Tocilizumab for Graft-versus-Host Disease Prevention

No longer recruiting at 1 trial location

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Memorial Sloan Kettering Cancer Center

Must not be taking: Checkpoint inhibitors

Disqualifiers: Myelofibrosis, 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 adding the drug Tocilizumab to standard treatment can help prevent graft-versus-host disease (GVHD) after stem cell transplants. GVHD occurs when transplanted cells attack the recipient's body, causing complications. Tocilizumab blocks a protein that causes inflammation, which often rises after transplants. Individuals with certain types of leukemia or lymphoma at high risk of relapse might be suitable for this trial. As a Phase 2 trial, the research measures the treatment's effectiveness in an initial, smaller group of participants.

Do I have to stop taking my current medications for the trial?

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 to get specific guidance.

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

Research has shown that tocilizumab, the treatment under study, is generally well-tolerated. In one study, only one out of 29 patients developed a specific gut issue related to GVHD when tocilizumab was used for prevention. This suggests the drug might help prevent such problems. Tocilizumab is already approved for treating some conditions, indicating that its safety is well-understood. However, since this is a Phase 2 trial, while the drug's safety appears promising, more research is needed to fully confirm its safety for this specific use. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Tocilizumab is unique because it targets the interleukin-6 (IL-6) receptor, which plays a key role in the inflammatory response associated with graft-versus-host disease (GVHD). Unlike the standard treatments for GVHD, such as corticosteroids or calcineurin inhibitors which broadly suppress the immune system, tocilizumab specifically blocks the signaling of IL-6, potentially reducing inflammation more precisely and with fewer side effects. Researchers are excited about tocilizumab because it could offer a more targeted approach to prevent GVHD, potentially improving outcomes for patients with high-risk hematologic malignancies.

What evidence suggests that Tocilizumab might be an effective treatment for GVHD?

Research has shown that tocilizumab, the investigational treatment in this trial, might help prevent graft-versus-host disease (GVHD) by reducing inflammation. Tocilizumab blocks a protein called interleukin-6, which increases during inflammation. Studies indicate that tocilizumab shows promise in preventing GVHD, particularly in the lower digestive system. It has also effectively treated GVHD cases unresponsive to steroids. Overall, early evidence suggests that tocilizumab could make transplants safer by lowering the risk of GVHD. ^{6 7 8 9 10}

Who Is on the Research Team?

Ioannis Politikos, MD

Principal Investigator

Memorial Sloan Kettering Cancer Center

Are You a Good Fit for This Trial?

This trial is for people with certain blood cancers or disorders who haven't had recent immunosuppressive chemo, stem cell transplants within specific time frames, or high-dose radiation. They need to be in good health otherwise, not pregnant or breastfeeding, and able to follow the study plan.

Inclusion Criteria

I have acute myelogenous leukemia.

My condition is MDS/MPD, but not myelofibrosis.

My blood disorder has genetic markers indicating a high risk of turning into acute leukemia.

Exclusion Criteria

I do not have an active, uncontrolled infection at the time of transplantation.

I haven't had immunosuppressive chemotherapy in the last 3 months.

I have received radiation therapy with a dose of 400cGy or more.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Transplantation and Treatment

Participants receive a double unit cord blood transplantation and a dose of tocilizumab to prevent GVHD

6 weeks

Follow-up

Participants are monitored for safety and effectiveness after transplantation and treatment

12 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Tocilizumab

Trial Overview The trial tests if Tocilizumab can make cord blood transplants safer by lowering the risk of Graft Versus Host Disease (GVHD). It's given alongside standard GVHD prevention treatments like Mycophenolate Mofetil and Cyclosporine.

How Is the Trial Designed?

Group I: Adult Participants With High Risk Hematologic Malignancies Experimental Treatment 5 Interventions

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?

Memorial Sloan Kettering Cancer Center

Lead Sponsor

Trials

1,998

Recruited

602,000+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC4698359/

Tocilizumab for steroid refractory acute graft-versus-host ... Tocilizumab is a humanized anti-IL-6 receptor monoclonal antibody that has evidence of activity in patients with steroid refractory (SR) GVHD.

2. clinicaltrials.gov clinicaltrials.gov/study/NCT01475162

Tocilizumab for Treatment of Steroid Refractory Acute Graft ... This trial designed to evaluate the toxicity and efficacy of tocilizumab in the treatment of steroid refractory acute graft versus host disease (GVHD). Detailed ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC5865423/

Tocilizumab, tacrolimus and methotrexate for the ... We conclude that tocilizumab has promising activity in preventing acute graft-versus-host disease, particularly in the lower gastrointestinal tract, and ...

4. haematologica.org haematologica.org/article/view/10798

host disease with tocilizumab prophylaxis is accompanied ... In summary, we conclude that an extended course of tocilizumab administration is effective for the prevention of lower GI-tract GvHD, and that loss of microbial ...

5. sciencedirect.com sciencedirect.com/science/article/pii/S1083879111002862

Tocilizumab for the Treatment of Steroid Refractory Graft- ... These results indicate that tocilizumab has activity in the treatment of steroid refractory GVHD and warrants further investigation as a therapeutic option for ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC3716013/

Tocilizumab for the Treatment of Steroid Refractory Graft- ... These results indicate that tocilizumab has activity in the treatment of steroid refractory GVHD and warrants further investigation as a therapeutic option for ...

7. clinicaltrials.gov clinicaltrials.gov/study/NCT01475162

Tocilizumab for Treatment of Steroid Refractory Acute Graft ... This trial designed to evaluate the toxicity and efficacy of tocilizumab in the treatment of steroid refractory acute graft versus host disease (GVHD). Detailed ...

8. uhcprovider.com uhcprovider.com/content/dam/provider/docs/public/policies/medicaid-comm-plan/tocilizumab-cs.pdf

Tocilizumab (Actemra®, Tofidence™, and Tyenne®) Acute Graft-Versus-Host Disease (GVHD). Tocilizumab is proven and medically necessary for the treatment of acute graft-versus-host disease (GVHD) when all of ...

9. haematologica.org haematologica.org/article/view/haematol.2022.281309

host disease with tocilizumab prophylaxis is accompanied ... This study demonstrated that tocilizumab prophylaxis was associated with a low incidence of GI-tract GvHD as only one of 29 patients developed ...

10. modahealth.com modahealth.com/pdfs/med_criteria/tocilizumab.pdf

Tocilizumab: Actemra®; Tofidence™ Acute Graft Versus Host Disease (aGVHD) 21-23,29. • Response to therapy with an improvement in one or more of the following: o Clinician ...