Phase 1/2a Study of Umbilical Cord Lining Stem Cells (ULSC) in Patients With ARDS Due to COVID-19
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- Restem, LLC.
- 入组人数
- 17
- 试验地点
- 4
- 主要终点
- Treatment-emergent adverse events (AE) and serious adverse events (SAE)
研究概览
简要总结
ULSC-CV-01 is a clinical trial that comprises both Phase 1 and Phase 2a, which will be conducted sequentially. This trial will evaluate the safety and potential efficacy of allogeneic Umbilical Cord Lining Stem Cells (ULSC), which are a type of umbilical cord tissue derived mesenchymal stem cells (MSC), with intravenous (IV) administration in hospitalized patients with acute respiratory distress syndrome (ARDS) due to COVID-19.
详细描述
The Phase 1, open-label, non-controlled trial in this study will investigate the safety of intravenous (IV) infusion of ULSC in a total of 20 patients with COVID-19-related ARDS that will include patients that are not intubated and not on a ventilator (NV) and patients that are intubated and on a ventilator (V) for respiratory support. Separate cohorts of each group (NV and V) will receive either a single dose (one infusion) or repeat dose (two infusions separated by 48-hour interval).
The Phase 2a randomized and placebo-controlled trial in this study will investigate the potential efficacy of IV infusion of ULSC in a total of 40 patients with COVID-19-related ARDS that will all be EITHER NV or V; the determination of that eligibility criterion and the ULSC dosing regimen will be based on Phase 1 data of safety and tolerability. Phase 2a will evaluate EITHER single dose (one infusion) or repeat dose (two infusions separated by 48-hour interval). The randomization will be 3:1 with 30 patients receiving investigational product (ULSC) and 10 patients receiving placebo (carrier control).
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 18 Years 至 —（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 the presence of the COVID-19 agent with confirmation of COVID-19 by standard reverse transcriptase polymerase chain reaction (RT-PCR) or equivalent test.
- •Patient with diagnosis of COVID-related ARDS, classified as either:
- •Not requiring mechanical ventilation (NV) or
- •Requiring mechanical ventilation (V).
- •According to Berlin Definition of Acute Respiratory Distress Syndrome (ARDS), patients will be categorized based on degrees of hypoxemia [arterial partial pressure of oxygen (PaO2)/oxygen concentration (FiO2)]:
- •Mild ARDS: 200 mm Hg < PaO2/FIO2 ≤ 300 mm Hg
- •Moderate ARDS: 100 mm Hg < PaO2/FIO2 ≤ 200 mm Hg
- •Severe ARDS: PaO2/FIO2 ≤ 100 mm Hg
- •Patient who has exhibited deterioration in condition during the past 72 hours prior to the informed consent.
- •Patient receiving standard of care in-hospital therapy, including appropriate critical oxygenation, fluid, and hemodynamic support as indicated clinically.
- •Patient or responsible family member or surrogate signs informed consent.
- •Exclusion Criteria
- •Hypersensitivity to study product components. History of hypersensitivity to dimethyl sulfoxide (DMSO).
- •Active cancer or prior diagnosis of cancer within the past year; however, patients with basal and squamous cell cancer of skin will not be excluded.
- •Organ transplant recipient.
- •Chronic renal failure being treated by renal replacement therapy (dialysis) before development of COVID-
- •Any other condition that, in the judgment of the Investigator or Sponsor, would be a contraindication to enrollment, study product administration, or follow-up.
- •Pregnancy or lactation; a negative pregnancy test between screening and day 1 (before administration of treatment) will be required of women with childbearing potential, and they will be advised of the requirement to use an effective means of contraception. A woman is considered to be of childbearing potential unless she is postmenopausal and without menses for 12 months or without a uterus and/or both ovaries.
排除标准
- 未提供
结局指标
主要结局
Treatment-emergent adverse events (AE) and serious adverse events (SAE)
时间窗: 12 months
Treatment-emergent adverse events (AE; incidence, grade, and assessment of relatedness or causality) and serious adverse events (SAE) during the study and up to the 12-month follow-up
Incidence of Dose Limiting Toxicity (DLT)
时间窗: 24 hours
Number of subjects with a DLT event during or within 24 hours after ULSC infusion \[Dose Limiting Toxicities are treatment-emergent suspected adverse reactions graded as severe, such as severe infusion-related hypersensitivity toxicities of grade ≥3, and any treatment-emergent serious adverse event (SAE) will be investigated to determine if DLT.\]
Incidence of Dose Limiting Toxicity (DLT), suspected adverse reaction (SAR), or serious adverse event (SAE)
时间窗: 1 week
Number of subjects with a DLT event, suspected adverse reaction, or any serious adverse event (SAE) within 1 week of each ULSC infusion
次要结局
- Changes in levels of blood glucose (mg/dL) from baseline(1 month, 2 months, 3 months, 6 months, and 12 months)
- Changes in levels of potassium (mEq/L) from baseline(1 month, 2 months, 3 months, 6 months, and 12 months)
- Changes in levels of alanine transaminase (ALT; U/L) from baseline(1 month, 2 months, 3 months, 6 months, and 12 months)
- Changes in Complete Blood Count (CBC) with differential from baseline(1 month, 2 months, 3 months, 6 months, and 12 months)
- Changes in levels of blood urea nitrogen (BUN; mg/dL) from baseline(1 month, 2 months, 3 months, 6 months, and 12 months)
- Change in Urinalysis (UA) from baseline(1 month)
- Number of ventilator-free days (VFD)(1 month)
- Levels of COVID-19 related ARDS as defined by the Berlin Definition of ARDS(1 month)
- Changes from baseline pulse oximetric saturation SpO2/FiO2 ratio or arterial oxygen pressure pAO2/FiO2 ratio(1 month)
- Changes in levels of sodium (mEq/L) from baseline(1 month, 2 months, 3 months, 6 months, and 12 months)