Evaluating belumosudil for treating chronic graft versus host disease in Chinese adolescents

A Multicenter, Open-label, Single-arm, Phase 4 Clinical Study to Evaluate the Pharmacokinetics, Efficacy, and Safety of Belumosudil Mesylate Tablets in Chinese Adolescents (Aged From 12 to Less Than 18 Years) With Chronic Graft-versus-host Disease (cGVHD) Who Have Had an Inadequate Response to Glucocorticoids or Other Systemic Therapies

This study is testing if belumosudil can help Chinese teenagers with chronic graft versus host disease who haven't improved with other treatments.

Quick facts

• Phase: Phase 4
• Study type: Interventional
• Enrollment: 6 (estimated)
• Ages: 12 Years to 17 Years
• Sex: All
• Sponsor: Sanofi (Industry-sponsored)
• Locations: 2 sites (Beijing and 1 other locations)
• Trial ID: NCT06616415 on ClinicalTrials.gov

What this trial studies

This clinical study aims to assess the pharmacokinetics, efficacy, and safety of belumosudil mesylate tablets in Chinese adolescents aged 12 to less than 18 who have chronic graft versus host disease (cGVHD) and have not responded adequately to glucocorticoids or other systemic therapies. Participants will receive a daily dose of 200 mg of belumosudil in 28-day cycles. The study is designed as a single-arm, Phase 4 post-marketing evaluation, focusing on the treatment outcomes in this specific patient population.

Who should consider this trial

Good fit: Ideal candidates for this study are Chinese adolescents aged 12 to less than 18 with active moderate to severe cGVHD who have previously received systemic therapy.

Not a fit: Patients who have not undergone allogeneic hematopoietic stem cell transplantation or those with mild cGVHD may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide an effective treatment option for adolescents suffering from cGVHD who have not responded to existing therapies.

How similar studies have performed: While this study focuses on a specific demographic and treatment, similar studies have shown promise in evaluating the efficacy of belumosudil in other populations.

Eligibility criteria

Inclusion Criteria:

* Participant must be 12 to less than 18 years of age at the time of signing the informed consent.
* Participant has undergone allogeneic hematopoietic stem cell transplantation (allo-HSCT).
* Has active moderate to severe cGVHD.
* Has received at least one line of prior systemic therapy for cGVHD.
* Participant must receive a corticosteroid therapy for cGVHD with a stable dose for at least 2 weeks prior to the first dose of the IMP.
* Has a Lansky-Play performance score of ≥60.
* Participants should have an expected survival of longer than 6 months.
* Body weight of 30 kg and above.
* Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
* The participant or their legally authorized representative (LAR) must be capable of giving signed informed consent.

Exclusion Criteria:

Participants are excluded from the study if any of the following criteria apply:

* Recurrence of hematologic neoplasms (according to the corresponding criteria for recurrence of primary hematologic neoplasms) or post-transplant lymphoproliferative disease at screening.
* Received investigational systemic therapy for cGVHD within 28 days prior to enrollment, unless the prior treatment had been washed out for at least 28 days or 5 half-lives prior to enrollment, whichever is shorter.
* Absolute neutrophil count (ANC) \<1.0 × 10\^9/L.
* Platelet count \<50 × 10\^9/L.
* Alanine aminotransferase (ALT) \>3× the upper limit of normal (ULN), aspartate aminotransferase (AST) \>3 × ULN.
* Total bilirubin (TBIL) \>1.5 × ULN (\>3 ULN if Gilbert's syndrome).
* Estimated Glomerular Filtration Rate (eGFR) \<30 mL/min/1.73 m\^2 using the revised Bedside Schwartz formula . Revised Schwartz equation: CrCl (mL/min/1.73 m\^2) = 0.413 × (height \[in cm\])/Creatinine (in mg/dL) at screening visit.
* Participant not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or participants potentially at risk of noncompliance to study procedures.

Where this trial is running

Beijing and 1 other locations

• Investigational Site Number: 1560001 — Beijing, China (Recruiting)
• Investigational Site Number: 1560002 — Shanghai, China (Recruiting)

Study contacts

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

Conditions Chronic Graft Versus Host Disease