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To provide efficacy and safety data comparing two dosing schedules of Tobramycin Inhalation Powder (TIP) for the treatment of pulmonary Pseudomonas aeruginosa in patients with cystic fibrosis.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Arm 1 | Experimental | Tobramycin Inhalation Powder (112 mg) once daily during 168 days |
|
| Arm 2 | Active Comparator | Tobramycin Inhalation Powder (112 mg) twice daily on days 1-28, days 57-84 and days 113-140 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Tobramycin Inhalation Powder | Drug | Tobramycin Inhalation Powder 112 mg (four 28-mg capsules) taken via inhaler once or twice a day, depending on study arm |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Forced Expiratory Volume in 1 Second ( FEV1) Percent Predicted | The Forced Expiratory Volume in 1 second (FEV1) percent predicted expresses FEV1 as a percentage of the "predicted values" for participants of similar characteristics (height, age, sex, and sometimes race and weight). A positive change from baseline in FEV1 percent predicted indicates improvement in lung function. | Baseline and Day 168 |
| Measure | Description | Time Frame |
|---|---|---|
| Percent Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) Percent Predicted | The Forced Expiratory Volume in 1 second (FEV1) percent predicted expresses FEV1 as a percentage of the "predicted values" for participants of similar characteristics (height, age, sex, and sometimes race and weight). A positive change from baseline in FEV1 percent predicted indicates improvement in lung function. |
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Inclusion Criteria:
Exclusion Criteria:
Other protocol-defined inclusion/exclusion criteria may apply.
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| Name | Affiliation | Role |
|---|---|---|
| Novartis Pharmaceuticals | Novartis Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Novartis Investigative Site | Mobile | Alabama | 36608-1128 | United States | ||
| Novartis Investigative Site |
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The study intended to randomize 200 patients in 18 months; however, after 9 months, only 25 patients were randomized, with a screen fail rate of 50%. Due to premature termination, summaries and analyses planned in the protocol were eliminated in the statistical analysis plan prior to database lock. No inferential analysis will be provided.
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| ID | Title | Description |
|---|---|---|
| FG000 | Tobramycin Inhalation Powder Once Daily | Tobramycin Inhalation Powder (112 mg) once daily during 168 days |
| FG001 | Tobramycin Inhalation Powder Twice Daily | Tobramycin Inhalation Powder (112 mg) twice daily on days 1-28, days 57-84 and days 113-140 |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Baseline and Day 168 |
| Percent Change From Baseline in Forced Vital Capacity (FVC) Percent Predicted | Forced Vital Capacity (FVC) is the amount of air which can be forcibly exhaled from the lungs after taking the deepest breath possible. FVC will be assessed via spirometry. A positive change from baseline in FVC indicates improvement in lung function. | Baseline and Day 168 |
| Percent Change From Baseline in Forced Expiratory Flow (FEF) 25%-75% Predicted | The Forced Expiratory Flow (FEF) 25%-75% measurement describes the amount of air expelled from the lungs during the middle half (25% - 75%) of the forced vital capacity test and is measured using spirometry. A positive change from baseline in FEF indicates improvement in lung function. The predicted percent will be assessed. | Baseline and day 168 |
| Change From Baseline in Pseudomonas Aeruginosa Sputum Density | Change from baseline in Pseudomonas aeruginosa sputum density will be measured by log10 colony forming units per gram of sputum. | Baseline and day 168 |
| Time to First Hospitalization Due to Respiratory-related Events | Time to the first hospitalization due to respiratory-related events (number of days) per patient. | Day 1 to day 168 |
| Percentage of Patients With Hospitalizations Due to Respiratory-related Events | Percentage of patients with hospitalization due to respiratory-related events | Day 1 to day 168 |
| Length of Hospital Stay Due to Respiratory-related Events | The number of days in length of hospital stay per patient due to respiratory-related events will be measured. | Day 1 to day 168 |
| Time to First Usage of Anti-pseudomonal Antibiotic | Time to first usage of anti-pseudomonal antibiotic per patient will be assessed by number of days | Day 1 to day 168 |
| Percentage of Patients Who Use Anti-pseudomonal Antibiotic | Percentage of patients who use anti-pseudomonal antibiotic will be assessed. | Day 1 to day 168 |
| Duration of Use of Anti-pseudomonal Antibiotic | Number of days of use of anti-pseudomonal antibiotic per patient will be assessed. | Day 1 to day 168 |
| Change From Baseline in Tobramycin Minimal Inhibitory Concentration for Pseudomonas Aeruginosa | Change from baseline in tobramycin minimal inhibitory concentration for Pseudomonas aeruginosa will be measured by laboratory testing. | Baseline and day 168 |
| Los Angeles |
| California |
| 90027 |
| United States |
| Novartis Investigative Site | Sacramento | California | 95819 | United States |
| Novartis Investigative Site | Ventura | California | 93003 | United States |
| Novartis Investigative Site | Altamonte Springs | Florida | 32701 | United States |
| Novartis Investigative Site | Jacksonville | Florida | 32207 | United States |
| Novartis Investigative Site | Miami | Florida | 33136 | United States |
| Novartis Investigative Site | Orlando | Florida | 32806 | United States |
| Novartis Investigative Site | Pensacola | Florida | 32504 | United States |
| Novartis Investigative Site | Glenview | Illinois | 60025 | United States |
| Novartis Investigative Site | Indianapolis | Indiana | 46202 | United States |
| Novartis Investigative Site | Iowa City | Iowa | 52242 | United States |
| Novartis Investigative Site | St Louis | Missouri | 63104 | United States |
| Novartis Investigative Site | New Hyde Park | New York | 11040 | United States |
| Novartis Investigative Site | New York | New York | 10595 | United States |
| Novartis Investigative Site | Akron | Ohio | 44308 | United States |
| Novartis Investigative Site | Oklahoma City | Oklahoma | 73112 | United States |
| Novartis Investigative Site | Portland | Oregon | 92772 | United States |
| Novartis Investigative Site | Hershey | Pennsylvania | 17033-8050 | United States |
| Novartis Investigative Site | Austin | Texas | 78723 | United States |
| Novartis Investigative Site | Salt Lake City | Utah | 84132 | United States |
| COMPLETED |
|
| NOT COMPLETED |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Tobramycin Inhalation Powder Once Daily | Tobramycin Inhalation Powder (112 mg) once daily during 168 days |
| BG001 | Tobramycin Inhalation Powder Twice Daily | Tobramycin Inhalation Powder (112 mg) twice daily on days 1-28, days 57-84 and days 113-140 |
| 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 | year |
| |||||||||||||||
| Sex: Female, Male | 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 | Change From Baseline in Forced Expiratory Volume in 1 Second ( FEV1) Percent Predicted | The Forced Expiratory Volume in 1 second (FEV1) percent predicted expresses FEV1 as a percentage of the "predicted values" for participants of similar characteristics (height, age, sex, and sometimes race and weight). A positive change from baseline in FEV1 percent predicted indicates improvement in lung function. | The study intended to randomize 200 patients within 18 months; however, after 9 months, only 32 patients were successfully randomized, with a screen fail rate of 50%. Study was terminated with only safety data analyzed. No data collected met the pre-specified powering of 200 patients needed for analysis (only 32 patients randomized) | Posted | Baseline and Day 168 |
|
| ||||||||||||||||||||||
| Secondary | Percent Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) Percent Predicted | The Forced Expiratory Volume in 1 second (FEV1) percent predicted expresses FEV1 as a percentage of the "predicted values" for participants of similar characteristics (height, age, sex, and sometimes race and weight). A positive change from baseline in FEV1 percent predicted indicates improvement in lung function. | The study intended to randomize 200 patients within 18 months; however, after 9 months, only 32 patients were successfully randomized, with a screen fail rate of 50%. Study was terminated with only safety data analyzed. No data collected met the pre-specified powering of 200 patients needed for analysis (only 32 patients randomized) | Posted | Baseline and Day 168 |
|
| ||||||||||||||||||||||
| Secondary | Percent Change From Baseline in Forced Vital Capacity (FVC) Percent Predicted | Forced Vital Capacity (FVC) is the amount of air which can be forcibly exhaled from the lungs after taking the deepest breath possible. FVC will be assessed via spirometry. A positive change from baseline in FVC indicates improvement in lung function. | The study intended to randomize 200 patients within 18 months; however, after 9 months, only 32 patients were successfully randomized, with a screen fail rate of 50%. Study was terminated with only safety data analyzed. No data collected met the pre-specified powering of 200 patients needed for analysis (only 32 patients randomized) | Posted | Baseline and Day 168 |
|
| ||||||||||||||||||||||
| Secondary | Percent Change From Baseline in Forced Expiratory Flow (FEF) 25%-75% Predicted | The Forced Expiratory Flow (FEF) 25%-75% measurement describes the amount of air expelled from the lungs during the middle half (25% - 75%) of the forced vital capacity test and is measured using spirometry. A positive change from baseline in FEF indicates improvement in lung function. The predicted percent will be assessed. | The study intended to randomize 200 patients within 18 months; however, after 9 months, only 32 patients were successfully randomized, with a screen fail rate of 50%. Study was terminated with only safety data analyzed. No data collected met the pre-specified powering of 200 patients needed for analysis (only 32 patients randomized) | Posted | Baseline and day 168 |
|
| ||||||||||||||||||||||
| Secondary | Change From Baseline in Pseudomonas Aeruginosa Sputum Density | Change from baseline in Pseudomonas aeruginosa sputum density will be measured by log10 colony forming units per gram of sputum. | The study intended to randomize 200 patients within 18 months; however, after 9 months, only 32 patients were successfully randomized, with a screen fail rate of 50%. Study was terminated with only safety data analyzed. No data collected met the pre-specified powering of 200 patients needed for analysis (only 32 patients randomized) | Posted | Baseline and day 168 |
|
| ||||||||||||||||||||||
| Secondary | Time to First Hospitalization Due to Respiratory-related Events | Time to the first hospitalization due to respiratory-related events (number of days) per patient. | The study intended to randomize 200 patients within 18 months; however, after 9 months, only 32 patients were successfully randomized, with a screen fail rate of 50%. Study was terminated with only safety data analyzed. No data collected met the pre-specified powering of 200 patients needed for analysis (only 32 patients randomized) | Posted | Day 1 to day 168 |
|
| ||||||||||||||||||||||
| Secondary | Percentage of Patients With Hospitalizations Due to Respiratory-related Events | Percentage of patients with hospitalization due to respiratory-related events | The study intended to randomize 200 patients within 18 months; however, after 9 months, only 32 patients were successfully randomized, with a screen fail rate of 50%. Study was terminated with only safety data analyzed. No data collected met the pre-specified powering of 200 patients needed for analysis (only 32 patients randomized) | Posted | Day 1 to day 168 |
|
| ||||||||||||||||||||||
| Secondary | Length of Hospital Stay Due to Respiratory-related Events | The number of days in length of hospital stay per patient due to respiratory-related events will be measured. | The study intended to randomize 200 patients within 18 months; however, after 9 months, only 32 patients were successfully randomized, with a screen fail rate of 50%. Study was terminated with only safety data analyzed. No data collected met the pre-specified powering of 200 patients needed for analysis (only 32 patients randomized) | Posted | Day 1 to day 168 |
|
| ||||||||||||||||||||||
| Secondary | Time to First Usage of Anti-pseudomonal Antibiotic | Time to first usage of anti-pseudomonal antibiotic per patient will be assessed by number of days | The study intended to randomize 200 patients within 18 months; however, after 9 months, only 32 patients were successfully randomized, with a screen fail rate of 50%. Study was terminated with only safety data analyzed. No data collected met the pre-specified powering of 200 patients needed for analysis (only 32 patients randomized) | Posted | Day 1 to day 168 |
|
| ||||||||||||||||||||||
| Secondary | Percentage of Patients Who Use Anti-pseudomonal Antibiotic | Percentage of patients who use anti-pseudomonal antibiotic will be assessed. | The study intended to randomize 200 patients within 18 months; however, after 9 months, only 32 patients were successfully randomized, with a screen fail rate of 50%. Study was terminated with only safety data analyzed. No data collected met the pre-specified powering of 200 patients needed for analysis (only 32 patients randomized) | Posted | Day 1 to day 168 |
|
| ||||||||||||||||||||||
| Secondary | Duration of Use of Anti-pseudomonal Antibiotic | Number of days of use of anti-pseudomonal antibiotic per patient will be assessed. | The study intended to randomize 200 patients within 18 months; however, after 9 months, only 32 patients were successfully randomized, with a screen fail rate of 50%. Study was terminated with only safety data analyzed. No data collected met the pre-specified powering of 200 patients needed for analysis (only 32 patients randomized) | Posted | Day 1 to day 168 |
|
| ||||||||||||||||||||||
| Secondary | Change From Baseline in Tobramycin Minimal Inhibitory Concentration for Pseudomonas Aeruginosa | Change from baseline in tobramycin minimal inhibitory concentration for Pseudomonas aeruginosa will be measured by laboratory testing. | The study intended to randomize 200 patients within 18 months; however, after 9 months, only 32 patients were successfully randomized, with a screen fail rate of 50%. Study was terminated with only safety data analyzed. No data collected met the pre-specified powering of 200 patients needed for analysis (only 32 patients randomized) | Posted | Baseline and day 168 |
|
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Tobramycin Inhalation Powder Once Daily | Tobramycin Inhalation Powder (112 mg) once daily during 168 days | 3 | 16 | 13 | 16 | ||
| EG001 | Tobramycin Inhalation Powder Twice Daily | Tobramycin Inhalation Powder (112 mg) twice daily on days 1-28, days 57-84 and days 113-140 | 1 | 15 | 12 | 15 | ||
| EG002 | Total Events | 4 | 31 | 25 | 31 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Endometriosis | Reproductive system and breast disorders | MedDRA | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Palpitations | Cardiac disorders | MedDRA | Systematic Assessment |
| |
| Hypoacusis | Ear and labyrinth disorders | MedDRA | Systematic Assessment |
| |
| Tinnitus | Ear and labyrinth disorders | MedDRA | Systematic Assessment |
| |
| Abdominal discomfort | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Dry mouth | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Post-tussive vomiting | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA | Systematic Assessment |
| |
| Chest discomfort | General disorders | MedDRA | Systematic Assessment |
| |
| Chills | General disorders | MedDRA | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA | Systematic Assessment |
| |
| Pain | General disorders | MedDRA | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA | Systematic Assessment |
| |
| Drug hypersensitivity | Immune system disorders | MedDRA | Systematic Assessment |
| |
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Otitis media | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Anaemia postoperative | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Gastrointestinal disorder postoperative | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Procedural pain | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Vaccination complication | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Hepatic enzyme increased | Investigations | MedDRA | Systematic Assessment |
| |
| Liver function test abnormal | Investigations | MedDRA | Systematic Assessment |
| |
| Oxygen saturation decreased | Investigations | MedDRA | Systematic Assessment |
| |
| Pulmonary function test decreased | Investigations | MedDRA | Systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Benign ovarian tumour | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA | Systematic Assessment |
| |
| Aphonia | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Dysgeusia | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Neuralgia | Nervous system disorders | MedDRA | Systematic Assessment |
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| Anxiety | Psychiatric disorders | MedDRA | Systematic Assessment |
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| Depression | Psychiatric disorders | MedDRA | Systematic Assessment |
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| Cervical dysplasia | Reproductive system and breast disorders | MedDRA | Systematic Assessment |
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| Fallopian tube cyst | Reproductive system and breast disorders | MedDRA | Systematic Assessment |
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| Haemorrhagic ovarian cyst | Reproductive system and breast disorders | MedDRA | Systematic Assessment |
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| Ovarian cyst | Reproductive system and breast disorders | MedDRA | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
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| Dry throat | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
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| Dysphonia | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
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| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
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| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
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| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
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| Pharyngeal oedema | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
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| Prolonged expiration | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
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| Pulmonary congestion | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
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| Rhonchi | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
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| Sinus congestion | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
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| Sputum increased | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Throat irritation | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Wheezing | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Dry skin | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
|
Due to premature termination, summaries and analyses planned in the protocol were eliminated in the statistical analysis plan prior to database lock. No inferential analysis will be provided.
The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (ie, data from all sites) in the clinical trial.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Clinical Disclosure Office | Novartis Pharmaceuticals | 862-778-8300 |
| 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 |
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| Male |
|