Evaluating the safety and effectiveness of oral belumosudil for chronic graft versus host disease in specific ethnic groups

A Phase 2, Open-label, Multicenter Study to Evaluate the Safety and Efficacy of Belumosudil in Black or African American, American Indian or Alaska Native, and Native Hawaiian or Other Pacific Islander Participants With Chronic Graft Versus Host Disease (cGVHD) After At Least 2 Prior Lines of Systemic Therapy

This study is testing if the oral medication belumosudil can safely help people aged 12 and older from certain ethnic backgrounds who have chronic graft versus host disease and haven't found relief from previous treatments.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 36 (estimated)
• Ages: 12 Years and up
• Sex: All
• Sponsor: Sanofi (Industry-sponsored)
• Drugs / interventions: rituximab, ibrutinib, ruxolitinib, methotrexate
• Locations: 5 sites (Miami, Florida and 4 other locations)
• Trial ID: NCT05567406 on ClinicalTrials.gov

What this trial studies

This study aims to assess the safety and efficacy of oral belumosudil in participants aged 12 years and older from specific ethnic backgrounds, including Black or African American, American Indian or Alaska Native, and Native Hawaiian or Other Pacific Islander individuals, who have chronic graft versus host disease (cGVHD) and have previously undergone at least two lines of systemic therapy. Participants will undergo a screening period of up to 4 weeks, followed by treatment until disease progression, and will have a 4-week safety follow-up along with long-term follow-up every 12 weeks. The study focuses on evaluating the drug's impact on cGVHD symptoms and overall patient health.

Who should consider this trial

Good fit: Ideal candidates include individuals aged 12 years and older from specific ethnic backgrounds who have persistent cGVHD and have received at least two prior lines of systemic therapy.

Not a fit: Patients who do not belong to the specified ethnic groups or who have not received the required prior treatments may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide a new treatment option for patients suffering from chronic graft versus host disease, particularly in underrepresented ethnic groups.

How similar studies have performed: While there have been studies on cGVHD treatments, this specific approach using belumosudil in these ethnic groups is novel and has not been extensively tested.

Eligibility criteria

Inclusion Criteria:

* Participants are included in the study if any of the following criteria apply:
* Participant is Black or African American, or American Indian or Alaska Native, or Native Hawaiian or Other Pacific Islander by self-identification.
* Previously received at least 2 and not more than 5 lines of systemic therapy for cGVHD.
* Receiving glucocorticoid therapy with a stable dose over the 2 weeks prior to screening.
* Have persistent cGVHD manifestations and systemic therapy is indicated.
* Karnofsky (if aged ≥ 16 years) / Lansky (if aged \< 16 years) Performance Score of ≥ 60.
* At least 12 years of age; weight ≥ 40 kilograms (kg).
* Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 3 x upper limit of normal (ULN).
* Total bilirubin ≤ 1.5 x ULN.
* Contraception (with double contraception methods) for male and female participants; not pregnant or breastfeeding for female participants
* Capable of giving signed informed consent.

Exclusion Criteria:

* Participants are excluded from the study if any of the following criteria apply:
* Participant has not been on a stable dose/regimen of systemic cGVHD treatment(s) for at least 2 weeks prior to screening. (Note: Concomitant corticosteroids, calcineurin inhibitors, sirolimus, MMF, methotrexate, rituximab, and ECP are acceptable. Systemic investigational GVHD treatments are not permitted).
* Histological relapse of the underlying cancer or post-transplant lymphoproliferative disease at the time of screening.
* Current treatment with ibrutinib or ruxolitinib. Prior treatment with ibrutinib or ruxolitinib is allowed with a washout of at least 28 days prior to enrollment.
* History or other evidence of severe illness or any other conditions that would make the participant, in the opinion of the Investigator, unsuitable for the study (such as malabsorption syndromes, poorly controlled psychiatric disease, or coronary artery disease).
* Corrected QT interval using Fridericia's formula (QTc\[F\]) \> 480 ms.
* Forced expiratory volume (in the first second; FEV1) ≤ 39% The above information is not intended to contain all considerations relevant to the potential participation in a clinical trial.

Where this trial is running

Miami, Florida and 4 other locations

• Nicklaus Children's Hospital - Miami - Southwest 62nd Avenue- Site Number : 129 — Miami, Florida, United States (Recruiting)
• University of Maryland School of Medicine - Baltimore- Site Number : 128 — Baltimore, Maryland, United States (Recruiting)
• Massachusetts General Hospital- Site Number : 002 — Boston, Massachusetts, United States (Recruiting)
• Dana-Farber Cancer Institute- Site Number : 004 — Boston, Massachusetts, United States (Recruiting)
• Westchester Medical Center- Site Number : 130 — Valhalla, New York, United States (Recruiting)

Study contacts

• Study coordinator: Trial Transparency email recommended (Toll free for US & Canada)

Conditions Chronic Graft Versus Host Disease