Tocilizumab for Blood Cancer Post-Transplant Care

Overseen By Meredith Mullane, RN

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Weill Medical College of Cornell University

Disqualifiers: Uncontrolled infection, Hepatitis, HIV, Pregnancy, others

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

Prior Safety Data This treatment has passed at least one previous human trial

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Approved in 6 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests whether tocilizumab, a medication that blocks part of the immune system, can safely replace another drug used during a specific type of blood cancer transplant. The researchers aim to determine if this switch aids immune system recovery and reduces cancer recurrence while still preventing transplant complications. Individuals with certain blood cancers, such as relapsed leukemia or multiple myeloma, who require a transplant and lack an immediate donor match, might be suitable candidates for this study. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of participants.

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 trial coordinators or your doctor.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that tocilizumab is generally safe for people. In past studies, patients who took tocilizumab did not experience major short-term side effects. For example, when used to treat cytokine release syndrome—a condition with fever and inflammation—after certain transplants, tocilizumab did not cause serious problems. This indicates its safety for short-term use.

Additionally, the FDA has approved tocilizumab for other conditions, further supporting its safety. This approval confirms it has been tested and found safe for those uses. However, discussing any concerns with a healthcare provider is always advisable. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatment?

Tocilizumab is unique because it offers a fresh approach to post-transplant care in blood cancer patients. Unlike traditional treatments that often focus on broad immunosuppression, Tocilizumab specifically targets the interleukin-6 (IL-6) pathway, which is a key player in inflammation and immune response. This targeted action could help reduce complications like graft-versus-host disease (GVHD) more effectively. Researchers are excited about this treatment because it represents a more precise way to manage the body's immune response after a transplant, potentially leading to better outcomes and fewer side effects compared to conventional therapies.

What evidence suggests that tocilizumab could be an effective treatment for blood cancer post-transplant care?

Research has shown that tocilizumab, which participants in this trial may receive, can improve outcomes after stem cell transplants. In one study, patients received tocilizumab before their transplant to prevent graft-versus-host disease (GVHD), where new immune cells attack the body. The results were promising, showing it might reduce complications. Another study on different conditions found high survival rates, suggesting tocilizumab's effectiveness in protecting transplants. Overall, these findings suggest that tocilizumab could aid post-transplant care for blood cancer patients by improving immune recovery and reducing the risk of relapse. ^{1 6 7 8 9}

Who Is on the Research Team?

Alexandra Gomez Arteaga, MD

Principal Investigator

Weill Medical College of Cornell University

Are You a Good Fit for This Trial?

Adults with certain blood cancers or disorders needing a bone marrow transplant, who can't find a matching donor quickly. They should be in relatively good health with decent organ function and no severe allergies to tocilizumab or similar drugs. Pregnant women, those with uncontrolled infections, HIV, or serious liver issues are excluded.

Exclusion Criteria

Life expectancy is severely limited by concomitant illness or uncontrolled infection, Evidence of chronic active hepatitis or cirrhosis, Uncontrolled HIV disease, Pregnancy or lactation, History of complicated diverticulitis, including fistulae, abscess formation or gastrointestinal perforation, History of allergic reactions attributed to compounds of similar chemical or biological composition as tocilizumab, including known allergies to Chinese hamster ovary cell products or other recombinant human or humanized antibodies.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Conditioning and Transplantation

Participants undergo conditioning with fludarabine, melphalan, total body irradiation, and receive tocilizumab and potentially anti-thymocyte globulin (ATG) as part of the transplant regimen

7 days

Daily visits for conditioning regimen

Post-Transplant Monitoring

Participants are monitored for successful neutrophil engraftment and absence of second nadir

3 weeks

Frequent visits for monitoring engraftment

Follow-up

Participants are monitored for progression-free survival, overall survival, and graft-versus-host disease

5 years

What Are the Treatments Tested in This Trial?

Interventions

• Tocilizumab

Trial Overview

The trial is testing if replacing anti-thymocyte globulin (ATG) with tocilizumab in the pre-transplant treatment regimen can improve immune recovery and reduce relapse while maintaining low graft failure and GVHD rates. It includes Fludarabine, Total Body Irradiation, Melphalan as part of the conditioning.

How Is the Trial Designed?

Group I: ATG Group IV Experimental Treatment 4 Interventions

* Fludarabine 30mg/m2 administered on Day -7 through Day -3 of transplant conditioning regimen (if under 60 years old), or on Day -5 through Day -3 of transplant conditioning regimen (if over 60 years old) * Melphalan 140 mg/m2 administered on Day -2 of transplant conditioning regimen. * Total Body Irradiation 2 Gray administered on Day -4, Day -3 of transplant conditioning regimen. * Tocilizumab 8 mg/kg administered on Day -1 of transplant conditioning regimen.

Group II: ATG Group III Experimental Treatment 5 Interventions

Anti-thymocyte Globulin (ATG) 1.5 mg/kg administered on Day -5 of the transplant conditioning regimen. * Fludarabine 30mg/m2 administered on Day -7 through Day -3 of transplant conditioning regimen (if under 60 years old), or on Day -5 through Day -3 of transplant conditioning regimen (if over 60 years old) * Melphalan 140 mg/m2 administered on Day -2 of transplant conditioning regimen. * Total Body Irradiation 2 Gray administered on Day -4, Day -3 of transplant conditioning regimen. * Tocilizumab 8 mg/kg administered on Day -1 of transplant conditioning regimen.

Group III: ATG Group II Experimental Treatment 5 Interventions

Anti-thymocyte Globulin (ATG) 1.5 mg/kg administered on Day -5, and Day -3 of the transplant conditioning regimen. * Fludarabine 30mg/m2 administered on Day -7 through Day -3 of transplant conditioning regimen (if under 60 years old), or on Day -5 through Day -3 of transplant conditioning regimen (if over 60 years old) * Melphalan 140 mg/m2 administered on Day -2 of transplant conditioning regimen. * Total Body Irradiation 2 Gray administered on Day -4, Day -3 of transplant conditioning regimen. * Tocilizumab 8 mg/kg administered on Day -1 of transplant conditioning regimen.

Group IV: ATG Group I Experimental Treatment 5 Interventions

Anti-thymocyte Globulin (ATG) 1.5 mg/kg administered on Day -5, Day -3 and Day -1 of the transplant conditioning regimen. * Fludarabine 30mg/m2 administered on Day -7 through Day -3 of transplant conditioning regimen (if under 60 years old), or on Day -5 through Day -3 of transplant conditioning regimen (if over 60 years old) * Melphalan 140 mg/m2 administered on Day -2 of transplant conditioning regimen. * Total Body Irradiation 2 Gray administered on Day -4, Day -3 of transplant conditioning regimen. * Tocilizumab 8 mg/kg administered on Day -1 of transplant conditioning regimen.

Tocilizumab is already approved in European Union, United States, Canada, Japan for the following indications:

Approved in European Union as Actemra for:

• Rheumatoid Arthritis
• Systemic Juvenile Idiopathic Arthritis
• Polyarticular Juvenile Idiopathic Arthritis
• Giant Cell Arteritis
• Cytokine Release Syndrome
• COVID-19

Approved in United States as Actemra for:

• Rheumatoid Arthritis
• Systemic Juvenile Idiopathic Arthritis
• Polyarticular Juvenile Idiopathic Arthritis
• Giant Cell Arteritis
• Cytokine Release Syndrome
• COVID-19

Approved in Canada as Actemra for:

• Rheumatoid Arthritis
• Systemic Juvenile Idiopathic Arthritis
• Polyarticular Juvenile Idiopathic Arthritis
• Giant Cell Arteritis
• Cytokine Release Syndrome
• COVID-19

Approved in Japan as Actemra for:

• Rheumatoid Arthritis
• Systemic Juvenile Idiopathic Arthritis
• Polyarticular Juvenile Idiopathic Arthritis
• Giant Cell Arteritis
• Cytokine Release Syndrome
• COVID-19

Who Is Running the Clinical Trial?

Weill Medical College of Cornell University

Lead Sponsor

Trials

1,103

Recruited

1,157,000+

Published Research Related to This Trial

Tocilizumab is an effective treatment for systemic and polyarticular juvenile idiopathic arthritis (JIA) in children aged 2 years and older, showing significant improvement in patient responses compared to placebo in two phase III trials involving patients aged 2-17 years.

The drug was generally well tolerated, with infections being the most common adverse events; however, there was an approximately 11% annual risk of serious infections, indicating the need for monitoring during treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Tocilizumab: a review of its use in the treatment of juvenile idiopathic arthritis. Frampton, JE.[2021]

In a study of 188 patients aged 2-17 with polyarticular-course juvenile idiopathic arthritis (JIA), tocilizumab (TCZ) demonstrated high efficacy over 2 years, with JIA-ACR50/70/90 response rates of 80.3%, 77.1%, and 59.6% respectively, and over half of the patients achieving inactive disease by week 104.

The safety profile of TCZ was consistent with previous reports, with adverse event rates of 406.5 per 100 patient-years and serious infections at 5.2 per 100 patient-years, indicating that TCZ is a relatively safe option for long-term treatment of JIA.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and Safety of Tocilizumab for Polyarticular-Course Juvenile Idiopathic Arthritis in the Open-Label Two-Year Extension of a Phase III Trial. Brunner, HI., Ruperto, N., Zuber, Z., et al.[2021]

Tocilizumab, an interleukin-6 receptor antagonist, has shown significant clinical improvements in rheumatoid arthritis patients at doses greater than 4 mg/kg, with the most effective dose being 8 mg/kg, which also reduces joint damage as seen in radiographic measures.

While tocilizumab is effective, it is associated with common adverse effects such as liver function abnormalities, infections, and neutropenia, highlighting the need for further research to better understand its safety profile and optimal use in treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Tocilizumab: an interleukin-6 receptor inhibitor for the treatment of rheumatoid arthritis. Plushner, SL.[2016]

Tocilizumab is a humanized monoclonal antibody that targets the interleukin-6 receptor, showing efficacy in treating conditions like Castleman disease and various types of arthritis, with approval in Japan and the European Union for these uses.

The drug is currently under review by the US FDA for moderate-to-severe rheumatoid arthritis and has potential applications for other IL-6 related disorders, such as Crohn's disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Tocilizumab. Venkiteshwaran, A.[2021]

In a study of 53 patients with rheumatoid arthritis, tocilizumab (TCZ) demonstrated high effectiveness, with 75.5% achieving remission or low disease activity at 24 weeks and 87.3% at 52 weeks.

The switch from intravenous to subcutaneous administration of TCZ did not affect its effectiveness, and the treatment was found to be safe with a low adverse event rate of 13.6 events per 100 patient-years.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Analysis of effectiveness, safety and optimization of tocilizumab in a cohort of patients with rheumatoid arthritis in clinical practice. Mena-Vázquez, N., Manrique-Arija, S., Rojas-Giménez, M., et al.[2022]

In a 2-year study involving 556 patients with active rheumatoid arthritis, tocilizumab (TCZ) combined with methotrexate was effective in achieving sustained remission, with 50.4% of patients able to discontinue TCZ after reaching this state.

While most patients (84.0%) who stopped TCZ experienced a flare in their condition, they responded well to reintroduction of the drug, and minimal radiographic progression was observed, indicating that TCZ is both effective and relatively safe for long-term management of rheumatoid arthritis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical and radiographic outcomes at 2 years and the effect of tocilizumab discontinuation following sustained remission in the second and third year of the ACT-RAY study. Huizinga, TW., Conaghan, PG., Martin-Mola, E., et al.[2022]

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