Trial Outcomes & Findings for Inhaled Sodium Nitrite as an Antimicrobial for Cystic Fibrosis (NCT NCT02694393)

NCT ID: NCT02694393

Last Updated: 2024-06-24

Results Overview

forced expiratory volume in one second (FEV1) after inhalation will assess safety

• Recruitment status: TERMINATED
• Study phase: PHASE1/PHASE2
• Target enrollment: 4 participants
• Primary outcome timeframe: Day 7
• Results posted on: 2024-06-24

Participant Flow

The study had a 2 week wash-out period during which inhaled antibiotics were held before the drug was dosed. If participants had a drop in percent predicted FEV1 between the screening visit and Visit 1 where the drug was dosed, they were excluded from the study. Of our 4 enrolled subjects, 1 subject withdrew from study participation during the washout period and 1 subject failed the wash-out period and was excluded from the study before being assigned to treatment arm.

Participant milestones

Measure	Nebulized Nitrite Nebulized nitrite, 40-80 mg
Overall Study STARTED	2
Overall Study COMPLETED	1
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Inhaled Sodium Nitrite as an Antimicrobial for Cystic Fibrosis

Baseline characteristics by cohort

Measure	Sodium Nitrite n=2 Participants Inhalation of 46 or 80 mg of sodium nitrite twice daily for four weeks sodium nitrite: inhalation of 46 or 80 mg of sodium nitrite by electronic nebulization
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	2 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	25.5 years n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	2 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	2 participants n=5 Participants
FEV1 (Percent predicted)	61.5 Percent predicted FEV1 n=5 Participants
exhaled Nitric Oxide	6.5 Parts per billion n=5 Participants

PRIMARY outcome

Timeframe: Day 7

forced expiratory volume in one second (FEV1) after inhalation will assess safety

Outcome measures

Measure	Nebulized Nitrite n=2 Participants Nebulized sodium nitrite, 40-80 mg
Pulmonary Function	59 percentage of predicted FEV1 Interval 37.0 to 81.0

PRIMARY outcome

Timeframe: Day 28

forced expiratory volume in one second (FEV1) after inhalation will assess safety

Outcome measures

Measure	Nebulized Nitrite n=1 Participants Nebulized sodium nitrite, 40-80 mg
Pulmonary Function	78 percentage of predicted FEV1

SECONDARY outcome

Timeframe: Day 7

concentration of nitric oxide in exhaled breath

Outcome measures

Measure	Nebulized Nitrite n=2 Participants Nebulized sodium nitrite, 40-80 mg
Exhaled Nitric Oxide	10 parts per billion Interval 8.0 to 12.0

SECONDARY outcome

Timeframe: Day 28

concentration of nitric oxide in exhaled breath

Outcome measures

Measure	Nebulized Nitrite n=1 Participants Nebulized sodium nitrite, 40-80 mg
Exhaled Nitric Oxide	12 parts per billion

SECONDARY outcome

Timeframe: day 7

Population: Sputum nitrite data not collected/measured

concentration of nitrite in sputum

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: day 28

Population: Sputum nitrite data not collected/measured

concentration of nitrite in sputum

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: day 28

Population: Pseudomonas density in sputum data not collected/measured

density of Pseudomonas in sputum

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: day 28

Population: respiratory symptom questionnaire data not collected/measured

respiratory symptom questionnaire

Outcome measures

Outcome data not reported

Adverse Events

Nebulized Nitrite

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Nebulized Nitrite n=2 participants at risk 40-80 mg nebulized nitrite administration
Blood and lymphatic system disorders Methemoglobinemia	0.00% 0/2 • Up to 42 days following first drug administration visit The adverse events predicted to be of interest were: methemoglobinemia, hypotension and bronchoconstriction (as measured by FEV1)
Cardiac disorders Hypotension	50.0% 1/2 • Number of events 1 • Up to 42 days following first drug administration visit The adverse events predicted to be of interest were: methemoglobinemia, hypotension and bronchoconstriction (as measured by FEV1)
Respiratory, thoracic and mediastinal disorders Decreased FEV1	50.0% 1/2 • Number of events 1 • Up to 42 days following first drug administration visit The adverse events predicted to be of interest were: methemoglobinemia, hypotension and bronchoconstriction (as measured by FEV1)

Additional Information

Dr. Joseph Pilewski

University of Pittsburgh

Phone: (412) 692-2210‬

Email: pilewskijm@upmc.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place