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This study is a multiple within participant crossover study to evaluate the effect of ivacaftor on lung function in participants aged 12 years and older with cystic fibrosis (CF) who have phenotypic or molecular evidence of residual CF transmembrane conductance regulator (CFTR) function.
CFTR Mutations associated with residual function or defective messenger ribonucleic acid (mRNA) splicing include the following:
R117H, E56K, P67L, D110E, D110H, R117C, R347H, R352Q, A455E, D579G, S945L, L206W, R1070W, F1074L, D1152H, S1235R, D1270N, 2789+5G->A, 3849+10kbC->T, 3272-26A->G, 711+5G->A, 3120G->A, 1811+1.6kbA->G, 711+3A->G, 1898+3A->G, 1898+1G->A, 1717-1G->A, 1717-8G->A, 1342-2A->C, 405+3A->C, 1716G/A 1811+1G->C, 1898+5G->T, 3850-3T->G, IVS14b+5G->A, 1898+1G->T, 4005+2T->C, 621+3A->G, 621+1G->T.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Ivacaftor, Placebo, Ivacaftor, Placebo (IPIP) | Experimental | During the Crossover Period, study drug was administered in 2-week alternating cycles with a minimum of 4-week washout period between Cycle 1 [(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)] and Cycle 2 [(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)] and between Cycle 2 and the Open-label Period (Day 1 to 57). During the Open-label Period, all participants received ivacaftor. |
|
| Ivacaftor, Placebo, Placebo, Ivacaftor (IPPI) | Experimental | During the Crossover Period, study drug was administered in 2-week alternating cycles with a minimum of 4-week washout period between Cycle 1 [(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)] and Cycle 2 [(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)] and between Cycle 2 and the Open-label Period (Day 1 to 57). During the Open-label Period, all participants received ivacaftor. |
|
| Placebo, Ivacaftor, Ivacaftor, Placebo (PIIP) | Experimental | During the Crossover Period, study drug was administered in 2-week alternating cycles with a minimum of 4-week washout period between Cycle 1 [(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)] and Cycle 2 [(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)] and between Cycle 2 and the Open-label Period (Day 1 to 57). During the Open-label Period, all participants received ivacaftor. |
|
| Placebo, Ivacaftor, Placebo, Ivacaftor (PIPI) |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Ivacaftor | Drug | 150 milligram (mg) tablet orally every 12 hours up to 12 weeks. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Cycle 1 and Cycle 2: Absolute Change From Cycle Baseline In Percent Predicted Forced Expiratory Volume In 1 Second (FEV1) After 2 Weeks of Treatment | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Hankinson and Wang standards were used to calculate percent predicted FEV1 (for age, gender, and height). The Hankinson standard was used for male participants 18 years and older and female participants 16 years and older. The Wang standard was used for male participants aged 12 to 17 years and for female participants aged 12 to 15 years. Data was to be reported for each cycle (Cycle 1 and Cycle 2) and as per drug treatment, for overall participants and as per genotype (residual function mutation and mRNA splice site mutation). | Cycle 1 baseline, Cycle 1 Day 15 (for Cycle 1 reporting arms); Cycle 2 baseline, Cycle 2 Day 15 (for Cycle 2 reporting arms) |
| Measure | Description | Time Frame |
|---|---|---|
| Cycle 1 and Cycle 2: Absolute Change From Cycle Baseline In Lung Clearance Index (LCI) After 2 Weeks of Treatment | LCI is a measure of ventilation inhomogeneity that is derived from a multiple-breath washout test. The LCI was calculated as the number of lung volume turnovers (cumulative expired volume divided by the functional residual capacity [FRC]) required to reduce end-tidal concentration of an inert gas to 1/40th of the starting value. Data was to be reported for each cycle (Cycle 1 and Cycle 2) and as per drug treatment. Data was to be reported for each cycle (Cycle 1 and Cycle 2) and as per drug treatment, for overall participants and as per genotype (residual function mutation and mRNA splice site mutation). |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Jerry Nick, MD | National Jewish Health | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Denver | Colorado | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 31784217 | Derived | Nick JA, St Clair C, Jones MC, Lan L, Higgins M; VX12-770-113 Study Team. Ivacaftor in cystic fibrosis with residual function: Lung function results from an N-of-1 study. J Cyst Fibros. 2020 Jan;19(1):91-98. doi: 10.1016/j.jcf.2019.09.013. Epub 2019 Nov 26. |
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| ID | Title | Description |
|---|---|---|
| FG000 | Ivacaftor, Placebo, Ivacaftor, Placebo (IPIP) | During the Crossover Period, study drug was administered in 2-week alternating cycles with a minimum of 4-week washout period between Cycle 1 [(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)] and Cycle 2 [(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)] and between Cycle 2 and the Open-label Period (Day 1 to 57). During the Open-label Period, all participants received ivacaftor. |
| FG001 | Ivacaftor, Placebo, Placebo, Ivacaftor (IPPI) | During the Crossover Period, study drug was administered in 2-week alternating cycles with a minimum of 4-week washout period between Cycle 1 [(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)] and Cycle 2 [(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)] and between Cycle 2 and the Open-label Period (Day 1 to 57). During the Open-label Period, all participants received ivacaftor. |
| FG002 | Placebo, Ivacaftor, Ivacaftor, Placebo (PIIP) | During the Crossover Period, study drug was administered in 2-week alternating cycles with a minimum of 4-week washout period between Cycle 1 [(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)] and Cycle 2 [(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)] and between Cycle 2 and the Open-label Period (Day 1 to 57). During the Open-label Period, all participants received ivacaftor. |
| FG003 | Placebo, Ivacaftor, Placebo, Ivacaftor (PIPI) | During the Crossover Period, study drug was administered in 2-week alternating cycles with a minimum of 4-week washout period between Cycle 1 [(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)] and Cycle 2 [(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)] and between Cycle 2 and the Open-label Period (Day 1 to 57). During the Open-label Period, all participants received ivacaftor. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Cycle 1 Period 1 (2 Weeks) |
| |||||||||||||
| Cycle 1 Period 2 (2 Weeks) |
| |||||||||||||
| Washout Period 1 (4 Weeks) |
| |||||||||||||
| Cycle 2 Period 1 (2 Weeks) |
| |||||||||||||
| Cycle 2 Period 2 (2 Weeks) |
| |||||||||||||
| Washout Period 2 (4 Weeks) |
| |||||||||||||
| Open-label Period (8 Weeks) |
|
Full Analysis Set (FAS) included all randomized participants who received at least 1 dose of study drug (ivacaftor or placebo).
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| ID | Title | Description |
|---|---|---|
| BG000 | IPIP | Detailed reporting group description is provided in Participant Flow module. |
| BG001 | IPPI | Detailed reporting group description is provided in Participant Flow module. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| 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 | Cycle 1 and Cycle 2: Absolute Change From Cycle Baseline In Percent Predicted Forced Expiratory Volume In 1 Second (FEV1) After 2 Weeks of Treatment | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Hankinson and Wang standards were used to calculate percent predicted FEV1 (for age, gender, and height). The Hankinson standard was used for male participants 18 years and older and female participants 16 years and older. The Wang standard was used for male participants aged 12 to 17 years and for female participants aged 12 to 15 years. Data was to be reported for each cycle (Cycle 1 and Cycle 2) and as per drug treatment, for overall participants and as per genotype (residual function mutation and mRNA splice site mutation). | Full analysis set (FAS) population. Here, number of participants analyzed signifies participant evaluable for this outcome measure and "n" signifies participants who were evaluable for the specified category in each arm, respectively. | Posted | Mean | Standard Deviation | Percent predicted of FEV1 | Cycle 1 baseline, Cycle 1 Day 15 (for Cycle 1 reporting arms); Cycle 2 baseline, Cycle 2 Day 15 (for Cycle 2 reporting arms) |
From first dose of study drug through completion of follow-up visit (up to 26 weeks)
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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 | Crossover Double-blind Period: Placebo | Placebo matched to ivacaftor tablet orally every 12 hours for 2 weeks either during the double blind period 1 or 2 of cycle 1 and cycle 2. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Monitor | Vertex Pharmaceuticals Incorporated | 617-341-6777 | medicalinfo@vrtx.com |
Not provided
| ID | Term |
|---|---|
| D003550 | Cystic Fibrosis |
| ID | Term |
|---|---|
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
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| ID | Term |
|---|---|
| C545203 | ivacaftor |
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During the Crossover Period, study drug was administered in 2-week alternating cycles with a minimum of 4-week washout period between Cycle 1 [(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)] and Cycle 2 [(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)] and between Cycle 2 and the Open-label Period (Day 1 to 57). During the Open-label Period, all participants received ivacaftor. |
|
|
| Placebo-matched-to-ivacaftor tablet | Drug | Orally every 12 hours up to 4 weeks. |
|
| Cycle 1 baseline, Cycle 1 Day 15 (for Cycle 1 reporting arms); Cycle 2 baseline, Cycle 2 Day 15 (for Cycle 2 reporting arms) |
| Open-label Period: Absolute Change From Open-label Baseline In Percent Predicted Forced Expiratory Volume In 1 Second (FEV1) at Day 57 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Hankinson and Wang standards were used to calculate percent predicted FEV1 (for age, gender, and height). The Hankinson standard was used for male participants 18 years and older and female participants 16 years and older. The Wang standard was used for male participants aged 12 to 17 years and for female participants aged 12 to 15 years. Data was to be reported for overall participants and as per genotype (residual function mutation and mRNA splice site mutation). | Open-label Baseline, Open-label Day 57 |
| Open-label Period: Absolute Change From Open-label Baseline In Lung Clearance Index (LCI) at Day 57 | LCI is a measure of ventilation inhomogeneity that is derived from a multiple-breath washout test. The LCI was calculated as the number of lung volume turnovers (cumulative expired volume divided by the functional residual capacity [FRC]) required to reduce end-tidal concentration of an inert gas to 1/40th of the starting value. Data was to be reported for overall participants and as per genotype (residual function mutation and mRNA splice site mutation). | Open-label Baseline, Open-label Day 57 |
| Open-label Period: Absolute Change From Study Baseline In Sweat Chloride at Day 57 | Sweat samples were collected using an approved Macroduct (Wescor, Logan, Utah) collection device. A volume of greater than or equal to (>=) 15 microliter was required for determination of sweat chloride. Data was to be reported for overall participants and as per genotype (residual function mutation and mRNA splice site mutation). | Study Baseline, Open-label Day 57 |
| Open-label Period: Absolute Change From Open-label Baseline In Weight at Day 57 | Data was to be reported for overall participants and as per genotype (residual function mutation and mRNA splice site mutation). | Open-label Baseline, Open-label Day 57 |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Adverse events (AEs) that started (or increased in severity) from first dose of study drug through completion of Follow-up were considered TEAEs, with exception that if an AE started during a Washout Period and was beyond 14 days from last dose date of preceding cycle, AE was considered as a "Washout Period" AE, and hence not TEAE. A TEAE was attributed to treatment in which it started or to the treatment in second cycling period of previous Crossover Period if it started during a Washout Period. SAE: medical event or condition, which falls into any of following categories, regardless of its relationship to study drug: death, life threatening adverse experience, in-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. Data was to be reported by drug treatment for double-blind crossover period (Cycle 1 up to Washout Period 2) and open-label period. | From first dose of study drug through completion of follow-up visit (up to 26 weeks) |
| COMPLETED |
|
| NOT COMPLETED |
|
|
| COMPLETED |
|
| NOT COMPLETED |
|
| COMPLETED |
|
| NOT COMPLETED |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
| COMPLETED |
|
| NOT COMPLETED |
|
| COMPLETED |
|
| NOT COMPLETED |
|
| BG002 | PIIP | Detailed reporting group description is provided in Participant Flow module. |
| BG003 | PIPI | Detailed reporting group description is provided in Participant Flow module. |
| BG004 | Total | Total of all reporting groups |
| years |
|
| Age, Customized | Number | participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Genotype | All participants were tested for cystic fibrosis transmembrane conductance regulator (CFTR) genotype. Participants with a documented medical history of either a residual function or a messenger ribonucleic acid (mRNA) splice site (Class V) CFTR mutation were reported. | Number | participants |
|
| ID |
|---|
| Title |
|---|
| Description |
|---|
| OG000 | Cycle 1: Placebo | Placebo-matched-to-ivacaftor tablet orally every 12 hours for 2 weeks during either in period 1 or 2 of Cycle 1. |
| OG001 | Cycle 1: Ivacaftor | Ivacaftor 150 mg tablet orally every 12 hours for 2 weeks during either in period 1 or 2 of Cycle 1. |
| OG002 | Cycle 2: Placebo | Placebo-matched-to-ivacaftor tablet orally every 12 hours for 2 weeks during either in period 1 or 2 of Cycle 2. |
| OG003 | Cycle 2: Ivacaftor | Ivacaftor 150 mg tablet orally every 12 hours for 2 weeks during either in period 1 or 2 of Cycle 2. |
|
|
|
| Secondary | Cycle 1 and Cycle 2: Absolute Change From Cycle Baseline In Lung Clearance Index (LCI) After 2 Weeks of Treatment | LCI is a measure of ventilation inhomogeneity that is derived from a multiple-breath washout test. The LCI was calculated as the number of lung volume turnovers (cumulative expired volume divided by the functional residual capacity [FRC]) required to reduce end-tidal concentration of an inert gas to 1/40th of the starting value. Data was to be reported for each cycle (Cycle 1 and Cycle 2) and as per drug treatment. Data was to be reported for each cycle (Cycle 1 and Cycle 2) and as per drug treatment, for overall participants and as per genotype (residual function mutation and mRNA splice site mutation). | FAS population. Here, number of participants analyzed signifies participant evaluable for this outcome measure and "n" signifies participants who were evaluable for the specified category in each arm, respectively. | Posted | Mean | Standard Deviation | ratio | Cycle 1 baseline, Cycle 1 Day 15 (for Cycle 1 reporting arms); Cycle 2 baseline, Cycle 2 Day 15 (for Cycle 2 reporting arms) |
|
|
|
| Secondary | Open-label Period: Absolute Change From Open-label Baseline In Percent Predicted Forced Expiratory Volume In 1 Second (FEV1) at Day 57 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Hankinson and Wang standards were used to calculate percent predicted FEV1 (for age, gender, and height). The Hankinson standard was used for male participants 18 years and older and female participants 16 years and older. The Wang standard was used for male participants aged 12 to 17 years and for female participants aged 12 to 15 years. Data was to be reported for overall participants and as per genotype (residual function mutation and mRNA splice site mutation). | FAS population. Here, number of participants analyzed signifies participant evaluable for this outcome measure and "n" signifies participants who were evaluable for the specified category. | Posted | Mean | Standard Deviation | Percent predicted of FEV1 | Open-label Baseline, Open-label Day 57 |
|
|
|
| Secondary | Open-label Period: Absolute Change From Open-label Baseline In Lung Clearance Index (LCI) at Day 57 | LCI is a measure of ventilation inhomogeneity that is derived from a multiple-breath washout test. The LCI was calculated as the number of lung volume turnovers (cumulative expired volume divided by the functional residual capacity [FRC]) required to reduce end-tidal concentration of an inert gas to 1/40th of the starting value. Data was to be reported for overall participants and as per genotype (residual function mutation and mRNA splice site mutation). | FAS population. Here, number of participants analyzed signifies participant evaluable for this outcome measure and "n" signifies participants who were evaluable for the specified category. | Posted | Mean | Standard Deviation | ratio | Open-label Baseline, Open-label Day 57 |
|
|
|
| Secondary | Open-label Period: Absolute Change From Study Baseline In Sweat Chloride at Day 57 | Sweat samples were collected using an approved Macroduct (Wescor, Logan, Utah) collection device. A volume of greater than or equal to (>=) 15 microliter was required for determination of sweat chloride. Data was to be reported for overall participants and as per genotype (residual function mutation and mRNA splice site mutation). | FAS population. Here, number of participants analyzed signifies participant evaluable for this outcome measure and "n" signifies participants who were evaluable for the specified category. | Posted | Mean | Standard Deviation | millimole per liter (mmol/L) | Study Baseline, Open-label Day 57 |
|
|
|
| Secondary | Open-label Period: Absolute Change From Open-label Baseline In Weight at Day 57 | Data was to be reported for overall participants and as per genotype (residual function mutation and mRNA splice site mutation). | FAS population. Here, number of participants analyzed signifies participant evaluable for this outcome measure and "n" signifies participants who were evaluable for the specified category. | Posted | Mean | Standard Deviation | kilograms (kg) | Open-label Baseline, Open-label Day 57 |
|
|
|
| Secondary | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Adverse events (AEs) that started (or increased in severity) from first dose of study drug through completion of Follow-up were considered TEAEs, with exception that if an AE started during a Washout Period and was beyond 14 days from last dose date of preceding cycle, AE was considered as a "Washout Period" AE, and hence not TEAE. A TEAE was attributed to treatment in which it started or to the treatment in second cycling period of previous Crossover Period if it started during a Washout Period. SAE: medical event or condition, which falls into any of following categories, regardless of its relationship to study drug: death, life threatening adverse experience, in-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. Data was to be reported by drug treatment for double-blind crossover period (Cycle 1 up to Washout Period 2) and open-label period. | Safety set included all participants who received at least 1 dose of study drug. Here, number of participants analyzed signifies participant evaluable for this outcome measure. | Posted | Number | participants | From first dose of study drug through completion of follow-up visit (up to 26 weeks) |
|
|
|
| 0 |
| 24 |
| 17 |
| 24 |
| EG001 | Crossover Double-blind Period: Ivacaftor | Ivacaftor 150 mg tablet orally every 12 hours for 2 weeks either during the double blind period 1 or 2 of cycle 1 and cycle 2. | 1 | 24 | 18 | 24 |
| EG002 | Open-label Period: Ivacaftor | Ivacaftor 150 mg tablet orally every 12 hours for 8 weeks during the open-label period after washout period 2. | 0 | 21 | 20 | 21 |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| Paranasal sinus hypersecretion | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| Sputum increased | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| Respiration abnormal | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| Sinus congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| Sneezing | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| Throat irritation | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| Wheezing | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| Pleuritic pain | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| Respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| Upper-airway cough syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| Dysphonia | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| Dyspnoea exertional | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| Respiratory tract irritation | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
|
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
|
| Acute sinusitis | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
|
| Incision site infection | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
|
| Laryngitis | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
|
| Oral viral infection | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
|
| Respiratory tract infection viral | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
|
| Sinusitis | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
|
| Viral upper respiratory tract infection | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
|
| Influenza | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
|
| Oral candidiasis | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
|
| Pharyngitis streptococcal | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
|
| Rhinitis | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
|
| Viral infection | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
|
| Fatigue | General disorders | MedDRA 15.0 | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA 15.0 | Systematic Assessment |
|
| Chest discomfort | General disorders | MedDRA 15.0 | Systematic Assessment |
|
| Chest pain | General disorders | MedDRA 15.0 | Systematic Assessment |
|
| Chills | General disorders | MedDRA 15.0 | Systematic Assessment |
|
| Sinus headache | Nervous system disorders | MedDRA 15.0 | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA 15.0 | Systematic Assessment |
|
| Dizziness | Nervous system disorders | MedDRA 15.0 | Systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment |
|
| Intervertebral disc protrusion | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment |
|
| Joint swelling | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment |
|
| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment |
|
| Musculoskeletal discomfort | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment |
|
| Osteopenia | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment |
|
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment |
|
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
|
| Heat rash | Skin and subcutaneous tissue disorders | MedDRA 15.0 | Systematic Assessment |
|
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 15.0 | Systematic Assessment |
|
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA 15.0 | Systematic Assessment |
|
| Contusion | Injury, poisoning and procedural complications | MedDRA 15.0 | Systematic Assessment |
|
| Excoriation | Injury, poisoning and procedural complications | MedDRA 15.0 | Systematic Assessment |
|
| Wrist fracture | Injury, poisoning and procedural complications | MedDRA 15.0 | Systematic Assessment |
|
| Cardiac flutter | Cardiac disorders | MedDRA 15.0 | Systematic Assessment |
|
| Lacrimation increased | Eye disorders | MedDRA 15.0 | Systematic Assessment |
|
| Ocular hyperaemia | Eye disorders | MedDRA 15.0 | Systematic Assessment |
|
| Hypersensitivity | Immune system disorders | MedDRA 15.0 | Systematic Assessment |
|
| Liver function test abnormal | Investigations | MedDRA 15.0 | Systematic Assessment |
|
| Decreased appetite | Metabolism and nutrition disorders | MedDRA 15.0 | Systematic Assessment |
|
| Dehydration | Metabolism and nutrition disorders | MedDRA 15.0 | Systematic Assessment |
|
| Anxiety | Psychiatric disorders | MedDRA 15.0 | Systematic Assessment |
|
| Postmenopausal haemorrhage | Reproductive system and breast disorders | MedDRA 15.0 | 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.
| D030342 |
| Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |
| mRNA Splice Site(ClassV)Mutations (n=10, 10, 9, 9) |
|
| Residual Function Mutations (n=13, 14, 14, 13) |
|
|
|
|
| Title | Measurements |
|---|---|
|
| Title | Measurements |
|---|---|
|