A Rollover Safety Study of Lumacaftor/Ivacaftor in Subjec... | NCT03125395

NCT03125395

ID	Type	Description	Link
2019-003112-31	EudraCT Number

Status Module

Sponsor/Collaborators Module

Oversight Module

Description Module

Conditions Module

Design Module

Interventional Study Design

Biospecimen

No data available

No data is available for this block.

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
LUM/IVA	Experimental	LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.	Drug: LUM/IVA

Name	Type	Description	Arm Group Labels	Other Names
LUM/IVA	Drug	Participants received LUM/IVA every q12h.	LUM/IVA	VX-809/VX-770

Outcomes Module

Measure	Description	Time Frame
Safety as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)		Day 1 up to Week 98

Secondary Outcomes

Measure	Description	Time Frame
Absolute Change in Sweat Chloride	Sweat samples were collected using an approved collection device.	From Parent Study 115B Baseline at Week 96
Absolute Change in Body Mass Index (BMI)	BMI was defined as weight in kilogram (kg) divided by height in square meter (m^2).

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Subjects entering the Treatment Cohort must meet the following criteria:

Completed 24 weeks of LUM/IVA treatment and the Safety Follow-up Visit in Study VX15-809-115 Part B (Study 115B, NCT02797132)
Willing to remain on a stable CF medication regimen through the Safety Follow-up Visit

Subjects entering the Observational Cohort must meet 1 of the following criteria:

Completed 24 weeks of LUM/IVA treatment and the Safety Follow-up Visit in Study 115B, but do not want to enroll in the Treatment Cohort.
Received at least 4 weeks of LUM/IVA treatment and completed visits up to Week 24 and the Safety Follow-up Visit, if required, of Study 115B but are not taking LUM/IVA at the end of the Study 115B Treatment Period (i.e., Week 24) because of a drug interruption and either did not receive Vertex approval to enroll in the Treatment Cohort or do not want to enroll in the Treatment Cohort.
Permanently discontinued LUM/IVA in Study 115B after receiving at least 4 weeks of treatment and remained in the study from the time of treatment discontinuation through the Week 24 Visit and Safety Follow-up Visit, if required.

Exclusion Criteria (Treatment Cohort Only):

Prematurely discontinued LUM/IVA treatment in Study 115B.
History of any comorbidity or laboratory abnormality that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering LUM/IVA to the subject
History of drug intolerance or other serious reactions to LUM/IVA in Study 115B that would pose an additional risk to the subject in the opinion of investigator, and which should be discussed with the Vertex medical monitor.
Subjects with a history of allergy or hypersensitivity to LUM/IVA.
Liver function test (LFT) abnormality meeting criteria for LUM/IVA treatment interruption at the completion of Study 115B, for which no convincing alternative etiology is identified.
QTc value at the completion of Study 115B that would pose an additional risk to the subject in the opinion of investigator, and which should be discussed with the Vertex medical monitor
History of poor compliance with LUM/IVA and/or procedures in Study 115B as deemed by the investigator.
Participation in an investigational drug trial (including studies investigating LUM and/or IVA) other than Study 115B.

Contacts/Locations Module

Facility	Status	City	State	ZIP	Country	Contacts
Stanford University		Palo Alto	California	94305	United States
Children's Hospital Colorado

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
33965000	Derived	Hoppe JE, Chilvers M, Ratjen F, McNamara JJ, Owen CA, Tian S, Zahigian R, Cornell AG, McColley SA. Long-term safety of lumacaftor-ivacaftor in children aged 2-5 years with cystic fibrosis homozygous for the F508del-CFTR mutation: a multicentre, phase 3, open-label, extension study. Lancet Respir Med. 2021 Sep;9(9):977-988. doi: 10.1016/S2213-2600(21)00069-2. Epub 2021 May 6.

IPD Sharing Statement Module

No data available

No data is available for this block.

Results Section

Participant Flow Module

Arm/Group Information

ID	Title	Description
FG000	LUM/IVA	LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.

Baseline Characteristics Module

Outcome Measures Module

Adverse Events Module

More Info Module

Annotation Section

No data available

No data is available for this block.

Document Section

Large Document Module

Type	Includes Protocol	Includes SAP	Includes ICF	Document Label	Document Date	Document Uploaded Date	Document File Name
Prot	Yes	No	No	Study Protocol	Oct 10, 2016	Jul 15, 2020

Derived Section

Miscellaneous Info Module

Submission Tracking

No data available

No data is available for this block.

Condition Browse Module

Intervention Browse Module

Masking Info

lumacaftor/ivacaftor

Type of Units Analyzed

Arm/Group Information

ID	Title	Description
BG000	LUM/IVA	LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.

Baseline Measures

Title

Description

Population Description

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Denominator Units Selected

Denominators

Classes

Age, Continuous

Mean

Standard Deviation

months

Title

Denominators

Categories

Title	Measurements
	BG00043.2± 12.17

Sex: Female, Male

Count of Participants

Participants

Title

Denominators

Categories

Title	Measurements
Female	BG00028
Male	BG00029

Ethnicity (NIH/OMB)

Count of Participants

Participants

Title

Denominators

Categories

Title	Measurements
Hispanic or Latino	BG0003
Not Hispanic or Latino	BG00054

Race (NIH/OMB)

Count of Participants

Participants

Title

Denominators

Categories

Title	Measurements
American Indian or Alaska Native	BG0000
Asian	BG0000

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

Safety as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

Study 116 safety set included all participants dosed in study 115B who were exposed to any amount of study drug in current study 116 treatment cohort.

Posted

Number

participants

Day 1 up to Week 98

ID	Title	Description
OG000	LUM/IVA	LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.

Units	Counts
Participants	OG00057

Title

Denominators

Categories

Participants with AEs

Title	Measurements
	OG00056

Participants with SAEs

Title	Measurements
	OG00015

Secondary

Absolute Change in Sweat Chloride

Sweat samples were collected using an approved collection device.

Study 116 Full Analysis Set (FAS) included all participants who were enrolled and exposed to any amount of study drug in current study 116.

Posted

Mean

Standard Deviation

millimole per liter (mmol/L)

From Parent Study 115B Baseline at Week 96

ID	Title	Description
OG000	LUM/IVA	LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.

Units	Counts
Participants	OG000

Secondary

Absolute Change in Body Mass Index (BMI)

BMI was defined as weight in kilogram (kg) divided by height in square meter (m^2).

Study 116 FAS.

Posted

Mean

Standard Deviation

kg/m^2

From Parent Study 115B Baseline at Week 96

ID	Title	Description
OG000	LUM/IVA	LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.

Units	Counts
Participants	OG000

Secondary

Absolute Change in BMI-for-age Z-score

BMI was defined as weight in kilograms divided by height in m^2. z-score is a statistical measure to describe whether a mean was above or below the standard. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean.

Study 116 FAS.

Posted

Mean

Standard Deviation

z-score

From Parent Study 115B Baseline at Week 96

ID	Title	Description
OG000	LUM/IVA	LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.

Units	Counts
Participants	OG000

Secondary

Absolute Change in Weight

Study 116 FAS.

Posted

Mean

Standard Deviation

From Parent Study 115B Baseline at Week 96

ID	Title	Description
OG000	LUM/IVA	LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.

Units	Counts
Participants	OG00057

Secondary

Absolute Change in Weight-for-age Z-score

Z-score is a statistical measure to describe whether a mean was above or below the standard. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean.

Study 116 FAS.

Posted

Mean

Standard Error

z-score

From Parent Study 115B Baseline at Week 96

ID	Title	Description
OG000	LUM/IVA	LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.

Units	Counts
Participants	OG000

Secondary

Absolute Change From Baseline in Stature (Height)

Study 116 FAS.

Posted

Mean

Standard Deviation

centimeter

From Parent Study 115B Baseline at Week 96

ID	Title	Description
OG000	LUM/IVA	LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.

Units	Counts
Participants	OG00057

Secondary

Absolute Change in Stature-for-age Z-score

Study 116 FAS.

Posted

Mean

Standard Deviation

z-score

From Parent Study 115B Baseline at Week 96

ID	Title	Description
OG000	LUM/IVA	LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.

Units	Counts
Participants	OG000

Secondary

Time-to-first Pulmonary Exacerbation

Pulmonary exacerbation was defined as new or changed treatment with oral, inhaled, or intravenous antibiotics and fulfillment of pre-specified protocol-defined criteria.

Study 115B FAS included all participants who were enrolled and exposed to any amount of study drug in parent study 115B

Posted

Median

Inter-Quartile Range

days

From Parent Study 115B Baseline through Week 96

ID	Title	Description
OG000	LUM/IVA	LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.

Units	Counts
Participants	OG000

Secondary

Number of Pulmonary Exacerbations (PEx)

Pulmonary exacerbation was defined as new or changed treatment with oral, inhaled, or intravenous antibiotics and fulfillment of pre-specified protocol-defined criteria.

Study 115B FAS.

Posted

Number

pulmonary exacerbation events

From Parent Study 115B Baseline through Week 96

ID	Title	Description
OG000	LUM/IVA	LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.

Units	Counts
Participants	OG000

Secondary

Number of Cystic Fibrosis (CF) Related Hospitalizations

Study 115B FAS.

Posted

Number

hospitalizations

From Parent Study 115B Baseline through Week 96

ID	Title	Description
OG000	LUM/IVA	LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.

Units	Counts
Participants	OG00060

Secondary

Absolute Change in Fecal Elastase-1 (FE-1) Levels

Study 116 FAS.

Posted

Mean

Standard Deviation

microgram per gram (mcg/g)

From Parent Study 115B Baseline at Week 96

ID	Title	Description
OG000	LUM/IVA	LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.

Units	Counts
Participants	OG00057

Secondary

Absolute Change in Immunoreactive Trypsinogen (IRT) Serum Levels

Study 116 FAS.

Posted

Mean

Standard Deviation

nanogram per milliliter (ng/mL)

From Parent Study 115B Baseline at Week 96

ID	Title	Description
OG000	LUM/IVA	LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.

Units	Counts
Participants	OG00057

Secondary

Number of Participants With Microbiology Culture Status (Positive or Negative)

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.

Study 116 FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome at Week 96.

Posted

Number

participants

Week 96

ID	Title	Description
OG000	LUM/IVA	LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.

Units	Counts
Participants	OG000

Secondary

Absolute Change in Lung Clearance Index (LCI) 2.5

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.

Study 116 LCI substudy set included all participants who signed the informed consent/assent to the optional LCI substudy and were enrolled and exposed to any amount of study drug in current study 116.

Posted

Mean

Standard Deviation

lung clearance index

From Parent Study 115B Baseline at Week 96

ID	Title	Description
OG000	LUM/IVA	LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.

Units	Counts
Participants	OG000

Secondary

Absolute Change in Lung Clearance Index (LCI) 5.0

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.

Study 116 LCI substudy set.

Posted

Mean

Standard Deviation

lung clearance index

From Parent Study 115B Baseline at Week 96

ID	Title	Description
OG000	LUM/IVA	LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.

Units	Counts
Participants	OG000

Arm/Groups

ID	Title	Description	Deaths (Affected)	Deaths (At Risk)	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	LUM/IVA	LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.	0	57	15	57	55	57

Serious Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Constipation	Gastrointestinal disorders	Meddra 22.0	Systematic Assessment	EG0001 affected57 at risk
Haematemesis	Gastrointestinal disorders	Meddra 22.0	Systematic Assessment	EG0001 affected57 at risk
Pancreatitis	Gastrointestinal disorders	Meddra 22.0	Systematic Assessment	EG0001 affected57 at risk
Appendicitis	Infections and infestations	Meddra 22.0	Systematic Assessment	EG0001 affected57 at risk
Chronic sinusitis	Infections and infestations	Meddra 22.0	Systematic Assessment	EG0001 affected57 at risk
Gastritis viral	Infections and infestations	Meddra 22.0	Systematic Assessment	EG0001 affected57 at risk
Gastroenteritis adenovirus	Infections and infestations	Meddra 22.0	Systematic Assessment	EG0001 affected57 at risk
Gastroenteritis viral	Infections and infestations	Meddra 22.0	Systematic Assessment	EG0001 affected57 at risk
Infective pulmonary exacerbation of cystic fibrosis	Infections and infestations	Meddra 22.0	Systematic Assessment	EG0006 affected57 at risk
Pneumonia	Infections and infestations	Meddra 22.0	Systematic Assessment	EG0002 affected57 at risk
Respiratory tract infection viral	Infections and infestations	Meddra 22.0	Systematic Assessment	EG0001 affected57 at risk
Alanine aminotransferase increased	Investigations	Meddra 22.0	Systematic Assessment	EG0001 affected57 at risk
Aspartate aminotransferase increased	Investigations	Meddra 22.0	Systematic Assessment	EG0001 affected57 at risk
Weight gain poor	Metabolism and nutrition disorders	Meddra 22.0	Systematic Assessment	EG0001 affected57 at risk
Sleep apnoea syndrome	Respiratory, thoracic and mediastinal disorders	Meddra 22.0	Systematic Assessment	EG0001 affected57 at risk

Other Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Ear pain	Ear and labyrinth disorders	MedDRA 22.0	Systematic Assessment	EG0003 affected57 at risk
Abdominal pain	Gastrointestinal disorders	MedDRA 22.0	Systematic Assessment	EG0007 affected57 at risk
Abdominal pain upper	Gastrointestinal disorders	MedDRA 22.0	Systematic Assessment	EG0004 affected57 at risk
Constipation	Gastrointestinal disorders	MedDRA 22.0	Systematic Assessment	EG0007 affected57 at risk
Diarrhoea	Gastrointestinal disorders	MedDRA 22.0	Systematic Assessment	EG0006 affected57 at risk
Vomiting	Gastrointestinal disorders	MedDRA 22.0	Systematic Assessment	EG00017 affected57 at risk
Fatigue	General disorders	MedDRA 22.0	Systematic Assessment	EG0005 affected57 at risk
Pyrexia	General disorders	MedDRA 22.0	Systematic Assessment	EG00023 affected57 at risk
Vessel puncture site pain	General disorders	MedDRA 22.0	Systematic Assessment	EG0004 affected57 at risk
Conjunctivitis	Infections and infestations	MedDRA 22.0	Systematic Assessment	EG0004 affected57 at risk
Ear infection	Infections and infestations	MedDRA 22.0	Systematic Assessment	EG00012 affected57 at risk
Hand-foot-and-mouth disease	Infections and infestations	MedDRA 22.0	Systematic Assessment	EG0003 affected57 at risk
Infective pulmonary exacerbation of cystic fibrosis	Infections and infestations	MedDRA 22.0	Systematic Assessment	EG00010 affected57 at risk
Influenza	Infections and infestations	MedDRA 22.0	Systematic Assessment	EG0004 affected57 at risk
Nasopharyngitis	Infections and infestations	MedDRA 22.0	Systematic Assessment	EG0008 affected57 at risk
Otitis media	Infections and infestations	MedDRA 22.0	Systematic Assessment	EG0007 affected57 at risk
Pharyngitis streptococcal	Infections and infestations	MedDRA 22.0	Systematic Assessment	EG0006 affected57 at risk
Pneumonia	Infections and infestations	MedDRA 22.0	Systematic Assessment	EG0003 affected57 at risk
Sinusitis	Infections and infestations	MedDRA 22.0	Systematic Assessment	EG00012 affected57 at risk
Upper respiratory tract infection	Infections and infestations	MedDRA 22.0	Systematic Assessment	EG00013 affected57 at risk
Viral upper respiratory tract infection	Infections and infestations	MedDRA 22.0	Systematic Assessment	EG0004 affected57 at risk
Alanine aminotransferase increased	Investigations	MedDRA 22.0	Systematic Assessment	EG00010 affected57 at risk
Aspartate aminotransferase increased	Investigations	MedDRA 22.0	Systematic Assessment	EG0005 affected57 at risk
Forced expiratory volume decreased	Investigations	MedDRA 22.0	Systematic Assessment	EG0003 affected57 at risk
Gamma-glutamyltransferase increased	Investigations	MedDRA 22.0	Systematic Assessment	EG0004 affected57 at risk
Pseudomonas test positive	Investigations	MedDRA 22.0	Systematic Assessment	EG0009 affected57 at risk
Staphylococcus test positive	Investigations	MedDRA 22.0	Systematic Assessment	EG00012 affected57 at risk
Decreased appetite	Metabolism and nutrition disorders	MedDRA 22.0	Systematic Assessment	EG0006 affected57 at risk
Cough	Respiratory, thoracic and mediastinal disorders	MedDRA 22.0	Systematic Assessment	EG00047 affected57 at risk
Dyspnoea	Respiratory, thoracic and mediastinal disorders	MedDRA 22.0	Systematic Assessment	EG0003 affected57 at risk
Lower respiratory tract congestion	Respiratory, thoracic and mediastinal disorders	MedDRA 22.0	Systematic Assessment	EG0004 affected57 at risk
Nasal congestion	Respiratory, thoracic and mediastinal disorders	MedDRA 22.0	Systematic Assessment	EG00025 affected57 at risk
Nasal discharge discolouration	Respiratory, thoracic and mediastinal disorders	MedDRA 22.0	Systematic Assessment	EG0003 affected57 at risk
Oropharyngeal pain	Respiratory, thoracic and mediastinal disorders	MedDRA 22.0	Systematic Assessment	EG00010 affected57 at risk
Productive cough	Respiratory, thoracic and mediastinal disorders	MedDRA 22.0	Systematic Assessment	EG0005 affected57 at risk
Rhinorrhoea	Respiratory, thoracic and mediastinal disorders	MedDRA 22.0	Systematic Assessment	EG00018 affected57 at risk
Sinus congestion	Respiratory, thoracic and mediastinal disorders	MedDRA 22.0	Systematic Assessment	EG0003 affected57 at risk
Sputum increased	Respiratory, thoracic and mediastinal disorders	MedDRA 22.0	Systematic Assessment	EG0005 affected57 at risk
Upper respiratory tract congestion	Respiratory, thoracic and mediastinal disorders	MedDRA 22.0	Systematic Assessment	EG0003 affected57 at risk
Wheezing	Respiratory, thoracic and mediastinal disorders	MedDRA 22.0	Systematic Assessment	EG0003 affected57 at risk
Rash	Skin and subcutaneous tissue disorders	MedDRA 22.0	Systematic Assessment	EG0003 affected57 at risk

ID	Term
D003550	Cystic Fibrosis

Ancestor Terms

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

ID	Term
C000599212	lumacaftor, ivacaftor drug combination

Title

Denominators

Categories

Title	Measurements
	OG0006.0± 2.0

Title

Denominators

Categories

Title	Measurements
	OG00016.1± 2.3

Title

Denominators

Categories

Title	Measurements
	OG00028

Title

Denominators

Categories

Title	Measurements
	OG000132.6± 174.2