What side effects are associated with this type of intervention?

The most common treatments for Graft-versus-Host Disease (GVHD) include corticosteroids and immune modulators such as Belumosudil and cyclosporine. Corticosteroids can cause a range of side effects including increased risk of infections, high blood sugar, hypertension, osteoporosis, and mood swings. Belumosudil, an investigational drug, may have side effects such as diarrhea, liver enzyme elevation, and potential for pancreatitis or bone marrow suppression. Cyclosporine, another immune modulator, can lead to nephrotoxicity, hypertension, and increased susceptibility to infections. These treatments aim to modulate the immune response but come with significant risks that need to be managed carefully.

What prior FDA approvals exist?

The FDA has approved several treatments for Graft-versus-Host Disease (GVHD) that focus on immune modulation. Notable drugs include ruxolitinib, a JAK1/2 inhibitor, approved for steroid-refractory acute GVHD, and ibrutinib, a BTK inhibitor, approved for chronic GVHD after failure of one or more lines of systemic therapy. Additionally, corticosteroids like prednisone are commonly used as first-line treatment for GVHD. These treatments aim to modulate the immune response to reduce the severity of GVHD symptoms.

What other types of research exist?

Promising novel alternatives for the treatment of chronic Graft-versus-Host Disease (cGVHD) in 2024 include: 1) Belumosudil combined with corticosteroids, as studied in a Phase 3 trial for newly diagnosed moderate or severe cGVHD. 2) JAK inhibitors, such as ruxolitinib, which have shown efficacy in reducing symptoms and improving outcomes in cGVHD patients. 3) BTK inhibitors, like ibrutinib, which target B-cell signaling pathways and have been approved for cGVHD treatment. 4) Proteasome inhibitors, such as bortezomib, which are being investigated for their potential to modulate immune responses in cGVHD. These therapies represent promising advancements in the management of cGVHD and should be discussed with a healthcare provider to determine the best treatment approach.

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Belumosudil + Corticosteroids for Chronic Graft Versus Host Disease (ROCKnrol-1 Trial)

Phase 3

Recruiting

Research Sponsored by Sanofi

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Patients must be at least 12 years of age inclusive, at the time of signing the informed consent

Participants who have undergone allogenic HCT with newly diagnosed moderate to severe cGVHD according to NIH consensus diagnosis and staging criteria (2014)

Must not have

Female participants who are pregnant or breastfeeding

Unable to tolerate a prednisone equivalent dose of corticosteroids ≥ 1 mg/kg/day

Timeline

Screening 3 weeks

Treatment Varies

Follow Up until the end of study (up to 5 years since first patient in)

Awards & highlights

Pivotal Trial

Summary

This trial is testing a new treatment for people with moderate or severe chronic GVHD. It targets newly diagnosed patients who may not respond well to existing treatments. The goal is to manage or reduce the symptoms of chronic GVHD.

Who is the study for?

This trial is for people at least 12 years old with a body weight of ≥40kg who have moderate to severe chronic Graft Versus Host Disease (cGVHD) after an allogeneic HCT. They must need systemic corticosteroid treatment and not have had prior cGVHD treatments, except possibly corticosteroids within the last 7 days. Participants should be able to consent and follow contraception guidelines.

What is being tested?

The study tests Belumosudil combined with corticosteroids against a placebo plus corticosteroids in treating newly diagnosed cGVHD. It's a Phase 3 trial where participants are randomly assigned to one of two groups and treated until their condition progresses or they experience unacceptable side effects.

What are the potential side effects?

While specific side effects for Belumosudil aren't listed, common ones from similar medications may include stomach issues, liver enzyme changes, blood pressure variations, fatigue, risk of infection, and potential allergic reactions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am 12 years old or older.

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I have had a stem cell transplant and now have moderate to severe chronic graft-versus-host disease.

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I need steroids for chronic graft-versus-host disease.

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I have not had any treatment for chronic GVHD.

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My body weight is 40kg or more.

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I can sign the consent form myself or have someone who can legally do it for me.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am not pregnant or breastfeeding.

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I cannot tolerate high doses of steroids.

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I haven't used any experimental drugs or devices for this study in the last 28 days or longer.

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My lung function is severely reduced or I have a severe lung condition.

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I have been diagnosed with HIV.

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My white blood cell count is low and I haven't used medication to increase it.

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My kidney function is low, with an eGFR below 30 mL/min.

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I have previously been treated with belumosudil.

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I do not have active hepatitis B or C.

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I cannot swallow pills.

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I need assistance with daily activities due to my health condition.

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My disease has returned after my last bone marrow transplant from a donor.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ until the end of study (up to 5 years since first patient in)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~until the end of study (up to 5 years since first patient in)

This trial's timeline: 3 weeks for screening, Varies for treatment, and until the end of study (up to 5 years since first patient in) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Event-Free Survival (EFS)

Secondary study objectives

Change in patient reported outcome (PRO)

Dose reduction in corticosteroid

Durable overall response rate

+9 more

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: BelumosudilExperimental Treatment3 Interventions

Participants will receive belumosudil 200 mg tablets per os(PO) once daily (QD) per 28-day cycles starting on Day 1 until discontinuation criteria are met or until end of study Note: 200mg two times a day (BID) is used in some cases, when the subject is taking a proton pump inhibitor or a strong CYP3A4 inducer)

Group II: PlaceboPlacebo Group3 Interventions

Participants will receive matching placebo tablets PO QD per 28-day cycles starting on Day 1 until discontinuation criteria are met or until end of study

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Belumosudil

2018

Completed Phase 2

~320

Prednisone

2014

Completed Phase 4

~2500

Prednisolone

2005

Completed Phase 4

~3570

0 / 18Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Graft-versus-Host Disease (GVHD) include corticosteroids and other immunosuppressive agents. Corticosteroids, such as prednisone, suppress the immune system to reduce inflammation and immune response. Agents like belumosudil target specific pathways, such as ROCK2, to inhibit immune cell activation and proliferation. These treatments are crucial for GVHD patients as they help control the immune response, preventing donor immune cells from attacking the recipient's tissues and reducing the risk of severe complications and organ damage.