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

Launched by KADMON, A SANOFI COMPANY · Oct 3, 2022

Keywords

ClinConnect Summary

This clinical trial is studying the safety and effectiveness of a medication called belumosudil for individuals aged 12 and older who have chronic graft versus host disease (cGVHD). The trial specifically focuses on Black or African American, American Indian or Alaska Native, and Native Hawaiian or Other Pacific Islander participants who have already received at least two different treatments for cGVHD. Participants must have ongoing symptoms from cGVHD and be stable on their current medication regimen for at least two weeks before starting the trial.

If you or a family member joins this trial, the participation will involve a screening period of up to four weeks, followed by treatment that lasts until the disease worsens significantly, and then a safety follow-up for four additional weeks. After that, there will be long-term check-ins every three months. To be eligible, participants need to weigh at least 40 kilograms and have specific health conditions, including stable liver function. It’s also important for participants to use double contraception if they are of childbearing age. Overall, this study aims to find out if belumosudil can help manage cGVHD effectively and safely in these specific communities.

Gender

ALL

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.