572 Participants Needed

Tacrolimus/Methotrexate/Ruxolitinib vs Cyclophosphamide/Tacrolimus/Mycophenolate Mofetil for Graft-versus-Host Disease

Recruiting at 27 trial locations
IC
IC
Overseen ByIncyte Corporation Call Center (ex-US)
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human trial

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to determine which treatment better prevents graft-versus-host disease, a condition where donated cells attack the recipient's body after a stem cell transplant. It compares two treatment combinations: one with Tacrolimus, Methotrexate, and Ruxolitinib (a Janus kinase inhibitor), and another with Cyclophosphamide, Tacrolimus, and Mycophenolate Mofetil. Ideal participants have conditions such as acute leukemia or certain types of lymphoma and are planning a specific type of stem cell transplant. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants the opportunity to contribute to potentially groundbreaking treatment advancements.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. However, it mentions that treatment with any investigational medications is not allowed during the study.

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

Research has shown that the combination of Tacrolimus, Methotrexate, and Ruxolitinib is generally well-tolerated for preventing graft-versus-host disease (GVHD). One study found that taking Ruxolitinib at a dose of 5 mg twice a day with Tacrolimus and Methotrexate is safe and effective. Patients reported it as helpful in protecting against GVHD without major safety issues.

Other studies have demonstrated that Ruxolitinib is safe during and after hematopoietic cell transplantation, a procedure to replace damaged bone marrow. It was associated with low rates of serious side effects.

These findings suggest that the treatment is likely safe for most people, based on previous research and patient experiences. However, individual reactions can vary, so discussing any concerns with the medical team running the trial is important.12345

Why are researchers excited about this trial's treatments?

Researchers are excited about Tacrolimus/Methotrexate/Ruxolitinib for graft-versus-host disease because it combines the strengths of three potent drugs in a novel way. Unlike standard treatments like cyclophosphamide, mycophenolate mofetil, and tacrolimus, this combination includes Ruxolitinib, a JAK inhibitor that targets specific cellular pathways involved in inflammation and immune response. This mechanism may offer more effective control of the disease with fewer side effects. Additionally, by using Tacrolimus and Methotrexate together with Ruxolitinib, the treatment could potentially reduce the severity and duration of symptoms faster than current options.

What evidence suggests that this trial's treatments could be effective for graft-versus-host disease?

In this trial, participants will receive different treatment combinations for graft-versus-host disease (GVHD). One group will receive Tacrolimus, Methotrexate, and Ruxolitinib, a combination that previous studies have shown to be promising. Research indicates that adding Ruxolitinib to Tacrolimus and Methotrexate is effective and safe for preventing GVHD, with an overall response rate of up to 85% in some cases. Another group will receive Cyclophosphamide, Tacrolimus, and Mycophenolate Mofetil. Tacrolimus and Methotrexate are already known for their roles in managing GVHD after transplantation, and Ruxolitinib is especially noted for its success in treating GVHD that doesn't respond to other treatments. This suggests that the Tacrolimus, Methotrexate, and Ruxolitinib combination could be a strong option for managing GVHD symptoms.12346

Who Is on the Research Team?

IM

Incyte Medical Monitor

Principal Investigator

Incyte Corporation

Are You a Good Fit for This Trial?

This trial is for adults over 18 with certain blood cancers like acute leukemia, myelodysplasia, or lymphoma without high levels of cancer cells in the blood or bone marrow. They must have a functioning heart and kidneys. Participants need a sibling or unrelated donor who matches specific genetic markers and agrees to donate stem cells.

Inclusion Criteria

My sibling, child, or parent is a perfect match for my stem cell transplant.
My leukemia has minimal presence in my blood and bone marrow.
My blood disorder has less than 10% immature cells in my bone marrow.
See 6 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Conditioning

Participants undergo non-myeloablative/reduced intensity conditioning prior to transplantation

1-2 weeks

Transplantation

Participants receive allogeneic peripheral blood stem cell transplantation

1 day

Treatment

Participants receive Tacrolimus/Methotrexate/Ruxolitinib or Post-Transplant Cyclophosphamide/Tacrolimus/Mycophenolate Mofetil

Up to 24 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

Up to 24 months

What Are the Treatments Tested in This Trial?

Interventions

  • Cyclophosphamide
  • Methotrexate
  • Mycophenolate mofetil
  • Ruxolitinib
  • Tacrolimus
Trial Overview The study compares two drug combinations after non-myeloablative/reduced intensity conditioning allogeneic peripheral blood stem cell transplantation. One group receives Tacrolimus/Methotrexate/Ruxolitinib, while the other gets Cyclophosphamide/Tacrolimus/Mycophenolate Mofetil.
How Is the Trial Designed?
4Treatment groups
Experimental Treatment
Active Control
Group I: Main Study Group A: Tac/MTX/RuxolitnibExperimental Treatment3 Interventions
Group II: Dose Finding Run-In Group 2: Tac/MTX/Ruxolitnib Dose 2Experimental Treatment3 Interventions
Group III: Dose Finding Run-In Group 1: Tac/MTX/Ruxolitnib Dose 1Experimental Treatment3 Interventions
Group IV: Main Study Group B: PTCy/Tac/MMFActive Control3 Interventions

Find a Clinic Near You

Who Is Running the Clinical Trial?

Incyte Corporation

Lead Sponsor

Trials
408
Recruited
66,800+
Steven Stein profile image

Steven Stein

Incyte Corporation

Chief Medical Officer since 2015

MD from University of Witwatersrand

Hervé Hoppenot profile image

Hervé Hoppenot

Incyte Corporation

Chief Executive Officer since 2014

MBA from ESSEC Business School

National Institutes of Health (NIH)

Collaborator

Trials
2,896
Recruited
8,053,000+

National Cancer Institute (NCI)

Collaborator

Trials
14,080
Recruited
41,180,000+

National Heart, Lung, and Blood Institute (NHLBI)

Collaborator

Trials
3,987
Recruited
47,860,000+

Blood and Marrow Transplant Clinical Trials Network

Collaborator

Trials
51
Recruited
14,600+

Citations

Steroid Refractory Chronic Graft-Versus-Host Disease: Cost ...ORR rates were higher with a median of 50% and range of 30% to 85%. Response rates were not statistically significant between the agents, supporting ...
A Randomized, Multicenter, Phase III Trial of Tacrolimus/ ...Study Design: The study is designed as a randomized, Phase III, open label multicenter trial comparing two acute graft-versus-host disease.
Safe and effective addition of ruxolitinib to graft-versus-host ...In the meantime, this report suggests that Rux 5 mg twice daily is a safe and effective GVHD prophylactic dose when combined with Tac/MTX in ...
Will Ruxolitinib Emerge as an Alternative to Post-Transplant ...Prior clinical trials confirmed RUX's ability to induce high response rates in resistant GVHD cases with manageable hematologic toxicities.2-4.
NCT06615050 | A Study of Tacrolimus/Methotrexate/ ...A Randomized, Multicenter, Phase III Trial of Tacrolimus/Methotrexate/Ruxolitinib Versus Post-Transplant Cyclophosphamide/Tacrolimus/Mycophenolate Mofetil ...
Phase II Study of Peri- and Post-Transplant Ruxolitinib for ...In summary, continued ruxolitinib administration during and after HCT demonstrates safety and tolerability, and is associated with low rates of ...
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