- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03473314
Prolonged-Use of Inhaled Gaseous Nitric Oxide (gNO) for Adult With Non-Tuberculous Mycobacteria Infection
Open Label Prolonged-Use of Inhaled Gaseous Nitric Oxide (gNO) for a Single Adult With Non-Tuberculous Mycobacteria Infection
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
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
• as evaluated by the number of unanticipated adverse events during home delivery in clinical labs (hematology, coagulation, and serum chemistries); in vitals; in inspired concentration of NO, O2 and NO2 delivered to subject and; in methemoglobin and oxygen saturation levels.
Efficacy
- as determined by improvement in lung function as measured by spirometry, endurance as measured by six minute walk-test and quality of life as determined by self-reporting quality of life questionnaire (CFQ-R) on Day 7, 14, 21 and every 21 days thereafter for 90 days as compared to pre-treatment baseline data.
- as assessed by recurrence of NTM in sputum as confirmed by measurement of semi-quantitative culture sputum growth on Day 30 and 60 post treatment.
Study Type
Enrollment (Actual)
Phase
- Phase 2
Contacts and Locations
Study Locations
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British Columbia
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Vancouver, British Columbia, Canada, V5Z-1L8
- Gordon Leslie Diamond Health Care Centre
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
- Allocation: NA
- Interventional Model: SINGLE_GROUP
- Masking: NONE
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
EXPERIMENTAL: Nitric Oxide gas at 160ppm
Nitric Oxide 160ppm for 50-80 minutes two -three times a day for 365 days
|
Nitric oxide gas at 160 ppm inhaled three times daily for 50-80 min delivered with air as the carrier via inhalation for a maximum total of 90 days (extended 365 days twice).
Total dose of 480 ppm hours per day.
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Eradication of NTM in sputum
Time Frame: 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.
|
365 days
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Mean absolute change in forced expiratory volume at one second (FEV1)% from baseline.
Time Frame: 365 days
|
Clinical Measurement of Mean absolute change in FEV1% from baseline to Day 365 (within group test).
|
365 days
|
Mean change in distance walked in the six-minute walk test from baseline
Time Frame: 365 days
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Clinical Measurement of Mean change in distance walked in the six-minute walk test from baseline.
|
365 days
|
Mean change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) scores for each domain from baseline
Time Frame: 365 days
|
Clinical Measurement of Mean change in CFQ-R scores for each domain from baseline.
Each domain measure the magnitude of severity for each of the 8 items.
Respondents score each item using a 5-point likert scale ranging from 0 (no symptom) to 4 (the highest magnitude of severity).
For item 1 (difficult to breathe), item 2 (feel feverish), item 3 (tired), item 6 (mucus), and item 7 (chest tightness) the response options are: 0=No symptom, 1=a little, 2=somewhat, 3=a good deal, 4=a great deal.
For item 4 (chills/sweats), item 5 (cough), and item 8 (wheezing) the response options are: 0=no symptom, 1=slightly, 2=moderately, 3=very, 4=extremely.
|
365 days
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Recurrence of NTM in sputum culture post NTM eradication.
Time Frame: 30 and 60 days post NTM eradication
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Measurement of recovered NTM organisms in sputum colony forming unit (CFU) g (log 10) post eradication.
Eradication will be defined as two negative sputum cultures post nitric oxide gas treatment over 60 days.
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30 and 60 days post NTM eradication
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Nitric oxide delivery effect on clinical values in home delivery
Time Frame: 365 days
|
Safety measured as evaluated by the number of unanticipated adverse events in clinical labs (hematology, coagulation, and serum chemistries).
|
365 days
|
Nitric oxide delivery effect on key physiologic vital signs in home delivery
Time Frame: 365 days
|
Safety measured as evaluated by the number of unanticipated adverse events in vitals signs (blood pressure, respiratory rate) and oxygen saturation levels during NO delivery.
|
365 days
|
Nitric oxide effect on delivery parameter concentrations in home delivery
Time Frame: 365 days
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Safety measured as evaluated by the number of unanticipated adverse events with inspired concentration of nitric oxide (NO), oxygen (O2) and nitrogen dioxide (NO2) delivered to subject.
|
365 days
|
Nitric oxide delivery effect on systemic methemoglobin levels in home delivery
Time Frame: 365 days
|
Safety measured as evaluated by the number of unanticipated adverse events in pulseoixmetric arterial methemoglobin percent levels during nitric oxide delivery.
|
365 days
|
Collaborators and Investigators
Sponsor
Collaborators
Investigators
- Principal Investigator: Jeremy Road, MD, University of British Columbia
Study record dates
Study Major Dates
Study Start (ACTUAL)
Primary Completion (ACTUAL)
Study Completion (ACTUAL)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (ACTUAL)
Study Record Updates
Last Update Posted (ACTUAL)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Infections
- Bacterial Infections
- Bacterial Infections and Mycoses
- Gram-Positive Bacterial Infections
- Actinomycetales Infections
- Mycobacterium Infections
- Mycobacterium Infections, Nontuberculous
- Physiological Effects of Drugs
- Neurotransmitter Agents
- Molecular Mechanisms of Pharmacological Action
- Vasodilator Agents
- Autonomic Agents
- Peripheral Nervous System Agents
- Protective Agents
- Bronchodilator Agents
- Anti-Asthmatic Agents
- Respiratory System Agents
- Antioxidants
- Free Radical Scavengers
- Endothelium-Dependent Relaxing Factors
- Gasotransmitters
- Nitric Oxide
Other Study ID Numbers
- NTM-SPU-01; H18-00512
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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