A Phase I, Trial to Evaluate the Safety, Tolerability, and Potential Efficacy of Allogeneic Human Mesenchymal Stem Cell Infusion in Patients With Mild Asthma
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 3
- 试验地点
- 2
- 主要终点
- Number of Participant with treatment emergent serious adverse events
研究概览
简要总结
A Phase 1 investigation will be performed to test the safety of two doses of bone marrow-derived MSCs (20,000,000 and 100,000,000) administered via peripheral intravenous infusion.
详细描述
A Phase 1 investigation will be performed to test the safety of two doses of bone marrow-derived MSCs (20,000,000 and 100,000,000) administered via peripheralintravenous infusion.
Group 1: 3 subjects will receive a single administration of allogeneic hMSCs: 20 million cells delivered via peripheral intravenous infusion Group 2: 3 subjects will receive a single administration of allogeneic hMSCs: 100 million cells delivered via peripheral intravenous infusion Interim safety analysis will be performed four weeks after the 1st subject is enrolled in each cohort. Continued safety and tolerability with review of adverse events (AEs) will be assessed at each visit. Efficacy parameters (pulmonary function tests, diffusing capacity (DLCO), lung volumes, 6-minute walk test (6MWT), and dyspnea/quality of life [QOL] questionnaires) will be assessed every 12 weeks until study completion. Clinical laboratory tests to assess safety will be performed at every visit.
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 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Provide written informed consent
- •be between 18 and 65 years at the time of signing the Informed Consent
- •have a clinical diagnosis of asthma prior to screening in accordance with the guidelines of the American Thoracic Society/European Respiratory Society
- •ACQ over 1.25
- •have a smoking history of less than 10 pack-years total and have not been smoking for at least the last 12 months
- •Perform a positive methacholine challenge at screening and repeat positive methacholine challenge at baseline visit (14 days later)
- •Have normal or mild obstructive spirometry
- •Have normal right heart function as documented by Doppler echo or right heart catheterization
- •If female, be surgically sterile, post-menopausal (more than 1 year), or practice double barrier methods of birth control
- •Subjects may receive non-drug therapies including oxygen supplementation no greater than 2L/minute, and pulmonary rehabilitation
- •Subjects may be on standard of care asthma medications including inhaled corticosteroids-long acting beta agonist at a dose not greater than 1 mg of a fluticasone equivalent
排除标准
- •Have any active infection that is not treated
- •Be unable to perform any of the assessments required for endpoint analysis.
- •currently receive (or have received within four weeks of screening) experimental agents for the treatment of asthma
- •be actively listed (or expecting to be listed in the near future) for transplant of any organ
- •Have clinically important abnormal screening laboratory values : blood screening tests (Hematology, Chemistry, CBC including Eosinophil count) results that are not within normal limits (according to UMHC Laboratory Reference Ranges) Have a serious comorbid illness that, in the opinion of the investigator, may compromise the safety or compliance of the patient or preclude successful completion of the study
- •Have known allergies to penicillin or streptomycin
- •Be an organ transplant recipient
- •Have a clinical history of malignancy within 5 years (i.e., patients with prior malignancy must be disease free for 5 years), except curatively-treated basal cell carcinoma
- •Have a non-pulmonary condition that limits lifespan to less than a year.
- •Have a history of drug or alcohol abuse within the past 24 months.
- •Be serum positive for HIV, hepatitis BsAg or Viremia hepatitis C
- •Be currently participating (or have participated within the previous 30 days) in an investigational therapeutic or device trial.
- •Have hypersensitivity to dimethyl sulfoxide (DMSO)
- •Have a resting oxygen saturation (SpO2) on room air of more than 93% at sea level or more than 88% at an altitude above 5,000 feet above sea level (1524 meters)
结局指标
主要结局
Number of Participant with treatment emergent serious adverse events
时间窗: Week 4 post infusion
as defined as the incidence of any treatment-emergent serious adverse events; these are a composite of death, non-fatal pulmonary embolism, stroke, hospitalization for worsening dyspnea and clinically significant laboratory test abnormalities
次要结局
- Difference in subject reported dyspnea and quality of life assessments(Participants will be followed from 1 week to an expected average of 48 weeks following infusion)
- Death from any cause(Participants will be followed from 1 week to an expected average of 48 weeks following infusion)
- Difference in lung function(Participants will be followed from 1 week to an expected average of 48 weeks following infusion)
- Decrease in peripheral eosinophilia(Participants will be followed from 1 week to an expected average of 48 weeks following infusion)
研究者
Marilyn Glassberg
Professor of Medicine, Surgery, and Pediatrics
University of Miami