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| ID | Type | Description | Link |
|---|---|---|---|
| 2017-003761-99 | EudraCT Number |
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This study will explore the impact of lumacaftor/ivacaftor (LUM/IVA) on disease progression in subjects aged 2 through 5 years with cystic fibrosis (CF), homozygous for F508del (F/F).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Part 1: Placebo | Placebo Comparator | Participants received placebo matched to LUM/IVA in placebo-controlled period for 48 weeks. |
|
| Part 1: LUM/IVA | Experimental | Participants weighing less than (<)14 kilograms (kg) at screening received LUM 100 milligrams (mg)/IVA 125 mg fixed-dose combination (FDC) every 12 hours (q12h) in placebo-controlled period for 48 weeks. Participants weighing greater than or equals to (>=)14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h in placebo-controlled period for 48 weeks. |
|
| Part 2: Overall LUM/IVA | Experimental | Participants who received either placebo or LUM/IVA in placebo-controlled period administered LUM/IVA (either LUM 100 mg/IVA 125 mg FDC q12h or LUM 150 mg/IVA 188 mg FDC q12h as per their body weight for participants <6 years of age at week 48 and LUM 200 mg/IVA 250 mg FDC q12h regardless of their body weight for participants >=6 years of age at week 48) in open-label period for 48 weeks. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| LUM/IVA | Drug | FDC tablets or granules for oral administration. |
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| Measure | Description | Time Frame |
|---|---|---|
| Part 1: Absolute Change From Baseline in MRI Global Chest Score at Week 48 | MRI scans assessed semi-quantitatively via a standardized chest MRI scoring system. Each participant had 6 lobes scored using 7 scoring parameters:1) Bronchiectasis/wall thickening 2) Mucus plugging 3) Abscesses/sacculations 4) Consolidations 5) Special findings 6)Mosaic pattern 7) Perfusion abnormalities. For each of 7 parameter, there were scores of 6 lobes (score of each lobe : 0= normal value, 1 = <50% of lobe involved and 2 = >=50% of lobe involved). MRI global score was calculated as sum of parameters 1 to 7. MRI total score is ranged from 0-84. Higher score indicate more lobe involvement. | From Baseline at Week 48 |
| Measure | Description | Time Frame |
|---|---|---|
| Part 1: Absolute Change in Lung Clearance Index2.5 (LCI2.5) Through Week 48 | LCI2.5 represents the number of lung turnovers required to reduce the end-tidal inert gas concentration to 1/40th of its starting value. | From Baseline Through Week 48 |
| Part 1: Absolute Change in Weight-for-age Z-score at Week 48 |
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Key Inclusion Criteria:
Key Exclusion Criteria:
Other protocol defined Inclusion/Exclusion criteria may apply.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Charite Paediatric Pulmonology Department | Berlin | Germany | ||||
| Justus-Leibig-Universitat Zentrum fur Kinderheilkunde und Jugendmedizin |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 39173175 | Derived | Stahl M, Roehmel J, Eichinger M, Doellinger F, Naehrlich L, Kopp MV, Dittrich AM, Sommerburg O, Ray P, Maniktala A, Xu T, Conner S, Joshi A, Mascia M, Wielputz MO, Mall MA. Long-Term Impact of Lumacaftor/Ivacaftor Treatment on Cystic Fibrosis Disease Progression in Children 2-5 Years of Age Homozygous for F508del-CFTR: A Phase 2, Open-Label Clinical Trial. Ann Am Thorac Soc. 2024 Nov;21(11):1550-1559. doi: 10.1513/AnnalsATS.202402-201OC. | |
| 36943405 |
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This study was conducted in participants with cystic fibrosis (CF) aged 2 to 5 years.
This study was planned in 2 parts: Part 1 (Placebo-controlled Period) and Part 2 (Open-label Period). The primary and secondary efficacy analyses were planned for only Part 1.
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| ID | Title | Description |
|---|---|---|
| FG000 | Part 1: Placebo | Participants received placebo matched to LUM/IVA in placebo-controlled period for 48 weeks. |
| FG001 | Part 1: LUM/IVA | Participants weighing less than (<)14 kilograms (kg) at screening received LUM 100 milligrams (mg)/IVA 125 mg fixed-dose combination (FDC) every 12 hours (q12h) in placebo-controlled period for 48 weeks. Participants weighing greater than or equals to (>=)14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h in placebo-controlled period for 48 weeks. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Placebo-controlled Period (48 Weeks) |
|
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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 | Jul 24, 2019 | Oct 8, 2021 |
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Double blinded in Part 1, and Open label in Part 2
| LUM/IVA | Drug | FDC granules for oral administration. |
|
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| Placebo | Drug | Placebo matched to LUM/IVA for oral administration. |
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The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard. |
| From Baseline at Week 48 |
| Part 1: Absolute Change in Stature-for-age Z-score at Week 48 | The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard. | From Baseline at Week 48 |
| Part 1: Absolute Change in Body Mass Index (BMI)-For-age Z-score at Week 48 | BMI was defined as weight in kilogram (kg) divided by squared height in meters (m^2). The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard. | From Baseline at Week 48 |
| Giessen |
| Germany |
| Hannover Medical School | Hanover | Germany |
| Heidelberg Cystic Fibrosis Center | Heidelberg | Germany |
| Universitatsklinikum Schleswig-Holstein, Klinik für Kinder- und Jugendmedizin | Lübeck | Germany |
| Derived |
| Stahl M, Roehmel J, Eichinger M, Doellinger F, Naehrlich L, Kopp MV, Dittrich AM, Lee C, Sommerburg O, Tian S, Xu T, Wu P, Joshi A, Ray P, Duncan ME, Wielputz MO, Mall MA. Effects of Lumacaftor/Ivacaftor on Cystic Fibrosis Disease Progression in Children 2 through 5 Years of Age Homozygous for F508del-CFTR: A Phase 2 Placebo-controlled Clinical Trial. Ann Am Thorac Soc. 2023 Aug;20(8):1144-1155. doi: 10.1513/AnnalsATS.202208-684OC. |
| FG002 | Part 2: Overall LUM/IVA | Participants who received either placebo or LUM/IVA in placebo-controlled period administered LUM/IVA (either LUM 100 mg/IVA 125 mg FDC q12h or LUM 150 mg/IVA 188 mg FDC q12h as per their body weight for participants <6 years of age at week 48 and LUM 200 mg/IVA 250 mg FDC q12h regardless of their body weight for participants >=6 years of age at week 48) in open-label period for 48 weeks. |
| COMPLETED |
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| NOT COMPLETED |
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| Open-label Period (48 Weeks) |
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Baseline population included all randomized participants who have received at least 1 dose of study drug.
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| ID | Title | Description |
|---|---|---|
| BG000 | Part 1: Placebo | Participants received placebo matched to LUM/IVA in placebo-controlled period for 48 weeks. |
| BG001 | Part 1: LUM/IVA | Participants weighing <14 kg at screening received LUM 100 mg/IVA 125 mg FDC every q12h in placebo-controlled period for 48 weeks. Participants weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h in placebo-controlled period for 48 weeks. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Magnetic Resonance Imaging (MRI) Global Chest Score | MRI scans assessed semi-quantitatively via a standardized chest MRI scoring system. Each participant had 6 lobes scored using 7 scoring parameters:1)Bronchiectasis/wall thickening 2)Mucus plugging 3)Abscesses/sacculations 4)Consolidations 5)Special findings 6)Mosaic pattern 7)Perfusion abnormalities. For each of 7 parameter, there were scores of 6 lobes (score of each lobe :0=normal value, 1=<50 percent(%) of lobe involved and 2=>=50% of lobe involved). MRI global score was calculated as sum of parameters 1 to 7. MRI total score is ranged from 0-84. Higher score indicate more lobe involvement. | Baseline population included all randomized participants who have received at least 1 dose of study drug. Here "number analyzed" signifies participants who were evaluable for this outcome measure at the specified time point. | Mean | Standard Deviation | score on a scale |
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| 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 | Part 1: Absolute Change From Baseline in MRI Global Chest Score at Week 48 | MRI scans assessed semi-quantitatively via a standardized chest MRI scoring system. Each participant had 6 lobes scored using 7 scoring parameters:1) Bronchiectasis/wall thickening 2) Mucus plugging 3) Abscesses/sacculations 4) Consolidations 5) Special findings 6)Mosaic pattern 7) Perfusion abnormalities. For each of 7 parameter, there were scores of 6 lobes (score of each lobe : 0= normal value, 1 = <50% of lobe involved and 2 = >=50% of lobe involved). MRI global score was calculated as sum of parameters 1 to 7. MRI total score is ranged from 0-84. Higher score indicate more lobe involvement. | Full Analysis Set (FAS) included all randomized participants who carried the intended CF transmembrane conductance regulator gene (CFTR) allele mutation and received at least 1 dose of study drug in Part 1. Here "overall number of participants analyzed" signifies participants who were evaluable for this outcome measure. | Posted | Mean | Standard Deviation | score on a scale | From Baseline at Week 48 |
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| Secondary | Part 1: Absolute Change in Lung Clearance Index2.5 (LCI2.5) Through Week 48 | LCI2.5 represents the number of lung turnovers required to reduce the end-tidal inert gas concentration to 1/40th of its starting value. | FAS. | Posted | Mean | 95% Confidence Interval | lung clearance index | From Baseline Through Week 48 |
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| Secondary | Part 1: Absolute Change in Weight-for-age Z-score at Week 48 | The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard. | FAS. | Posted | Mean | 95% Confidence Interval | z-score | From Baseline at Week 48 |
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| Secondary | Part 1: Absolute Change in Stature-for-age Z-score at Week 48 | The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard. | FAS. | Posted | Mean | 95% Confidence Interval | z-score | From Baseline at Week 48 |
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| Secondary | Part 1: Absolute Change in Body Mass Index (BMI)-For-age Z-score at Week 48 | BMI was defined as weight in kilogram (kg) divided by squared height in meters (m^2). The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard. | FAS. | Posted | Mean | 95% Confidence Interval | z-score | From Baseline at Week 48 |
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From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)
MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.
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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 | Part 1: Placebo | Participants received placebo matched to LUM/IVA in placebo-controlled period for 48 weeks. | 0 | 16 | 2 | 16 | 16 | 16 |
| EG001 | Part 1: LUM/IVA | Participants weighing <14 kg at screening received LUM 100 mg/IVA 125 mg FDC q12h in placebo-controlled period for 48 weeks. Participants weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h in placebo-controlled period for 48 weeks. | 0 | 35 | 7 | 35 | 33 | 35 |
| EG002 | Part 2: Overall LUM/IVA | Participants who received either placebo or LUM/IVA in placebo-controlled period administered LUM/IVA (either LUM 100 mg/IVA 125 mg FDC q12h or LUM 150 mg/IVA 188 mg FDC q12h as per their body weight for participants <6 years of age at week 48 and LUM 200 mg/IVA 250 mg FDC q12h regardless of their body weight for participants >=6 years of age at week 48) in open-label period for 48 weeks. | 0 | 49 | 12 | 49 | 41 | 49 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Coeliac disease | Gastrointestinal disorders | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Distal intestinal obstruction syndrome | Gastrointestinal disorders | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Bronchopulmonary aspergillosis allergic | Infections and infestations | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Chronic sinusitis | Infections and infestations | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Gastroenteritis | Infections and infestations | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Otitis media | Infections and infestations | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Pseudomonas bronchitis | Infections and infestations | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Tonsillitis | Infections and infestations | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Sedation complication | Injury, poisoning and procedural complications | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Lung infiltration | Respiratory, thoracic and mediastinal disorders | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Haematemesis | Gastrointestinal disorders | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Intussusception | Gastrointestinal disorders | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Constipation | Gastrointestinal disorders | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Pneumonia | Infections and infestations | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain | Gastrointestinal disorders | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Abdominal pain upper | Gastrointestinal disorders | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Constipation | Gastrointestinal disorders | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Post-tussive vomiting | Gastrointestinal disorders | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Bacterial disease carrier | Infections and infestations | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Bronchitis | Infections and infestations | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Conjunctivitis | Infections and infestations | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Enterobiasis | Infections and infestations | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Gastroenteritis | Infections and infestations | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Otitis media | Infections and infestations | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Rhinitis | Infections and infestations | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Sinusitis | Infections and infestations | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Upper respiratory tract infection | Infections and infestations | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Viral infection | Infections and infestations | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Arthropod bite | Injury, poisoning and procedural complications | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Traumatic haematoma | Injury, poisoning and procedural complications | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Alanine aminotransferase increased | Investigations | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Aspartate aminotransferase increased | Investigations | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Staphylococcus test positive | Investigations | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Rash | Skin and subcutaneous tissue disorders | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Tachycardia | Cardiac disorders | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Otorrhoea | Ear and labyrinth disorders | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Astigmatism | Eye disorders | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Blepharospasm | Eye disorders | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Abnormal faeces | Gastrointestinal disorders | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Faeces pale | Gastrointestinal disorders | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Frequent bowel movements | Gastrointestinal disorders | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Peripheral swelling | General disorders | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Diarrhoea infectious | Infections and infestations | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Febrile infection | Infections and infestations | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Otitis media acute | Infections and infestations | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Pharyngitis streptococcal | Infections and infestations | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Pneumonia | Infections and infestations | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Pneumonia pseudomonal | Infections and infestations | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Skin abrasion | Injury, poisoning and procedural complications | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Skin laceration | Injury, poisoning and procedural complications | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Pseudomonas test positive | Investigations | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Lung infiltration | Respiratory, thoracic and mediastinal disorders | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Nasal obstruction | Respiratory, thoracic and mediastinal disorders | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Nasal polyps | Respiratory, thoracic and mediastinal disorders | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Wheezing | Respiratory, thoracic and mediastinal disorders | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Dry skin | Skin and subcutaneous tissue disorders | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Tonsillitis | Infections and infestations | MedDRA 23.1, 24.0 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Monitor | Vertex Pharmaceuticals Incorporated | 617-341-6777 | medicalinfo@vrtx.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Oct 16, 2020 | Oct 8, 2021 | 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 |
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| ID | Term |
|---|---|
| C000599212 | lumacaftor, ivacaftor drug combination |
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