Open Label Prolonged-Use of Inhaled Gaseous Nitric Oxide (gNO) for a Single Adult With Non-Tuberculous Mycobacteria Infection
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 1
- 试验地点
- 1
- 主要终点
- Eradication of NTM in sputum
研究概览
简要总结
An open labeled Study (NCT03331445) is demonstrating encouraging safety and efficacy results for most subjects receiving 160ppm nitric oxide gas (gNO) for treatment of non-tuberculous mycobacteria (NTM) over a 15 day treatment regimen. In one subject, who had a reduction in sputum culture concentration of Bacterium bolletii from plus 3 to plus 1 corresponding to a 2-3 log10 cfu/gm reduction during the treatment, the one-week post treatment follow-up sputum culture had increased to plus 2. It is hypothesized that a longer treatment period may be necessary to fully eradicate NTM from the sputum culture in chronic lung disease. This study will extend the period of gNO exposure for a prolonged period of time (3 months) to attempt to fully eradicate the NTM in this single subject. This study will transition from the medical clinic to supervised delivery in the patient's home environment.
详细描述
Primary Objective: Determine the efficacy of prolonged delivery of inhaled nitric oxide to treat an adult patient with pulmonary NTM Primary Endpoint: Eradication of NTM growth in sputum cultures. Efficacy will be assessed by the antimicrobial effect of inhaled NO on the density of NTM species and other microorganisms in the sputum.
• as confirmed by measurement of semi-quantitative culture sputum growth which has been verified with serial dilution technique on Day 7, 14, 21 and every 21 days thereafter for 90 days as compared to pre-treatment baseline sputum culture.
Secondary Objective(s): Determine the safety & efficacy of inhaled nitric oxide
Secondary Endpoint(s):
- Safety
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
- 19 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Written informed consent.
- •Has been previously diagnosed with NTM. [NTM defined as positive culture(s) of at least one species of Mycobacterium avium Complex (MAC) or Mycobacterium abscessus Complex (MABSC)]
- •Has been previously treated with gNO for 15 days without complete eradication of NTM but with a decrease of at least 1-2 points on cultures.
- •Male or female ≥19 years of age.
- •Female not pregnant at time of study.
- •Oxygen saturation on room air ≥92% at screening. (able to breathe without supplemental oxygen for 60 minutes)
- •Non-smoker for at least 6 months prior to screening and agrees not to smoke during the study.
- •Willing and able to comply with the treatment schedule and procedures.
排除标准
- •History of frequent epistaxis (>1 episode/month)
- •History of reactive pulmonary vascular hypertension
- •Methemoglobin >3% at screening
- •Liver function insufficiency aspartate aminotransferase/alanine aminotransferase (AST/ALT) >3 of normal values)
- •Hemoglobin <10 g/dl
- •Thrombocytopenia (platelet count <100,000/mm3) at screening
- •Prothrombin time international ratio (INR) > 1.3 at screening
- •On supplemental oxygen during gNO treatment (SaO2 < 90% for 50 minutes while resting in a chair).
- •For women of child bearing potential:
- •positive pregnancy test at screening or
- •lactating or
- •unwilling to practice a medically acceptable form of contraception from screening to Day 36 (acceptable forms of contraception: abstinence, hormonal birth control, intrauterine device, or barrier method plus a spermicidal agent)
- •Presence of a condition or abnormality that in the opinion of the Investigator would compromise the safety of the patient or the quality of the data.
研究组 & 干预措施
Nitric Oxide gas at 160ppm
Nitric Oxide 160ppm for 50-80 minutes two -three times a day for 365 days
干预措施: Nitric Oxide gas at 160ppm (Drug)
结局指标
主要结局
Eradication of NTM in sputum
时间窗: 365 days
The primary efficacy variable for this study is eradication of recovered NTM organisms in sputum colony forming unit (CFU) g (log 10) from baseline. Eradication will be defined as two negative sputum cultures post nitric oxide gas treatment over 60 days.
次要结局
- Mean change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) scores for each domain from baseline(365 days)
- Nitric oxide delivery effect on systemic methemoglobin levels in home delivery(365 days)
- Mean absolute change in forced expiratory volume at one second (FEV1)% from baseline.(365 days)
- Nitric oxide delivery effect on key physiologic vital signs in home delivery(365 days)
- Mean change in distance walked in the six-minute walk test from baseline(365 days)
- Nitric oxide effect on delivery parameter concentrations in home delivery(365 days)
- Recurrence of NTM in sputum culture post NTM eradication.(30 and 60 days post NTM eradication)
- Nitric oxide delivery effect on clinical values in home delivery(365 days)
研究者
Jeremy Road
Principal Investigator
University of British Columbia