Recruiting

Phase 2

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

Sponsor:

Fred Hutchinson Cancer Center

Code:

NCT05996627

Conditions

Chronic Graft Versus Host Disease

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

Belumosudil

Biospecimen Collection

Electronic Health Record Review

Placebo Administration

Study Details

Brief summary:

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.

Conditions

Chronic Graft Versus Host Disease

Study ID

NCT05996627

Start date

Dec 6, 2023

Status verified date

Aug, 2026

Completion date

Nov 30, 2028

Anticipated

Primary completion date

Apr 30, 2028

Anticipated

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

82 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: 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.

placebo comparator: 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.

Interventions

Belumosudil

Given PO

Biospecimen Collection

Undergo blood sample collection

Electronic Health Record Review

Ancillary studies

Placebo Administration

Given PO

Primary outcome measure

Time to start of subsequent systemic immune suppressive treatment for chronic graft versus host disease (cGVHD) [ Time Frame: From first dose of study medication to starting a new systemic immunosuppressive agent for cGVHD therapy, up to 12 months ]

Central Contacts and Locations

Central contacts

Gaby Desatnik

206-667-1356 gdesatni@fredhutch.org

Locations

Moffitt Cancer Center

Recruiting

Tampa, Florida, United States, 33612

Principal Investigator:

Joseph Pidala, MD, PhD

Dana-Farber Cancer Institute

Recruiting

Boston, Massachusetts, United States, 02215

Principal Investigator:

Corey Cutler, MD, MPH

Memorial Sloan Kettering Cancer Center

Recruiting

New York, New York, United States, 10065

Principal Investigator:

Doris Ponce, MD

Fred Hutch/University of Washington Cancer Consortium

Recruiting

Seattle, Washington, United States, 98109

Contacts

Gaby Desatnik

206-667-1356 gdesatni@fredhutch.org

Principal Investigator:

Stephanie Lee, MD, MPH

More Information

Sponsor

Fred Hutchinson Cancer Center

Last update posted

Aug 21, 2026

Last verified

Aug, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Fred Hutchinson Cancer Center on 2026-08-21.