Trial Outcomes & Findings for Aztreonam Aerosol to Treat Cystic Fibrosis Nasal Disease (NCT NCT02730793)
NCT ID: NCT02730793
Last Updated: 2021-01-28
Results Overview
Protocol-Defined Pulmonary Exacerbation includes events that are characterized by change or worsening pulmonary symptoms (increased cough, increased sputum production and chest congestion, decreased appetite and exercise tolerance), loss of weight, and lung function decline that prompt initation of antibiotic therapy. Protocol-defined exacerbations in subjects that warrant treatment with IV antibiotics will be determined from the medical record during the course of this study.
TERMINATED
PHASE2
5 participants
140 days
2021-01-28
Participant Flow
Participant milestones
| Measure |
Standard Therapy
Oral Inhalation via PARI Altera Device: Standard Therapy Comparator (Oral Aztreonam 75mg three times a day) AND Nasal Inhalation via PARI PAS Device: Nasal Placebo-Normal Saline twice per day
Oral Aztreonam: Standard Therapy Comparator (Oral Cayston 75mg three times a day)
Nasal Placebo: Placebo (nasal saline twice per day)
|
Study Therapy
Oral Inhalation via PARI Altera Device: Standard Therapy Comparator (Oral Aztreonam 75mg three times a day) AND Nasal Inhalation via PARI PAS Device: Experimental- Nasal Aztreonam 75 mg twice per day
Oral Aztreonam: Standard Therapy Comparator (Oral Cayston 75mg three times a day)
Nasal Aztreonam: Study Therapy (nasal Aztreonam 75mg twice per day)
|
|---|---|---|
|
Overall Study
STARTED
|
2
|
3
|
|
Overall Study
COMPLETED
|
2
|
1
|
|
Overall Study
NOT COMPLETED
|
0
|
2
|
Reasons for withdrawal
| Measure |
Standard Therapy
Oral Inhalation via PARI Altera Device: Standard Therapy Comparator (Oral Aztreonam 75mg three times a day) AND Nasal Inhalation via PARI PAS Device: Nasal Placebo-Normal Saline twice per day
Oral Aztreonam: Standard Therapy Comparator (Oral Cayston 75mg three times a day)
Nasal Placebo: Placebo (nasal saline twice per day)
|
Study Therapy
Oral Inhalation via PARI Altera Device: Standard Therapy Comparator (Oral Aztreonam 75mg three times a day) AND Nasal Inhalation via PARI PAS Device: Experimental- Nasal Aztreonam 75 mg twice per day
Oral Aztreonam: Standard Therapy Comparator (Oral Cayston 75mg three times a day)
Nasal Aztreonam: Study Therapy (nasal Aztreonam 75mg twice per day)
|
|---|---|---|
|
Overall Study
Physician Decision
|
0
|
1
|
|
Overall Study
Withdrawal by Subject
|
0
|
1
|
Baseline Characteristics
Aztreonam Aerosol to Treat Cystic Fibrosis Nasal Disease
Baseline characteristics by cohort
Baseline data not reported
PRIMARY outcome
Timeframe: 140 daysPopulation: Because of the low number of enrollees with only 1 in the treatment group, results are not reported to protect the confidentiality of the participants
Protocol-Defined Pulmonary Exacerbation includes events that are characterized by change or worsening pulmonary symptoms (increased cough, increased sputum production and chest congestion, decreased appetite and exercise tolerance), loss of weight, and lung function decline that prompt initation of antibiotic therapy. Protocol-defined exacerbations in subjects that warrant treatment with IV antibiotics will be determined from the medical record during the course of this study.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Up to 6 monthsPopulation: Because of the low number of enrollees with only 1 in the treatment group, results are not reported to protect the confidentiality of the participants
Protocol-Defined Pulmonary Exacerbation includes events that are characterized by change or worsening pulmonary symptoms (increased cough, increased sputum production and chest congestion, decreased appetite and exercise tolerance), loss of weight, and lung function decline that prompt initation of antibiotic therapy. Protocol-defined exacerbations in subjects that warrant treatment with IV antibiotics will be determined from the medical record during the course of this study. The time of beginning the study to first protocol-defined pulmonary exacerbation will also be determined from the medical record during the course of this study.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Day 140 and Day 168Population: Because of the low number of enrollees with only 1 in the treatment group, results are not reported to protect the confidentiality of the participants
The Sino-Nasal Outcome Test 20 (SNOT-20) is a validated health-related QOL questionnarie designed to determine the impact of sinonasal dysfunction. Patients will assess nasal symptoms, emotion, and activity on a scale of worsening symptoms scored 1 through 7 to provide a quantifiable score capable of disease severity. It has been shown as a responsive measure of health-related quality of life and suitable for use in outcomes studies and routine clinical care. Survey responses from Day 140 and Day 168 will be compared to the SNOT20 survey responses from previous visits (V1, V2, and V4)
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Day 140Population: Because of the low number of enrollees with only 1 in the treatment group, results are not reported to protect the confidentiality of the participants
The CFQ-R is a validated patient-reported outcome measuring health-related quality of life for children and adults with CF. The CFQ-R contains both general and CF-specific scales. The CFQ-R was administered at Visits 1, 6, and 7. The endpoint was change in CFQR on day 140 compared to V1 and V5.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Day 140Population: Because of the low number of enrollees with only 1 in the treatment group, results are not reported to protect the confidentiality of the participants
Routine spirometry will be performed according to American Thoracic Society (ATS) guidelines. A minimum of three maneuvers will be performed. The largest FVC and FEV1 will be reported after examining data from all acceptable curves even if they did not originate from the same maneuver. Data will be expressed both in absolute values and as percent (%) predicted based upon NHANES predicted values.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 1 yearPopulation: Because of the low number of enrollees with only 1 in the treatment group, results are not reported to protect the confidentiality of the participants
Expectorated sputum and nasal swabs will be cultured at each visit using standardized procedures to identify CF pathogens as well as susceptibility to a standard panel of antibiotics.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 1 yearPopulation: Because of the low number of enrollees with only 1 in the treatment group, results are not reported to protect the confidentiality of the participants
Acoustic rhinometry is used to measure cross-sectional volume of the nasal cavity allowing the calculation of nasal volume. Reflected sound waves are painlessly introduced through nasal adaptors into the nasal passages allowing the production of area-distance graphs.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 1 yearPopulation: Because of the low number of enrollees with only 1 in the treatment group, results are not reported to protect the confidentiality of the participants
The frequency, severity and duration of all nasal and pulmonary adverse events, regardless of cause, will be recorded in REDCap as an electronic case report form. Serious adverse events will be captured in OnCore. The frequency and severity of adverse events will be calculated for each patient, with each patient counted once using the most severe grade experienced. The duration of adverse events will be calculated by the number of days each event persisted. Tables will be generated for all adverse events including serious adverse events and withdraws from the study.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 1 yearPopulation: Because of the low number of enrollees with only 1 in the treatment group, results are not reported to protect the confidentiality of the participants
Protocol-Defined Pulmonary Exacerbation includes events that are characterized by change or worsening pulmonary symptoms (increased cough, increased sputum production and chest congestion, decreased appetite and exercise tolerance), loss of weight, and lung function decline that prompt initation of antibiotic therapy. Protocol-defined exacerbations in subjects that warrant treatment with oral antibiotics will be determined from the medical record during the course of this study.
Outcome measures
Outcome data not reported
Adverse Events
Standard Therapy
Study Therapy
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Bruce K. Rubin MEngr, MD, MBA, FRCPC
Virginia Commonwealth University
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place