NCT04723303已完成早期 1 期
Phase 1 Study of Mesenchymal Stromal Cells, Umbilical Cord Lining Stem Cells (ULSC), in Patients With Polymyositis (PM) and Dermatomyositis (DM)
适应症
干预措施
试验速览
- 阶段
- 早期 1 期
- 状态
- 已完成
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- Number of Subjects with Dose Limiting Toxicity (DLT) that begins during or following ULSC infusion as assessed within 24 hours.
研究概览
简要总结
This study will investigate Umbilical Cord Lining Stem Cells (ULSC) as an investigational medicinal product and its use in patients with polymyositis (PM) or dermatomyositis (DM) to see if a single intravenous (IV) infusion of allogeneic umbilical cord lining stem cells (ULSC) safe, tolerable, and feasible to administer.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 90 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Adult, male or female, age ≥18 years old
- •Diagnosis of definite or probable DM or PM, according to the criteria of Bohan and Peter
- •Patients with PM will either be positive for a myositis-associated antibody or have undergone evaluation to exclude mimics, as deemed appropriate by the Investigator (See Note below).
- •Signs informed consent.
排除标准
- •A diagnosis of inclusion body myositis, juvenile DM or PM, myositis in the context of significant overlap with another systemic autoimmune rheumatologic disease.
- •Non immune myopathies.
- •Cancer associated myositis.
- •Hypersensitivity to study product components. History of hypersensitivity to dimethyl sulfoxide (DMSO).
- •Pregnant or lactating women.
- •Concomitant severe cardiac, pulmonary disease, active infection or other conditions that preclude assessment of safety and efficacy of the study product.
- •Patients with predominant muscle atrophy secondary to uncontrolled or chronic DM or PM, based on clinical, biochemical, and/or radiologic assessment, despite previous optimized treatment.
- •Anticipated need for surgery during the trial period.
- •A history of prevalent noncompliance with medical therapy.
- •Recipient of an organ transplant.
- •Neutropenia (absolute neutrophil count<1,800/mm3 [or <1,000/mm3 in African-American subjects]).
- •Severe impairment in renal function (estimated glomerular filtration rate <30 ml/kg*min).
- •Recent or planned use of vaccination with live attenuated viruses.
- •Active cancer or prior diagnosis of cancer within the past 2 years (patients with basal and squamous cell cancer of skin will not be excluded).
- •Condition that would impair an assessment of muscle strength, including neurological disorders such as Parkinson's disease or severe musculoskeletal condition.
- •Any other condition that, in the judgment of the Investigator or Sponsor, would be a contraindication to enrollment, study product administration, or follow-up.
- •History of Atrial septal defect or ventricular septal defect
研究组 & 干预措施
Treatment Arm
Experimental
A single IV infusion of ULSC's in patients with DM or PM
干预措施: Umbilical Cord Lining Stem Cells (Drug)
结局指标
主要结局
Number of Subjects with Dose Limiting Toxicity (DLT) that begins during or following ULSC infusion as assessed within 24 hours.
时间窗: Within 24 hours
Dose Limiting Toxicities are treatment-emergent suspected adverse reactions graded severe, such as severe infusion-related hypersensitivity toxicities of grade ≥3, and any serious adverse event (SAE). (Note: DLT during an infusion will stop that infusion in that subject.)
次要结局
未报告次要终点
研究者
研究点 (1)
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