A Phase 1/2a Study of the Safety and Efficacy of BX-U001 for the Treatment of Severe COVID-19 Pneumonia With Moderate to Severe Acute Respiratory Distress Syndrome (ARDS).
试验速览
- 阶段
- 1 期
- 状态
- 撤回
- 发起方
- Baylx Inc.
- 入组人数
- 39
- 主要终点
- Incidence of any treatment-emergent adverse events (TEAEs) and treatment emergent serious adverse events (TESAEs)
研究概览
简要总结
This is a phase 1/2a study including 2 parts, phase 1 and phase 2a. The phase 1 part is an open-label, single-arm, dose-escalating study to evaluate the safety and explore the dose limiting toxicity and maximum tolerated dose of a human umbilical cord derived mesenchymal stem cell product (BX-U001) in severe COVID-19 pneumonia patients with acute respiratory distress syndrome (ARDS). Qualified subjects after the screening will be divided into low, medium, or high dose groups to receive a single intravenous infusion of BX-U001 at the dose of 0.5×10^6, 1.0×10^6, or 1.5×10^6 cells/kg of body weight, respectively. The Phase 2a part is a randomized, placebo-controlled, double-blind clinical trial examining the safety and biological effects of BX-U001 at the appropriate dose selected from phase 1 for severe COVID-19 pneumonia patients with the same inclusion/exclusion criteria as the phase 1 part.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female, aged between 18 and 80;
- •Laboratory (RT-PCR, Gene Sequencing or Antibody) confirmed cases of severe COVID-19 pneumonia with mild to moderate ARDS (Berlin definition);
- •Patients are intubated;
- •Patients who voluntarily adhere to the research procedures and ensure good compliance during the research period;
- •Patients who fully understand the research nature of this study and sign written informed consent.
排除标准
- •Subjects who have received investigational drug (except for Remdesivir) for the treatment of COVID-19 within 30 days before screening;
- •Subjects who are pregnant, breastfeeding or whose urinary pregnancy test is positive before participation in the study; subjects who are pregnant, breastfeeding, have a birth plan, or are unwillingness to use contraception during the study period and within 12 months of infusion; except for subjects who have sterilization surgery or menopause during the study period;
- •Within 3 days before screening/randomization, subjects who have used high-dose corticosteroids that is equivalent to methylprednisolone >240 mg/day or irregular use of systemic corticosteroids to treat other diseases that could affect the efficacy evaluated by the investigator;
- •Subjects receiving extracorporeal membrane oxygenation (ECMO) support.
- •Subjects who are allergic to low-molecular-weight heparin calcium or human albumin;
- •Subjects with ongoing malignant tumors.
研究组 & 干预措施
Phase 1 Low-dose Group
Eligible subjects will receive a single infusion of hUC-MCS product at the dose of 0.5 million cells/kg in addition to standard of care treatment.
干预措施: Human umbilical cord mesenchymal stem cells + best supportive care (Biological)
Phase 1 Middle-dose Group
Eligible subjects will receive a single infusion of hUC-MCS product at the dose of 1 million cells/kg in addition to standard of care treatment.
干预措施: Human umbilical cord mesenchymal stem cells + best supportive care (Biological)
Phase 1 High-dose Group
Eligible subjects will receive a single infusion of hUC-MCS product at the dose of 1.5 million cells/kg in addition to standard of care treatment.
干预措施: Human umbilical cord mesenchymal stem cells + best supportive care (Biological)
Phase 2a Treatment Group
Eligible subjects will receive a single infusion of hUC-MCS product at the selected dose from phase 1 in addition to standard of care treatment.
干预措施: Human umbilical cord mesenchymal stem cells + best supportive care (Biological)
Phase 2a Control Group
Eligible subjects will receive a single infusion of placebo control and standard of care treatment.
干预措施: Placebo control + best supportive care (Other)
结局指标
主要结局
Incidence of any treatment-emergent adverse events (TEAEs) and treatment emergent serious adverse events (TESAEs)
时间窗: Day 28
Safety will be defined by the incidence of TEAEs and TESAEs as assessed by the treating physician
Incidence of infusion-related adverse events
时间窗: Day 3
Safety will be defined by the incidence of infusion-related adverse events as assessed by the treating physician
次要结局
- Duration of ICU stay(Day 28)
- Changes in blood cytokine levels(Day 28)
- Selection of an appropriate dose of BX-U001 for the following Phase 2 study(Day 28)
- All-cause mortality(Day 28)
- Duration of hospital stay(Day 28)
- Proportion of patients achieving clinical response by the improvement of at least 2 points on the 7-point ordinal scale.(Day 14)