Low-Dose Tocilizumab for Acute Chest Syndrome in Sickle Cell Disease

Plain English Summary

Tocilizumab for Acute Chest Syndrome is a Phase 2 clinical trial sponsored by University of Chicago studying Sickle Cell Disease, Acute Chest Syndrome. This trial tests if a low dose of the drug tocilizumab can help treat acute chest syndrome in people with sickle cell disease. It is for adults and adolescents aged 12 and older who have sickle cell disease and are experiencing acute chest syndrome. Participants will receive tocilizumab intravenously (through an IV) and will be monitored for their oxygen levels, clinical outcomes, and lab markers. Alternative treatments for acute chest syndrome may include blood transfusions and supportive care. The trial aims to enroll 200 participants.

Official Summary

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.

Who Can Participate

Here is what you need to know about eligibility for this trial. You can join if you are 12 years or older and have been diagnosed with sickle cell disease. You cannot join if you are pregnant or breastfeeding, have had gene therapy or a stem cell transplant, or are currently in another drug trial. You also cannot join if you have very low white blood cell or platelet counts, severe liver problems, a history of or current tuberculosis, or are taking certain other medications that affect the immune system. This trial is studying Sickle Cell Disease, Acute Chest Syndrome, so participants generally need a confirmed diagnosis. The trial is currently accepting new participants.

What They're Measuring

The main goal is to see how well your oxygen levels improve over a few days after receiving the medication, indicating better breathing and lung function. The specific primary outcome measures are: Time-weighted SaO2/FiO2 ratio (Total of 4 days (Day 0 to Day 4)). These endpoints are how researchers determine whether the treatment is effective and will form the basis of any future regulatory submissions.

About This Phase

This trial is in Phase 2, which tests whether the treatment actually works against the target condition. Phase 2 trials involve 100-300 patients and continue to monitor safety while evaluating effectiveness. This phase often tests different dosages to find the optimal amount. About 33% of Phase 2 drugs advance to Phase 3. If successful, the treatment will move to large-scale Phase 3 trials needed for FDA approval.

Why This Trial Matters

This trial addresses a critical need for better treatments for acute chest syndrome, a life-threatening complication of sickle cell disease, by investigating a drug that targets inflammation. Phase 2 success would typically lead to larger Phase 3 trials needed for regulatory approval. This research targets Sickle Cell Disease, Acute Chest Syndrome, where improved treatment options are needed.

Investor Insight

This trial explores a novel use for an existing drug in a rare but severe condition, potentially opening a new treatment avenue and representing a significant unmet medical need. Phase 2 trials have approximately a 15-20% chance of eventually gaining FDA approval.

Is This Trial Right for Me?

Ask your doctor about the potential benefits and risks of tocilizumab, and what to expect during the trial. Participation involves receiving the study drug and regular monitoring of your health, including blood tests and oxygen levels, while in the hospital. This trial is currently recruiting participants. The trial is being conducted at 1 site. Always discuss clinical trial participation with your healthcare provider before making any decisions. This information is for educational purposes only and is not medical advice.

AI-generated analysis for educational purposes only. This is not medical advice. Discuss clinical trial participation with your doctor. Data sourced from ClinicalTrials.gov.

Study Design

• Study Type: INTERVENTIONAL
• Allocation: RANDOMIZED
• Model: PARALLEL
• Masking: QUADRUPLE
• Enrollment: 200 participants

Interventions

• DRUG: Tocilizumab — Tocilizumab 80 mg IV dose (one time per patient)

Primary Outcomes

• Time-weighted SaO2/FiO2 ratio (Total of 4 days (Day 0 to Day 4))

Secondary Outcomes

• Red cell exchange transfusion rate (Total of 9 days (Day 0 to Day 8))
• Intensive Care Unit (ICU) transfer rate (Total of 9 days (Day 0 to Day 8))
• Length of stay (Up to 3 months (Admission Date to Discharge Date))
• Readmission rate (Total of 29 days (Discharge Date to 28 days after discharge))
• Mortality rate (Total of 29 days (Day 0 to Day 28))

Full 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

Trial Locations

• University of Chicago, Chicago, Illinois, United States

Frequently Asked Questions

Related Conditions

• Sickle Cell Disease
• Acute Chest Syndrome
• Anemia
• Inflammation
• Cytokine Release Syndrome
• Rheumatoid Arthritis
• Autoimmune Diseases
• Vasculitis
• COVID-19
• Immunosuppression

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