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To evaluate the clinical mechanisms of action in lung and extrapulmonary systems of VX-661 (tezacaftor; TEZ) in combination with ivacaftor (IVA) (TEZ/IVA) in participants with cystic fibrosis (CF) who are homozygous for the F508del mutation on the CF transmembrane conductance regulator (CFTR) gene.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Placebo | Placebo Comparator | Participants received placebo matched to tezacaftor (TEZ)/ivacaftor (IVA) fixed dose combination (FDC) tablet orally once daily in the morning followed by placebo matched to IVA tablet orally once daily in the evening for 29 days. |
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| TEZ/IVA | Experimental | Participants received 100 milligram (mg) TEZ/150 mg IVA FDC tablet orally once daily in the morning followed by 150 mg IVA tablet orally once daily in the evening for 29 days. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Tezacaftor/Ivacaftor | Drug | Tezacaftor/Ivacaftor FDC |
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| Ivacaftor |
| Measure | Description | Time Frame |
|---|---|---|
| Absolute Change From Baseline in Mucociliary Clearance (MCC) at Day 28 | MCC was assessed using an imaging technique that enables the tracking of mucus within the airways. MCC was expressed as the percentage of whole-lung clearance through 60 minutes at Baseline and Day 28. | Baseline, Day 28 |
| Measure | Description | Time Frame |
|---|---|---|
| Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 28 | Percent predicted FEV1 is the ratio of FEV1 to the predicted FEV1, expressed as a percentage. FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. | Baseline, Day 28 |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Birmingham | Alabama | United States | ||||
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| ID | Title | Description |
|---|---|---|
| FG000 | Placebo | Participants received placebo matched to tezacaftor (TEZ)/ivacaftor (IVA) fixed dose combination (FDC) tablet orally once daily in the morning followed by placebo matched to IVA tablet orally once daily in the evening for 29 days. |
| FG001 | TEZ/IVA |
| 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 | Sep 19, 2016 | Jun 29, 2018 |
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| Drug |
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| Tezacaftor/Ivacaftor matching placebo | Drug |
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| Ivacaftor matching placebo | Drug |
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| Absolute Change From Baseline in Small-bowel Area Under the Curve (AUC) Over 1-minute Mean pH Increments at Day 29 |
Absolute change from Baseline in small bowel AUC over 1-minute mean pH increments through 30 minutes at Day 29 was assessed. |
| Baseline, Day 29 |
| Absolute Change From Baseline in Sweat Chloride at Day 29 | Baseline, Day 29 |
| Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | Baseline up to Day 57 |
| Indianapolis |
| Indiana |
| United States |
| Baltimore | Maryland | United States |
| Chapel Hill | North Carolina | United States |
| Cincinnati | Ohio | United States |
| Toledo | Ohio | United States |
| Pittsburgh | Pennsylvania | United States |
Participants received 100 milligram (mg) TEZ/150 mg IVA FDC tablet orally once daily in the morning followed by 150 mg IVA tablet orally once daily in the evening for 29 days. |
| COMPLETED |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | Placebo | Participants received placebo matched to TEZ/IVA FDC tablet orally once daily in the morning followed by placebo matched to IVA tablet orally once daily in the evening for 29 days. |
| BG001 | TEZ/IVA | Participants received 100 mg TEZ/150 mg IVA FDC tablet orally once daily in the morning followed by 150 mg IVA tablet orally once daily in the evening for 29 days. |
| 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 |
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| Mucociliary Clearance (MCC): Percentage of Whole-lung Clearance | MCC was assessed using an imaging technique that enables the tracking of mucus within the airways. MCC was expressed as the percentage of whole-lung clearance through 60 minutes at Baseline. | Mean | Standard Deviation | percentage of whole-lung clearance |
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| Small-bowel Area Under the Curve (AUC) Over 1-minute Mean pH Increments | Small-bowel AUC through 30 minutes of 1 minute mean pH profile through gastric emptying at Baseline was reported. | Mean | Standard Deviation | pH minutes |
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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 | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Primary | Absolute Change From Baseline in Mucociliary Clearance (MCC) at Day 28 | MCC was assessed using an imaging technique that enables the tracking of mucus within the airways. MCC was expressed as the percentage of whole-lung clearance through 60 minutes at Baseline and Day 28. | The Full Analysis Set (FAS) included all randomized participants who carry the relevant cystic fibrosis transmembrane conductance regulator (CFTR) allele and received at least 1 dose of study drug. | Posted | Mean | Standard Deviation | percentage of whole-lung clearance | Baseline, Day 28 |
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| Secondary | Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 28 | Percent predicted FEV1 is the ratio of FEV1 to the predicted FEV1, expressed as a percentage. FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. | The FAS was used. | Posted | Mean | Standard Deviation | percentage of predicted FEV1 | Baseline, Day 28 |
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| Secondary | Absolute Change From Baseline in Small-bowel Area Under the Curve (AUC) Over 1-minute Mean pH Increments at Day 29 | Absolute change from Baseline in small bowel AUC over 1-minute mean pH increments through 30 minutes at Day 29 was assessed. | The FAS was used. Here, "Overall Number of Participants Analyzed" signifies those participants who were evaluable for this outcome measure. | Posted | Mean | Standard Deviation | pH minutes | Baseline, Day 29 |
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| Secondary | Absolute Change From Baseline in Sweat Chloride at Day 29 | The FAS was used. Here, "Overall Number of Participants Analyzed" signifies those participants who were evaluable for this outcome measure. | Posted | Mean | Standard Deviation | millimoles per liter | Baseline, Day 29 |
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| Secondary | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | The Safety Set included all participants who received at least 1 dose of study drug. | Posted | Count of Participants | Participants | Baseline up to Day 57 |
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Baseline up to Day 57: 29 days of dosing + 28 days of safety follow up
Other adverse events table reports the participants with adverse events that exceed a frequency threshold of at least 5% within any arm of the study.
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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 | Placebo | Participants received placebo matched to TEZ/IVA FDC tablet orally once daily in the morning followed by placebo matched to IVA tablet orally once daily in the evening for 29 days. | 0 | 7 | 0 | 7 | 2 | 7 |
| EG001 | TEZ/IVA | Participants received 100 mg TEZ/150 mg IVA FDC tablet orally once daily in the morning followed by 150 mg IVA tablet orally once daily in the evening for 29 days. | 0 | 27 | 0 | 27 | 18 | 27 |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
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| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
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| Sputum increased | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
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| Renal impairment | Renal and urinary disorders | MedDRA 20.0 | Systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
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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 | Jul 27, 2017 | Jun 29, 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 |
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| ID | Term |
|---|---|
| C000654124 | tezacaftor, ivacaftor drug combination |
| C545203 | ivacaftor |
| C000625213 | tezacaftor |
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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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