Phase 2 Randomized Parallel-Group Double-Blind Placebo-Controlled Multiple-Dose Proof-of-Concept Study to Evaluate the Efficacy/Safety of Hyaluronic Acid Inhalation Solution for Treatment of Emphysema
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 27
- 试验地点
- 6
- 主要终点
- Assessment of pulmonary function tests
研究概览
简要总结
The purpose of the study is to evaluate the safety and efficacy of administering repeated doses of Hyaluronic Acid Inhalation Solution to subjects with Emphysema that have Alpha-1-Antitrypsin deficiency
详细描述
The study primarily aims to establish desmosine and isodesmosine concentrations in plasma, sputum and urine measured as markers of elastin degradation systemically in the lung and also markers of inflammation and fibrinogen. Assessment of vital signs, lab tests, carbon monoxide diffusing capacity, oxygen saturation, pulmonary function tests, ECGs, physical exams and adverse events.
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
- Double (Participant, Investigator)
盲法说明
treatment has blinded labeling
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
- •Able and willing to provide written informed consent and comply with study requirements
- •Men or women aged 18 through 80 years at the time of consent
- •Diagnosis of emphysema at screening consistent with National Institutes of Health guidelines 19 GOLD COPD classification stages I, II or III
- •Evidence of emphysema on radiographic imaging.
- •A ratio of pre-bronchodilator FEV1 to forced vital capacity (FVC) of ≤ 80% at screening
- •FEV1 ≥ 30% and ≤ 79% (post-bronchodilator) of predicted normal at screening
- •Clinical laboratory tests (complete blood count, serum chemistry, and urinalysis) within normal limits or clinically acceptable to the PI and sponsor at screening
- •Women of childbearing potential and men who are sexually active must agree to use an adequate method of contraception (oral contraceptives, depot progesterone, condom plus spermicidal, or IUD) during the study and for 1 month after the final dose of study drug.
- •Evidence of alpha-1 antitrypsin deficiency (AATD) with any genotype except PiMZ deficiency. Individuals with PiMZ deficiency are not allowed in the study.
- •Patients must have stopped using Intravenous alpha-1 antitrypsin protein (AAT) augmentation therapy at least 3 months before entering study.
排除标准
- •Subjects with measured DLCO of ≤ 35%, or unable to perform a reproducible DLCO
- •Subjects unable to perform 3 reproducible spirometry tests after 8 attempts
- •Upper or lower respiratory tract infection within 2 weeks prior to screening and baseline (day1)
- •Presence of clinically relevant abnormality on chest x-ray (other than evidence of emphysema) within the previous 12 months
- •Use of supplemental oxygen therapy
- •Requirement for ventilator support within the last year
- •Exacerbation requiring treatment with systemic corticosteroids within the last 3 months
- •History of lung transplant or liver transplant.
- •Presence of clinically relevant abnormality on electrocardiogram (ECG)
- •Any medical condition that, in the investigator's judgment, would compromise study participation or the evaluation of the study drug
- •Women who are pregnant or breastfeeding
- •Receipt of an investigational drug within 30 days prior to screening
- •Patients who are current smokers or have smoked within the last 3 months -
研究组 & 干预措施
Hyaluronic Acid inhalation solution
3mL of 0.03% Hyaluronic Acid inhalation solution BID for 28 days
干预措施: Hyaluronic Acid Inhalation Solution (Drug)
Placebo Inhalation Solution
3mL matching placebo inhalation solution BID for 28 days
干预措施: Placebo Inhalation Solution (Drug)
结局指标
主要结局
Assessment of pulmonary function tests
时间窗: 28 days
measurement of pulmonary function
Measurement of sputum, plasma and urine concentrations of desmosine and isodesmosine
时间窗: 28 days
measurement of biomarkers
次要结局
- Assessment of St. George Respiratory Questionnaire(28 days)
研究者
Gerard Turino
Sponsor-Investigator
Turino, Gerard, M.D.