Use of Bone Marrow-Derived Mesenchymal Stem Cells (Mesencell) for the Treatment of Patients Undergoing Hematopoietic Progenitor Cell Transplantation Who Developed Refractory Moderate or Severe Chronic Graft-versus-Host Disease: A Phase I Clinical Trial
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 20
- 试验地点
- 4
- 主要终点
- Number of Participants With Treatment-Related Adverse Events
研究概览
简要总结
The purpose of this Phase I clinical trial is to evaluate the safety and potential treatment response of bone marrow-derived mesenchymal stem cells (MesenCell) in adult patients with moderate or severe chronic graft-versus-host disease (cGVHD) that is refractory to corticosteroid treatment. The main questions it seeks to answer are:
- Is MesenCell infusion safe in patients with corticosteroid-refractory chronic graft-versus-host disease?
- Can MesenCell treatment improve the clinical response and disease control in these patients?
Participants will receive one to three intravenous infusions of MesenCell, according to the study protocol and be followed for 12 months after the last MesenCell infusion.
详细描述
This is a prospective, single-arm, open-label Phase I clinical trial designed to evaluate the safety and treatment response of bone marrow-derived mesenchymal stem cells (MesenCell) in adult patients with moderate or severe chronic graft-versus-host disease (cGVHD) refractory to corticosteroid treatment.
Approximately 20 participants will be enrolled in the study. Participants will receive one to three intravenous infusions of MesenCell at a dose of 2 × 10⁶ cells/kg, administered at seven-day intervals. The number of infusions will depend on the dose-escalation level to which each participant is assigned.
MesenCell will be administered in combination with standard treatment consisting of corticosteroids at a dose of 1 mg/kg/day and cyclosporine. Participants will undergo clinical and laboratory monitoring throughout the study to evaluate the safety of the intervention and the occurrence, frequency, severity, and causal relationship of adverse events following MesenCell infusion.
Treatment response will be assessed using standardized criteria for chronic graft-versus-host disease, including overall and organ-specific response. The study will also evaluate all-cause mortality, failure-free survival, and changes in corticosteroid and immunosuppressive medication use, including dose reductions, treatment discontinuation, and reduction in the number of immunosuppressive agents.
Participants will be followed for 12 months after administration of the last MesenCell dose. During this period, clinical assessments and review of medical records will be performed to monitor treatment response, disease progression, safety outcomes, need for additional systemic therapy, mortality, and changes in concomitant immunosuppressive treatment.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age from 18 to 70 years;
- •Male or female sex;
- •Patients after allogeneic hematopoietic stem cell transplantation (any donor or stem cell source);
- •Presenting with moderate or severe chronic Graft versus host Disease refractory to treatment with corticosteroids and calcineurin inhibitors;
- •Patients who agree to participate in this study and who sign the Informed Consent Form.
排除标准
- •Patients with any concomitant clinical condition that, in the physician's opinion, contraindicates the infusion of bone marrow-containing stem cells;
- •Pregnancy;
- •Patients (or their legal guardians) who do not agree to participate in this study or who do not sign the Informed Consent Form;
- •Patients with recurrent malignant neoplasm.
研究组 & 干预措施
MesenCell
Patient will receive 1 to 3 intravenous infusions of 2 × 10⁶/kg of Mesenchymal stem cells (MSCs) at 7 day intervals
干预措施: Mesenchymal Stem Cell Infusion (Biological)
结局指标
主要结局
Number of Participants With Treatment-Related Adverse Events
时间窗: 30 days after the last MesenCell infusion
Number of participants experiencing unexpected adverse events or adverse events considered probably or definitely attributable to MesenCell infusion within 30 days after the last dose. Adverse events will be classified according to the Common Terminology Criteria for Adverse Events (CTCAE) version 6.0 (2025). Grade 3 or higher events will be considered for toxicity assessment.
次要结局
- All-cause mortality(From the first MesenCell infusion through 12 months after the last MesenCell infusion)
- Overall Treatment Response Rate(6 months after treatment initiation)
- Change in Overall Response(From baseline through 12 months after treatment initiation)
- Change in Organ-Specific Response Scores(From baseline through 12 months after treatment initiation)
- Failure-Free Survival(From the first MesenCell infusion through 12 months after the last MesenCell infusion)
- Reduction in Corticosteroid Therapy(From treatment initiation through 12 months after the last MesenCell infusion)
- Discontinuation of Immunosuppressive Therapy(From treatment initiation through 12 months after the last MesenCell infusion)
- Time to Best Overall Response(From the first MesenCell infusion through 12 months after the last MesenCell infusion)
研究者
Carmen Rebelatto
Research Coordinator
Pontifícia Universidade Católica do Paraná