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Non-Tuberculous Mycobacterial PneumoniaPhase 2NCT03473314

Prolonged-Use of Inhaled Gaseous Nitric Oxide (gNO) for Adult With Non-Tuberculous Mycobacteria Infection

A Phase 2 study testing Nitric Oxide gas at 160ppm in people with Non-Tuberculous Mycobacterial Pneumonia, run by University of British Columbia at 1 site in Canada.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

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. Read more about this study

Who is it for?
Ages 19 years and older
Conditions the study is for
Non-Tuberculous Mycobacterial Pneumonia
What is being tested?
Nitric Oxide gas at 160ppm
Could I receive a placebo?
No, everyone receives an active treatment
How many people
1 participants
Where
1 siteCanada
Study dates
9 Mar 2018to 21 Jul 2020
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 19 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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.

Who cannot join

  • 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
Show all 13Show fewer
  • 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.

Trial locations and contacts

1 site in 1 country.

Canada1 site

  • Gordon Leslie Diamond Health Care Centre

    Vancouver, British Columbia, V5Z-1L8

Other trials in Canada

About this study

Official title: Open Label Prolonged-Use of Inhaled Gaseous Nitric Oxide (gNO) for a Single Adult With Non-Tuberculous Mycobacteria Infection

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.

Read the 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):

  1. 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.
  1. 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.
Being tested
Nitric Oxide gas at 160ppm
Keywords
Inhaled nitric oxide, Non-tuberculous mycobacteria, Drug resistant bacteria, Antimicrobial drug resistance

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Nitric Oxide gas at 160ppmDrug

    Also called Thiolanox

    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.

Study groups in NCT03473314
Study groupRoleWhat they receive
Nitric Oxide gas at 160ppmExperimentalNitric Oxide 160ppm for 50-80 minutes two -three times a day for 365 days
Receives: Drug: Nitric Oxide gas at 160ppm

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Eradication of NTM in sputum

    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.

    Measured: 365 days

Secondary outcomes (8)

  1. Mean absolute change in forced expiratory volume at one second (FEV1)% from baseline.

    Clinical Measurement of Mean absolute change in FEV1% from baseline to Day 365 (within group test).

    Measured: 365 days

  2. Mean change in distance walked in the six-minute walk test from baseline

    Clinical Measurement of Mean change in distance walked in the six-minute walk test from baseline.

    Measured: 365 days

  3. Mean change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) scores for each domain from baseline

    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.

    Measured: 365 days

  4. Recurrence of NTM in sputum culture post NTM eradication.

    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.

    Measured: 30 and 60 days post NTM eradication

  5. Nitric oxide delivery effect on clinical values in home delivery

    Safety measured as evaluated by the number of unanticipated adverse events in clinical labs (hematology, coagulation, and serum chemistries).

    Measured: 365 days

  6. Nitric oxide delivery effect on key physiologic vital signs in home delivery

    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.

    Measured: 365 days

  7. Nitric oxide effect on delivery parameter concentrations in home delivery

    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.

    Measured: 365 days

  8. Nitric oxide delivery effect on systemic methemoglobin levels in home delivery

    Safety measured as evaluated by the number of unanticipated adverse events in pulseoixmetric arterial methemoglobin percent levels during nitric oxide delivery.

    Measured: 365 days

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted22 Mar 2018
  2. Study start9 Mar 2018
  3. Primary completion11 Jul 2020
  4. Study completion21 Jul 2020

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Model description
Open label study
Masking
None (open label)
Enrolment
1 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03473314
Sponsor's protocol ID
NTM-SPU-01; H18-00512
Status last verified
Jul 2020
First submitted
14 Mar 2018
Last updated
29 Jul 2020
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Undecided

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Nitric Oxide gas at 160ppm in people with Non-Tuberculous Mycobacterial Pneumonia, run by University of British Columbia at 1 site in Canada.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 8 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 29 Jul 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Eradication of NTM in sputum (time frame: 365 days). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03473314.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Jul 2020.

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