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December 8, 2025Blood

Investigating belumosudil in children with refractory moderate-to-severe chronic graft-versus-host disease: Design of a phase 1/2 study

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Authors

ABAdriana Balduzzi

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Overview

Phase 1/2 trial assesses treatment efficacy, safety, and overall response rate in children with cGvHD, indicating its potential benefits.

Key Points

  • To investigate the safety and efficacy of belumosudil in children with moderate-to-severe chronic graft-versus-host disease (cGvHD).
  • Phase 1/2, open-label, multicenter trial
  • Administration of belumosudil in children ages 1 to <18 years with refractory cGvHD
  • Evaluation of the overall response rate by Week 24, with safety follow-up for 4 weeks post-treatment
  • Determination of the recommended pediatric equivalent dose and overall response rate as primary endpoints
  • Key safety and pharmacokinetic parameters will also be assessed

Cite This Study

Adriana Balduzzi (2025) studied this question.

synapsesocial.com/papers/69362f3a4fa91c937236d404https://doi.org/10.1182/blood-2025-7753
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Efficacy and safety of the ROCK2-inhibitor belumosudil for chronic gvhd treatment: Multicenter retrospective analysis of a large german-austrian cohort of the belumosudil managed-access program.2025
  2. 2Belumosudil in pediatric patients with steroid-refractory chronic graft-versus-host disease2025
  3. 3FDA Approval Summary: Belumosudil for Adult and Pediatric Patients 12 Years and Older with Chronic GvHD after Two or More Prior Lines of Systemic Therapy2022 · 58 citations
  4. 4Belumosudil in the Management of Heavily Pretreated Prolonged Chronic GVHD: A Retrospective Multicenter Real‐World Study2026
  5. 5Belumosudil for chronic graft-versus-host disease in children under 12: A retrospective analysis of effectiveness and safety2025