Belumosudil for Graft-Versus-Host Disease

Recruiting at 6 trial locations

Overseen By Miguel-Angel Perales, MD

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Memorial Sloan Kettering Cancer Center

Must not be taking: Corticosteroids, Investigational agents

Disqualifiers: Uncontrolled infection, Cardiovascular conditions, 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

Compensation: Varies

Number of Visits: Unknown

Duration: 1 year

46 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores whether adding a drug called belumosudil can help prevent graft-versus-host disease (GVHD) in people with blood cancer who have undergone a stem cell transplant. Researchers aim to determine if this new approach is safe and effective compared to usual methods. The trial seeks participants with blood cancer in remission who have received a stem cell transplant from a matched donor. Those with stable health conditions, not currently facing uncontrolled infections or certain heart or lung issues, may be suitable for the study. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to important medical 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 you cannot be on certain investigational agents or high-dose corticosteroids. It's best to discuss your specific medications with the trial team.

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

Research has shown that belumosudil has been tested for safety in treating chronic graft-versus-host disease (GVHD), a condition where donor cells attack the recipient's body after a stem cell transplant. Studies have found that belumosudil can help patients while being as safe as the best current treatments.

In one study, most participants tolerated belumosudil well, with side effects usually mild or moderate. Common side effects included tiredness, nausea, and diarrhea, which are typical for many treatments and usually manageable. Serious side effects were less common.

Although belumosudil is still being tested to prevent GVHD, its previous use in treating the chronic form of the disease provides some confidence in its safety for humans. However, anyone considering joining a trial should discuss the possible risks and benefits with their doctor. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for graft-versus-host disease?

Belumosudil is unique because it targets a specific enzyme called ROCK2, which plays a role in the immune response involved in graft-versus-host disease (GVHD). Unlike standard treatments that often involve corticosteroids or immunosuppressants, which can have broad and harsh side effects, Belumosudil offers a more targeted approach. This specificity might lead to fewer side effects and improved outcomes for patients with GVHD, making researchers optimistic about its potential.

What evidence suggests that belumosudil might be an effective treatment for graft-versus-host disease?

Research has shown that belumosudil, the investigational treatment in this trial, offers promising results for treating chronic graft-versus-host disease (cGVHD). This condition occurs when transplanted cells attack the body's own tissues. Studies have found that about 74% of patients, who had tried several other treatments, responded well to belumosudil. Additionally, belumosudil's effects last longer, and patients have tolerated it well over time. These findings suggest that belumosudil could effectively prevent graft-versus-host disease in individuals undergoing stem cell transplants for blood cancer. ^{1 3 6 7 8}

Who Is on the Research Team?

Doris Ponce, MD

Principal Investigator

Memorial Sloan Kettering Cancer Center

Are You a Good Fit for This Trial?

This trial is for individuals with blood cancers who are undergoing a stem cell transplant and at risk of developing graft-versus-host disease (GVHD). Specific eligibility criteria details were not provided, so it's important to contact the study organizers for more information.

Inclusion Criteria

ALT and AST ≤ 3x upper limit of normal

I have had a bone marrow transplant and am currently in remission.

I recently had a stem cell transplant.

Exclusion Criteria

Uncontrolled psychosis, active suicidal ideation, or recent psychiatric hospitalization

I am currently on high-dose corticosteroids.

I do not have an uncontrolled infection or active hepatitis B or C.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive belumosudil as part of a prophylactic regimen to prevent graft-versus-host disease after stem cell transplantation

1 year

Follow-up

Participants are monitored for safety and effectiveness after treatment

1 year

What Are the Treatments Tested in This Trial?

Interventions

• Belumosudil

Trial Overview The study is testing if adding belumosudil to standard treatments can prevent GVHD in patients receiving stem cell transplants. It aims to determine the effectiveness and safety of this potential new prevention strategy.

How Is the Trial Designed?

Group I: Participants with hematologic malignancy in morphologic remission Experimental Treatment 1 Intervention

Participants will be diagnosed with a hematologic malignancy in morphologic remission

Who Is Running the Clinical Trial?

Memorial Sloan Kettering Cancer Center

Lead Sponsor

Trials

1,998

Recruited

602,000+

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/41046308/

Real-world experience of belumosudil and ... - PubMed Overall, belumosudil was associated with high treatment response and survival outcomes. Notably, deeper responses were observed with ongoing ...

2. nature.com nature.com/articles/s41409-025-02554-w

Efficacy and safety of belumosudil for treatment of cGVHD Median failure-free survival (FFS) was not reached, with 6- and 12-month FFS rates of 89.1% and 80.4%, respectively. Nine patients died, mainly ...

3. astctjournal.org astctjournal.org/article/S2666-6367(25)01159-5/fulltext

Analysis of Long-Term Results from the KD025-208 and ... Durability of response was shown through an increase in DOR, FFS, and TTNT. •. Belumosudil was well tolerated in patients with cGVHD after long-term follow-up.

4. ashpublications.org ashpublications.org/bloodadvances/article/doi/10.1182/bloodadvances.2025015832/546987/Efficacy-and-Safety-of-Belumosudil-as-Compared

Efficacy and Safety of Belumosudil as Compared with Best ... Belumosudil increased 6-month overall response rate compared to best available therapy in cGVHD patients with 2-5 prior lines of therapy.

5. sciencedirect.com sciencedirect.com/science/article/pii/S266663672500483X

410 Real World Outcomes of Belumosudil for Treatment ... High overall response rates (ORR) of 74% were seen in patients on the pivotal Rockstar trial. Herein, we report our single-institution experience with using ...

6. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40318736/

Belumosudil for Chronic Graft-Versus-Host Disease - PubMed The median FFS in the mITT population was 15.1 months (range, 11.3 to 20.6 months). The 1- and 2-year FFS rates were 56% and 40%, respectively.

7. astctjournal.org astctjournal.org/article/S2666-6367(25)01520-9/fulltext

Efficacy And Safety Of Belumosudil In The Treatment ... Efficacy And Safety Of Belumosudil In The Treatment Of Refractory Chronic Graft-Versus-Host Disease: A Systematic Review And Meta-analysis.

8. sciencedirect.com sciencedirect.com/science/article/pii/S2666636725011595

Analysis of Long-Term Results from the KD025-208 and ... The long-term results from this pooled analysis of these two phase 2 studies demonstrated belumosudil was associated with durable responses, and it remained ...