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This is a Phase 3, randomized, double blind, placebo controlled, parallel group, multicenter study in people with cystic fibrosis (CF) who are homozygous for the F508del CF transmembrane conductance regulator (CFTR) gene mutation.
This is a Phase 3, randomized, double-blind, placebo-controlled, parallel-group, multicenter study in people with CF who are homozygous for the F508del-CFTR mutation. This study is designed to evaluate the efficacy and safety of VX-661 in combination with Ivacaftor (IVA, VX-770). The active treatment regimen comprised of a morning dose of a fixed-dose combination (FDC) tablet of 100 milligram (mg) VX-661/150 mg IVA once daily (qd) and an evening dose of IVA 150 mg to be taken approximately 12 hours after the morning dose. The placebo regimen was visually matched tablets to be taken with the same schedule as the active treatment.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Placebo | Placebo Comparator | Placebo matched to VX-661 plus IVA FDC tablet administered orally in the morning and placebo matched to IVA tablet administered orally in the evening up to Week 24. |
|
| VX-661/IVA | Experimental | VX-661 100 mg plus IVA 150 mg FDC tablet administered orally in the morning and IVA 150 mg tablet administered orally in the evening up to Week 24. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| VX-661 Plus Ivacaftor Combination | Drug | FDC tablet, oral use |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Absolute Change From Baseline (Day 1) in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) Through Week 24 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. | Day 1, Through Week 24 |
| Measure | Description | Time Frame |
|---|---|---|
| Relative Change From Baseline (Day 1) in ppFEV1 Through Week 24 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. | Day 1, Through Week 24 |
| Number of Pulmonary Exacerbations Per Year |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Little Rock | Arkansas | United States | ||||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 36509366 | Derived | 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. | |
| 29099344 |
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A total of 510 participants were randomized and 509 participants were treated in the study.
The study was conducted across 91 sites in 12 countries.
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| ID | Title | Description |
|---|---|---|
| FG000 | Placebo | Placebo matched to VX-661 plus Ivacaftor (IVA, VX-770) fixed dose combination (FDC) tablet administered orally in the morning and placebo matched to IVA tablet administered orally in the evening up to Week 24. |
| FG001 | VX-661/IVA |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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 | May 6, 2016 | Mar 14, 2018 |
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| Ivacaftor |
| Drug |
Tablet, oral use |
|
| VX-661 Plus Ivacaftor Combination Placebo | Drug | FDC tablet, oral use |
|
| Ivacaftor placebo | Drug | Tablet, oral use |
|
Pulmonary exacerbation was defined as a new event or change in antibiotic therapy for greater than or equal to 4 sinopulmonary signs/symptoms. Pulmonary exacerbation events per year (48 weeks) were reported.
| Day 1 through Week 24 |
| Absolute Change From Baseline (Day 1) Body Mass Index (BMI) at Week 24 | BMI was defined as weight in kilograms (kg) divided by height in square meter (m^2). | Day 1, Week 24 |
| Absolute Change From Baseline (Day 1) in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 24 | The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life. | Day 1, Through Week 24 |
| Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | AE: any untoward medical occurrence in a participant during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after the informed consent form is signed. AE includes serious as well as non-serious AEs. SAE (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, inpatient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. Any AE that increased in severity or newly developed at or after initial dosing of study drug to Week 28 was considered treatment-emergent. | Day 1 up to Week 28 |
| Number of Participants With at Least One Pulmonary Exacerbation Pulmonary Exacerbation Through Week 24 | Pulmonary exacerbation was defined as a new event or change in antibiotic therapy for greater than or equal to 4 sinopulmonary signs/symptoms. Time to event data was not collected and instead, Number of Subjects with first event were collected and are reported. Time-to-first pulmonary exacerbation was planned to be estimated using Kaplan-Meier (KM) estimates. However, due to less than 50% of events, time-to-first event data was not estimated. Instead, number of participants with at least one pulmonary exacerbation event were collected and are reported. | Day 1 through Week 24 |
| Absolute Change From Baseline (Day 1) in Sweat Chloride Through Week 24 | Sweat samples were collected using an approved collection device. | Day 1, Through Week 24 |
| Absolute Change From Baseline (Day 1) in BMI Z-score at Week 24 in Participants Less Than (<) 20 Years Old at the Time of Screening) | BMI was defined as weight in kg divided by height in m^2. z-score is a statistical measure to describe whether a mean was above or below the standard. BMI, adjusted for age and sex, was analyzed as BMI-for-age z-score (BMI z-score). | Day 1, Week 24 |
| Absolute Change From Baseline (Day 1) in Body Weight at Week 24 | Day 1, Week 24 |
| Trough Plasma Concentrations (Ctrough) of VX-661, VX-661 Metabolites (M1 VX-661 and M2-VX-661), Ivacaftor (IVA) and IVA Metabolite (M1-IVA) | This outcome was not planned to be assessed in Placebo arm. | Pre-morning dose on Week 16 |
| Long Beach |
| California |
| United States |
| Denver | Colorado | United States |
| Orlando | Florida | United States |
| Tampa | Florida | United States |
| Chicago | Illinois | United States |
| Peoria | Illinois | United States |
| Boston | Massachusetts | United States |
| Minneapolis | Minnesota | United States |
| St Louis | Missouri | United States |
| Manchester | New Hampshire | United States |
| Long Branch | New Jersey | United States |
| Albuquerque | New Mexico | United States |
| Albany | New York | United States |
| Buffalo | New York | United States |
| New Hyde Park | New York | United States |
| Rochester | New York | United States |
| Syracuse | New York | United States |
| Akron | Ohio | United States |
| Columbus | Ohio | United States |
| Oklahoma City | Oklahoma | United States |
| Charleston | South Carolina | United States |
| Sioux Falls | South Dakota | United States |
| Fort Worth | Texas | United States |
| Tyler | Texas | United States |
| Seattle | Washington | United States |
| Milwaukee | Wisconsin | United States |
| Calgary | Alberta | Canada |
| Vancouver | British Columbia | Canada |
| Halifax | Nova Scotia | Canada |
| Toronto | Ontario | Canada |
| Montreal | Quebec | Canada |
| Copenhagen O | Denmark |
| Marseille | Bouches-du-Rhone | France |
| Roscoff | Finistere | France |
| Bordeaux | Girande | France |
| Lille | Nord | France |
| Paris | Paris | France |
| Pierre-Bénite | Rhone | France |
| Heidelberg | Baden-Wurttemberg | Germany |
| Munich | Bavaria | Germany |
| Würzburg | Bavaria | Germany |
| Frankfurt am Main | Hesse | Germany |
| Giessen | Hesse | Germany |
| Belfast | State of Berlin | Germany |
| Berlin | Germany |
| Cork | Ireland |
| Dublin | Ireland |
| Genova | Italy |
| Palermo | Italy |
| Roma | Italy |
| Torino | Italy |
| Verona | Italy |
| Groningen | Netherlands |
| Nijmegen | Netherlands |
| Rotterdam | Netherlands |
| The Hague | Netherlands |
| Utrecht | Netherlands |
| Barcelona | Spain |
| Madrid | Spain |
| Seville | Spain |
| Valencia | Spain |
| Gothenburg | Sweden |
| Stockholm | Sweden |
| Uppsala | Sweden |
| Bern | Switzerland |
| Zurich | Switzerland |
| Exeter | Devon | United Kingdom |
| Liverpool | Lancashire | United Kingdom |
| Sheffield | South Yorkshire | United Kingdom |
| Newcastle upon Tyne | Tyne & Wear | United Kingdom |
| Birmingham | West Midlands | United Kingdom |
| Belfast | United Kingdom |
| London | United Kingdom |
| Taylor-Cousar JL, Munck A, McKone EF, van der Ent CK, Moeller A, Simard C, Wang LT, Ingenito EP, McKee C, Lu Y, Lekstrom-Himes J, Elborn JS. Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del. N Engl J Med. 2017 Nov 23;377(21):2013-2023. doi: 10.1056/NEJMoa1709846. Epub 2017 Nov 3. |
VX-661 100 milligram (mg) plus IVA 150 mg FDC tablet administered orally in the morning and IVA 150 mg tablet administered orally in the evening up to Week 24. |
| Treated |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
Full Analysis Set (FAS) included all randomized participants who carried the intended cystic fibrosis transmembrane conductance regulator (CFTR) allele mutation and had received at least 1 dose of study drug.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Placebo | Placebo matched to VX-661 plus IVA FDC tablet administered orally in the morning and placebo matched to IVA tablet administered orally in the evening up to Week 24. |
| BG001 | VX-661/IVA | VX-661 100 mg plus IVA 150 mg FDC tablet administered orally in the morning and IVA 150 mg tablet administered orally in the evening up to Week 24. |
| 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 |
| ||||||||||||||||
| 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 Baseline (Day 1) in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) Through Week 24 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. | FAS included all randomized participants who carry the intended CFTR allele mutation and have received at least 1 dose of study drug. Here 'Number of participants analyzed' signifies those participants who were evaluable for this outcome measure. | Posted | Least Squares Mean | 95% Confidence Interval | Percentage of predicted FEV1 | Day 1, Through Week 24 |
|
|
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Relative Change From Baseline (Day 1) in ppFEV1 Through Week 24 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. | FAS included all randomized participants who carry the intended CFTR allele mutation and have received at least 1 dose of study drug. Here 'Number of participants analyzed' signifies those participants who were evaluable for this outcome measure. | Posted | Least Squares Mean | 95% Confidence Interval | percent change | Day 1, Through Week 24 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Number of Pulmonary Exacerbations Per Year | Pulmonary exacerbation was defined as a new event or change in antibiotic therapy for greater than or equal to 4 sinopulmonary signs/symptoms. Pulmonary exacerbation events per year (48 weeks) were reported. | FAS included all randomized participants who carry the intended CFTR allele mutation and have received at least 1 dose of study drug. | Posted | Number | pulmonary exacerbation events per year | Day 1 through Week 24 |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Absolute Change From Baseline (Day 1) Body Mass Index (BMI) at Week 24 | BMI was defined as weight in kilograms (kg) divided by height in square meter (m^2). | FAS included all randomized participants who carry the intended CFTR allele mutation and have received at least 1 dose of study drug. Here 'Number of participants analyzed' signifies those participants who were evaluable for this outcome measure. | Posted | Least Squares Mean | 95% Confidence Interval | kilogram per square meter (kg/m^2) | Day 1, Week 24 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Absolute Change From Baseline (Day 1) in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 24 | The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life. | FAS included all randomized participants who carry the intended CFTR allele mutation and have received at least 1 dose of study drug. Here 'Number of participants analyzed' signifies those participants who were evaluable for this outcome measure. | Posted | Least Squares Mean | 95% Confidence Interval | units on a scale | Day 1, Through Week 24 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | AE: any untoward medical occurrence in a participant during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after the informed consent form is signed. AE includes serious as well as non-serious AEs. SAE (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, inpatient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. Any AE that increased in severity or newly developed at or after initial dosing of study drug to Week 28 was considered treatment-emergent. | Safety Set included all participants who received at least 1 dose of the study drug. | Posted | Count of Participants | Participants | Day 1 up to Week 28 |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants With at Least One Pulmonary Exacerbation Pulmonary Exacerbation Through Week 24 | Pulmonary exacerbation was defined as a new event or change in antibiotic therapy for greater than or equal to 4 sinopulmonary signs/symptoms. Time to event data was not collected and instead, Number of Subjects with first event were collected and are reported. Time-to-first pulmonary exacerbation was planned to be estimated using Kaplan-Meier (KM) estimates. However, due to less than 50% of events, time-to-first event data was not estimated. Instead, number of participants with at least one pulmonary exacerbation event were collected and are reported. | FAS included all randomized participants who carry the intended CFTR allele mutation and have received at least 1 dose of study drug. | Posted | Count of Participants | Participants | Day 1 through Week 24 |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Absolute Change From Baseline (Day 1) in Sweat Chloride Through Week 24 | Sweat samples were collected using an approved collection device. | FAS included all randomized participants who carry the intended CFTR allele mutation and have received at least 1 dose of study drug. Here 'Number of participants analyzed' signifies those participants who were evaluable for this outcome measure. | Posted | Least Squares Mean | 95% Confidence Interval | millimole per liter (mmol/L) | Day 1, Through Week 24 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Absolute Change From Baseline (Day 1) in BMI Z-score at Week 24 in Participants Less Than (<) 20 Years Old at the Time of Screening) | BMI was defined as weight in kg divided by height in m^2. z-score is a statistical measure to describe whether a mean was above or below the standard. BMI, adjusted for age and sex, was analyzed as BMI-for-age z-score (BMI z-score). | Analysis population included all randomized participants who received at least 1 dose of study drug and were <20 years of age at the time of screening. Here 'Number of participants analyzed' signifies those participants who were evaluable for this outcome measure. | Posted | Least Squares Mean | 95% Confidence Interval | z-score | Day 1, Week 24 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Absolute Change From Baseline (Day 1) in Body Weight at Week 24 | FAS included all randomized participants who carry the intended CFTR allele mutation and have received at least 1 dose of study drug. Here 'Number of participants analyzed' signifies those participants who were evaluable for this outcome measure. | Posted | Least Squares Mean | 95% Confidence Interval | kg | Day 1, Week 24 |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Trough Plasma Concentrations (Ctrough) of VX-661, VX-661 Metabolites (M1 VX-661 and M2-VX-661), Ivacaftor (IVA) and IVA Metabolite (M1-IVA) | This outcome was not planned to be assessed in Placebo arm. | Pharmacokinetic (PK) set included all randomized participants who received any amount of study drug and had a PK assessment. Here 'Number of participants analyzed' signifies those participants who were evaluable for this outcome measure. | Posted | Mean | Standard Deviation | nanogram per milliliter (ng/mL) | Pre-morning dose on Week 16 |
|
|
Day 1 up to Week 28
Safety Set included all participants who received at least 1 dose of the study drug.
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 plus IVA FDC tablet administered orally in the morning and placebo matched to IVA tablet administered orally in the evening up to Week 24. | 0 | 258 | 47 | 258 | 243 | 258 |
| EG001 | VX-661/IVA | VX-661 100 mg plus IVA 150 mg FDC tablet administered orally in the morning and IVA 150 mg tablet administered orally in the evening up to Week 24. | 0 | 251 | 31 | 251 | 227 | 251 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Clostridium difficile colitis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Lung abscess | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Respiratory tract infection viral | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Acarodermatitis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Bronchopulmonary aspergillosis allergic | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Benign intracranial hypertension | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Generalised tonic-clonic seizure | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Migraine | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Paranasal cyst | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Inguinal hernia | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Coeliac disease | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Faecaloma | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Gastric ulcer | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pancreatitis acute | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Blood creatine phosphokinase increased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Blood glucose abnormal | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Electrocardiogram ST segment elevation | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Pulmonary function test decreased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Haemolytic anaemia | Blood and lymphatic system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Chest discomfort | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Alcohol poisoning | Injury, poisoning and procedural complications | MedDRA (19.1) | Systematic Assessment |
| |
| Toxicity to various agents | Injury, poisoning and procedural complications | MedDRA (19.1) | Systematic Assessment |
| |
| Wrist fracture | Injury, poisoning and procedural complications | MedDRA (19.1) | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA (19.1) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| 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 |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Bacterial test positive | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue 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 9, 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 |
|---|---|
| C000625213 | tezacaftor |
| C545203 | ivacaftor |
Not provided
Not provided
Not provided
| 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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