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Clinical Trial Results:
A Phase 3b Open-label Extension Study Evaluating the Safety of Elexacaftor/Tezacaftor/Ivacaftor Combination Therapy in Cystic Fibrosis Subjects

Summary
EudraCT number	2019-003455-11
Trial protocol	GB DE BE
Global end of trial date	21 Dec 2022

Results information
Results version number	v1(current)
This version publication date	06 Jul 2023
First version publication date	06 Jul 2023
Other versions

Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information

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Trial information

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Sponsor protocol code

VX19-445-115

ISRCTN number

US NCT number

NCT04362761

WHO universal trial number (UTN)

Sponsor organisation name

Vertex Pharmaceuticals Incorporated

Sponsor organisation address

50 Northern Avenue , Boston, Massachusetts, United States,

Public contact

Medical Information, Vertex Pharmaceuticals Incorporated, +1 617-341-6777, medicalinfo@vrtx.com

Scientific contact

Medical Information, Vertex Pharmaceuticals Incorporated, +1 617-341-6777, medicalinfo@vrtx.com

Is trial part of an agreed paediatric investigation plan (PIP)

Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?

Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?

Yes

Analysis stage

Final

Date of interim/final analysis

18 Jan 2023

Is this the analysis of the primary completion data?

Yes

Primary completion date

21 Dec 2022

Global end of trial reached?

Yes

Global end of trial date

21 Dec 2022

Was the trial ended prematurely?

Main objective of the trial

To evaluate the safety and tolerability of elexacaftor (VX-445; ELX)/tezacaftor (TEZ)/ivacaftor (IVA)

Protection of trial subjects

The study was conducted in accordance with the ethical principles stated in the Declaration of Helsinki and the International Conference on Harmonization (ICH) Guideline for Good Clinical Practice (GCP).

Background therapy

Evidence for comparator

Actual start date of recruitment

04 May 2020

Long term follow-up planned

Independent data monitoring committee (IDMC) involvement?

Number of subjects enrolled per country

Country: Number of subjects enrolled

United Kingdom: 86

Country: Number of subjects enrolled

Belgium: 20

Country: Number of subjects enrolled

Germany: 36

Country: Number of subjects enrolled

Australia: 30

Worldwide total number of subjects

172

EEA total number of subjects

Number of subjects enrolled per age group

In utero

Preterm newborn - gestational age < 37 wk

Newborns (0-27 days)

Infants and toddlers (28 days-23 months)

Children (2-11 years)

Adolescents (12-17 years)

Adults (18-64 years)

121

From 65 to 84 years

85 years and over

Subject disposition

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Recruitment details

Screening details

A total 172 subjects were enrolled from the parent study VX18-445-109 (NCT04105972). The study was conducted in 2 parts, Part A and Part B.

Period 1 title

Part A

Is this the baseline period?

Yes

Allocation method

Not applicable

Blinding used

Not blinded

Part A: ELX/TEZ/IVA

Arm description

Subjects received ELX (elexacaftor) 200 milligram (mg) once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the treatment period for 48 weeks.

Arm type

Experimental

Investigational medicinal product name

Elexacaftor/Tezacaftor/Ivacaftor

Investigational medicinal product code

VX-445/VX-661/VX-770

Other name

ELX/TEZ/IVA

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Subjects received ELX/TEZ/IVA fixed dose combination once daily in the morning.

Investigational medicinal product name

Ivacaftor

Investigational medicinal product code

VX-770

Other name

IVA

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Subjects received IVA once daily in the evening.

Number of subjects in period 1	Part A: ELX/TEZ/IVA
Started	172
Completed	159
Not completed	13
Adverse Event	2
Other	5
Withdrawal of consent (not due to AE)	4
Commercial drug is available for subject	2

Period 2 title

Part B

Is this the baseline period?

Allocation method

Not applicable

Blinding used

Not blinded

Part B: ELX/TEZ/IVA

Arm description

Subjects received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period up to 86 weeks.

Arm type

Experimental

Investigational medicinal product name

Elexacaftor/Tezacaftor/Ivacaftor

Investigational medicinal product code

VX-445/VX-661/VX-770

Other name

ELX/TEZ/IVA

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Subjects received ELX/TEZ/IVA fixed dose combination once daily in the morning.

Investigational medicinal product name

Ivacaftor

Investigational medicinal product code

VX-770

Other name

IVA

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Subjects received IVA once daily in the evening.

Number of subjects in period 2 ^[1]	Part B: ELX/TEZ/IVA
Started	50
Completed	0
Not completed	50
Other	4
Commercial drug is available for subject	46

Notes
[1] - The number of subjects starting the period is not consistent with the number completing the preceding period. It is expected the number of subjects starting the subsequent period will be the same as the number completing the preceding period.
Justification: A total of 172 subjects were enrolled in the parent study on Part A. However, only 50 subjects rolled over to Part B from Part A of the study.

Baseline characteristics

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Reporting group title

Part A

Reporting group description

Subjects received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 48 weeks.

Reporting group values	Part A	Total
Number of subjects	172	172
Age categorical
Units: Subjects
Age continuous
Units: years
arithmetic mean (standard deviation)	27.9 ( 11.4 )	-
Gender categorical
Units: Subjects
Female	87	87
Male	85	85
Ethnicity
Units: Subjects
Hispanic or Latino	3	3
Not Hispanic or Latino	167	167
Not collected per local regulations	2	2
Race
Units: Subjects
White	169	169
Black or African American	0	0
Asian	2	2
American Indian or Alaska Native	0	0
Native Hawaiian or Other Pacific Islander	0	0
Not collected per local regulations	0	0
White, Asian	1	1

End points

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Reporting group title

Part A: ELX/TEZ/IVA

Reporting group description

Subjects received ELX (elexacaftor) 200 milligram (mg) once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the treatment period for 48 weeks.

Reporting group title

Part B: ELX/TEZ/IVA

Reporting group description

Subjects received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period up to 86 weeks.

Primary: Part A: Safety and Tolerability as Assessed by Number of Subjects With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

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End point title

Part A: Safety and Tolerability as Assessed by Number of Subjects With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) ^[1]

End point description

Safety set included all subjects who received at least 1 dose of study drug.

End point type

Primary

End point timeframe

From Day 1 up to Week 52

Notes
[1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: Only descriptive statistics were planned. No statistical comparisons were planned for this endpoint.

End point values	Part A: ELX/TEZ/IVA
Number of subjects analysed	172
Units: Subjects
Subjects with TEAEs	160
Subjects with SAEs	26

No statistical analyses for this end point

Primary: Part B: Safety and Tolerability as Assessed by Number of Subjects With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

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End point title

Part B: Safety and Tolerability as Assessed by Number of Subjects With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) ^[2]

End point description

Safety set included all subjects who received at least 1 dose of study drug.

End point type

Primary

End point timeframe

From Day 1 up to Week 86

Notes
[2] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: Only descriptive statistics were planned. No statistical comparisons were planned for this endpoint.

End point values	Part B: ELX/TEZ/IVA
Number of subjects analysed	50
Units: Subjects
Subjects with TEAEs	50
Subjects with SAEs	8

No statistical analyses for this end point

Adverse events

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Timeframe for reporting adverse events

Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B

Adverse event reporting additional description

MedDRA 24.0 applied for Part A and MedDRA 25.1 applied for Part B.

Assessment type

Systematic

Dictionary name

MedDRA

Dictionary version

24.0,25.1

Reporting group title

Part A: ELX/TEZ/IVA

Reporting group description

Subjects received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg qd in the treatment period for 48 weeks.

Reporting group title

Part B: ELX/TEZ/IVA

Reporting group description

Subjects received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg qd in the treatment period up to 86 weeks.

Serious adverse events	Part A: ELX/TEZ/IVA	Part B: ELX/TEZ/IVA
Total subjects affected by serious adverse events
subjects affected / exposed	26 / 172 (15.12%)	8 / 50 (16.00%)
number of deaths (all causes)	0	0
number of deaths resulting from adverse events
General disorders and administration site conditions
Vascular device occlusion
subjects affected / exposed	1 / 172 (0.58%)	0 / 50 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Reproductive system and breast disorders
Heavy menstrual bleeding
subjects affected / exposed	1 / 172 (0.58%)	0 / 50 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Uterine polyp
subjects affected / exposed	1 / 172 (0.58%)	0 / 50 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Respiratory, thoracic and mediastinal disorders
Haemoptysis
subjects affected / exposed	1 / 172 (0.58%)	0 / 50 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Nasal polyps
subjects affected / exposed	1 / 172 (0.58%)	0 / 50 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Productive cough
subjects affected / exposed	0 / 172 (0.00%)	1 / 50 (2.00%)
occurrences causally related to treatment / all	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0
Psychiatric disorders
Aggression
subjects affected / exposed	1 / 172 (0.58%)	0 / 50 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Dissociative disorder
subjects affected / exposed	1 / 172 (0.58%)	0 / 50 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Investigations
Blood bilirubin increased
subjects affected / exposed	1 / 172 (0.58%)	0 / 50 (0.00%)
occurrences causally related to treatment / all	1 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Aspartate aminotransferase increased
subjects affected / exposed	2 / 172 (1.16%)	0 / 50 (0.00%)
occurrences causally related to treatment / all	2 / 3	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Alanine aminotransferase increased
subjects affected / exposed	2 / 172 (1.16%)	0 / 50 (0.00%)
occurrences causally related to treatment / all	2 / 3	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Forced expiratory volume decreased
subjects affected / exposed	0 / 172 (0.00%)	1 / 50 (2.00%)
occurrences causally related to treatment / all	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0
Gamma-glutamyltransferase increased
subjects affected / exposed	1 / 172 (0.58%)	0 / 50 (0.00%)
occurrences causally related to treatment / all	2 / 2	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Injury, poisoning and procedural complications
Rib fracture
subjects affected / exposed	1 / 172 (0.58%)	0 / 50 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Limb traumatic amputation
subjects affected / exposed	0 / 172 (0.00%)	1 / 50 (2.00%)
occurrences causally related to treatment / all	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0
Cardiac disorders
Atrial fibrillation
subjects affected / exposed	1 / 172 (0.58%)	0 / 50 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Sinus arrest
subjects affected / exposed	1 / 172 (0.58%)	0 / 50 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Myocardial infarction
subjects affected / exposed	1 / 172 (0.58%)	0 / 50 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Nervous system disorders
Headache
subjects affected / exposed	1 / 172 (0.58%)	0 / 50 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Loss of consciousness
subjects affected / exposed	1 / 172 (0.58%)	0 / 50 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Seizure
subjects affected / exposed	1 / 172 (0.58%)	0 / 50 (0.00%)
occurrences causally related to treatment / all	0 / 2	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Gastrointestinal disorders
Ileus
subjects affected / exposed	1 / 172 (0.58%)	0 / 50 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Constipation
subjects affected / exposed	1 / 172 (0.58%)	0 / 50 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Hepatobiliary disorders
Cholecystitis
subjects affected / exposed	0 / 172 (0.00%)	1 / 50 (2.00%)
occurrences causally related to treatment / all	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0
Skin and subcutaneous tissue disorders
Erythema nodosum
subjects affected / exposed	1 / 172 (0.58%)	0 / 50 (0.00%)
occurrences causally related to treatment / all	1 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Renal and urinary disorders
Nephrolithiasis
subjects affected / exposed	1 / 172 (0.58%)	0 / 50 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Musculoskeletal and connective tissue disorders
Dupuytren's contracture
subjects affected / exposed	1 / 172 (0.58%)	0 / 50 (0.00%)
occurrences causally related to treatment / all	1 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Myalgia
subjects affected / exposed	1 / 172 (0.58%)	0 / 50 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Infections and infestations
Chronic sinusitis
subjects affected / exposed	1 / 172 (0.58%)	0 / 50 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Infective pulmonary exacerbation of cystic fibrosis
subjects affected / exposed	8 / 172 (4.65%)	5 / 50 (10.00%)
occurrences causally related to treatment / all	1 / 9	0 / 5
deaths causally related to treatment / all	0 / 0	0 / 0
Lower respiratory tract infection
subjects affected / exposed	1 / 172 (0.58%)	0 / 50 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Pneumonia
subjects affected / exposed	1 / 172 (0.58%)	0 / 50 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Pneumonia pseudomonal
subjects affected / exposed	1 / 172 (0.58%)	0 / 50 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Metabolism and nutrition disorders
Hyperglycaemia
subjects affected / exposed	1 / 172 (0.58%)	0 / 50 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Electrolyte imbalance
subjects affected / exposed	1 / 172 (0.58%)	0 / 50 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0

Frequency threshold for reporting non-serious adverse events: 5%

Non-serious adverse events	Part A: ELX/TEZ/IVA	Part B: ELX/TEZ/IVA
Total subjects affected by non serious adverse events
subjects affected / exposed	127 / 172 (73.84%)	47 / 50 (94.00%)
Investigations
Blood bilirubin increased
subjects affected / exposed	11 / 172 (6.40%)	1 / 50 (2.00%)
occurrences all number	12	1
Aspartate aminotransferase increased
subjects affected / exposed	11 / 172 (6.40%)	0 / 50 (0.00%)
occurrences all number	13	0
Alanine aminotransferase increased
subjects affected / exposed	9 / 172 (5.23%)	1 / 50 (2.00%)
occurrences all number	18	1
Injury, poisoning and procedural complications
Vaccination complication
subjects affected / exposed	24 / 172 (13.95%)	0 / 50 (0.00%)
occurrences all number	36	0
Procedural pain
subjects affected / exposed	2 / 172 (1.16%)	3 / 50 (6.00%)
occurrences all number	2	3
Nervous system disorders
Dizziness
subjects affected / exposed	11 / 172 (6.40%)	0 / 50 (0.00%)
occurrences all number	17	0
Headache
subjects affected / exposed	42 / 172 (24.42%)	10 / 50 (20.00%)
occurrences all number	73	18
General disorders and administration site conditions
Pyrexia
subjects affected / exposed	7 / 172 (4.07%)	8 / 50 (16.00%)
occurrences all number	9	8
Fatigue
subjects affected / exposed	13 / 172 (7.56%)	1 / 50 (2.00%)
occurrences all number	14	1
Malaise
subjects affected / exposed	3 / 172 (1.74%)	3 / 50 (6.00%)
occurrences all number	4	3
Immune system disorders
Immunisation reaction
subjects affected / exposed	0 / 172 (0.00%)	7 / 50 (14.00%)
occurrences all number	0	10
Gastrointestinal disorders
Abdominal pain upper
subjects affected / exposed	12 / 172 (6.98%)	2 / 50 (4.00%)
occurrences all number	15	3
Constipation
subjects affected / exposed	10 / 172 (5.81%)	4 / 50 (8.00%)
occurrences all number	11	5
Diarrhoea
subjects affected / exposed	13 / 172 (7.56%)	2 / 50 (4.00%)
occurrences all number	27	2
Gastrooesophageal reflux disease
subjects affected / exposed	8 / 172 (4.65%)	3 / 50 (6.00%)
occurrences all number	20	3
Abdominal pain
subjects affected / exposed	16 / 172 (9.30%)	3 / 50 (6.00%)
occurrences all number	32	4
Nausea
subjects affected / exposed	9 / 172 (5.23%)	1 / 50 (2.00%)
occurrences all number	13	1
Vomiting
subjects affected / exposed	8 / 172 (4.65%)	3 / 50 (6.00%)
occurrences all number	8	3
Respiratory, thoracic and mediastinal disorders
Sputum increased
subjects affected / exposed	19 / 172 (11.05%)	5 / 50 (10.00%)
occurrences all number	20	6
Productive cough
subjects affected / exposed	4 / 172 (2.33%)	6 / 50 (12.00%)
occurrences all number	7	10
Oropharyngeal pain
subjects affected / exposed	16 / 172 (9.30%)	5 / 50 (10.00%)
occurrences all number	19	7
Cough
subjects affected / exposed	23 / 172 (13.37%)	5 / 50 (10.00%)
occurrences all number	29	7
Skin and subcutaneous tissue disorders
Rash
subjects affected / exposed	12 / 172 (6.98%)	2 / 50 (4.00%)
occurrences all number	17	2
Musculoskeletal and connective tissue disorders
Arthralgia
subjects affected / exposed	12 / 172 (6.98%)	4 / 50 (8.00%)
occurrences all number	15	4
Infections and infestations
Infective pulmonary exacerbation of cystic fibrosis
subjects affected / exposed	17 / 172 (9.88%)	21 / 50 (42.00%)
occurrences all number	22	29
COVID-19
subjects affected / exposed	2 / 172 (1.16%)	17 / 50 (34.00%)
occurrences all number	2	17
Influenza
subjects affected / exposed	2 / 172 (1.16%)	3 / 50 (6.00%)
occurrences all number	2	3
Nasopharyngitis
subjects affected / exposed	28 / 172 (16.28%)	16 / 50 (32.00%)
occurrences all number	39	23
Upper respiratory tract infection
subjects affected / exposed	10 / 172 (5.81%)	11 / 50 (22.00%)
occurrences all number	13	15

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Were there any global substantial amendments to the protocol? Yes

04 Jan 2021

Amended to extend treatment period.

19 Mar 2021

Amended to allow subjects who depart this study to enroll in another qualified Vertex study to return to this study if they did not receive study drug in the Treatment Period of the other qualified Vertex study.

Were there any global interruptions to the trial? No

Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.

None reported

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Clinical trials

Clinical Trial Results:
A Phase 3b Open-label Extension Study Evaluating the Safety of Elexacaftor/Tezacaftor/Ivacaftor Combination Therapy in Cystic Fibrosis Subjects

Trial information

Trial information

Subject disposition

Subject disposition

Baseline characteristics

Baseline characteristics

End points

End points

Primary: Part A: Safety and Tolerability as Assessed by Number of Subjects With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

Primary: Part A: Safety and Tolerability as Assessed by Number of Subjects With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

Primary: Part B: Safety and Tolerability as Assessed by Number of Subjects With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

Primary: Part B: Safety and Tolerability as Assessed by Number of Subjects With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

Adverse events

Adverse events

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Substantial protocol amendments (globally)

Interruptions (globally)

Limitations and caveats

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Clinical trials

Clinical Trial Results: A Phase 3b Open-label Extension Study Evaluating the Safety of Elexacaftor/Tezacaftor/Ivacaftor Combination Therapy in Cystic Fibrosis Subjects

Trial information

Trial information

Subject disposition

Subject disposition

Baseline characteristics

Baseline characteristics

End points

End points

Primary: Part A: Safety and Tolerability as Assessed by Number of Subjects With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

Primary: Part A: Safety and Tolerability as Assessed by Number of Subjects With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

Primary: Part B: Safety and Tolerability as Assessed by Number of Subjects With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

Primary: Part B: Safety and Tolerability as Assessed by Number of Subjects With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

Adverse events

Adverse events

More information

Substantial protocol amendments (globally)

Interruptions (globally)

Limitations and caveats

More information

Clinical Trial Results:
A Phase 3b Open-label Extension Study Evaluating the Safety of Elexacaftor/Tezacaftor/Ivacaftor Combination Therapy in Cystic Fibrosis Subjects