Safety and Efficacy of Oral Belumosudil in Black or African American, American Indian or Alaska Native, and Native Hawaiian or Other Pacific Islander Male and Female Participants Aged 12 Years and Above With Chronic Graft Versus Host Disease (cGVHD) After At Least 2 Prior Lines of Systemic Therapy

Investigating the Safety and Efficacy of an Investigational Medication in Participants with Chronic Graft-Versus-Host Disease (cGVHD)

Recruiting
12 years or above
All
Phase 2
36 participants needed
7 Locations

Study Overview

The purpose of this study is to measure safety and efficacy of oral belumosudil in Black or African American, American Indian or Alaska Native, and Native Hawaiian or Other Pacific Islander male and female participants with cGVHD who have previously been treated with at least 2 prior lines of systemic therapy aged 12 years and above.

The duration of participants participation will be up to 4 weeks for screening, treatment until clinically significant progression of disease, and 4 weeks of safety follow-up, and then long-term follow-up every 12 weeks.1 Cycle = 28 days.

Study Details

Up to 4 weeks for screening, treatment until clinically significant progression of disease, 4 weeks of safety follow-up and then long-term follow-up every 12 weeks.

Eligibility Criteria

You may be eligible for this study if you meet the following criteria:

  • Conditions: Chronic Graft Versus Host Disease
  • Age: 12 years or above
  • Gender: All

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.

Updated on 22 Apr 2025. Study ID: NCT05567406

This study investigates the safety and effectiveness of an investigational medication in male and female participants aged 12 years and older with chronic graft-versus-host disease (cGVHD). cGVHD is a condition where the donated cells from a transplant attack the recipient's body. The study focuses on individuals who identify as Black or African American, American Indian or Alaska Native, and Native Hawaiian or Other Pacific Islander, who have already tried at least two other treatments.

Participants will receive the investigational medication and continue their current glucocorticoid therapy, which helps control inflammation. The study will monitor the participants' health and how well the medication works in managing cGVHD symptoms. Participants will undergo regular assessments to track their response to the treatment.

  • Who can participate: Participants must be at least 12 years old, weigh 40 kg or more, and have received 2 to 5 prior treatments for cGVHD. They must self-identify as Black or African American, American Indian or Alaska Native, or Native Hawaiian or Other Pacific Islander, and have stable glucocorticoid therapy with a performance score of 60 or higher.
  • Study details: Participants will take an investigational medication while continuing their current glucocorticoid therapy. They will be regularly monitored for safety and effectiveness in managing cGVHD symptoms. An inactive substance that looks like the investigational medication but does not contain any medicine may be used as a placebo.
  • Study Timelines: The study will last up to 4 weeks for screening, treatment until disease progression, and 4 weeks of safety follow-up.

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