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| Name | Class |
|---|---|
| Cystic Fibrosis Foundation | OTHER |
| Dartmouth-Hitchcock Medical Center | OTHER |
| University of Washington | OTHER |
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Despite the increasingly common use of cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies in treating cystic fibrosis (CF), it is still largely unknown whether or not other chronic therapies can be safely stopped. This SIMPLIFY sub-study is being done to test whether or not it is safe to stop taking dornase alfa (Dnase) in those people that are also taking elexacaftor/tezacaftor/ivacaftor (ETI).
ETI is a combination CFTR modulator therapy that was approved by the Food and Drug Administration for people with CF who have at least one F508del mutation. The three drugs that make up ETI work together to allow many more chloride ions to move into and out of the cells, improving the balance of salt and water in the lungs. These changes result in better clearance of mucus from the lungs and improvements in lung function.
Dornase alfa (Dnase) also improves clearance of mucus from the lungs to support lung function and has been available to people with CF for many years. Dnase is considered to be relatively burdensome and it is not known whether Dnase can improve or maintain lung function above what is already gained through ETI use.
The goal of this SIMPLIFY sub-study is to get information about whether or not it is safe to stop Dnase by testing if there is a change in lung function in participants with cystic fibrosis (CF) who are assigned to stop taking Dnase as compared to those who are assigned to keep taking Dnase while continuing to take ETI.
This is a sub study of master protocol SIMPLIFY-IP-19, NCT04378153.
The sub study investigating the impact of discontinuing and continuing hypertonic saline is registered under NCT06350461.
This SIMPLIFY sub-study (Dornase Alfa (Dnase) Trial) is designed to evaluate the effects of discontinuing Dnase in people with cystic fibrosis (CF) age 12 and older currently taking the highly effective modulator elexacaftor/tezacaftor/ivacaftor (ETI). This is an open label two-arm randomized non-inferiority trial consisting of a 2-week screening period, randomization to continue or discontinue dornase alfa, followed by a 6-week study period. Participants at trial entry will be randomized 1:1 to either continue or discontinue their Dnase therapy.
Clinical outcomes (forced expiratory volume in 1 second [FEV1], antibiotic use, pulmonary exacerbations, and patient reported outcomes), safety (adverse events) and patient reported outcomes to evaluate respiratory symptoms and the participant's perception of how stopping Dnase would impact their daily life will be evaluated in all subjects. Additionally, a subset of participants at selected study sites will participate in Multiple Breath Washout (MBW) to evaluate changes in lung clearance index (LCI).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Dnase - Discontinue | Experimental | Discontinuation of current dornase alfa (dnase) therapy |
|
| Dnase - Continue | Active Comparator | Continuation of current dornase alfa (dnase) therapy |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Discontinuation of dornase alfa (Dnase) | Other | Discontinuation of current dornase alfa (Dnase) therapy during 6-week study period. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Absolute Change in FEV1 % Predicted From Week 0 to Week 6 | Non-Inferiority statistical testing compared difference between study arms (discontinue - continue) in the absolute change in FEV1 % predicted from Week 0 to Week 6. | Week 0 to Week 6 |
| Measure | Description | Time Frame |
|---|---|---|
| Absolute Change in LCI 2.5 From Baseline to Week 6 | Statistical testing compared difference between study arms (discontinue - continue) in the absolute change in LCI 2.5 (Lung Clearance Index) from Baseline (Week 0, if available, or else Week -2) to Week 6. LCI 2.5 is the number of times the volume in the lungs needs to turn over to expel an inert gas. A higher value of LCI 2.5 indicates poorer lung function. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Nicole Mayer-Hamblett, PhD | University of Washington/Seattle Children's | Principal Investigator |
| Alex Gifford, MD, FCCP | Dartmouth-Hitchcock Medical Center | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Alabama at Birmingham | Birmingham | Alabama | 35294 | United States | ||
| Providence Alaska Medical Center |
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| ID | Title | Description |
|---|---|---|
| FG000 | Dnase - Discontinue | Discontinuation of current dornase alfa (dnase) therapy Discontinuation of dornase alfa (Dnase): Discontinuation of current dornase alfa (Dnase) therapy during 6-week study period. |
| FG001 | Dnase - Continue |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Feb 14, 2020 | Sep 30, 2024 |
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| Continuation of dornase alfa (Dnase) | Other | Continuation of current dornase alfa (dnase) therapy during 6-week study period. Therapy is taken at least once daily according to each participant's pre-existing, clinically prescribed regimen (e.g., daily, twice daily) |
|
| Baseline (Week 0 or Week -2) to Week 6 |
| Absolute Change in Respiratory Symptoms, as Measured by the CF Respiratory Symptoms Diary-Chronic Respiratory Infection Symptom Severity Score (CRISS) From Week 0 to Week 6 | Statistical testing compared the difference between study arms (discontinue - continue) in the absolute change in respiratory symptoms, as measured by the CF Respiratory Symptoms Diary-Chronic Respiratory Infection Symptom Severity Score (CRISS) from Week 0 to Week 6. The Cystic Fibrosis Respiratory Symptoms Daily Diary (CFRSD) asks a participant to state the extent of their 8 respiratory symptoms: difficulty breathing, feverishness, tiredness, chills or sweats, coughing, coughing up mucus, tightness in the chest and wheezing. Each respiratory symptom is assigned a score from 0-4 based on the response, with zero corresponding to the absence of the symptom and four corresponding to symptom being present 'a great deal' or 'extremely'. A summed score (range from 0-24) is calculated for each participant and converted to a final score with a range of 0 to 100 where the lowest scores indicate improvement of symptoms. Calculation of a score requires responses for at least 7 out of 8 symptoms | Week 0 to Week 6 |
| Absolute Change in Respiratory Symptoms, as Measured by CFQ-R Respiratory Domain From Week 0 to Week 6 | Statistical testing compared the difference between study arms (discontinue - continue) in the absolute change in respiratory symptoms, as measured by the Cystic Fibrosis Questionnaire-Revised Respiratory Domain Score from Week 0 to Week 6. The Cystic Fibrosis Questionnaire - Revised asks participants 6 questions related to respiratory symptoms which are each assigned a score 1-4. The Respiratory Domain Scaled Score is calculated as follows: 100*[{sum of responses}/{number of responses}-1]/3 only if number of responses ≥ 3; otherwise the score is set to missing. The scaled score ranges from 0 to 100 where higher scores indicate improvement of symptoms. | Week 0 to Week 6 |
| Absolute Change in FEV1 % Predicted From Week -2 to Week 0 | Statistical testing compared the difference between study arms (discontinue - continue) in the absolute change in FEV1 % predicted from Week -2 to Week 0. | Week -2 to Week 0 |
| Absolute Change in FEV1 % Predicted From Week 0 to Week 2 | Statistical testing compared the difference between study arms (discontinue - continue) in the absolute change in FEV1 % predicted from Week 0 to Week 2. | Week 0 to Week 2 |
| Number and Percent of Participants Initiating Acute Antibiotics From Week 0 to Week 6 | Difference between study arms (discontinue - continue) in the percent of subjects initiating acute oral, inhaled or intravenous antibiotics from Week 0 to Week 6. Includes antibiotics initiated for respiratory indications; excludes those taken as part of a chronic cycled regimen or for a UTI, skin infection, etc. | Week 0 to Week 6 |
| Number and Percent of Participants Hospitalized From Week 0 to Week 6 | Statistical testing compared the difference between study arms (discontinue - continue) in the percent of subjects hospitalized from Week 0 to Week 6. | Week 0 to Week 6 |
| Number and Percent of Participants Experiencing Pulmonary Exacerbations From Week 0 to Week 6 | Statistical testing compared the difference between study arms (discontinue - continue) in the percent of subjects experiencing a pulmonary exacerbation from Week 0 to Week 6. Pulmonary exacerbations defined using Fuchs criteria. | Week 0 to Week 6 |
| Number and Percent of Participants Experiencing Adverse Events (AEs) From Week 0 to Week 6 | Statistical testing compared the difference between study arms (discontinue - continue) in the percent of participants with at least one AE from Week 0 to Week 6. Includes serious and non-serious AEs. | Week 0 to Week 6 |
| Rate of Adverse Events (AEs) From Week 0 to Week 6 Therapy Arms | Statistical testing compared the compared the rate of AE occurrence (number of events divided by total follow-up weeks in each arm) between study arms from Week 0 to Week 6. Includes serious and non-serious AEs. | Week 0 to Week 6 |
| Number and Percent of Participants With Temporary or Permanent Changes From Assigned Therapy Regimen Due to Adverse Event From Week 0 to Week 6 | Statistical testing compared the difference between study arms (discontinue - continue) in the percent of subjects temporarily or permanently changing their assigned therapy regimen due to an adverse event Week 0 to Week 6 | Week 0 to Week 6 |
| Anchorage |
| Alaska |
| 99508 |
| United States |
| Tucson Cystic Fibrosis Center | Tucson | Arizona | 85724 | United States |
| Arkansas Children's Hospital | Little Rock | Arkansas | 72202 | United States |
| Miller Children's and Women's Hospital Long Beach | Long Beach | California | 90806 | United States |
| CHOC Children's Hospital | Orange | California | 92868 | United States |
| Stanford University Medical Center | Palo Alto | California | 94304 | United States |
| Rady Children's Hospital and Health Center at the University of California San Diego | San Diego | California | 92123 | United States |
| University of California, San Francisco - Adult Center | San Francisco | California | 94143 | United States |
| University of California, San Francisco - Peds Center | San Francisco | California | 94158 | United States |
| Children's Hospital Colorado | Aurora | Colorado | 80045 | United States |
| National Jewish Health | Denver | Colorado | 80206 | United States |
| Yale University School of Medicine | New Haven | Connecticut | 06520 | United States |
| University of Florida | Gainesville | Florida | 32610 | United States |
| Nemours Children's Clinic - Jacksonville | Jacksonville | Florida | 32207 | United States |
| Central Florida Pulmonary Group | Orlando | Florida | 32803 | United States |
| The Nemours Children's Clinic - Orlando | Orlando | Florida | 32827 | United States |
| Nemours Children's Clinic - Pensacola | Pensacola | Florida | 32514 | United States |
| All Children's Hospital | St. Petersburg | Florida | 33701 | United States |
| Tampa General Hospital | Tampa | Florida | 33606 | United States |
| Emory University | Atlanta | Georgia | 30324 | United States |
| Saint Luke's Cystic Fibrosis Center of Idaho | Boise | Idaho | 83702 | United States |
| Northwestern University | Chicago | Illinois | 60611 | United States |
| OSF Saint Francis Medical Center | Peoria | Illinois | 61637 | United States |
| Riley Hospital for Children | Indianapolis | Indiana | 46202 | United States |
| University of Iowa | Iowa City | Iowa | 52242 | United States |
| University of Kansas Medical Center | Kansas City | Kansas | 66160 | United States |
| University of Kentucky | Lexington | Kentucky | 40506 | United States |
| University of Louisville | Louisville | Kentucky | 40202 | United States |
| Tulane University | Metairie | Louisiana | 70001 | United States |
| Maine Medical Partners Pediatric Specialty Care | Portland | Maine | 04102 | United States |
| John Hopkins Hospital | Baltimore | Maryland | 21205 | United States |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | United States |
| University of Michigan, Michigan Medicine | Ann Arbor | Michigan | 48109 | United States |
| Wayne State University Harper University Hospital | Detroit | Michigan | 48201 | United States |
| Corewell Health Helen DeVos | Grand Rapids | Michigan | 49546 | United States |
| Children's Hospitals and Clinics of Minnesota | Minneapolis | Minnesota | 55404 | United States |
| The Minnesota Cystic Fibrosis Center | Minneapolis | Minnesota | 55455 | United States |
| Children's Mercy Kansas City | Kansas City | Missouri | 64108 | United States |
| Washington University School of Medicine | St Louis | Missouri | 63110 | United States |
| Billings Clinic | Billings | Montana | 59101 | United States |
| Dartmouth Hitchcock Medical Center | Lebanon | New Hampshire | 03756 | United States |
| Monmouth Medical Center | Eatontown | New Jersey | 07724 | United States |
| Morristown Medical Center | Morristown | New Jersey | 07960 | United States |
| Rutgers - Robert Wood Johnson Medical School | New Brunswick | New Jersey | 08903 | United States |
| Cohen Children's Medical Center of New York | New Hyde Park | New York | 11042 | United States |
| Beth Israel Medical Center | New York | New York | 10003 | United States |
| Lenox Hill Hospital Cystic Fibrosis Center | New York | New York | 10028 | United States |
| Columbia University Cystic Fibrosis Program | New York | New York | 10032 | United States |
| University of Rochester Medical Center Strong Memorial | Rochester | New York | 14642 | United States |
| SUNY Upstate Medical University | Syracuse | New York | 13210 | United States |
| New York Medical College at Westchester Medical Center | Valhalla | New York | 10595 | United States |
| University of North Carolina at Chapel Hill | Chapel Hill | North Carolina | 27517 | United States |
| Atrium Health Wake Forest Baptist | Winston-Salem | North Carolina | 27157 | United States |
| Children's Hospital Medical Center of Akron | Akron | Ohio | 44308 | United States |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
| Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center | Cleveland | Ohio | 44146 | United States |
| Cleveland Clinic Cystic Fibrosis Program | Cleveland | Ohio | 44195 | United States |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | United States |
| Dayton Children's Hospital | Dayton | Ohio | 45404 | United States |
| Oregon Health & Sciences University | Portland | Oregon | 97239 | United States |
| Hershey Medical Center Pennsylvania State University | Hershey | Pennsylvania | 17033 | United States |
| University of Pennsylvania | Philadelphia | Pennsylvania | 19104 | United States |
| University of Pittsburgh Medical Center | Pittsburgh | Pennsylvania | 15224 | United States |
| Medical University of South Carolina | Charleston | South Carolina | 29425 | United States |
| Dell Children's Medical Center of Central Texas | Austin | Texas | 78723 | United States |
| University of Texas Southwestern / Children's Health | Dallas | Texas | 75207 | United States |
| University of Texas Southwestern | Dallas | Texas | 75390 | United States |
| Cook Children's Medical Center | Fort Worth | Texas | 76104 | United States |
| University of Texas Health Center at Tyler | Tyler | Texas | 75708 | United States |
| Primary Children's Cystic Fibrosis Center | Salt Lake City | Utah | 84113 | United States |
| University of Vermont Medical Center | Burlington | Vermont | 05401 | United States |
| University of Virginia | Charlottesville | Virginia | 22904 | United States |
| Virginia Commonwealth University | Richmond | Virginia | 23219 | United States |
| Seattle Children's Hospital | Seattle | Washington | 98105 | United States |
| University of Washington Medical Center | Seattle | Washington | 98195 | United States |
| Providence Medical Group, Cystic Fibrosis Center | Spokane | Washington | 99204 | United States |
| West Virginia University - Morgantown | Morgantown | West Virginia | 26506 | United States |
| University of Wisconsin | Madison | Wisconsin | 53792 | United States |
| Children's Wisconsin | Milwaukee | Wisconsin | 53226 | United States |
| Froedtert & Medical College of Wisconsin | Milwaukee | Wisconsin | 53226 | United States |
Continuation of current dornase alfa (dnase) therapy
Continuation of dornase alfa (Dnase): Continuation of current dornase alfa (dnase) therapy during 6-week study period. Therapy is taken at least once daily according to each participant's pre-existing, clinically prescribed regimen (e.g., daily, twice daily)
| COMPLETED |
|
| NOT COMPLETED |
|
Primary analyses were done on the per-protocol (PP) population.
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| ID | Title | Description |
|---|---|---|
| BG000 | Dnase - Discontinue | Discontinuation of current dornase alfa (dnase) therapy Discontinuation of dornase alfa (Dnase): Discontinuation of current dornase alfa (Dnase) therapy during 6-week study period. |
| BG001 | Dnase - Continue | Continuation of current dornase alfa (dnase) therapy Continuation of dornase alfa (Dnase): Continuation of current dornase alfa (dnase) therapy during 6-week study period. Therapy is taken at least once daily according to each participant's pre-existing, clinically prescribed regimen (e.g., daily, twice daily) |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Age, Customized | Count of Participants | Participants |
| ||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
| ||||||||||||||||
| Cystic Fibrosis (CF) Genotype | Count of Participants | Participants |
| ||||||||||||||||
| Forced Expiratory Volume in 1 second (FEV1) | Mean | Standard Deviation | liters |
| |||||||||||||||
| FEV1 (% Predicted) | FEV1 % predicted is calculated using the Global Lung Initiative multi-ethnic reference equations for ages 3-95. | Mean | Standard Deviation | percent predicted |
| ||||||||||||||
| FEV1 (% Predicted) Distribution | Measure Description: FEV1 % predicted is calculated using the Global Lung Initiative multi-ethnic reference equations for ages 3-95. | Count of Participants | Participants |
| |||||||||||||||
| Current Dornase Alfa Use | Use as reported at screening prior to randomization. | Count of Participants | Participants |
| |||||||||||||||
| Current Airway Clearance Use | Includes airway clearance by route chest PT/vest. | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Absolute Change in FEV1 % Predicted From Week 0 to Week 6 | Non-Inferiority statistical testing compared difference between study arms (discontinue - continue) in the absolute change in FEV1 % predicted from Week 0 to Week 6. | Per-protocol (PP) population. | Posted | Mean | Standard Deviation | FEV1 % predicted | Week 0 to Week 6 |
|
|
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Absolute Change in LCI 2.5 From Baseline to Week 6 | Statistical testing compared difference between study arms (discontinue - continue) in the absolute change in LCI 2.5 (Lung Clearance Index) from Baseline (Week 0, if available, or else Week -2) to Week 6. LCI 2.5 is the number of times the volume in the lungs needs to turn over to expel an inert gas. A higher value of LCI 2.5 indicates poorer lung function. | Participants in the per-protocol (PP) population with an acceptable LCI 2.5 measurement at baseline and at Week 6. | Posted | Mean | Standard Deviation | number of lung volume turnovers | Baseline (Week 0 or Week -2) to Week 6 |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Absolute Change in Respiratory Symptoms, as Measured by the CF Respiratory Symptoms Diary-Chronic Respiratory Infection Symptom Severity Score (CRISS) From Week 0 to Week 6 | Statistical testing compared the difference between study arms (discontinue - continue) in the absolute change in respiratory symptoms, as measured by the CF Respiratory Symptoms Diary-Chronic Respiratory Infection Symptom Severity Score (CRISS) from Week 0 to Week 6. The Cystic Fibrosis Respiratory Symptoms Daily Diary (CFRSD) asks a participant to state the extent of their 8 respiratory symptoms: difficulty breathing, feverishness, tiredness, chills or sweats, coughing, coughing up mucus, tightness in the chest and wheezing. Each respiratory symptom is assigned a score from 0-4 based on the response, with zero corresponding to the absence of the symptom and four corresponding to symptom being present 'a great deal' or 'extremely'. A summed score (range from 0-24) is calculated for each participant and converted to a final score with a range of 0 to 100 where the lowest scores indicate improvement of symptoms. Calculation of a score requires responses for at least 7 out of 8 symptoms | Participants in the per-protocol (PP) population with a CRISS score at Week 0 and at Week 6. | Posted | Mean | Standard Deviation | score on a scale | Week 0 to Week 6 |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Absolute Change in Respiratory Symptoms, as Measured by CFQ-R Respiratory Domain From Week 0 to Week 6 | Statistical testing compared the difference between study arms (discontinue - continue) in the absolute change in respiratory symptoms, as measured by the Cystic Fibrosis Questionnaire-Revised Respiratory Domain Score from Week 0 to Week 6. The Cystic Fibrosis Questionnaire - Revised asks participants 6 questions related to respiratory symptoms which are each assigned a score 1-4. The Respiratory Domain Scaled Score is calculated as follows: 100*[{sum of responses}/{number of responses}-1]/3 only if number of responses ≥ 3; otherwise the score is set to missing. The scaled score ranges from 0 to 100 where higher scores indicate improvement of symptoms. | Participants in the per-protocol (PP) population with a score at Week 0 and at Week 6. | Posted | Mean | Standard Deviation | score on a scale | Week 0 to Week 6 |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Absolute Change in FEV1 % Predicted From Week -2 to Week 0 | Statistical testing compared the difference between study arms (discontinue - continue) in the absolute change in FEV1 % predicted from Week -2 to Week 0. | Per-protocol (PP) population. | Posted | Mean | Standard Deviation | FEV1 % predicted | Week -2 to Week 0 |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Absolute Change in FEV1 % Predicted From Week 0 to Week 2 | Statistical testing compared the difference between study arms (discontinue - continue) in the absolute change in FEV1 % predicted from Week 0 to Week 2. | Participants in the per-protocol (PP) population with FEV1 measurements at Week 0 and at Week 2. | Posted | Mean | Standard Deviation | FEV1 % predicted | Week 0 to Week 2 |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Number and Percent of Participants Initiating Acute Antibiotics From Week 0 to Week 6 | Difference between study arms (discontinue - continue) in the percent of subjects initiating acute oral, inhaled or intravenous antibiotics from Week 0 to Week 6. Includes antibiotics initiated for respiratory indications; excludes those taken as part of a chronic cycled regimen or for a UTI, skin infection, etc. | Intent-to-treat (ITT) population of all randomized participants. | Posted | Count of Participants | Participants | Week 0 to Week 6 |
|
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| Secondary | Number and Percent of Participants Hospitalized From Week 0 to Week 6 | Statistical testing compared the difference between study arms (discontinue - continue) in the percent of subjects hospitalized from Week 0 to Week 6. | Intent-to-treat (ITT) population of all randomized participants. | Posted | Count of Participants | Participants | Week 0 to Week 6 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Number and Percent of Participants Experiencing Pulmonary Exacerbations From Week 0 to Week 6 | Statistical testing compared the difference between study arms (discontinue - continue) in the percent of subjects experiencing a pulmonary exacerbation from Week 0 to Week 6. Pulmonary exacerbations defined using Fuchs criteria. | Intent-to-treat (ITT) population of all randomized participants. | Posted | Count of Participants | Participants | Week 0 to Week 6 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Number and Percent of Participants Experiencing Adverse Events (AEs) From Week 0 to Week 6 | Statistical testing compared the difference between study arms (discontinue - continue) in the percent of participants with at least one AE from Week 0 to Week 6. Includes serious and non-serious AEs. | Posted | Count of Participants | Participants | Week 0 to Week 6 |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Rate of Adverse Events (AEs) From Week 0 to Week 6 Therapy Arms | Statistical testing compared the compared the rate of AE occurrence (number of events divided by total follow-up weeks in each arm) between study arms from Week 0 to Week 6. Includes serious and non-serious AEs. | Intent-to-treat (ITT) population of all randomized participants. | Posted | Number | events per week | Week 0 to Week 6 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Number and Percent of Participants With Temporary or Permanent Changes From Assigned Therapy Regimen Due to Adverse Event From Week 0 to Week 6 | Statistical testing compared the difference between study arms (discontinue - continue) in the percent of subjects temporarily or permanently changing their assigned therapy regimen due to an adverse event Week 0 to Week 6 | Intent-to-treat (ITT) population of all randomized participants. | Posted | Count of Participants | Participants | Week 0 to Week 6 |
|
|
From randomization (Week 0) up to last study visit (Week 6), i.e., 6 weeks.
Not provided
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Dnase - Discontinue | Discontinuation of dornase alfa (Dnase): Discontinuation of current dornase alfa (Dnase) therapy during 6-week study period. | 0 | 240 | 0 | 240 | 25 | 240 |
| EG001 | Dnase - Continue | Continuation of dornase alfa (Dnase): Continuation of current dornase alfa (dnase) therapy during 6-week study period. Therapy is taken at least once daily according to each participant's pre-existing, clinically prescribed regimen (e.g., daily, twice daily) | 0 | 237 | 0 | 237 | 14 | 237 |
Not provided
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (23.0) | Non-systematic Assessment |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Margaret Kloster | Seattle Children's | 206-884-7862 | margaret.kloster@seattlechildrens.org |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jul 6, 2020 | Sep 30, 2024 | SAP_001.pdf |
| ID | Term |
|---|---|
| D003550 | Cystic Fibrosis |
| ID | Term |
|---|---|
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| D003851 | Deoxyribonucleases |
| ID | Term |
|---|---|
| D004950 | Esterases |
| D006867 | Hydrolases |
| D004798 | Enzymes |
| D045762 | Enzymes and Coenzymes |
Not provided
Not provided
| >=18 to <24 |
|
| >=24 to <30 |
|
| >=30 |
|
| Male |
|
| Black or African American |
|
| Asian |
|
| American Indian or Alaskan Native |
|
| Native Hawaiian or Other Pacific Islander |
|
| Other, More than One Race, or Unknown/Not Reported |
|
| F508del Heterozygous |
|
| Other or Unknown |
|
| >=60 to <70 |
|
| >=70 to <90 |
|
| >=90 to <100 |
|
| >=100 |
|
|
|
|
Continuation of dornase alfa (Dnase): Continuation of current dornase alfa (dnase) therapy during 6-week study period. Therapy is taken at least once daily according to each participant's pre-existing, clinically prescribed regimen (e.g., daily, twice daily) |
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|---|---|
| Participants |
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