Belumosudil for the Pre-emptive Treatment of Patients With Chronic Graft Versus Host Disease

NCT ID: NCT05996627

Last Updated: 2025-09-02

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

82 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-12-06

Study Completion Date

2027-12-31

Brief Summary

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This phase II trial compares the effect of belumosudil to a placebo in treating patients with chronic graft versus host disease. Chronic graft versus host disease remains a major complication of stem cell transplantation and can involve multiple organ systems. Belumosudil is a ROCK2 selective inhibitor that works to reduce the immune system response causing the chronic graft versus host disease. Giving belumosudil may better treat patients with chronic graft versus host disease and prevent the need for starting additional immune suppressive medications.

Detailed Description

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OUTLINE: Patients are randomized to 1 of 2 arms.

ARM I: Patients receive belumosudil orally (PO) daily (QD) or twice daily (BID) if taken with strong CYP3A inducers or proton pump inhibitors for days 1 through 28 of each cycle. Cycles repeat every 28 days for a total of 11 cycles, followed by one cycle of tapering prior to discontinuation, in the absence of disease progression or unacceptable toxicity.

ARM II: Patients receive a placebo PO QD or BID if taken with strong CYP3A inducers or proton pump inhibitors for days 1 through 28 of each cycle. Cycles repeat every 28 days for a total of 11 cycles, followed by one cycle of tapering prior to discontinuation, in the absence of disease progression or unacceptable toxicity.

Patients undergo blood sample collection on study.

After completion of study treatment, patients are followed up at 30 days, at 60 days if 12 cycles are completed, and then up to 18 months.

Conditions

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Chronic Graft Versus Host Disease

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators
The study will be conducted in a double-blinded fashion

Study Groups

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Arm I (Belumosudil)

Patients receive belumosudil PO QD or BID if taken with strong CYP3A inducers or proton pump inhibitors for days 1 through 28 of each cycle. Cycles repeat every 28 days for a total of 11 cycles, followed by one cycle of tapering prior to discontinuation, in the absence of disease progression or unacceptable toxicity. Patients undergo blood sample collection on study.

Group Type EXPERIMENTAL

Belumosudil

Intervention Type DRUG

Given PO

Biospecimen Collection

Intervention Type PROCEDURE

Undergo blood sample collection

Electronic Health Record Review

Intervention Type OTHER

Ancillary studies

Arm II (Placebo)

Patients receive a placebo PO QD or BID if taken with strong CYP3A inducers or proton pump inhibitors for days 1 through 28 of each cycle. Cycles repeat every 28 days for a total of 11 cycles, followed by one cycle of tapering prior to discontinuation, in the absence of disease progression or unacceptable toxicity. Patients undergo blood sample collection on study.

Group Type PLACEBO_COMPARATOR

Biospecimen Collection

Intervention Type PROCEDURE

Undergo blood sample collection

Electronic Health Record Review

Intervention Type OTHER

Ancillary studies

Placebo Administration

Intervention Type DRUG

Given PO

Interventions

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Belumosudil

Given PO

Intervention Type DRUG

Biospecimen Collection

Undergo blood sample collection

Intervention Type PROCEDURE

Electronic Health Record Review

Ancillary studies

Intervention Type OTHER

Placebo Administration

Given PO

Intervention Type DRUG

Other Intervention Names

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KD 025 KD-025 KD025 Rho-associated Coiled-coil Kinase 2 Inhibitor KD025 ROCK-II Inhibitor KD025 ROCK2 Inhibitor KD025 SLx 2119 SLx-2119 SLx2119 Biological Sample Collection Biospecimen Collected Specimen Collection

Eligibility Criteria

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Inclusion Criteria

* At least one diagnostic or distinctive cGVHD manifestation(s), with a clinical diagnosis of cGVHD,but patients do not need to meet National Institute of Health (NIH) criteria for cGVHD
* If eye involvement only, cGVHD must be confirmed on exam by an ophthalmologist or optometrist
* No new immune suppressive therapy added within preceding 2 weeks prior to study enrollment for any indication

* Continuation of agents previously given as either GVHD prophylaxis or acute/late acute GVHD therapy are permitted. Modification of dose of these agents for targeting of therapeutic drug levels is permitted, as are decreases in existing prednisone or prednisone equivalent dose based on routine clinical tapering practices. Increases in prednisone or prednisone equivalents are not allowed in the 2 weeks prior to enrollment
* Age 18 and older
* Karnofsky performance score \>= 70
* Able to take oral medications
* Signed informed consent
* Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) =\< 3 x upper limit of normal (ULN)
* Total bilirubin =\< 1.5 x ULN, unless due to Gilbert's disease
* Glomerular filtration rate (estimated glomerular filtration rate \[eGFR\]) \>= 30 mL/min/1.73 m\^2
* Female subjects of childbearing potential have a negative serum or urine pregnancy test at screening. Females of childbearing potential are defined as sexually mature females without prior hysterectomy or who have had any evidence of menses in the past 12 months. However, females who have been amenorrheic for 12 or more months are still considered to be of childbearing potential if the amenorrhea is possibly due to prior chemotherapy, anti-estrogens, or ovarian suppression
* Sexually active females of childbearing potential enrolled in the study must agree to use two forms of accepted methods of contraception during the course of the study and for 3 months after their last dose of study drug. Effective birth control includes:

* Intrauterine device (IUD) plus one barrier method
* Stable doses of hormonal contraception for at least 3 months (eg, oral, injectable, implant, transdermal) plus one barrier method
* 2 barrier methods. Effective barrier methods are male or female condoms, diaphragms, and spermicides (creams or gel that contain a chemical to kill sperm)
* Surgical sterilization (tubal ligation)
* A vasectomized partner
* For male subjects who are sexually active and who are partners of females of childbearing potential: Agreement to use two forms of contraception as per above and to not donate sperm during the treatment period and for at least 3 months after the last dose of study drug
* No evidence of active malignancy

Exclusion Criteria

* Any systemic immune suppressive treatment for cGVHD (topical or local therapies are allowed)
* Plan to start systemic immune suppressive therapy for cGVHD or increase steroid dose within 14 days after planned start of study medication
* 0.25 mg/kg/day or higher prednisone or prednisone equivalent dose at time of screening
* History of non-compliance that in the investigator's opinion would interfere with study participation
* Uncontrolled psychiatric illness
* Female subject who is pregnant or breast feeding
* Previous therapy with belumosudil
* Known allergy/sensitivity to belumosudil or any other ROCK2 inhibitor
* Treatment with another investigational agent within 28 days (or 5 half-lives, whichever is greater) of enrollment
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Sanofi

INDUSTRY

Sponsor Role collaborator

Fred Hutchinson Cancer Center

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Stephanie Lee, MD, MPH

Role: PRINCIPAL_INVESTIGATOR

Fred Hutch/University of Washington Cancer Consortium

Locations

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Moffitt Cancer Center

Tampa, Florida, United States

Site Status RECRUITING

Dana-Farber Cancer Institute

Boston, Massachusetts, United States

Site Status RECRUITING

Memorial Sloan Kettering Cancer Center

New York, New York, United States

Site Status RECRUITING

Fred Hutch/University of Washington Cancer Consortium

Seattle, Washington, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Gaby Desatnik

Role: CONTACT

206-667-1356

Facility Contacts

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Gaby Desatnik

Role: primary

206-667-1356

Other Identifiers

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NCI-2023-05293

Identifier Type: REGISTRY

Identifier Source: secondary_id

RG1123523

Identifier Type: OTHER

Identifier Source: secondary_id

20163

Identifier Type: OTHER

Identifier Source: secondary_id

RG1123523

Identifier Type: -

Identifier Source: org_study_id

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