Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2014-004788-18 | EudraCT Number |
Not provided
Not provided
Not provided
Not provided
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The purpose of this study is to evaluate the efficacy and safety of VX-661 in combination with ivacaftor (IVA, VX-770) and IVA monotherapy in participants with Cystic Fibrosis (CF) who are heterozygous for F508del-CFTR allele and a second allele with a CFTR mutation predicted to have residual function.
Not provided
Not provided
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| VX-661/Ivacaftor combination | Experimental |
| |
| Ivacaftor monotherapy | Experimental |
| |
| Placebo | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| VX-661/Ivacaftor | Drug | Fixed dose combination tablet, oral use |
|
| Measure | Description | Time Frame |
|---|---|---|
| Absolute Change From Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Average of Week 4 and Week 8 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. | Baseline, Week 4 and Week 8 of each treatment period |
| Measure | Description | Time Frame |
|---|---|---|
| Absolute Change From Study Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score at Average of Week 4 and Week 8 | The CFQ-R assessed respiratory symptoms on a scale with scores ranging from 0 to 100; where higher scores indicated fewer symptoms and better health-related quality of life. | Baseline, Week 4 and Week 8 of each treatment period |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Birmingham | Alabama | United States | ||||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37983082 | Derived | Heneghan M, Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2023 Nov 20;11(11):CD010966. doi: 10.1002/14651858.CD010966.pub4. | |
| 36509366 | Derived |
Not provided
Not provided
Participants were randomized to 1 of 6 treatment sequences, each of which included 2 treatment periods and 2 of 3 potential treatments (placebo, VX-661/ivacaftor [IVA], IVA). Treatment periods were separated by an 8 week wash-out period.
A total of 248 participants were enrolled. Out of which, 246 received at least 1 dose of study drug were included in safety set and 244 participants who carried intended cystic fibrosis transmembrane conductance regulator (CFTR) mutations were included in Full analysis set.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | First VX-661/IVA, Then IVA | VX-661 100 milligram (mg) plus IVA 150 mg fixed dose combination (FDC) tablet and placebo matched to IVA in the morning, IVA 150 mg tablet in the evening for 8 weeks in treatment period 1 followed by IVA 150 mg tablet and placebo matched to VX-661 plus IVA FDC tablet in the morning, IVA 150 mg tablet in the evening for 8 weeks in treatment period 2. Each treatment period was separated by a minimum 8 weeks of washout period. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Treatment Period 1 (8 Weeks) |
|
Not provided
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jun 10, 2016 | Mar 14, 2018 |
Not provided
Not provided
Not provided
Not provided
Not provided
| Ivacaftor | Drug | Tablet, oral use |
|
|
| Placebo matched to VX-661/ ivacaftor | Drug | Fixed dose combination tablet, oral use |
|
| Placebo matched to Ivacaftor | Drug | Tablet, oral use |
|
| Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Day 1 up to Week 28 |
| Relative Change From Study Baseline in ppFEV1 at Average of Week 4 and Week 8 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. | Baseline, Week 4 and Week 8 of each treatment period |
| Absolute Change From Study Baseline in Sweat Chloride at Average of Week 4 and Week 8 | Baseline, Week 4 and Week 8 of each treatment period |
| Trough Plasma Concentrations (Ctrough) of VX-661, VX-661 Metabolites (M1 VX-661), IVA and IVA Metabolite (M1 IVA) After Administration of VX-661/IVA Combination Therapy | Pre-morning dose on Week 8 of each treatment period |
| Ctrough of IVA and IVA Metabolite (M1 IVA) After Administration of IVA Monotherapy | Pre-morning dose on Week 8 of each treatment period |
| Phoenix |
| Arizona |
| United States |
| Tucson | Arizona | United States |
| Long Beach | California | United States |
| Oakland | California | United States |
| Palo Alto | California | United States |
| Sacramento | California | United States |
| Aurora | Colorado | United States |
| Denver | Colorado | United States |
| Gainesville | Florida | United States |
| Miami | Florida | United States |
| Orlando | Florida | United States |
| Pensacola | Florida | United States |
| Tampa | Florida | United States |
| Atlanta | Georgia | United States |
| Chicago | Illinois | United States |
| Park Ridge | Illinois | United States |
| Iowa City | Iowa | United States |
| Baltimore | Maryland | United States |
| Boston | Massachusetts | United States |
| Ann Arbor | Michigan | United States |
| Grand Rapids | Michigan | United States |
| Minneapolis | Minnesota | United States |
| St Louis | Missouri | United States |
| Lebanon | New Hampshire | United States |
| New York | New York | United States |
| Syracuse | New York | United States |
| Valhalla | New York | United States |
| Chapel Hill | North Carolina | United States |
| Akron | Ohio | United States |
| Cincinnati | Ohio | United States |
| Cleveland | Ohio | United States |
| Toledo | Ohio | United States |
| Portland | Oregon | United States |
| Philadelphia | Pennsylvania | United States |
| Pittsburgh | Pennsylvania | United States |
| Charleston | South Carolina | United States |
| Sioux Falls | South Dakota | United States |
| Memphis | Tennessee | United States |
| Austin | Texas | United States |
| Dallas | Texas | United States |
| Tyler | Texas | United States |
| Salt Lake City | Utah | United States |
| Norfolk | Virginia | United States |
| Seattle | Washington | United States |
| Spokane | Washington | United States |
| Milwaukee | Wisconsin | United States |
| Adelaide | Australia |
| Melbourne | Australia |
| South Brisbane | Australia |
| Westmead | Australia |
| Ghent | Belgium |
| Montreal | Canada |
| Québec | Canada |
| Toronto | Canada |
| Vancouver | Canada |
| Marseille | Bouches-du-Rhone | France |
| Montpellier | Herault | France |
| Rennes | Ille Et Vilaine | France |
| Lille | Nord | France |
| Paris | Paris | France |
| Bron | Rhone | France |
| Bordeaux | France |
| Munich | Bavaria | Germany |
| München | Bavaria | Germany |
| Hanover | Lower Saxony | Germany |
| Essen | North Rhine-Westphalia | Germany |
| Jena | Thuringia | Germany |
| Berlin | Germany |
| Haifa | Israel |
| Jerusalem | Israel |
| Petah Tikva | Israel |
| Ramat Gan | Israel |
| Ancona | Italy |
| Milan | Italy |
| Orbassano | Italy |
| Potenza | Italy |
| Roma | Italy |
| Verona | Italy |
| Amsterdam | Netherlands |
| Rotterdam | Netherlands |
| The Hague | Netherlands |
| Utrecht | Netherlands |
| Bern | Switzerland |
| Sankt Gallen | Switzerland |
| Zurich | Switzerland |
| Exeter | Devon | United Kingdom |
| London | Greater London | United Kingdom |
| Manchester | Greater Manchester | United Kingdom |
| Southampton | Hampshire | United Kingdom |
| Liverpool | Lancashire | United Kingdom |
| Glasgow | Strathclyde | United Kingdom |
| Leeds | West Yorkshire | United Kingdom |
| Acaster S, Mukuria C, Rowen D, Brazier JE, Wainwright CE, Quon BS, Duckers J, Quittner AL, Lou Y, Sosnay PR, McGarry LJ. Development of the Cystic Fibrosis Questionnaire-Revised-8 Dimensions: Estimating Utilities From the Cystic Fibrosis Questionnaire-Revised. Value Health. 2023 Apr;26(4):567-578. doi: 10.1016/j.jval.2022.12.002. Epub 2022 Dec 9. |
| 33331662 | Derived | Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3. |
| 29099333 | Derived | Rowe SM, Daines C, Ringshausen FC, Kerem E, Wilson J, Tullis E, Nair N, Simard C, Han L, Ingenito EP, McKee C, Lekstrom-Himes J, Davies JC. Tezacaftor-Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis. N Engl J Med. 2017 Nov 23;377(21):2024-2035. doi: 10.1056/NEJMoa1709847. Epub 2017 Nov 3. |
| FG001 | First VX-661/IVA, Then Placebo | VX-661 100 mg plus IVA 150 mg FDC tablet and placebo matched to IVA in the morning, IVA 150 mg tablet in the evening for 8 weeks in treatment period 1 followed by placebo matched to VX-661 plus IVA FDC tablet and placebo matched to IVA tablet in the morning, placebo matched to IVA tablet in the evening for 8 weeks in treatment period 2. Each treatment period was separated by a minimum 8 weeks of washout period. |
| FG002 | First IVA, Then Placebo | IVA 150 mg tablet and placebo matched to VX-661 plus IVA FDC tablet in the morning, IVA 150 mg tablet in the evening for 8 weeks in treatment period 1 followed by placebo matched to VX-661 plus IVA FDC tablet and placebo matched to IVA tablet in the morning, placebo matched to IVA tablet in the evening for 8 weeks in treatment period 2. Each treatment period was separated by a minimum 8 weeks of washout period. |
| FG003 | First IVA, Then VX- 661/IVA | IVA 150 mg tablet and placebo matched to VX-661 plus IVA FDC tablet in the morning, IVA 150 mg tablet in the evening for 8 weeks in treatment period 1 followed by VX-661 100 mg plus IVA 150 mg FDC tablet and placebo matched to IVA tablet in the morning, IVA 150 mg tablet in the evening for 8 weeks in treatment period 2. Each treatment period was separated by a minimum 8 weeks of washout period. |
| FG004 | First Placebo, Then VX- 661/IVA | Placebo matched to VX-661 plus IVA FDC tablet and placebo matched to IVA tablet in the morning, placebo matched to IVA tablet in the evening for 8 weeks in treatment period 1 followed by VX-661 100 mg plus IVA 150 mg FDC tablet and placebo matched to IVA tablet in the morning, IVA 150 mg tablet in the evening for 8 weeks in treatment period 2. Each treatment period was separated by a minimum 8 weeks of washout period. |
| FG005 | First Placebo, Then IVA | Placebo matched to VX-661 plus IVA FDC tablet and placebo matched to IVA tablet in the morning, placebo matched to IVA tablet in the evening for 8 weeks in treatment period 1 followed by IVA 150 mg tablet and placebo matched to VX-661 plus IVA FDC tablet in the morning, IVA 150 mg tablet in the evening for 8 weeks in treatment period 2. Each treatment period was separated by a minimum 8 weeks of washout period. |
| Treated |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
| Treatment Period 2 (8 Weeks) |
|
|
The Full Analysis Set (FAS) was defined as all randomized subjects who carry the intended cystic fibrosis transmembrane conductance regulator (CFTR) mutations and had received at least 1 dose of study drug.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | First VX- 661/IVA, Then IVA | VX-661 100 mg plus IVA 150 mg FDC tablet and placebo matched to IVA in the morning, IVA 150 mg tablet in the evening for 8 weeks in treatment period 1 followed by IVA 150 mg tablet and placebo matched to VX-661 plus IVA FDC tablet in the morning, IVA 150 mg tablet in the evening for 8 weeks in treatment period 2. Each treatment period was separated by a minimum 8 weeks of washout period. |
| BG001 | First VX-661/IVA, Then Placebo | VX-661 100 mg plus IVA 150 mg FDC tablet and placebo matched to IVA in the morning, IVA 150 mg tablet in the evening for 8 weeks in treatment period 1 followed by placebo matched to VX-661 plus IVA FDC tablet and placebo matched to IVA tablet in the morning, placebo matched to IVA tablet in the evening for 8 weeks in treatment period 2. Each treatment period was separated by a minimum 8 weeks of washout period. |
| BG002 | First IVA, Then Placebo | IVA 150 mg tablet and placebo matched to VX-661 plus IVA FDC tablet in the morning, IVA 150 mg tablet in the evening for 8 weeks in treatment period 1 followed by placebo matched to VX-661 plus IVA FDC tablet and placebo matched to IVA tablet in the morning, placebo matched to IVA tablet in the evening for 8 weeks in treatment period 2. Each treatment period was separated by a minimum 8 weeks of washout period. |
| BG003 | First IVA, Then VX- 661/IVA | IVA 150 mg tablet and placebo matched to VX-661 plus IVA FDC tablet in the morning, IVA 150 mg tablet in the evening for 8 weeks in treatment period 1 followed by VX-661 100 mg plus IVA 150 mg FDC tablet and placebo matched to IVA tablet in the morning, IVA 150 mg tablet in the evening for 8 weeks in treatment period 2. Each treatment period was separated by a minimum 8 weeks of washout period. |
| BG004 | First Placebo, Then VX- 661/IVA | Placebo matched to VX-661 plus IVA FDC tablet and placebo matched to IVA tablet in the morning, placebo matched to IVA tablet in the evening for 8 weeks in treatment period 1 followed by VX-661 100 mg plus IVA 150 mg FDC tablet and placebo matched to IVA tablet in the morning, IVA 150 mg tablet in the evening for 8 weeks in treatment period 2. Each treatment period was separated by a minimum 8 weeks of washout period. |
| BG005 | First Placebo, Then IVA | Placebo matched to VX-661 plus IVA FDC tablet and placebo matched to IVA tablet in the morning, placebo matched to IVA tablet in the evening for 8 weeks in treatment period 1 followed by IVA 150 mg tablet and placebo matched to VX-661 plus IVA FDC tablet in the morning, IVA 150 mg tablet in the evening for 8 weeks in treatment period 2. Each treatment period was separated by a minimum 8 weeks of washout period. |
| BG006 | 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, Categorical | Count of Participants | Participants |
| ||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | 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 From Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Average of Week 4 and Week 8 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. | Full Analysis Set (FAS) included all randomized participants who carry the protocol specified cystic fibrosis transmembrane conductance regulator gene (CFTR) mutations and had received at least 1 dose of study drug. Here 'Overall Number of participants analyzed' signifies those participants who had evaluable data for this outcome measure. | Posted | Least Squares Mean | 95% Confidence Interval | percentage of predicted FEV1 | Baseline, Week 4 and Week 8 of each treatment period |
|
|
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Absolute Change From Study Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score at Average of Week 4 and Week 8 | The CFQ-R assessed respiratory symptoms on a scale with scores ranging from 0 to 100; where higher scores indicated fewer symptoms and better health-related quality of life. | FAS was used. Here 'Overall Number of participants analyzed' signifies those participants who had evaluable data for this outcome measure. | Posted | Least Squares Mean | 95% Confidence Interval | units on a scale | Baseline, Week 4 and Week 8 of each treatment period |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| 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 | Number | participants | Day 1 up to Week 28 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Relative Change From Study Baseline in ppFEV1 at Average of Week 4 and Week 8 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. | FAS was used. Here 'Overall Number of participants analyzed' signifies those participants who had evaluable data for this outcome measure. | Posted | Least Squares Mean | 95% Confidence Interval | percent change | Baseline, Week 4 and Week 8 of each treatment period |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Absolute Change From Study Baseline in Sweat Chloride at Average of Week 4 and Week 8 | FAS was used. Here 'Overall Number of participants analyzed' signifies those participants who had evaluable data for this outcome measure. | Posted | Least Squares Mean | 95% Confidence Interval | millimoles per liter (mmol/L) | Baseline, Week 4 and Week 8 of each treatment period |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Trough Plasma Concentrations (Ctrough) of VX-661, VX-661 Metabolites (M1 VX-661), IVA and IVA Metabolite (M1 IVA) After Administration of VX-661/IVA Combination Therapy | Pharmacokinetic (PK) set included all randomized participants who received any amount of study drug and had a PK assessment. | Posted | Mean | Standard Deviation | nanogram per milliliter (ng/mL) | Pre-morning dose on Week 8 of each treatment period |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Ctrough of IVA and IVA Metabolite (M1 IVA) After Administration of IVA Monotherapy | PK set was used. Here 'Overall Number of participants analyzed' signifies those participants who had evaluable data for this outcome measure. | Posted | Mean | Standard Deviation | ng/mL | Pre-morning dose on Week 8 of each treatment period |
|
|
Day 1 up to Week 28
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 | Placebo | Placebo matched to VX-661/Ivacaftor (IVA) fixed dose combination (FDC) tablet and placebo matched to IVA tablet in the morning, placebo matched to IVA tablet in the evening for 8 weeks in treatment period 1 and 2. | 0 | 162 | 14 | 162 | 87 | 162 |
| EG001 | Ivacaftor | IVA 150 milligram (mg) tablet and placebo matched to VX-661/IVA FDC tablet in the morning, IVA 150 mg tablet in the evening for 8 weeks in treatment period 1 and 2. | 0 | 157 | 10 | 157 | 54 | 157 |
| EG002 | VX-661/IVA | VX-661 100 mg/IVA 150 mg FDC tablet and placebo matched to IVA in the morning, IVA 150 mg tablet in the evening for 8 weeks in treatment period 1 and 2. | 0 | 162 | 8 | 162 | 80 | 162 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Ankle fracture | Injury, poisoning and procedural complications | MedDRA (19.1) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pneumothorax | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Distal intestinal obstruction syndrome | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pancreatitis | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Blood creatine phosphokinase increased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Pulmonary function test decreased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Mental status changes | Psychiatric disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Urethral stenosis | Renal and urinary disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Acute febrile neutrophilic dermatosis | Skin and subcutaneous tissue disorders | MedDRA (19.1) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Sputum increased | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (19.1) | Systematic Assessment |
|
PI is free to publish results of the study after (1) the first multi-center publication, (2) if the sponsor elects not to publish the results, or (3) 18 months after close of the study, whichever occurs first. Proposed publications are to be submitted to the sponsor for review and comment for a period of at least 45 days (which may be extended under certain circumstances related to protection of intellectual property); the sponsor cannot require changes to the proposed publications.
| 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 | Feb 24, 2017 | Mar 14, 2018 | 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 |
|---|---|
| C545203 | ivacaftor |
Not provided
Not provided
Not provided
| Between 18 and 65 years |
|
| >=65 years |
|
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| LS Mean Difference |
| 6.8 |
| 2-Sided |
| 95 |
| 5.7 |
| 7.8 |
| Superiority |
|
|
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
|
| Participants |
|
|
|
|
| Title | Denominators | Categories | ||||
|---|---|---|---|---|---|---|
| IVA |
|
| ||||
| M1 IVA |
|
|