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The European Union Clinical Trials Register allows you to search for protocol and results information on:

interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC

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EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
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The EU Clinical Trials Register currently displays 43865 clinical trials with a EudraCT protocol, of which 7286 are clinical trials conducted with subjects less than 18 years old. The register also displays information on 18700 older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).

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Clinical Trial Results:
A Phase 3b Open-label Study to Assess the Effect of Elexacaftor/Tezacaftor/Ivacaftor on Glucose Tolerance in Cystic Fibrosis Subjects with Abnormal Glucose Metabolism

Summary
EudraCT number	2020-003170-44
Trial protocol	BE CZ FR NL IT
Global end of trial date	14 Jul 2022

Results information
Results version number	v1
This version publication date	29 Jan 2023
First version publication date	29 Jan 2023
Other versions	v2

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

VX19-445-117

ISRCTN number

-

US NCT number

NCT04599465

WHO universal trial number (UTN)

-

Sponsor organisation name

Vertex Pharmaceuticals Incorporated

Sponsor organisation address

50 Northern Avenue, Boston, Massachusetts, United States,

Public contact

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

Scientific contact

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

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

No

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

No

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

Yes

Analysis stage

Final

Date of interim/final analysis

19 Aug 2022

Is this the analysis of the primary completion data?

Yes

Primary completion date

14 Jul 2022

Global end of trial reached?

Yes

Global end of trial date

14 Jul 2022

Was the trial ended prematurely?

No

Main objective of the trial

To evaluate the effect of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) on glucose tolerance in Cystic Fibrosis (CF) subjects with impaired glucose tolerance (IGT) or CF-related diabetes (CFRD)

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

15 Jan 2021

Long term follow-up planned

No

Independent data monitoring committee (IDMC) involvement?

No

Number of subjects enrolled per country

Country: Number of subjects enrolled

Australia: 11

Country: Number of subjects enrolled

Netherlands: 9

Country: Number of subjects enrolled

Spain: 14

Country: Number of subjects enrolled

Belgium: 7

Country: Number of subjects enrolled

Czechia: 3

Country: Number of subjects enrolled

France: 13

Country: Number of subjects enrolled

Italy: 12

Worldwide total number of subjects

69

EEA total number of subjects

58

Number of subjects enrolled per age group

In utero

0

Preterm newborn - gestational age < 37 wk

0

Newborns (0-27 days)

0

Infants and toddlers (28 days-23 months)

0

Children (2-11 years)

0

Adolescents (12-17 years)

19

Adults (18-64 years)

50

From 65 to 84 years

0

85 years and over

0

Subject disposition

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Recruitment details

-

Screening details

This study was conducted in subjects with CF aged 12 years and older who are heterozygous for the F508del mutation and a minimal function mutation (F/MF genotypes), with abnormal glucose metabolism.

Period 1 title

Overall Period

Is this the baseline period?

Yes

Allocation method

Not applicable

Blinding used

Not blinded

ELX/TEZ/IVA

Arm description

Subjects received ELX/TEZ/IVA fixed dose combination (FDC) in the morning and ivacaftor (IVA) in the evening.

Arm type

Experimental

Investigational medicinal product name

ELX/TEZ/IVA

Investigational medicinal product code

VX-445/VX-661/VX-770

Other name

elexacaftor/tezacaftor/ivacaftor

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

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

Investigational medicinal product name

IVA

Investigational medicinal product code

VX-770

Other name

ivacaftor

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Subjects received IVA dose once daily in the evening.

Number of subjects in period 1	ELX/TEZ/IVA
Started	69
Completed	66
Not completed	3
Physician decision	1
Withdrawal of consent (not due to AE)	2

Baseline characteristics

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Reporting group title

Overall Period

Reporting group description

Subjects received ELX/TEZ/IVA fixed dose combination (FDC) in the morning and ivacaftor (IVA) in the evening.

Reporting group values	Overall Period	Total
Number of subjects	69	69
Age categorical
Units: Subjects
Less than (<)18 years	19	19
More than or equal to (≥)18 years	50	50
Age continuous
Units: years
arithmetic mean (standard deviation)	25.1 ( 9.5 )	-
Gender categorical
Units: Subjects
Female	31	31
Male	38	38

End points

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Reporting group title

ELX/TEZ/IVA

Reporting group description

Subjects received ELX/TEZ/IVA fixed dose combination (FDC) in the morning and ivacaftor (IVA) in the evening.

Primary: Change From Baseline in 2-hour Blood Glucose Levels Following an Oral Glucose Tolerance Test (OGTT) to the Average of Week 36 and Week 48

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End point title

Change From Baseline in 2-hour Blood Glucose Levels Following an Oral Glucose Tolerance Test (OGTT) to the Average of Week 36 and Week 48 ^[1]

End point description

End point type

Primary

End point timeframe

Baseline, Week 36 and 48

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: This is a single arm study, and thus no between-group comparisons were planned. However, participants’ post-baseline values were compared to their pre-treatment baseline values with a mixed model for repeated measures (MMRM) with change from baseline in 2-hour post-OGTT blood glucose levels at each post-baseline visit as the dependent variable. The primary result obtained from the model was the estimated mean change from baseline to the average of Week 36 and Week 48.

End point values	ELX/TEZ/IVA
Number of subjects analysed	66
Units: milligrams per deciliter (mg/dl)
least squares mean (confidence interval 95%)	-35.0 (-49.2 to -20.7)

No statistical analyses for this end point

Adverse events

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Timeframe for reporting adverse events

Day 1 up to Week 52

Assessment type

Systematic

Dictionary name

MedDRA

Dictionary version

25.0

Reporting group title

ELX/TEZ/IVA

Reporting group description

Subjects received ELX/TEZ/IVA fixed dose combination (FDC) in the morning and ivacaftor (IVA) in the evening.

Serious adverse events	ELX/TEZ/IVA
Total subjects affected by serious adverse events
subjects affected / exposed	6 / 69 (8.70%)
number of deaths (all causes)	0
number of deaths resulting from adverse events
Injury, poisoning and procedural complications
Clavicle fracture
subjects affected / exposed	1 / 69 (1.45%)
occurrences causally related to treatment / all	0 / 1
deaths causally related to treatment / all	0 / 0
Nervous system disorders
Headache
subjects affected / exposed	1 / 69 (1.45%)
occurrences causally related to treatment / all	0 / 1
deaths causally related to treatment / all	0 / 0
Gastrointestinal disorders
Gastritis
subjects affected / exposed	1 / 69 (1.45%)
occurrences causally related to treatment / all	0 / 1
deaths causally related to treatment / all	0 / 0
Reproductive system and breast disorders
Heavy menstrual bleeding
subjects affected / exposed	1 / 69 (1.45%)
occurrences causally related to treatment / all	0 / 1
deaths causally related to treatment / all	0 / 0
Respiratory, thoracic and mediastinal disorders
Pulmonary haemorrhage
subjects affected / exposed	1 / 69 (1.45%)
occurrences causally related to treatment / all	0 / 2
deaths causally related to treatment / all	0 / 0
Infections and infestations
Infective pulmonary exacerbation of cystic fibrosis
subjects affected / exposed	1 / 69 (1.45%)
occurrences causally related to treatment / all	0 / 1
deaths causally related to treatment / all	0 / 0
Influenza
subjects affected / exposed	1 / 69 (1.45%)
occurrences causally related to treatment / all	0 / 1
deaths causally related to treatment / all	0 / 0
Sinusitis
subjects affected / exposed	1 / 69 (1.45%)
occurrences causally related to treatment / all	0 / 1
deaths causally related to treatment / all	0 / 0

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

Non-serious adverse events	ELX/TEZ/IVA
Total subjects affected by non serious adverse events
subjects affected / exposed	62 / 69 (89.86%)
Investigations
Aspartate aminotransferase increased
subjects affected / exposed	5 / 69 (7.25%)
occurrences all number	5
Alanine aminotransferase increased
subjects affected / exposed	6 / 69 (8.70%)
occurrences all number	6
Blood bilirubin increased
subjects affected / exposed	7 / 69 (10.14%)
occurrences all number	7
Blood creatine phosphokinase increased
subjects affected / exposed	5 / 69 (7.25%)
occurrences all number	6
Nervous system disorders
Headache
subjects affected / exposed	16 / 69 (23.19%)
occurrences all number	22
General disorders and administration site conditions
Fatigue
subjects affected / exposed	5 / 69 (7.25%)
occurrences all number	5
Pyrexia
subjects affected / exposed	16 / 69 (23.19%)
occurrences all number	22
Immune system disorders
Immunisation reaction
subjects affected / exposed	6 / 69 (8.70%)
occurrences all number	9
Gastrointestinal disorders
Abdominal pain
subjects affected / exposed	9 / 69 (13.04%)
occurrences all number	13
Diarrhoea
subjects affected / exposed	13 / 69 (18.84%)
occurrences all number	16
Abdominal pain upper
subjects affected / exposed	8 / 69 (11.59%)
occurrences all number	9
Nausea
subjects affected / exposed	6 / 69 (8.70%)
occurrences all number	6
Vomiting
subjects affected / exposed	7 / 69 (10.14%)
occurrences all number	9
Respiratory, thoracic and mediastinal disorders
Cough
subjects affected / exposed	11 / 69 (15.94%)
occurrences all number	12
Dyspnoea
subjects affected / exposed	4 / 69 (5.80%)
occurrences all number	4
Oropharyngeal pain
subjects affected / exposed	8 / 69 (11.59%)
occurrences all number	9
Productive cough
subjects affected / exposed	5 / 69 (7.25%)
occurrences all number	7
Sputum increased
subjects affected / exposed	12 / 69 (17.39%)
occurrences all number	13
Skin and subcutaneous tissue disorders
Acne
subjects affected / exposed	4 / 69 (5.80%)
occurrences all number	4
Rash
subjects affected / exposed	7 / 69 (10.14%)
occurrences all number	10
Infections and infestations
COVID-19
subjects affected / exposed	22 / 69 (31.88%)
occurrences all number	22
Infective pulmonary exacerbation of cystic fibrosis
subjects affected / exposed	10 / 69 (14.49%)
occurrences all number	14
Influenza
subjects affected / exposed	6 / 69 (8.70%)
occurrences all number	6
Nasopharyngitis
subjects affected / exposed	15 / 69 (21.74%)
occurrences all number	17
Rhinitis
subjects affected / exposed	7 / 69 (10.14%)
occurrences all number	11
Tonsillitis
subjects affected / exposed	4 / 69 (5.80%)
occurrences all number	4
Upper respiratory tract infection
subjects affected / exposed	4 / 69 (5.80%)
occurrences all number	5
Metabolism and nutrition disorders
Hypoglycaemia
subjects affected / exposed	4 / 69 (5.80%)
occurrences all number	8

More information

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Were there any global substantial amendments to the protocol? Yes

22 Sep 2020

Removed option for use of remote measures at certain study visits such that those visits are to be performed in the clinic; clarified different glomerular filtration rates for subjects ≥18 years of age and subjects <18 years of age; clarified that during in-clinic visits, spirometry assessments may be performed on more than 1 spirometer, as applicable; removed sweat chloride assessment at Week 4.

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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The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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