A Phase 1 Study Evaluating the Safety and Tolerability of Autologous Non-hematopoietic Peripheral Blood Stem Cells Therapy in Healthy Adult Volunteers in Abu Dhabi, 2020 (SANTANA Study).
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 18
- 试验地点
- 2
- 主要终点
- St. George's Respiratory Questionnaire (SGRQ) scoring.
研究概览
简要总结
SANTANA Study is a phase 1, prospective, monocentric, open-label clinical trial involving healthy adult volunteers, with the objective to evaluate the safety and tolerability of the compressor nebulization of autologous non-hematopoietic peripheral blood stem cells (NHPBSC).
详细描述
The study was conducted in Abu Dhabi Stem Cells Center (ADSCC), and the cell processing and investigational product formulation were performed at the same center according to Good Laboratory Practices (GLPs) and Good Manufacturing Practices (GMPs). Participants received the investigational therapy through compressor (jet) nebulization (two doses divided on Days 0 and 1). The primary endpoint was the safety assessment, to be measured as Adverse Reactions (ARs) incidence [by the World Health Organization - Uppsala Monitoring Centre (WHO-UMC) causality assessment system], and the nebulization tolerability rate (Days 0 and 1). The Laboratory testing profile was evaluated before treatment (Day 0 or -1) and on Days 14 and 28. Two thorax radiography tests (chest X-rays - CXR) were assessed: before the therapy (Day 0 or -1) and on Day 7. An electrocardiogram (ECG) assessment was performed on Days 0 (or -1), 14, and 28. The application of the validated St. George's Respiratory Questionnaire (SGRQ) on Days 0 and 28 was a surrogate safety endpoint. The whole clinical assessment in SANTANA Study was daily recorded until Day 28, taking into account the ARs incidence. The secondary endpoint was the tolerability assessment, measured by nebulization tolerability rate (Days 0 and 1), and Adverse Events (AEs) tolerability within 28-days. The trial was approved by the ADSCC Research Ethics Committee, and written informed consent was obtained from all participants. SANTANA Study was conducted following the Declaration of Helsinki principles and Good Clinical Practices (GCPs), and the authors are responsible for designing the trial, compiling, and analyzing the data.
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
- 18 Years 至 50 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Healthy adult male or female volunteers (18-50 years inclusive). Healthy subjects: volunteers not having any mental or physical diagnosed disorder or requiring regular or frequent medication, who is not known to suffer any significant illness, who should be within the ordinary range of body measurements)
- •Non-smoking volunteers, based on self-reporting who have not used tobacco or nicotine-containing products (or less than 100 cigarettes or equivalent in his / her lifetime), including commercially available cigarettes, hand-rolled cigarettes, bidis, cigars, chewing tobacco, pipes, hookahs, shishas, snuff, snus, electronic cigarettes and similar devices, and nicotine replacement therapy within the last 12 months.
- •Body mass index (BMI) ≥ 19 kg/m².
- •For females: premenopausal, non-lactating, and non-pregnant.
- •Ability to comply with test requirements and peripheral blood stem cell collection.
- •The subject agrees to participate in the study and signs the SANTANA Study informed consent form.
排除标准
- •Pediatric subjects (aged < 18 years) or older than 50 years' volunteers.
- •Any clinically relevant gastrointestinal, renal, hepatic, neurological, hematological, endocrine, oncological, urological, immunological, and cardiovascular diseases or any other condition including clinically significant abnormal Laboratory parameters that, in the opinion of the Investigator, could jeopardize the safety of the subject.
- •Current smoking subjects (or former smokers who have quit smoking three months before eligibility assessment).
- •Body mass index (BMI) < 19 kg/m².
- •For females: Postmenopausal, lactating or pregnant women.
- •Donation or receipt of whole blood or blood products within three months before the screening visit.
- •Inability to comply with test requirements or peripheral blood stem cell collection.
- •Organ or cell transplants in the past three months.
- •History of malignancies in the past five years.
- •Have participated in other clinical trials in the past three months.
- •Employee of Abu Dhabi Stem Cells Center (ADSCC) or their first-degree relatives (parent, sibling, or child).
- •Inability to provide informed consent.
结局指标
主要结局
St. George's Respiratory Questionnaire (SGRQ) scoring.
时间窗: Days 0, 28.
Assessment of scoring changes.
Adverse reactions (AR) incidence.
时间窗: Days 0-28.
Proportion of participants with treatment-related adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE) v5.0 and the WHO-UMC system for the causality assessment.
Nebulization tolerability rate.
时间窗: Days 0, 1.
Proportion of participants that tolerate the NHPBSC nebulization successfully.
Adverse Events (AEs) tolerability rate.
时间窗: Days 0-28.
Proportion of participants that tolerate AEs -if any, and finish the study.
次要结局
未报告次要终点