Anakinra for Leukemia

(PACER Trial)

Overseen By Kevin O McNerney, MD

Age: < 65

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Ann & Robert H Lurie Children's Hospital of Chicago

Must be taking: Anakinra

Disqualifiers: Pregnancy, Inadequate organ function, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 3 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 30

Duration: 7 days post-CAR T infusion

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores whether anakinra can reduce severe side effects, known as cytokine release syndrome (CRS), in young patients receiving CAR T-cell therapy for B-acute lymphoblastic leukemia (B-ALL). CRS may occur when the immune system becomes overly activated by the treatment. The trial seeks to determine if early administration of anakinra can make CAR T-cell therapy safer and more effective. This study suits children and young adults under 25 battling challenging cases of B-ALL, particularly those with high bone marrow involvement or cancerous cells in the blood. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the chance to be among the first to receive this potentially groundbreaking therapy.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the study team or your doctor.

Is there any evidence suggesting that anakinra is likely to be safe for humans?

Research has shown that anakinra is generally safe for people. In studies involving patients with acute leukemia, anakinra proved safe and even helped improve some symptoms. The most common side effects were mild, such as reactions at the injection site. Other studies also indicate that anakinra is usually well-tolerated by patients. While some side effects have been reported, they are generally manageable and not serious. This suggests that anakinra could be a promising option for treating conditions like cytokine release syndrome (CRS) in leukemia. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Anakinra is unique because it targets leukemia by blocking the activity of interleukin-1, which is a protein that can promote cancer cell growth. Unlike traditional chemotherapy treatments that attack rapidly dividing cells indiscriminately, Anakinra offers a more targeted approach, potentially reducing side effects and improving patient quality of life. Researchers are excited about Anakinra because it represents a novel mechanism of action for leukemia treatment, which could lead to new ways to combat this condition more effectively.

What evidence suggests that anakinra might be an effective treatment for severe CRS in B-ALL patients?

Studies have shown that anakinra, which participants in this trial will receive, can help reduce inflammation in various conditions. Specifically, research suggests it may help with cytokine release syndrome (CRS), a serious side effect of CAR T-cell therapy. One study found that anakinra quickly improved symptoms in patients with CRS. Although more research is needed, these findings suggest that anakinra could effectively manage CRS in patients receiving CAR T-cell therapy for leukemia. ^{2 6 7 8 9}

Are You a Good Fit for This Trial?

This trial is for children and young adults under 25 with B-acute lymphoblastic leukemia (B-ALL) who are undergoing CAR T-cell therapy. They must have a significant presence of cancer cells in their bone marrow or detectable cancer cells in the blood recently.

Inclusion Criteria

My leukemia has returned or didn't respond to treatment.

I (or my guardian) can understand and am willing to sign the consent form.

I am between 1 and 26 years old.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive anakinra pre-emptively to reduce the rate of severe CRS following CAR T-cell therapy

7 days post-CAR T infusion

Daily monitoring during hospitalization and clinical visits within the first 30 days

Follow-up

Participants are monitored for safety and effectiveness after treatment, including CRS and ICANS severity, infection rates, and survival

12 months

What Are the Treatments Tested in This Trial?

Interventions

• Anakinra

Trial Overview The study tests if giving Anakinra before symptoms start can prevent severe cytokine release syndrome after CAR T-cell therapy in patients with high levels of leukemia cells. It's a pilot study where all participants receive Anakinra when they develop a persistent fever.

How Is the Trial Designed?

Group I: Treatment Arm Experimental Treatment 1 Intervention

Anakinra is already approved in European Union, United States for the following indications:

Approved in European Union as Kineret for:

• Rheumatoid arthritis
• Cryopyrin-associated periodic syndromes
• Deficiency of interleukin-1 receptor antagonist
• COVID-19

Approved in United States as Kineret for:

• Rheumatoid arthritis
• Deficiency of interleukin-1 receptor antagonist
• Neonatal-onset multisystem inflammatory disease (NOMID)
• COVID-19

Who Is Running the Clinical Trial?

Ann & Robert H Lurie Children's Hospital of Chicago

Lead Sponsor

Trials

275

Recruited

5,182,000+

National Center for Advancing Translational Sciences (NCATS)

Collaborator

Trials

394

Recruited

404,000+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT04691765?term=anakinra&viewType=Table&rank=1

Anakinra in Previously Untreated Chronic Lymphocytic ... Anakinra will be given daily for seven 4-week cycles based on experience with other immunomodulatory drugs that suggest an average time to best response is ~7 ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC6232613/

Anakinra Therapy for Non-cancer Inflammatory Diseases - PMC The SchS International Registry reports nearly 100% efficacy with anakinra treatment, which leads to clinical improvement within hours and remission within days ...

3. idcmjournal.org idcmjournal.org/wp-content/uploads/2024/03/IDCM-2024-287.pdf

Individualized High Dose Intravenous Anakinra Treatment ... In this study, we aimed to evaluate the outcomes of high-dose intrave- nous anakinra treatment in patients with severe and critically ill COVID- ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11022673/

Improved survival outcomes with anakinra over etoposide ... We aim to compare survival outcomes of adult patients with HLH who were treated with ruxolitinib, anakinra, and etoposide-based therapies.

5. ashpublications.org ashpublications.org/bloodadvances/article/4/13/3123/461283/Clinical-efficacy-of-anakinra-to-mitigate-CAR-T

Clinical efficacy of anakinra to mitigate CAR T-cell therapy ... Despite encouraging data, the potential effects of anakinra on CAR T-cell amplification and function remain to be fully investigated in humans.

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC1798263/

Safety of extended treatment with anakinra in patients ... The most frequent AEs were injection site reactions (122.26 events/100 patient‐years), rheumatoid arthritis progression (67.80 events/100 patient‐years), and ...

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC7280623/

Interleukin‐1 blockade with anakinra in acute leukaemia ... We demonstrate that anakinra is safe in haematology patients and resulted in a clinical improvement in three patients with acute leukaemia and confirmed or ...

8. frontiersin.org frontiersin.org/journals/pharmacology/articles/10.3389/fphar.2025.1483669/full

Post-marketing safety of anakinra and canakinumab: a real ... This study confirms the favorable safety profiles of anakinra and canakinumab, offering critical insights into rational drug usage and safety regulations.

9. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0022356524170589

Comparison of Safety and Biological Efficacy of Anakinra ... There were no differences in the rate of HF hospitalization or cardiovascular death in patients who received anakinra in plastic or glass syringes. Fewer cases ...