length of stay | Patients will be assessed for their admission and discharge dates. Length of stay will be calculated based on those two dates. | Up to 3 months (Admission Date to Discharge Date) | |
Secondary | 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. | Total of 29 days (Discharge Date to 28 days after discharge) | |
Secondary | Mortality rate | Patients will be assessed for mortality from Day 0 to Day 28. The date of death will be recorded if applicable. | Total of 29 days (Day 0 to Day 28) |
Status | Clinical Trial | Phase | |
---|---|---|---|
Completed |
NCT02227472 -
Working Memory and School Readiness in Preschool-Aged Children With Sickle Cell Disease
|
||
Recruiting |
NCT06301893 -
Uganda Sickle Surveillance Study (US-3)
|
||
Recruiting |
NCT04398628 -
ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders
|
||
Completed |
NCT02522104 -
Evaluation of the Impact of Renal Function on the Pharmacokinetics of SIKLOS ® (DARH)
|
Phase 4 | |
Recruiting |
NCT04688411 -
An mHealth Strategy to Improve Medication Adherence in Adolescents With Sickle Cell Disease
|
N/A | |
Terminated |
NCT03615924 -
Effect of Ticagrelor vs. Placebo in the Reduction of Vaso-occlusive Crises in Pediatric Patients With Sickle Cell Disease
|
Phase 3 | |
Not yet recruiting |
NCT06300723 -
Clinical Study of BRL-101 in Severe SCD
|
N/A | |
Recruiting |
NCT03937817 -
Collection of Human Biospecimens for Basic and Clinical Research Into Globin Variants
|
||
Completed |
NCT04917783 -
Health Literacy - Neurocognitive Screening in Pediatric SCD
|
N/A | |
Completed |
NCT04134299 -
To Assess Safety, Tolerability and Physiological Effects on Structure and Function of AXA4010 in Subjects With Sickle Cell Disease
|
N/A | |
Completed |
NCT02580565 -
Prevalence of Problematic Use of Equimolar Mixture of Oxygen and Nitrous Oxide and Analgesics in the Sickle-cell Disease
|
||
Recruiting |
NCT04754711 -
Interest of Nutritional Care of Children With Sickle Cell Disease on Bone Mineral Density and Body Composition
|
N/A | |
Completed |
NCT04388241 -
Preliminary Feasibility and Efficacy of Behavioral Intervention to Reduce Pain-Related Disability in Pediatric SCD
|
N/A | |
Recruiting |
NCT05431088 -
A Phase 2/3 Study in Adult and Pediatric Participants With SCD
|
Phase 2/Phase 3 | |
Completed |
NCT01158794 -
Genes Influencing Iron Overload State
|
||
Recruiting |
NCT03027258 -
Point-of-Delivery Prenatal Test Results Through mHealth to Improve Birth Outcome
|
N/A | |
Withdrawn |
NCT02960503 -
Macrolide Therapy to Improve Forced Expiratory Volume in 1 Second in Adults With Sickle Cell Disease
|
Phase 1/Phase 2 | |
Completed |
NCT02565082 -
Evaluation of the Hemostatic Potential in Sickle Cell Disease Patients
|
N/A | |
Withdrawn |
NCT02630394 -
A Pilot Study of Azithromycin Prophylaxis for Acute Chest Syndrome in Sickle Cell Disease
|
Phase 1 | |
Completed |
NCT02620488 -
A Brief Laboratory-Based Hypnosis Session for Pain in Sickle Cell Disease
|
N/A |