A Single-Arm, Single-Center Exploratory Study of Amimestrocel Injection for the Prevention of Gastrointestinal Mucositis Induced by Conditioning Regimens Containing TBI and/or Melphalan
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 22
- 试验地点
- 1
- 主要终点
- Incidence of Grade 3-4 Oral Mucositis within 28 Days Post-Transplant
研究概览
简要总结
This study aims to see if a single intravenous infusion of a cell therapy called Amimestrocel injection can help prevent severe mouth sores (oral mucositis) in patients receiving a stem cell transplant.
Patients getting a stem cell transplant often receive strong chemotherapy (with radiation and/or a drug called melphalan) that can cause painful mouth and gut sores, making eating difficult and increasing infection risk. Amimestrocel injection is made from human umbilical cord cells that may help reduce inflammation and promote healing.
About 22 adult patients scheduled for this type of transplant at one hospital in China will receive the infusion 1-2 days before their stem cell transplant. Researchers will closely check for mouth sores, pain, and side effects for the first 28 days, and continue safety monitoring for 100 days.
The main goal is to see if the treatment lowers the rate of severe (Grade 3-4) mouth sores. The study will also track pain levels, need for pain medication, diarrhea, time for blood counts to recover, and overall safety.
详细描述
Background and Rationale:
Myeloablative conditioning regimens containing total body irradiation (TBI) and/or high-dose melphalan are standard prior to allogeneic hematopoietic stem cell transplantation (HSCT) but frequently cause severe gastrointestinal mucositis. This condition leads to significant morbidity, including pain, dysphagia, diarrhea, nutritional impairment, infection risk, prolonged hospitalization, and increased costs. Effective preventive strategies are limited. Mesenchymal stem cells (MSCs) have demonstrated immunomodulatory and tissue-repair properties via paracrine secretion of anti-inflammatory and growth factors, showing potential in mitigating mucosal injury in preclinical and early clinical contexts.
Study Design:
This is a single-arm, single-center, open-label, exploratory interventional study. It will enroll approximately 22 subjects to preliminarily evaluate the efficacy and safety of prophylactic Amimestrocel infusion.
Intervention:
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Prevention
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Aged 18 to 65 years.
- •Planned to undergo myeloablative allogeneic hematopoietic stem cell transplantation.
- •The conditioning regimen must contain total body irradiation (TBI) and/or melphalan.
- •Eastern Cooperative Oncology Group (ECOG) performance status of 0 or
- •Adequate organ function defined as:Left ventricular ejection fraction (LVEF) ≥ 50%.Serum total bilirubin ≤ 1.5 × upper limit of normal (ULN).Serum creatinine ≤ 1.5 × ULN or creatinine clearance ≥ 60 mL/min.
- •Voluntarily signs the informed consent form.
排除标准
- •History of allergy to mesenchymal stem cells or any component of the Amimestrocel injection preparation.
- •Presence of active, uncontrolled bacterial, fungal, or viral infection.
- •History of other malignancies within the past 5 years, except for cured carcinoma in situ or basal cell skin cancer.
- •Pregnant or lactating women, or those planning pregnancy during the study period.
- •Previous receipt of any cell therapy product.
- •Severe psychiatric disorder or cognitive impairment that would limit the ability to provide informed consent or comply with study procedures.
- •Any condition that, in the investigator's judgment, makes the subject unsuitable for study participation.
研究组 & 干预措施
Amimestrocel Injection
All enrolled participants will receive a single, fixed-dose intravenous infusion of Amimestrocel injection (6.0×10⁷ cells in 12mL). The infusion is administered within 24 to 48 hours after the last dose of cyclophosphamide (part of the standard conditioning regimen) and before the infusion of hematopoietic stem cells (Day 0). All participants will also undergo standard myeloablative conditioning (containing TBI and/or melphalan), allogeneic hematopoietic stem cell transplantation, and institutional standard supportive care.
干预措施: Amimestrocel injection (Biological)
结局指标
主要结局
Incidence of Grade 3-4 Oral Mucositis within 28 Days Post-Transplant
时间窗: From stem cell infusion (Day 0) up to Day 28 post-transplant
The proportion of subjects who develop severe (Grade 3 or 4) oral mucositis from the transplant day (Day 0) up to Day 28 post-transplant. The severity of oral mucositis is assessed daily and the highest grade recorded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version 5.0.
次要结局
- Maximum Severity Grade of Oral Mucositis(From Day 0 up to Day 28 post-transplant.)
- Duration of Oral Mucositis(From Day 0 up to Day 28 post-transplant.)
- Maximum Severity Grade of Diarrhea(From Day 0 up to Day 28 post-transplant.)
- Total Duration of ≥ Grade 2 Diarrhea(From Day 0 up to Day 28 post-transplant.)
- Peak Oral Pain Score(The highest score recorded in the subject's daily self-assessed oral pain Numerical Rating Scale (NRS, 0-10) from Day 0 to Day 28 post-transplant. Results are described as median and interquartile range.)
- Total Opioid Use from Day 0 to Day 28 Post-Transplant(From Day 0 up to Day 28 post-transplant.)
- Time to Neutrophil Engraftment(From Day 0 up to Day 60 post-transplant.)
- Incidence of Any Adverse Event (AE) within 100 Days Post-Transplant(From after investigational product infusion up to Day 100 post-transplant.)
- Incidence of Serious Adverse Events (SAEs) within 100 Days Post-Transplant(From after investigational product infusion up to Day 100 post-transplant.)