Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2016-001004-33 | EudraCT Number |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
This is a Phase 3, 2-part (Part A and Part B), open-label, multicenter study evaluating the pharmacokinetics (PK), safety, tolerability, and pharmacodynamics (PD) of multiple doses of lumacaftor/ivacaftor (LUM/IVA) in subjects 2 through 5 years of age (inclusive) with cystic fibrosis (CF), homozygous for F508del. Subjects who participate in Part A may participate in Part B, if they meet the eligibility criteria.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Lumacaftor/Ivacaftor (LUM/IVA) | Experimental | Part A (<14 kg): Participants weighing less than (<) 14 kilograms (kg) at screening received LUM 100 milligram (mg)/IVA 125 mg fixed-dose combination every 12 hours for 15 days in Part A. Part A (>=14 kg): Participants weighing greater than or equal to (>=) 14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 15 days in Part A. Part B (<14 kg): Participants weighing <14 kg at screening received LUM 100 mg/IVA 125 mg fixed-dose combination every 12 hours for 24 weeks in Part B. Part B (>=14 kg): Participants weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 24 weeks in Part B. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| LUM/IVA | Drug |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Part A: Pre-dose Concentration (Ctrough) of LUM and IVA | Day 15 | |
| Part B: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Day 1 up to Week 26 |
| Measure | Description | Time Frame |
|---|---|---|
| Part A: Pre-dose Concentration (Ctrough) of LUM and IVA Metabolites | Day 15 | |
| Part A: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Day 1 up to Day 25 |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Palo Alto | California | United States | ||||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 30686767 | Derived | McNamara JJ, McColley SA, Marigowda G, Liu F, Tian S, Owen CA, Stiles D, Li C, Waltz D, Wang LT, Sawicki GS. Safety, pharmacokinetics, and pharmacodynamics of lumacaftor and ivacaftor combination therapy in children aged 2-5 years with cystic fibrosis homozygous for F508del-CFTR: an open-label phase 3 study. Lancet Respir Med. 2019 Apr;7(4):325-335. doi: 10.1016/S2213-2600(18)30460-0. Epub 2019 Jan 24. |
Not provided
Not provided
Not provided
The study was conducted in 2 parts, Part A and Part B. In Parts A and B, participants received doses of lumacaftor/ivacaftor (LUM/IVA) based on weight. Participants from Part A may have also participated in Part B.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Lumacaftor/Ivacaftor (LUM/IVA) | Part A (<14 kg): Participants weighing less than (<) 14 kilograms (kg) at screening received LUM 100 milligram (mg)/IVA 125 mg fixed-dose combination every 12 hours for 15 days in Part A. Part A (>=14 kg): Participants weighing greater than or equal to (>=) 14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 15 days in Part A. Part B (<14 kg): Participants weighing <14 kg at screening received LUM 100 mg/IVA 125 mg fixed-dose combination every 12 hours for 24 weeks in Part B. Part B (>=14 kg): Participants weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 24 weeks in Part B. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Part A (15 Days) |
|
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 | Apr 13, 2017 | Sep 28, 2018 |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Part B: Absolute Change From Baseline in Sweat Chloride at Week 24 | Sweat samples were collected using an approved collection device. | Baseline, Week 24 |
| Part B: Absolute Change From Baseline in Body Mass Index (BMI) at Week 24 | BMI was defined as weight in kilograms (kg) divided by height in square meter (m^2). | Baseline, Week 24 |
| Part B: Absolute Change From Baseline in Body Mass Index (BMI) For-Age Z-Score at Week 24 | 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). | Baseline, Week 24 |
| Part B: Absolute Change From Baseline in Weight at Week 24 | Baseline, Week 24 |
| Part B: Absolute Change From Baseline in Weight-for-age Z-Score at Week 24 | z-score is a statistical measure to describe whether a mean was above or below the standard. Weight, adjusted for age and sex, was analyzed as weight-for-age z-score (Weight z-score). | Baseline, Week 24 |
| Part B: Absolute Change From Baseline in Stature (Height) at Week 24 | Baseline, Week 24 |
| Part B: Absolute Change From Baseline in Stature-for-Age Z-Score | z-score is a statistical measure to describe whether a mean was above or below the standard. Stature (height), adjusted for age and sex, was analyzed as Stature-for-age z-score (Stature z-score). | Baseline, Week 24 |
| Part B: Number of Pulmonary Exacerbations | Pulmonary exacerbation was defined as new or changed treatment with oral, inhaled, or intravenous antibiotics and fulfillment of pre-specified protocol defined criteria. | Through Week 24 |
| Part B: Number of Participants With at Least One Pulmonary Exacerbation Pulmonary Exacerbation Through Week 24 | Pulmonary exacerbation was defined as new or changed treatment with oral, inhaled, or intravenous antibiotics and fulfillment of pre-specified protocol defined criteria. Time to event data was not collected and instead, number of participants 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. | Through Week 24 |
| Part B: Number of Cystic Fibrosis (CF)-Related Hospitalizations | Through Week 24 |
| Part B: Absolute Change From Baseline in Fecal Elastase-1 (FE-1) Levels at Week 24 | Baseline, Week 24 |
| Part B: Absolute Change From Baseline in Serum Levels of Immunoreactive Trypsinogen (IRT) Through Week 24 | Baseline, Through Week 24 |
| Part B: Number of Participants With Microbiology Culture Status (Positive or Negative) at Week 24 | Following microbial tests were performed: Burkholderia, Methicillin Resistant Staphylococcus Aureus (MRSA), Methicillin Susceptible Staphylococcus Aureus (MSSA), Pseudomonas Aeruginosa Mucoid (P. Aeruginosa Mucoid), P. Aeruginosa Non-Mucoid, P. Aeruginosa Small Colony Variant and Stenotrophomonas Maltophilia. | Baseline and Week 24 |
| Part B: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Week 24 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. | Baseline, Week 24 |
| Part B: Absolute Change in Sweat Chloride From Week 24 at Week 26 | Sweat samples were collected using an approved collection device. | Week 24, Week 26 |
| Part B: Acceptability/Palatability of LUM/IVA Granules Measured Using Hedonic Scale | The acceptability and palatability of LUM/IVA granules was assessed by a visual analog scale that incorporates a 5 point facial hedonic scale (Liked it Very Much, Liked it a Little, Not sure, Disliked it a Little, Disliked it Very Much). The assessment was conducted in 2 steps: assessment of approved food/liquid (Evaluation 1), and assessment of approved food/liquid with LUM/IVA granules (Evaluation 2). | Day 1 |
| Part B: Absolute Change From Baseline in Lung Clearance Index (LCI) 2.5 at Week 24 | Lung clearance index (LCI) is a measure of ventilation inhomogeneity that is derived from a multiple breath washout test using Nitrogen (N2). LCI 2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value. | Baseline, Week 24 |
| Part B: Absolute Change From Baseline in Lung Clearance Index (LCI) 5.0 at Week 24 | LCI is a measure of ventilation inhomogeneity that is derived from a multiple breath washout test using Nitrogen (N2). LCI 5.0 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/20th of its starting value. | Baseline, Week 24 |
| Part B: Pre-dose Concentration (Ctrough) of LUM and IVA and Its Metabolites | Week 24 |
| Aurora |
| Colorado |
| United States |
| Chicago | Illinois | United States |
| Indianapolis | Indiana | United States |
| Boston | Massachusetts | United States |
| Minneapolis | Minnesota | United States |
| Kansas City | Missouri | United States |
| Buffalo | New York | United States |
| Chapel Hill | North Carolina | United States |
| Cincinnati | Ohio | United States |
| Cleveland | Ohio | United States |
| Columbus | Ohio | United States |
| Philadelphia | Pennsylvania | United States |
| Charleston | South Carolina | United States |
| Houston | Texas | United States |
| Norfolk | Virginia | United States |
| Seattle | Washington | United States |
| Vancouver | British Columbia | Canada |
| Toronto | Ontario | Canada |
| Montreal | Canada |
| Part A (<14 kg Weight) |
|
| Part A (>=14 kg Weight) |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
| Part B (24 Weeks) |
|
|
The Safety Set included all participants who received at least 1 dose of LUM/IVA in the respective study part.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Lumacaftor/Ivacaftor (LUM/IVA) | Part A (<14 kg): Participants weighing <14 kg at screening received LUM 100 mg/IVA 125 mg fixed-dose combination every 12 hours for 15 days in Part A. Part A (>=14 kg): Participants weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 15 days in Part A. Part B (<14 kg): Participants weighing <14 kg at screening received LUM 100 mg/IVA 125 mg fixed-dose combination every 12 hours for 24 weeks in Part B. Part B (>=14 kg): Participants weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 24 weeks in Part B. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Data were planned to be reported separately for Part A and Part B of the study. Here "number analyzed" signifies participants who were evaluable for specified part of the study. | Mean | Standard Deviation | months |
| ||||||||||||||||
| Sex: Female, Male | Data were planned to be reported separately for Part A and Part B of the study. Here "number analyzed" signifies participants who were evaluable for specified part of the study. | Count of Participants | Participants |
| |||||||||||||||||
| Ethnicity (NIH/OMB) | Data were planned to be reported separately for Part A and Part B of the study. Here "number analyzed" signifies participants who were evaluable for specified part of the study. | Count of Participants | Participants |
| |||||||||||||||||
| Race (NIH/OMB) | Data were planned to be reported separately for Part A and Part B of the study. Here "number analyzed" signifies participants who were evaluable for specified part of the study. | 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 | Part A: Pre-dose Concentration (Ctrough) of LUM and IVA | The pharmacokinetic (PK) set for Part A included all participants who received at least 1 dose of LUM/IVA in Part A. | Posted | Mean | Standard Deviation | nanogram per milliliter (ng/mL) | Day 15 |
|
|
| ||||||||||||||||||||||||||||||||||||
| Primary | Part B: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | The Safety Set included all participants who received at least 1 dose of LUM/IVA in Part B. | Posted | Count of Participants | Participants | Day 1 up to Week 26 |
|
| ||||||||||||||||||||||||||||||||||||||
| Secondary | Part A: Pre-dose Concentration (Ctrough) of LUM and IVA Metabolites | The PK set for Part A included all participants who received at least 1 dose of LUM/IVA in Part A. | Posted | Mean | Standard Deviation | ng/mL | Day 15 |
|
| |||||||||||||||||||||||||||||||||||||
| Secondary | Part A: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | The Safety Set included all participants who received at least 1 dose of LUM/IVA in Part A. | Posted | Count of Participants | Participants | Day 1 up to Day 25 |
|
| ||||||||||||||||||||||||||||||||||||||
| Secondary | Part B: Absolute Change From Baseline in Sweat Chloride at Week 24 | Sweat samples were collected using an approved collection device. | The Full Analysis Set (FAS) included all enrolled participants in Part B who were exposed to any amount of LUM/IVA in Part B. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome. | Posted | Mean | Standard Deviation | millimole per liter (mmol/L) | Baseline, Week 24 |
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Part B: Absolute Change From Baseline in Body Mass Index (BMI) at Week 24 | BMI was defined as weight in kilograms (kg) divided by height in square meter (m^2). | The FAS included all enrolled participants in Part B who were exposed to any amount of LUM/IVA in Part B. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome. | Posted | Mean | Standard Deviation | Kilogram per meter square (kg/m^2) | Baseline, Week 24 |
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Part B: Absolute Change From Baseline in Body Mass Index (BMI) For-Age Z-Score at Week 24 | 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). | The FAS included all enrolled participants in Part B who were exposed to any amount of LUM/IVA in Part B. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluable for this outcome. | Posted | Mean | Standard Deviation | Z-score | Baseline, Week 24 |
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Part B: Absolute Change From Baseline in Weight at Week 24 | The FAS included all enrolled participants in Part B who were exposed to any amount of LUM/IVA in Part B. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome. | Posted | Mean | Standard Deviation | Kilogram (kg) | Baseline, Week 24 |
|
| |||||||||||||||||||||||||||||||||||||
| Secondary | Part B: Absolute Change From Baseline in Weight-for-age Z-Score at Week 24 | z-score is a statistical measure to describe whether a mean was above or below the standard. Weight, adjusted for age and sex, was analyzed as weight-for-age z-score (Weight z-score). | The FAS included all enrolled subjects in Part B who were exposed to any amount of LUM/IVA in Part B. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome. | Posted | Mean | Standard Deviation | Z-score | Baseline, Week 24 |
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Part B: Absolute Change From Baseline in Stature (Height) at Week 24 | The FAS included all enrolled participants in Part B who were exposed to any amount of LUM/IVA in Part B. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome. | Posted | Mean | Standard Deviation | Centimeter (cm) | Baseline, Week 24 |
|
| |||||||||||||||||||||||||||||||||||||
| Secondary | Part B: Absolute Change From Baseline in Stature-for-Age Z-Score | z-score is a statistical measure to describe whether a mean was above or below the standard. Stature (height), adjusted for age and sex, was analyzed as Stature-for-age z-score (Stature z-score). | The FAS included all enrolled participants in Part B who were exposed to any amount of LUM/IVA in Part B. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome. | Posted | Mean | Standard Deviation | Z-score | Baseline, Week 24 |
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Part B: Number of Pulmonary Exacerbations | Pulmonary exacerbation was defined as new or changed treatment with oral, inhaled, or intravenous antibiotics and fulfillment of pre-specified protocol defined criteria. | The FAS included all enrolled participants in Part B who were exposed to any amount of LUM/IVA in Part B. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome. | Posted | Number | pulmonary exacerbations | Through Week 24 |
|
| |||||||||||||||||||||||||||||||||||||
| Secondary | Part B: Number of Participants With at Least One Pulmonary Exacerbation Pulmonary Exacerbation Through Week 24 | Pulmonary exacerbation was defined as new or changed treatment with oral, inhaled, or intravenous antibiotics and fulfillment of pre-specified protocol defined criteria. Time to event data was not collected and instead, number of participants 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. | The FAS included all enrolled participants in Part B who were exposed to any amount of LUM/IVA in Part B. | Posted | Count of Participants | Participants | Through Week 24 |
| ||||||||||||||||||||||||||||||||||||||
| Secondary | Part B: Number of Cystic Fibrosis (CF)-Related Hospitalizations | The FAS included all enrolled participants in Part B who were exposed to any amount of LUM/IVA in Part B. | Posted | Number | hospitalizations | Through Week 24 |
|
| ||||||||||||||||||||||||||||||||||||||
| Secondary | Part B: Absolute Change From Baseline in Fecal Elastase-1 (FE-1) Levels at Week 24 | The FAS included all enrolled subjects in Part B who were exposed to any amount of LUM/IVA in Part B. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome. | Posted | Mean | Standard Deviation | microgram per gram | Baseline, Week 24 |
|
| |||||||||||||||||||||||||||||||||||||
| Secondary | Part B: Absolute Change From Baseline in Serum Levels of Immunoreactive Trypsinogen (IRT) Through Week 24 | The FAS included all enrolled participants in Part B who were exposed to any amount of LUM/IVA in Part B. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome. | Posted | Mean | Standard Deviation | ng/mL | Baseline, Through Week 24 |
|
| |||||||||||||||||||||||||||||||||||||
| Secondary | Part B: Number of Participants With Microbiology Culture Status (Positive or Negative) at Week 24 | Following microbial tests were performed: Burkholderia, Methicillin Resistant Staphylococcus Aureus (MRSA), Methicillin Susceptible Staphylococcus Aureus (MSSA), Pseudomonas Aeruginosa Mucoid (P. Aeruginosa Mucoid), P. Aeruginosa Non-Mucoid, P. Aeruginosa Small Colony Variant and Stenotrophomonas Maltophilia. | The FAS analysis set was used. Here "Number Analyzed" signifies those participants who were evaluated for this outcome at the specified time point.. | Posted | Count of Participants | Participants | Baseline and Week 24 |
|
| |||||||||||||||||||||||||||||||||||||
| Secondary | Part B: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Week 24 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. | Analysis population: participants >=3 years of age with data available for Baseline and Week 24. Only participants from "Part B (>=14 Kg Weight)" arm met the eligibility criteria for the specified time point and were included in the analysis. | Posted | Mean | Standard Deviation | percentage of predicted FEV1 | Baseline, Week 24 |
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Part B: Absolute Change in Sweat Chloride From Week 24 at Week 26 | Sweat samples were collected using an approved collection device. | The FAS included all enrolled subjects in Part B who were exposed to any amount of LUM/IVA in Part B. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome. | Posted | Mean | Standard Deviation | mmol/L | Week 24, Week 26 |
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Part B: Acceptability/Palatability of LUM/IVA Granules Measured Using Hedonic Scale | The acceptability and palatability of LUM/IVA granules was assessed by a visual analog scale that incorporates a 5 point facial hedonic scale (Liked it Very Much, Liked it a Little, Not sure, Disliked it a Little, Disliked it Very Much). The assessment was conducted in 2 steps: assessment of approved food/liquid (Evaluation 1), and assessment of approved food/liquid with LUM/IVA granules (Evaluation 2). | The FAS included all enrolled subjects in Part B who were exposed to any amount of LUM/IVA in Part B. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome. | Posted | Count of Participants | Participants | Day 1 |
|
| |||||||||||||||||||||||||||||||||||||
| Secondary | Part B: Absolute Change From Baseline in Lung Clearance Index (LCI) 2.5 at Week 24 | Lung clearance index (LCI) is a measure of ventilation inhomogeneity that is derived from a multiple breath washout test using Nitrogen (N2). LCI 2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value. | The LCI Substudy Set included all participants who had signed informed consent (and assent, if applicable) to the optional LCI Substudy in Part B and enrolled and dosed in Part B. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome. | Posted | Mean | Standard Deviation | ratio | Baseline, Week 24 |
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Part B: Absolute Change From Baseline in Lung Clearance Index (LCI) 5.0 at Week 24 | LCI is a measure of ventilation inhomogeneity that is derived from a multiple breath washout test using Nitrogen (N2). LCI 5.0 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/20th of its starting value. | The LCI Substudy Set included all subjects who had signed informed consent (and assent, if applicable) to the optional LCI Substudy in Part B and enrolled and dosed in Part B. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome. | Posted | Mean | Standard Deviation | ratio | Baseline, Week 24 |
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Part B: Pre-dose Concentration (Ctrough) of LUM and IVA and Its Metabolites | The PK set for Part B included all participants who received at least 1 dose of LUM/IVA in Part B. Here "Number Analyzed" signifies those participants who were evaluable for the specified category. | Posted | Mean | Standard Deviation | ng/mL | Week 24 |
|
|
Part A: Day 1 up to Day 25; Part B: Day 1 up to Week 26
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 | Part A: LUM 100 mg/IVA 125 mg | Participants weighing <14 kg at screening received LUM 100 milligram (mg)/IVA 125 mg fixed-dose combination every 12 hours for 15 days in Part A. | 0 | 4 | 0 | 4 | 4 | 4 |
| EG001 | Part A: LUM 150 mg/IVA 188 mg | Participants weighing >= 14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 15 days in Part A. | 0 | 8 | 0 | 8 | 6 | 8 |
| EG002 | Part B: LUM 100 mg/IVA 125 mg | Participants weighing less than <14 kg at screening received LUM 100 mg/IVA 125 mg fixed-dose combination every 12 hours for 24 weeks in Part B. | 0 | 19 | 2 | 19 | 19 | 19 |
| EG003 | Part B: LUM 150 mg/IVA 188 mg | Participants weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hurs for 24 weeks in Part B. | 0 | 41 | 2 | 41 | 40 | 41 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Constipation | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Seasonal allergy | Immune system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hypersensitivity | Immune system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Chills | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Discomfort | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Pain | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Enuresis | Psychiatric disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Sleep terror | Psychiatric disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Tearfulness | Psychiatric disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Joint dislocation | Injury, poisoning and procedural complications | MedDRA 20.0 | Systematic Assessment |
| |
| Radial head dislocation | Injury, poisoning and procedural complications | MedDRA 20.0 | Systematic Assessment |
| |
| Respiratory rate increased | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Forced expiratory volume decreased | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Pseudomonas test positive | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Activated partial thromboplastin time prolonged | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Burkholderia test positive | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Gamma-glutamyltransferase increased | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| International normalised ratio increased | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Prothrombin time prolonged | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Pulmonary function test decreased | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Staphylococcus test positive | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Streptococcus test positive | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Bacterial test positive | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Blood creatine phosphokinase increased | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Blood iron decreased | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Enterovirus test positive | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Respirovirus test positive | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Vitamin D decreased | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Ventricular extrasystoles | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Nasal discharge discolouration | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Wheezing | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Respiration abnormal | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Sinus congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Sputum discoloured | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Tonsillar inflammation | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Upper respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Anaemia | Blood and lymphatic system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Lymphocytosis | Blood and lymphatic system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Cognitive disorder | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Lethargy | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Eye irritation | Eye disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Lacrimation increased | Eye disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Faeces discoloured | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Faeces soft | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Flatulence | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Haematochezia | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Oral discomfort | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Oral pain | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Post-tussive vomiting | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Steatorrhoea | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Dyschezia | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Frequent bowel movements | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Urinary incontinence | Renal and urinary disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hepatomegaly | Hepatobiliary disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Eczema | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hyperhidrosis | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hypothyroidism | Endocrine disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Lice infestation | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Lower respiratory tract infection viral | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Otitis media | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Conjunctivitis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Impetigo | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Labyrinthitis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Pharyngitis streptococcal | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Viral rash | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Vulvovaginal mycotic infection | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Candida nappy rash | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Gastroenteritis rotavirus | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Hand-foot-and-mouth disease | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Oral candidiasis | Infections and infestations | MedDRA 20.0 | 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.
| 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 | Sep 23, 2016 | Sep 28, 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 |
|---|---|
| C000599212 | lumacaftor, ivacaftor drug combination |
Not provided
Not provided
Not provided
|
| Part B (<14 kg Weight) |
|
|
| Part B (>=14 kg Weight) |
|
|
| Male |
|
| Part A (>=14 kg Weight) |
|
|
| Part B (<14 kg Weight) |
|
|
| Part B (>=14 kg Weight) |
|
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Part A (>=14 kg Weight) |
|
|
| Part B (<14 kg Weight) |
|
|
| Part B (>=14 kg Weight) |
|
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Part A (>=14 kg Weight) |
|
|
| Part B (<14 kg Weight) |
|
|
| Part B (>=14 kg Weight) |
|
|
|
|
|
|
|
|
|
|
|
|
|
| Units | Counts |
|---|
| Participants |
|
|
|
|
|
|
|
|
| Participants |
|
|
| Participants |
|
|
|
|
|
| Positive |
|
| Positive |
|
| Positive |
|
| Positive |
|
| Positive |
|
| Positive |
|
| Positive |
|
| Positive |
|
| Positive |
|
| Positive |
|
| Positive |
|
| Positive |
|
| Positive |
|
| Not sure |
|
| Disliked it a Little |
|
| Disliked it Very Much |
|