Trial Outcomes & Findings for Safety and Tolerability of Inhaled Nitric Oxide in Patients With Cystic Fibrosis (NCT NCT00570349)
NCT ID: NCT00570349
Last Updated: 2019-11-13
Results Overview
Methemoglobin level assessments were measured through blood draws - hematology. This test measures the amount of methemoglobin (a type of hemoglobin that is unable to transport oxygen to tissues) in blood. Normal methemoglobin percentage range 1% - 2%.
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
18 participants
Primary outcome timeframe
Baseline and 48 hours
Results posted on
2019-11-13
Participant Flow
Participant milestones
| Measure |
Inhaled Nitric Oxide Low Dose
Low dose group received Inhaled Nitric Oxide at 20 parts per million (ppm) via nasal cannula for 44 hours
|
Inhaled Nitric Oxide High Dose
High dose group received Inhaled Nitric Oxide at 40 ppm via nasal cannula for 44 hours.
|
Nitrogen
Nitrogen (Placebo) administered at 20 ppm or 40 ppm via nasal cannula over a 44 hour period
|
|---|---|---|---|
|
Overall Study
STARTED
|
6
|
6
|
6
|
|
Overall Study
COMPLETED
|
6
|
6
|
6
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Safety and Tolerability of Inhaled Nitric Oxide in Patients With Cystic Fibrosis
Baseline characteristics by cohort
| Measure |
Inhaled Nitric Oxide Low Dose
n=6 Participants
Low dose group received Inhaled Nitric Oxide at 20 parts per million (ppm) via nasal cannula for 44 hours
|
Inhaled Nitric Oxide High Dose
n=6 Participants
High dose group received Inhaled Nitric Oxide at 40 ppm via nasal cannula for 44 hours.
|
Nitrogen
n=6 Participants
Nitrogen (Placebo) administered at 20 ppm or 40 ppm via nasal cannula over a 44 hour period
|
Total
n=18 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
5 Participants
n=99 Participants
|
5 Participants
n=107 Participants
|
3 Participants
n=206 Participants
|
13 Participants
n=7 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
1 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
3 Participants
n=206 Participants
|
5 Participants
n=7 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Age, Continuous
|
16.9 years
STANDARD_DEVIATION 2.14 • n=99 Participants
|
15.6 years
STANDARD_DEVIATION 2.33 • n=107 Participants
|
17.7 years
STANDARD_DEVIATION 5.14 • n=206 Participants
|
16.7 years
STANDARD_DEVIATION 3.40 • n=7 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
5 Participants
n=7 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=99 Participants
|
4 Participants
n=107 Participants
|
5 Participants
n=206 Participants
|
13 Participants
n=7 Participants
|
|
Region of Enrollment
United States
|
6 participants
n=99 Participants
|
6 participants
n=107 Participants
|
6 participants
n=206 Participants
|
18 participants
n=7 Participants
|
PRIMARY outcome
Timeframe: Baseline and 48 hoursMethemoglobin level assessments were measured through blood draws - hematology. This test measures the amount of methemoglobin (a type of hemoglobin that is unable to transport oxygen to tissues) in blood. Normal methemoglobin percentage range 1% - 2%.
Outcome measures
| Measure |
Inhaled Nitric Oxide Low Dose
n=6 Participants
Low dose group received Inhaled Nitric Oxide at 20 parts per million (ppm) via nasal cannula for 44 hours
|
Inhaled Nitric Oxide High Dose
n=6 Participants
High dose group received Inhaled Nitric Oxide at 40 ppm via nasal cannula for 44 hours.
|
Nitrogen
n=6 Participants
Nitrogen (Placebo) administered at 20 ppm or 40 ppm via nasal cannula over a 44 hour period
|
|---|---|---|---|
|
Safety and Tolerability of Drug, Assessed by Change in Methemoglobin Levels
Methemoglobin Levels at Baseline
|
0.4 Percent of Methemoglobin Level
Standard Deviation 0.14
|
0.5 Percent of Methemoglobin Level
Standard Deviation 0.48
|
0.3 Percent of Methemoglobin Level
Standard Deviation 0.19
|
|
Safety and Tolerability of Drug, Assessed by Change in Methemoglobin Levels
Methemoglobin Levels at 48 hours
|
0.3 Percent of Methemoglobin Level
Standard Deviation 0.14
|
0.1 Percent of Methemoglobin Level
Standard Deviation 0.10
|
0.4 Percent of Methemoglobin Level
Standard Deviation 0.21
|
PRIMARY outcome
Timeframe: Baseline and 48 hoursSafety and tolerability of drug assessed by decreased oxygen saturation was measured through pulse oximeter, which measure the amount of oxygen in the blood. Normal range percentage is 95 - 100%
Outcome measures
| Measure |
Inhaled Nitric Oxide Low Dose
n=6 Participants
Low dose group received Inhaled Nitric Oxide at 20 parts per million (ppm) via nasal cannula for 44 hours
|
Inhaled Nitric Oxide High Dose
n=6 Participants
High dose group received Inhaled Nitric Oxide at 40 ppm via nasal cannula for 44 hours.
|
Nitrogen
n=6 Participants
Nitrogen (Placebo) administered at 20 ppm or 40 ppm via nasal cannula over a 44 hour period
|
|---|---|---|---|
|
Change in Oxygen Saturation
Oxygen Saturation at 48 hours
|
95.7 Percent of oxygen saturation
Standard Deviation 2.16
|
95.8 Percent of oxygen saturation
Standard Deviation 3.19
|
97.0 Percent of oxygen saturation
Standard Deviation 1.10
|
|
Change in Oxygen Saturation
Oxygen Saturation at Baseline
|
95.5 Percent of oxygen saturation
Standard Deviation 1.87
|
94.8 Percent of oxygen saturation
Standard Deviation 2.14
|
95.5 Percent of oxygen saturation
Standard Deviation 2.66
|
PRIMARY outcome
Timeframe: Baseline and 48 hoursDecrease in forced expiratory volume in 1 second was measured through spirometer. Spirometer measures the volume of air inspired and expired by the lungs.
Outcome measures
| Measure |
Inhaled Nitric Oxide Low Dose
n=6 Participants
Low dose group received Inhaled Nitric Oxide at 20 parts per million (ppm) via nasal cannula for 44 hours
|
Inhaled Nitric Oxide High Dose
n=6 Participants
High dose group received Inhaled Nitric Oxide at 40 ppm via nasal cannula for 44 hours.
|
Nitrogen
n=6 Participants
Nitrogen (Placebo) administered at 20 ppm or 40 ppm via nasal cannula over a 44 hour period
|
|---|---|---|---|
|
Change in Forced Expiratory Volume in 1 Second (FEV1)
Forced Expiratory Volume in 1 second at Baseline
|
3.4 Liters
Standard Deviation 0.66
|
3.1 Liters
Standard Deviation 1.07
|
3.0 Liters
Standard Deviation 0.85
|
|
Change in Forced Expiratory Volume in 1 Second (FEV1)
Forced Expiratory Volume in 1 second at 48 hours
|
3.5 Liters
Standard Deviation 0.66
|
3.1 Liters
Standard Deviation 1.17
|
3.0 Liters
Standard Deviation 0.83
|
Adverse Events
Inhaled Nitric Oxide Low Dose
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Inhaled Nitric Oxide High Dose
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Nitrogen
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Inhaled Nitric Oxide Low Dose
n=6 participants at risk
Low dose group received Inhaled Nitric Oxide at 20 parts per million (ppm) via nasal cannula for 44 hours
|
Inhaled Nitric Oxide High Dose
n=6 participants at risk
High dose group received Inhaled Nitric Oxide at 40 ppm via nasal cannula for 44 hours.
|
Nitrogen
n=6 participants at risk
Nitrogen (Placebo) administered at 20 ppm or 40 ppm via nasal cannula over a 44 hour period
|
|---|---|---|---|
|
Infections and infestations
Otitis Media
|
16.7%
1/6 • Number of events 1
|
0.00%
0/6
|
0.00%
0/6
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
16.7%
1/6 • Number of events 1
|
0.00%
0/6
|
0.00%
0/6
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
0.00%
0/6
|
16.7%
1/6 • Number of events 1
|
0.00%
0/6
|
Additional Information
Medical Information Call Center
Mallinckrodt Pharmaceuticals
Phone: 800-556-3314
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: OTHER