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The primary purpose of study is to evaluate the treatment effect of tezacaftor in combination with ivacaftor (TEZ/IVA) on chest imaging endpoints using low-dose computed tomography (LDCT) at Week 72, and to evaluate the safety of TEZ/IVA through Week 72.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| TEZ/IVA | Experimental | Participants received TEZ 100 milligram (mg)/IVA 150 mg fixed dose combination tablet orally once daily in the morning and IVA 150 mg tablet orally once daily in the evening for 72 weeks. |
|
| Placebo | Placebo Comparator | Participants received placebo matched to TEZ/IVA fixed dose combination tablet orally once daily in the morning and placebo matched to IVA tablet orally once daily in the evening for 72 weeks. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Tezacaftor/Ivacaftor | Drug | TEZ 100 mg/IVA 150 mg fixed-dose combination tablet. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Absolute Change in Total Brody/CF-CT Score | The exploratory Brody/CF-CT score semi-quantitatively scores the degree of structural lung disease as shown on CT in participants with CF. The score ranges from a minimum of 0 to a maximum of 219 with higher scores indicating more severe structural lung disease. | From Baseline at Week 72 |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Day 1 up to Week 76 |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Chermside | Australia | |||||
A total of 41 participants were randomized and treated in the study.
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| ID | Title | Description |
|---|---|---|
| FG000 | TEZ/IVA | Participants received TEZ 100 milligram (mg)/IVA 150 mg fixed dose combination tablet orally once daily in the morning and IVA 150 mg tablet orally once daily in the evening for 72 weeks. |
| FG001 | Placebo |
| 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 26, 2018 | Jul 22, 2019 |
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| Ivacaftor | Drug | IVA 150 mg tablet. |
|
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| Placebo | Drug | Placebo matched to TEZ/IVA fixed-dose combination tablet. |
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| Placebo | Drug | Placebo matched to IVA tablet. |
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| Melbourne |
| Australia |
| Nedlands | Australia |
| New Lambton Heights | Australia |
| Parkville SIC | Australia |
| Randwick | Australia |
| South Brisbane | Australia |
| Subiaco | Australia |
| Westmead | Australia |
Participants received placebo matched to TEZ/IVA fixed dose combination tablet orally once daily in the morning and placebo matched to IVA tablet orally once daily in the evening for 72 weeks.
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | TEZ/IVA | Participants received TEZ 100 mg/IVA 150 mg fixed dose combination tablet orally once daily in the morning and IVA 150 mg tablet orally once daily in the evening for 72 weeks. |
| BG001 | Placebo | Participants received placebo matched to TEZ/IVA fixed dose combination tablet orally once daily in the morning and placebo matched to IVA tablet orally once daily in the evening for 72 weeks. |
| 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 |
| |||||||||||||||
| 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 |
| ||||||||||||||||
| Total Brody/Cystic Fibrosis - Computed Tomography (CF-CT) Score | The exploratory Brody/CF-CT score semi-quantitatively scores the degree of structural lung disease as shown on CT in participants with CF. The score ranges from a minimum of 0 to a maximum of 219 with higher scores indicating more severe structural lung disease. | Mean | Standard Deviation | scores 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 | Absolute Change in Total Brody/CF-CT Score | The exploratory Brody/CF-CT score semi-quantitatively scores the degree of structural lung disease as shown on CT in participants with CF. The score ranges from a minimum of 0 to a maximum of 219 with higher scores indicating more severe structural lung disease. | FAS included all participants who were randomized and received at least 1 dose of study drug. | Posted | Least Squares Mean | Standard Error | scores on a scale | From Baseline at Week 72 |
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| Secondary | Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Safety set included all participants who received at least 1 dose of study drug. | Posted | Count of Participants | Participants | Day 1 up to Week 76 |
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Day 1 up to Week 76
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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 | TEZ/IVA | Participants received TEZ 100 mg/IVA 150 mg fixed dose combination tablet orally once daily in the morning and IVA 150 mg tablet orally once daily in the evening for 72 weeks. | 0 | 20 | 8 | 20 | 19 | 20 |
| EG001 | Placebo | Participants received placebo matched to TEZ/IVA fixed dose combination tablet orally once daily in the morning and placebo matched to IVA tablet orally once daily in the evening for 72 weeks. | 0 | 21 | 13 | 21 | 20 | 21 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Lung infection pseudomonal | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Atypical mycobacterial lower respiratory tract infection | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
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| Gastroenteritis | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
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| Atrioventricular block first degree | Cardiac disorders | MedDRA 21.0 | Systematic Assessment |
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| Abdominal pain upper | Gastrointestinal disorders | MedDRA 21.0 | Systematic Assessment |
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| Abdominal pain | Gastrointestinal disorders | MedDRA 21.0 | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA 21.0 | Systematic Assessment |
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| Testicular torsion | Reproductive system and breast disorders | MedDRA 21.0 | Systematic Assessment |
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| Hepatic cirrhosis | Hepatobiliary disorders | MedDRA 21.0 | Systematic Assessment |
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| Type I hypersensitivity | Immune system disorders | MedDRA 21.0 | Systematic Assessment |
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| Bacterial test positive | Investigations | MedDRA 21.0 | Systematic Assessment |
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| Weight decreased | Investigations | MedDRA 21.0 | Systematic Assessment |
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| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA 21.0 | Systematic Assessment |
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| Atelectasis | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
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| Lower respiratory tract infection bacterial | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
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| Gastroenteritis | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
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| Influenza | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
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| Pharyngitis | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
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| Rhinitis | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
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| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
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| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
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| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
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| Sputum increased | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
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| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
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| Upper respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
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| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA 21.0 | Systematic Assessment |
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| Fatigue | General disorders | MedDRA 21.0 | Systematic Assessment |
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| Chest pain | General disorders | MedDRA 21.0 | Systematic Assessment |
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| Migraine | Nervous system disorders | MedDRA 21.0 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA 21.0 | Systematic Assessment |
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| Abdominal pain | Gastrointestinal disorders | MedDRA 21.0 | Systematic Assessment |
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| Constipation | Gastrointestinal disorders | MedDRA 21.0 | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | MedDRA 21.0 | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA 21.0 | Systematic Assessment |
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| Sunburn | Injury, poisoning and procedural complications | MedDRA 21.0 | Systematic Assessment |
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| Arthropod bite | Injury, poisoning and procedural complications | MedDRA 21.0 | Systematic Assessment |
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| Fungal test positive | Investigations | MedDRA 21.0 | Systematic Assessment |
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| Bacterial test positive | Investigations | MedDRA 21.0 | Systematic Assessment |
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| Blood alkaline phosphatase increased | Investigations | MedDRA 21.0 | 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 | May 7, 2018 | Jul 22, 2019 | 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 |
|---|---|
| C000654124 | tezacaftor, ivacaftor drug combination |
| C545203 | ivacaftor |
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| Male |
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| Not Hispanic or Latino |
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| Unknown or Not Reported |
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| Asian |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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