Cystic Fibrosis
Conditions
Keywords
Cystic Fibrosis, high flow 20 liters per minute (LPM) humidification therapy
Brief summary
This study is a single center, randomized pilot study to evaluate the clinical effectiveness of nasal high flow 20LPM humidification therapy in subjects with Cystic Fibrosis.
Interventions
DEVICEHigh-Flow, 20 LPM (via Optiflow cannula)
Nasal high flow humidification (20LPM) therapy will be administered using Optiflow with Airvo 2.
DEVICELow FLow, 5 LPM (via Optiflow cannula)
standard humidified wall medical air.
Sponsors
Virginia Commonwealth University
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
SUPPORTIVE_CARE
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
10 Years to 50 Years
Healthy volunteers
No
Inclusion criteria
* Confirmed diagnosis of CF * Hospital admission for acute pulmonary exacerbation of CF (using Fuchs criteria 4/10) * 10 years of age and older * Subject is able to comply with the procedures scheduled in the protocol * Signed informed consent form
Exclusion criteria
* Receipt of any aerosolized experimental or investigational drugs within 1 month of enrollment * Subject had ear, nose, and throat (ENT) surgery, nasal bleeding, or nasal polyps within 6 months prior to study * Subject is unlikely to comply with the procedures scheduled in the protocol * Inability to give informed consent * Subject requires supplemental oxygen * History of obstructive sleep apnea * History of pressure headaches requiring therapy within one month of enrollment * Any other medical or psychological condition in which the study doctor(s) believe(s) would inhibit the individual from being an appropriate study subject.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) | Day 0 to Day 6 or upon discharge from the hospital, whichever comes first | The Cystic Fibrosis Questionnaire-Revised (CFQ-R) is a validated health-related quality of life measure for CF that meets US Food and Drug Administration psychometric requirements for patient reported outcomes. It contains both generic and CF-specific scales and has demonstrated responsiveness in previous clinical studies. Scores range from 0 to 100 with higher scores indicating better health. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Sputum Collection | 10 minutes | Sputum is collected during pulmonary function test (PFT) to later measure the biophysical properties of cystic fibrosis sputum. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Experimental Therapy High-Flow, 20 LPM (via Optiflow cannula) Heated (34C) Humidified Air
High-Flow, 20 LPM (via Optiflow cannula): Nasal high flow humidification (20LPM) therapy will be administered using Optiflow with Airvo 2. | 12 |
| Control Therapy (Low Flow) Low FLow, 5 LPM (via Optiflow cannula) Room Temperature (23-26C) Ambient Air
Low FLow, 5 LPM (via Optiflow cannula): standard humidified wall medical air. | 12 |
| Total | 24 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Difficulty sleeping | 1 | 0 |
| Overall Study | Morning headache | 0 | 1 |
| Overall Study | Physician Decision | 0 | 1 |
| Overall Study | Protocol Violation | 4 | 0 |
| Overall Study | Withdrawal by Subject | 1 | 1 |
Baseline characteristics
| Characteristic | Experimental Therapy | Total | Control Therapy (Low Flow) |
|---|---|---|---|
| Age, Continuous | 19.4 years STANDARD_DEVIATION 5.4 | 20.5 years STANDARD_DEVIATION 6.1 | 21.7 years STANDARD_DEVIATION 6.7 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 1 Participants | 1 Participants | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 11 Participants | 23 Participants | 12 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 1 Participants | 1 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 4 Participants | 6 Participants | 2 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 8 Participants | 17 Participants | 9 Participants |
| Region of Enrollment United States | 12 participants | 24 participants | 12 participants |
| Sex: Female, Male Female | 5 Participants | 13 Participants | 8 Participants |
| Sex: Female, Male Male | 7 Participants | 11 Participants | 4 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 6 | 0 / 9 |
| other Total, other adverse events | 2 / 6 | 1 / 9 |
| serious Total, serious adverse events | 0 / 6 | 0 / 9 |
Outcome results
Primary
Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R)
The Cystic Fibrosis Questionnaire-Revised (CFQ-R) is a validated health-related quality of life measure for CF that meets US Food and Drug Administration psychometric requirements for patient reported outcomes. It contains both generic and CF-specific scales and has demonstrated responsiveness in previous clinical studies. Scores range from 0 to 100 with higher scores indicating better health.
Time frame: Day 0 to Day 6 or upon discharge from the hospital, whichever comes first
Population: Results could not be reported for 4 participants due to missing baseline data in one case, missing followup data in one case, and no CFQ-R data in two cases.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Experimental Therapy | Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) | Social limitations subscale | -0.056 change in score on a scale | Standard Deviation 0.096 |
| Experimental Therapy | Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) | Digestive symptoms subscale | 0.111 change in score on a scale | Standard Deviation 0.192 |
| Experimental Therapy | Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) | Eating disturbances subscale | 0.037 change in score on a scale | Standard Deviation 0.28 |
| Experimental Therapy | Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) | Physical functioning subscale | 0.056 change in score on a scale | Standard Deviation 0.105 |
| Experimental Therapy | Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) | Emotional state subscale | -0.089 change in score on a scale | Standard Deviation 0.139 |
| Experimental Therapy | Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) | Vitality subscale | 0.192 change in score on a scale | Standard Deviation 0.139 |
| Experimental Therapy | Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) | Body image subscale | 0.037 change in score on a scale | Standard Deviation 0.17 |
| Experimental Therapy | Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) | Treatment constraints subscale | -0.037 change in score on a scale | Standard Deviation 0.17 |
| Experimental Therapy | Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) | Weight subscale | -0.222 change in score on a scale | Standard Deviation 0.192 |
| Experimental Therapy | Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) | Role limitations subscale | -0.056 change in score on a scale | Standard Deviation 0.192 |
| Experimental Therapy | Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) | Health status subscale | -0.083 change in score on a scale | Standard Deviation 0.399 |
| Experimental Therapy | Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) | Respiratory symptoms subscale | 0.019 change in score on a scale | Standard Deviation 0.17 |
| Control Therapy (Low Flow) | Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) | Health status subscale | 0.000 change in score on a scale | Standard Deviation 0.103 |
| Control Therapy (Low Flow) | Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) | Emotional state subscale | 0.033 change in score on a scale | Standard Deviation 0.087 |
| Control Therapy (Low Flow) | Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) | Social limitations subscale | -0.028 change in score on a scale | Standard Deviation 0.154 |
| Control Therapy (Low Flow) | Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) | Role limitations subscale | 0.010 change in score on a scale | Standard Deviation 0.175 |
| Control Therapy (Low Flow) | Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) | Eating disturbances subscale | 0.097 change in score on a scale | Standard Deviation 0.162 |
| Control Therapy (Low Flow) | Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) | Treatment constraints subscale | -0.097 change in score on a scale | Standard Deviation 0.093 |
| Control Therapy (Low Flow) | Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) | Respiratory symptoms subscale | -0.125 change in score on a scale | Standard Deviation 0.856 |
| Control Therapy (Low Flow) | Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) | Digestive symptoms subscale | 0.028 change in score on a scale | Standard Deviation 0.098 |
| Control Therapy (Low Flow) | Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) | Physical functioning subscale | 0.052 change in score on a scale | Standard Deviation 0.152 |
| Control Therapy (Low Flow) | Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) | Body image subscale | 0.125 change in score on a scale | Standard Deviation 0.192 |
| Control Therapy (Low Flow) | Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) | Weight subscale | 0.083 change in score on a scale | Standard Deviation 0.236 |
| Control Therapy (Low Flow) | Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) | Vitality subscale | 0.194 change in score on a scale | Standard Deviation 0.115 |
Secondary
Sputum Collection
Sputum is collected during pulmonary function test (PFT) to later measure the biophysical properties of cystic fibrosis sputum.
Time frame: 10 minutes
Population: We were unable to collect sputum samples from the participants