A Study of Belumosudil in People at Risk of Developing Graft-Versus-Host Disease After a Stem Cell Transplant

NCT07006506 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: 25-033
• Study Type: interventional
• Target: 46
• Locations: 1 country
• Sites: 8

Brief Summary

The purpose of this study is to find out whether adding belumosudil to a usual approach for reducing the risk of graft-versus-host disease (GVHD) may be an effective GVHD prevention approach for people with blood cancer who have a stem cell transplant. The investigators will also look at the safety of the study approach.

Conditions

Graft Vs Host Disease; Graft Versus Host Disease; Hematologic Malignancy

Keywords

Graft Versus Host Disease; Graft Vs Host Disease; Hematologic Malignancy; Hematologic malignancy in morphologic remission; Belumosudil; Stem Cell Transplant; Memorial Sloan Kettering Cancer Center; 25-033

Outcome Measures

Primary Outcomes (1)

• Change in GVHD/relapse-free survival (GRFS) at 1-year post-Hematopoietic Cell Transplantation (HCT)

  The primary objective is to assess the efficacy of belumosudil in the improvement of GRFS at 1-year post-HCT for patients receiving PTCY GVHD prophylaxis and separately for participants receiving CNI-based (CNI without PTCY) plus abatacept GVHD prophylaxis.

  1 year

Study Arms (1)

Participants with hematologic malignancy in morphologic remission

EXPERIMENTAL

Participants will be diagnosed with a hematologic malignancy in morphologic remission

Drug: Belumosudil

Interventions

Belumosudil DRUG

Belumosudil is an oral selective inhibitor of Rho-GTPase-associated coiled-coil kinase 2 (ROCK2)

Participants with hematologic malignancy in morphologic remission

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients ≥ 18 years-old at time of consent.
• Diagnosis: hematologic malignancy in morphologic remission who will be treated with RI or NMA conditioning and GVHD prophylaxis CNI-based (CNI without PTCY) plus abatacept or PTCY-based (CNI with PTCY).
• Recipients of 7-8/8 related or unrelated HLA-matched or related haploidentical donor.
• Peripheral blood stem cell graft
• Allo-HCT day \<120 at time of consent
• Patient has received an allo-HCT transplant and is in morphologic remission (blasts \<5%, no evidence of extramedullary disease in AML or MDS). Patients with CR with incomplete count recovery (CRp or CRi) or minimal residual disease are allowed.
• Patient has achieved engraftment. Engraftment is defined as ANC≥500/μL and platelets ≥ 20000/μL on 3 consecutive measurements (each occurring at least 1 day apart). The patient must not have had a platelet transfusion within 7 days before the first measurement.
• Patient is ≥ 80 days and ≤ 20 days from allo-HCT infusion.
• Karnofsky score ≥ 70%.
• Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 3x upper limit of normal (ULN)
• Total bilirubin ≤1.5 x ULN (unless benign congenital hyperbilirubinemia).
• Glomerular filtration rate (GFR) ≥ 30 mL/min/1.73 m2
• Female subjects of childbearing potential (≤ 50 years old) have a negative serum or urine pregnancy test. Females of childbearing potential are defined as females without prior hysterectomy or who have had any evidence of menses in the past 12 months.
• ° Sexually active females of childbearing potential enrolled in the study must agree to consistently 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); or \* 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.

You may not qualify if:

• Recipient of CD34+ selected or engineered stem cell graft.
• Treatment with in vivo T cell depletion (e.g. anti-thymocyte globulin).
• Evidence of current uncontrolled cardiovascular conditions, uncontrolled cardiac arrhythmias, symptomatic congestive heart failure, unstable angina, or myocardial infarction within the past 6 months.
• Pulmonary dysfunction with DLCO \<50% corrected for hemoglobin
• Uncontrolled infection, including active hepatitis B and C. Definitive therapy for infection is required and must have no signs of progression within 7 days of the first day of study drug treatment.
• Use of investigational agent within 14 days pre-HCT or anytime thereafter.
• Active acute or chronic GVHD requiring systemic therapy (topical or local therapies are allowed).
• Active treatment with corticosteroids at a dose of ≥ 0.25 mg/kg/day for non-GVHD indication.
• Uncontrolled psychosis, active suicidal ideation, or psychiatric hospitalization within the past year
• Female patient who is pregnant or breastfeeding.
• Prior therapy with belumosudil.
• Known allergy or sensitivity to belumosudil or any other ROCK2 inhibitor.

Sponsors & Collaborators

• Memorial Sloan Kettering Cancer Center: lead

Study Sites (8)

Memorial Sloan Kettering Basking Ridge (Limited Protocol Activities)

Basking Ridge, New Jersey, 07920, United States

RECRUITING

Memorial Sloan Kettering Monmouth (Limited Protocol Activities)

Middletown, New Jersey, 07748, United States

RECRUITING

Memorial Sloan Kettering Bergen (Limited Protocol Activities)

Montvale, New Jersey, 07645, United States

RECRUITING

Memorial Sloan Kettering Suffolk-Commack (Limited protocol activity)

Commack, New York, 11725, United States

RECRUITING

Memorial Sloan Kettering Westchester (Limited Protocol Activities)

Harrison, New York, 10604, United States

RECRUITING

New York University (Data Collection Only)

New York, New York, 10010, United States

RECRUITING

Memorial Sloan Kettering Cancer Center (All Protocol Activities)

New York, New York, 10065, United States

RECRUITING

Memorial Sloan Kettering Nassau (Limited Protocol Activites)

Rockville Centre, New York, 11553, United States

RECRUITING

Related Links

• Memorial Sloan Kettering Cancer Center

MeSH Terms

Conditions

Graft vs Host Disease; Hematologic Neoplasms

Interventions

belumosudil

Condition Hierarchy (Ancestors)

Immune System Diseases; Neoplasms by Site; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Doris Ponce, MD

  Memorial Sloan Kettering Cancer Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Doris Ponce, MD

CONTACT

646-608-3739; ponced@mskcc.org

Gajan Raju, MD

CONTACT

646-608-3987

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 22, 2025
• First Posted: June 5, 2025
• Study Start: May 21, 2025
• Primary Completion (Estimated): May 21, 2029
• Study Completion (Estimated): May 21, 2029
• Last Updated: April 13, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share

Locations

US (8)