Trial Outcomes & Findings for A Proof of Concept Study of Inhaled Nitric Oxide for Adults With Pulmonary Non-Tuberculous Mycobacterial Infection (NCT NCT03748992)
NCT ID: NCT03748992
Last Updated: 2021-05-18
Results Overview
Sputum culture will be assessed for any sputum growth. A negative culture represents no mycobacterial growth.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
10 participants
Primary outcome timeframe
End of Treatment (Day 15)
Results posted on
2021-05-18
Participant Flow
Participant milestones
| Measure |
Inhaled Nitric Oxide (gNO)
Participants will be treated for 3 weeks (5 days per week), Monday through Friday, and followed monthly for 3 months.
The treatment period includes a physical exam, vital signs, blood tests, echocardiogram, spirometry test, sputum sample, questionnaires, and the inhalation gas treatment. During the inhalation gas treatment, participants will wear a face mask (similar to an oxygen mask) and will be given nitric oxide gas through the participants nose for three treatments with fifty minutes each time. They will then be followed up monthly during the Follow-up period with tests that include a physical exam and questionnaires.
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|---|---|
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Overall Study
STARTED
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10
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Overall Study
COMPLETED
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10
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Overall Study
NOT COMPLETED
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0
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Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Proof of Concept Study of Inhaled Nitric Oxide for Adults With Pulmonary Non-Tuberculous Mycobacterial Infection
Baseline characteristics by cohort
| Measure |
Inhaled Nitric Oxide (gNO)
n=10 Participants
Participants will be treated for 3 weeks (5 days per week), Monday through Friday, and followed monthly for 3 months.
The treatment period includes a physical exam, vital signs, blood tests, echocardiogram, spirometry test, sputum sample, questionnaires, and the inhalation gas treatment. During the inhalation gas treatment, participants will wear a face mask (similar to an oxygen mask) and will be given nitric oxide gas through the participants nose for three treatments with fifty minutes each time. They will then be followed up monthly during the Follow-up period with tests that include a physical exam and questionnaires.
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|---|---|
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Age, Continuous
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47.7 years
STANDARD_DEVIATION 24.6 • n=5 Participants
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Sex: Female, Male
Female
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3 Participants
n=5 Participants
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Sex: Female, Male
Male
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7 Participants
n=5 Participants
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Ethnicity (NIH/OMB)
Hispanic or Latino
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1 Participants
n=5 Participants
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Ethnicity (NIH/OMB)
Not Hispanic or Latino
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9 Participants
n=5 Participants
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Ethnicity (NIH/OMB)
Unknown or Not Reported
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0 Participants
n=5 Participants
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Race (NIH/OMB)
American Indian or Alaska Native
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0 Participants
n=5 Participants
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Race (NIH/OMB)
Asian
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0 Participants
n=5 Participants
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Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
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0 Participants
n=5 Participants
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Race (NIH/OMB)
Black or African American
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0 Participants
n=5 Participants
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Race (NIH/OMB)
White
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9 Participants
n=5 Participants
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Race (NIH/OMB)
More than one race
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0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
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Region of Enrollment
United States
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10 participants
n=5 Participants
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PRIMARY outcome
Timeframe: End of Treatment (Day 15)Sputum culture will be assessed for any sputum growth. A negative culture represents no mycobacterial growth.
Outcome measures
| Measure |
Inhaled Nitric Oxide (gNO)
n=10 Participants
Participants will be treated for 3 weeks (5 days per week), Monday through Friday, and followed monthly for 3 months.
The treatment period includes a physical exam, vital signs, blood tests, echocardiogram, spirometry test, sputum sample, questionnaires, and the inhalation gas treatment. During the inhalation gas treatment, participants will wear a face mask (similar to an oxygen mask) and will be given nitric oxide gas through the participants nose for three treatments with fifty minutes each time. They will then be followed up monthly during the Follow-up period with tests that include a physical exam and questionnaires.
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|---|---|
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Percent Patients With Negative Sputum Culture
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40 percentage of participants
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SECONDARY outcome
Timeframe: Treatment Day 1 through End of Treatment (3 Months)Adverse Events will be assessed by patient reporting and routine lab work
Outcome measures
| Measure |
Inhaled Nitric Oxide (gNO)
n=10 Participants
Participants will be treated for 3 weeks (5 days per week), Monday through Friday, and followed monthly for 3 months.
The treatment period includes a physical exam, vital signs, blood tests, echocardiogram, spirometry test, sputum sample, questionnaires, and the inhalation gas treatment. During the inhalation gas treatment, participants will wear a face mask (similar to an oxygen mask) and will be given nitric oxide gas through the participants nose for three treatments with fifty minutes each time. They will then be followed up monthly during the Follow-up period with tests that include a physical exam and questionnaires.
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|---|---|
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Number of Participants With Treatment Related Adverse Events as Assessed by the CTCAE (Common Terminology Criteria for Adverse Events)
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6 Participants
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SECONDARY outcome
Timeframe: At day 15 from baselineSemiquantitative Culture Score is an assessment of bacterial burden with a lower score suggesting a lesser bacterial burden. The Semiquantitative Culture Score at Day 15 was compared to baseline, and a reduction in the Semiquantitative Culture Score suggests a reduced bacterial burden with treatment. Cultures were reported as positive if growth occurs in broth medium only; growth on broth medium plus solid medium cultures with countable colonies will be reported as 0-49 colonies, 1+; solid medium growth with 50-99 colonies, 2+; solid medium growth with 100-199 colonies; 3+, solid medium growth with 200-299 colonies; and 4+, solid medium growth with at least 300 colonies. For data analysis, each culture was scored as follows: 0, no growth in broth or solid medium; 1, broth medium growth only; 2, countable colonies (\<50 colonies) on solid medium; and 3-6, 1+ to 4+ growth on solid medium, respectively.
Outcome measures
| Measure |
Inhaled Nitric Oxide (gNO)
n=10 Participants
Participants will be treated for 3 weeks (5 days per week), Monday through Friday, and followed monthly for 3 months.
The treatment period includes a physical exam, vital signs, blood tests, echocardiogram, spirometry test, sputum sample, questionnaires, and the inhalation gas treatment. During the inhalation gas treatment, participants will wear a face mask (similar to an oxygen mask) and will be given nitric oxide gas through the participants nose for three treatments with fifty minutes each time. They will then be followed up monthly during the Follow-up period with tests that include a physical exam and questionnaires.
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|---|---|
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Number of Participants With a Reduction in Semiquantitative Cultures
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4 participants
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Adverse Events
Inhaled Nitric Oxide (gNO)
Serious events: 1 serious events
Other events: 6 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Inhaled Nitric Oxide (gNO)
n=10 participants at risk
evaluate the efficacy and safety of open-label exposure of gNO in patients with NTM lung disease
gNO: This is a proof-of-concept study in which all subjects will be treated with gNO (i.e. no control) to see if there can be a microbiological effect by conversion to negative sputum cultures and how long that effect can be sustained following treatment.
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|---|---|
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Vascular disorders
Transient Ischemic Attack
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10.0%
1/10 • Number of events 1 • Patients were assessed for AE's throughout the duration of the study, approximately 3 months
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Other adverse events
| Measure |
Inhaled Nitric Oxide (gNO)
n=10 participants at risk
evaluate the efficacy and safety of open-label exposure of gNO in patients with NTM lung disease
gNO: This is a proof-of-concept study in which all subjects will be treated with gNO (i.e. no control) to see if there can be a microbiological effect by conversion to negative sputum cultures and how long that effect can be sustained following treatment.
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|---|---|
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Cardiac disorders
Chest tightness
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10.0%
1/10 • Number of events 2 • Patients were assessed for AE's throughout the duration of the study, approximately 3 months
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Respiratory, thoracic and mediastinal disorders
Cough increased
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10.0%
1/10 • Number of events 1 • Patients were assessed for AE's throughout the duration of the study, approximately 3 months
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Nervous system disorders
Headache
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20.0%
2/10 • Number of events 2 • Patients were assessed for AE's throughout the duration of the study, approximately 3 months
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Gastrointestinal disorders
Hematochezia
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10.0%
1/10 • Number of events 2 • Patients were assessed for AE's throughout the duration of the study, approximately 3 months
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Respiratory, thoracic and mediastinal disorders
Hemoptysis
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30.0%
3/10 • Number of events 9 • Patients were assessed for AE's throughout the duration of the study, approximately 3 months
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Gastrointestinal disorders
Nausea
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10.0%
1/10 • Number of events 1 • Patients were assessed for AE's throughout the duration of the study, approximately 3 months
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General disorders
Night sweats
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10.0%
1/10 • Number of events 1 • Patients were assessed for AE's throughout the duration of the study, approximately 3 months
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Nervous system disorders
Pain/pleurisy
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20.0%
2/10 • Number of events 2 • Patients were assessed for AE's throughout the duration of the study, approximately 3 months
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Infections and infestations
Shingles
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10.0%
1/10 • Number of events 1 • Patients were assessed for AE's throughout the duration of the study, approximately 3 months
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Respiratory, thoracic and mediastinal disorders
Sputum increased
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10.0%
1/10 • Number of events 1 • Patients were assessed for AE's throughout the duration of the study, approximately 3 months
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Gastrointestinal disorders
Vomiting
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10.0%
1/10 • Number of events 1 • Patients were assessed for AE's throughout the duration of the study, approximately 3 months
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Respiratory, thoracic and mediastinal disorders
Wheezing
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20.0%
2/10 • Number of events 2 • Patients were assessed for AE's throughout the duration of the study, approximately 3 months
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Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place