Study of Human Umbilical Cord Mesenchymal Stem Cells (UC-MSC) In Patients With Duchenne Muscular Dystrophy
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 35
- 试验地点
- 2
- 主要终点
- Primary Endpoints
研究概览
简要总结
The primary objective of this study is to provide UC-MSC treatment to patients with DMD.
Secondary objectives will be to further evaluate treatment-related adverse events as well as changes in DMD-related functional testing/assessments, blood laboratories, and inflammation related biomarker levels over time.
详细描述
The Phase 2 study will enroll up to 35 ambulatory male participants between the ages of 5 and 10, who will receive four, 3-day intravenous dose cycles of UC-MSC treatment, each administered every three months. Participants will be closely monitored throughout the study period for treatment-related adverse events and changes in DMD-related functional assessments at specific follow-up intervals through 12 months.
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
- 5 Years 至 10 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Male sex by birth with a genetically confirmed diagnosis of Duchenne Muscular Dystrophy (DMD).
- •Age is greater than or equal to 5 and less than or equal to 10 years.
- •Has a North Star Ambulatory Assessment (NSAA) score greater than 13 and less than
- •Demonstrates the ability to perform the "time to rise" test in under 10 seconds.
- •Is up-to-date on immunizations.
- •Is on a stable dose of glucocorticoids for at least 12 weeks prior to study participation, except for weight-based or toxicity-related adjustments.
- •Is on a stable dose of supplements for at least 12 weeks prior to study participation.
- •Has the ability to comply with the requirements of the study and the ability to understand and provide written informed assent and a guardian's consent.
- •Patient must be either a non-responder to or a poor candidate for treatment with another established therapy.
排除标准
- •Active cancer or prior diagnosis of cancer within the past year (patients with basal and squamous cell cancer of the skin will not be excluded).
- •BMI > 45 kg/m².
- •Any other condition (including concomitant treatment) that, in the judgment of the Investigator or Sponsor, would be a contraindication to enrollment, study product administration (e.g., known hypersensitivity to dimethyl sulfoxide (DMSO), Human Serum Albumin (HSA), or PlasmaLyte), or follow-up.
- •Treatment with an exon skipping therapy within 3 months of study start.
- •Cognitive delay or impairment that can confound motor development in the opinion of the investigator.
- •Major surgery within 3 months prior to Day 0 or planned surgery or procedures that could affect the conduct of the study.
研究组 & 干预措施
UC-MSC Treatment Group
Intravenous infusion of UC-MSC
干预措施: Human Umbilical Cord Mesenchymal Stem Cells (UC-MSC) (Biological)
结局指标
主要结局
Primary Endpoints
时间窗: From enrollment to the end of follow up at 12-months
The primary safety endpoint is the rate of treatment-related adverse events over time. The primary efficacy endpoint is the change in DMD-related functional assessments over time.
次要结局
- Secondary Endpoint(From enrollment through follow up at 12-months)