Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 005177D123 | Other Grant/Funding Number | Cystic Fibrosis Foundation |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Class |
|---|---|
| Cystic Fibrosis Foundation | OTHER |
Not provided
Not provided
Not provided
The purpose of this single arm clinical trial is to evaluate the effects of 7% hypertonic saline (HS) delivered by nebulizer on clearance of mucus from the lungs in people with bronchiectasis (dilated airways) not due to cystic fibrosis. Mucociliary clearance (MCC) to measure the rate at which a person's lungs can clear inhaled particles will be assessed at baseline, and after acute (single dose) HS treatment, as well as after two weeks of treatment with HS.
The study has two main questions:
Participants will participate in up to 5 study visits if completing both Aim 1 and Aim 2: 1 screening/enrollment visit, 2 baseline visits (1 baseline visit if only participating in Aim 2), 1 visit during which first dose of HS would be administered and assessed, and 1 visit after 2 weeks of treatment with HS.
The investigators will perform an open label, single arm pilot study of 7% HS as our treatment intervention in patients with NCFB. Participants will attend up to 5 study visits.
The first visit (V1) will be a screening visit to obtain informed consent if not already obtained, confirm that subjects meet all eligibility criteria through review of medical history and clinical records, and performance of a hypertonic saline tolerance test (HSTT). Participants meeting eligibility criteria will be enrolled in the study at that time, and subsequent study visits will be scheduled.
At each study visit, medical history will be reviewed. Pregnancy testing will be performed at all visits in participants who may become pregnant. Up to two baseline MCC scans (V2 and optional V3) will be performed under baseline conditions to assess variability, and expectorated sputum will be collected at either visit. Baseline spirometry and MBW will also be obtained at V2 and V3, and QOL-B will be assessed at V2 prior to any other study procedures that could impact their symptoms and responses.
At V4, the acute response to HS on MCC will be assessed after the first dose of HS is administered at the visit, marking the start of the two-week treatment period. Participants will be given a supply of HS to incorporate in their airway clearance regimen twice a day for two weeks (treatment period of up to 17 days, depending on exact timing of when V5 falls).
The evening prior to V5, patients will perform airway clearance and receive their final dose of HS. The following morning, they will hold airway clearance until after their study visit. At V5, they will complete post-treatment QOL-B, and Treatment Satisfaction Questionnaire for Medication (TSQM), MBW, spirometry, MCC (12 hours after last dose of HS, to assess sustained response to HS), and sputum collection.
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Single arm treatment group with 7% HS | Experimental | All study participants will receive 7% HS by nebulizer twice a day for two weeks as part of airway clearance. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 7% Hypertonic Saline via nebulization | Drug | Twice a day treatment with nebulized 7% HS for 2 weeks as part of airway clearance |
|
| Measure | Description | Time Frame |
|---|---|---|
| Repeatability of Mucociliary Clearance (MCC) at 60 minutes | The primary outcome for Aim 1 will be the correlation between the average rate of MCC measured in the whole right lung compartment over 60 minutes (MCC60), calculated using point estimates collected every 10 minutes, assessed from two separate visits (Visit 2 and Visit 3) | Day 1 (Visit 2) up to Day 14 +/- 3 (Visit 3) |
| Average change in MCC60 from baseline (Visit 2 and 3), after acute treatment with HS (Visit 4), and after two weeks of treatment with HS (Visit 5) | Mean baseline MCC60 from Visit 2 and optional Visit 3 (if participating in Aim 1) will be compared with MCC60 measured 30 minutes after first HS dose at Visit 4, and with MCC60 measured 12 hours after last dose of HS following 2 weeks of treatment with twice a day HS at Visit 5. MCC60 will be measured as the average rate of MCC measured in the whole right lung compartment over 60 minutes, calculated using point estimates collected every 10 minutes, at Visit 2 (mean Baseline from Visit 2 and Visit 3 if completing Aim 1), Visit 4, and Visit 5. | Day 1 (Visit 2) up to 7 weeks (Visit 5) |
| Measure | Description | Time Frame |
|---|---|---|
| Change in Forced Expiratory Volume in 1 Second (FEV1) from Baseline (mean from visit 2 and visit 3, pre-HS treatment) to Visit 5 (post-HS treatment period) | FEV1 is measured by spirometry. On each occasion, the best of 3 trials, based on American Thoracic Society (ATS)/European Respiratory Society (ERS) criteria. The best forced vital capacity (FVC), FEV1 and Forced expiratory flow between 25% and 75% of vital capacity (FEF25-75) (from trial with highest FVC+FEV1 sum) will be recorded (absolute value and % of predicted). Baseline measurements will occur at Visit 2 (and Visit 3 if completing Aim 1). The change in FEV1 from Baseline (either Visit 2, or mean from Visit 2 and Visit 3 if completing Aim 2) to Visit 5 will then be assessed. |
Not provided
Inclusion Criteria:
Exclusion Criteria
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Katherine A. Despotes, MD | Contact | 9199669198 | katherine.despotes@unchealth.unc.edu | |
| Alex Nesbit | Contact | alexandria_nesbit@med.unc.edu |
| Name | Affiliation | Role |
|---|---|---|
| Katherine A. Despotes, MD | University of North Carolina, Chapel Hill | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of North Carolina at Chapel Hill | Recruiting | Chapel Hill | North Carolina | 27514 | United States |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| ID | Term |
|---|---|
| D001987 | Bronchiectasis |
| ID | Term |
|---|---|
| D001982 | Bronchial Diseases |
| D012140 | Respiratory Tract Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| D012965 | Sodium Chloride |
| ID | Term |
|---|---|
| D002712 | Chlorides |
| D006851 | Hydrochloric Acid |
| D017606 | Chlorine Compounds |
| D007287 | Inorganic Chemicals |
Not provided
Not provided
This will be an open label, single arm pilot study of 7% hypertonic saline (HS) in patients with non-cystic fibrosis bronchiectasis (NCFB).
Not provided
Not provided
Not provided
Not provided
|
| Day 1 (Visit 2) up to 7 weeks (Visit 5) |
| Change in Quality of life for Bronchiectasis (QOL-B) Domain Scores from Visit 3 (2nd baseline visit, pre-HS treatment) to Visit 5 (post-HS treatment period) | The QOL-B Version 3.1 questionnaire is a self-administered patient reported outcome for symptom assessment and health-related quality of life in people with NCFB. It is a validated tool containing 37 items in 8 domains translated into multiple languages, with minimal important difference determined per each domain. Domains include Physical Functioning, Role Functioning, Vitality, Emotional Functioning, Social Functioning, Treatment Burden, Health Perceptions, Respiratory Symptoms. Items are on 4-point Likert scales, except for the Respiratory Symptoms domain which includes an item about sputum color, which has 6 answer choices (see separate outcome measure for sputum color). Each domain is scored separately, scores are scaled up to be in the range of 0 to 100 (higher scores correspond to better health outcomes). Participants will complete the QOL-B at baseline (Visit 2), and again post-HS treatment period (Visit 5), and changes in the domain scores will be assessed. | Day 1 (Visit 2) up to 5 weeks (Visit 5) |
| Change in Quality of life for Bronchiectasis (QOL-B) Respiratory Symptoms Domain Sputum Score from Visit 3 (2nd baseline visit, pre-HS treatment) to Visit 5 (post-HS treatment period) | The Respiratory Symptoms domain includes one item that assesses sputum color with 6 answer choices (unlike other items on the QOL-B which are scored on 4-point Likert scales). Answer choices include "clear" (1 point, reverse coded for scoring as 4 points), "clear to yellow" (2 points, reverse coded as 3 points), "yellowish-green" (3 points, reverse coded as 2 points), "brownish-dark" (4 points, reverse coded as 1 point), "green with traces of blood" (4 points, reverse coded as 1 point), and "don't know" (6 points, reverse coded to be not scored). The sputum score is reverse coded so that higher scores correspond to better health outcomes. Reverse coded score minimum 1, maximum 4. Participants will complete the QOL-B at baseline (Visit 2), and again post-HS treatment period (Visit 5), and changes in the domain scores will be assessed. | Day 1 (Visit 2) up to 5 weeks (Visit 5) |
| Change in Lung Clearance Index (LCI) as measured by Multiple Breath Washout (MBW) from Baseline (mean from Visit 2 and visit 3, pre-HS treatment) to Visit 5 (post-HS treatment period) | The LCI is the number of lung turnovers required to reduce exhaled nitrogen (N2) containing tracer gas concentration to 1/40th of its starting values. It is calculated by dividing the sum of exhaled tidal breaths (cumulative exhaled volume) by simultaneously measured functional residual capacity (FRC). LCI is able to detect abnormalities in lung function earlier than more traditional modalities such as spirometry. | Day 1 (Visit 2) up to 7 weeks (Visit 5) |
| D017670 |
| Sodium Compounds |