Belumosudil treatment for chronic graft versus host disease
Randomized Phase II Study of Belumosudil vs. Placebo for Preemptive Treatment of Chronic Graft Versus Host Disease
This study is testing if a new drug called belumosudil can help people with chronic graft versus host disease feel better compared to a placebo.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 82 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Fred Hutchinson Cancer Center Academic / other |
| Drugs / interventions | chemotherapy, prednisone |
| Locations | 4 sites (Tampa, Florida and 3 other locations) |
| Trial ID | NCT05996627 on ClinicalTrials.gov |
What this trial studies
This phase II trial evaluates the effectiveness of belumosudil compared to a placebo in patients suffering from chronic graft versus host disease (cGVHD), a significant complication following stem cell transplantation. Participants are randomly assigned to receive either belumosudil or a placebo for 28-day cycles, with a total of 11 cycles followed by a tapering phase. The study aims to assess the drug's ability to mitigate immune responses associated with cGVHD and potentially reduce the need for additional immunosuppressive therapies. Blood samples will be collected for analysis throughout the study, and patients will be monitored for up to 18 months post-treatment.
Who should consider this trial
Good fit: Ideal candidates are adults aged 18 and older with a clinical diagnosis of chronic graft versus host disease and at least one distinctive manifestation of the condition.
Not a fit: Patients who have recently started new immunosuppressive therapies or have only eye involvement without confirmation from an ophthalmologist may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve the management of chronic graft versus host disease and enhance patients' quality of life.
How similar studies have performed: Other studies have explored treatments for chronic graft versus host disease, but the specific use of belumosudil as a selective ROCK2 inhibitor represents a novel approach.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Tampa, Florida and 3 other locations
- Moffitt Cancer Center — Tampa, Florida, United States (Recruiting)
- Dana-Farber Cancer Institute — Boston, Massachusetts, United States (Recruiting)
- Memorial Sloan Kettering Cancer Center — New York, New York, United States (Recruiting)
- Fred Hutch/University of Washington Cancer Consortium — Seattle, Washington, United States (Recruiting)
Study contacts
- Principal investigator: Stephanie Lee, MD, MPH — Fred Hutch/University of Washington Cancer Consortium
- Study coordinator: Gaby Desatnik
- Email: gdesatni@fredhutch.org
- Phone: 206-667-1356
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.