Chronic Graft Versus Host DiseasePhase 2NCT05996627
Belumosudil for the Pre-emptive Treatment of Patients With Chronic Graft Versus Host Disease
A Phase 2 study testing Belumosudil, Biospecimen Collection and Electronic Health Record Review in people with Chronic Graft Versus Host Disease, run by Fred Hutchinson Cancer Center at 5 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Chronic Graft Versus Host Disease
- What is being tested?
- Belumosudil, Biospecimen Collection and Electronic Health Record Review
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 82 participants
- Where
- 5 sitesUnited States
- Study dates
- 6 Dec 2023to 30 Nov 2028
- Phase
- Phase 2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
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- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
5 sites in 1 country. Contact the study team or a site near you to ask about taking part.
5 sites in 1 country
United States5 sites, 5 recruiting
- Recruiting
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215
Corey Cutler, MD, MPH
- Recruiting
City of Hope
Duarte, California, 91010
Sunmin Park, MD
- Recruiting
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
Doris Ponce, MD
- Recruiting
Fred Hutch/University of Washington Cancer Consortium
Seattle, Washington, 98109
Gaby Desatnik, 206-667-1356, gdesatni@fredhutch.org
Stephanie Lee, MD, MPH
- Recruiting
Moffitt Cancer Center
Tampa, Florida, 33612
Joseph Pidala, MD, PhD
About this study
Official title: Randomized Phase II Study of Belumosudil vs. Placebo for Preemptive Treatment of Chronic Graft Versus Host Disease
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.
Read the detailed description
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
- Chronic Graft Versus Host Disease
- Being tested
- Belumosudil, Biospecimen Collection, Electronic Health Record Review, Placebo Administration
- Medical subject headings
- Bronchiolitis Obliterans Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BelumosudilDrug
Also called KD 025, KD-025, KD025, Rho-associated Coiled-coil Kinase 2 Inhibitor KD025
Given PO
Biospecimen CollectionProcedure or surgery
Also called Biological Sample Collection, Biospecimen Collected, Specimen Collection
Undergo blood sample collection
Electronic Health Record ReviewOther
Ancillary studies
Placebo AdministrationDrug
Given PO
| Study group | Role | What they receive |
|---|---|---|
| Arm I (Belumosudil) | Experimental | 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. Receives: Drug: Belumosudil; Procedure: Biospecimen Collection; Other: Electronic Health Record Review |
| Arm II (Placebo) | Placebo comparator | 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. Receives: Procedure: Biospecimen Collection; Other: Electronic Health Record Review; Drug: Placebo Administration |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time to start of subsequent systemic immune suppressive treatment for chronic graft versus host disease (cGVHD)
Systemic therapies include any systemic agent given for a cGVHD indication, including extracorporeal photopheresis. Will use Gray's test. Point estimates of new systemic immunosuppressive use will be obtained using cumulative incidence estimates.
Measured: From first dose of study medication to starting a new systemic immunosuppressive agent for cGVHD therapy, up to 12 months
Secondary outcomes (3)
Event-free survival
Point estimates will be obtained using the method of Kaplan and Meier and the log-rank test will be used to assess the difference between treatment groups.
Measured: From randomization to death, malignancy relapse or addition of a new systemic immune suppressive therapy, up to 12 months or end of study
Overall survival
Point estimates will be obtained using the method of Kaplan and Meier and the log-rank test will be used to assess the difference between treatment groups.
Measured: Up to 12 months or end of study
Rate of relapse
Point estimates will be obtained using the method of Kaplan and Meier and the log-rank test will be used to assess the difference between treatment groups.
Measured: Up to 12 months or end of study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Aug 2023
- Study start6 Dec 2023
- Primary completion30 Apr 2028 (estimated)
- Study completion30 Nov 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Chronic Graft Versus Host DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 82 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Fred Hutchinson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- Sanofi
- Responsible party
- Sponsor
- Investigators
- Stephanie Lee, MD, MPH, Principal investigator, Fred Hutch/University of Washington Cancer Consortium
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05996627
- Sponsor's protocol ID
- RG1123523
- Other IDs
- NCI-2023-05293, RG1123523, 20163
- Status last verified
- Aug 2026
- First submitted
- 10 Aug 2023
- Last updated
- 17 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No