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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT04353817




Registration number
NCT04353817
Ethics application status
Date submitted
16/04/2020
Date registered
20/04/2020
Date last updated
8/02/2021

Titles & IDs
Public title
A Study Evaluating Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis and F/MF Genotypes
Scientific title
A Phase 3b, Randomized, Placebo-controlled Study Evaluating the Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects 6 Through 11 Years of Age Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
Secondary ID [1] 0 0
2019-003554-86
Secondary ID [2] 0 0
VX19-445-116
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Cystic fibrosis
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Inflammatory and Immune System 0 0 0 0
Connective tissue diseases
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - ELX/TEZ/IVA
Treatment: Drugs - IVA
Other interventions - Placebo
Other interventions - Placebo

Experimental: ELX/TEZ/IVA - Subjects will receive ELX/TEZ/IVA in the morning and IVA in the evening.

Placebo Comparator: Placebo - Subjects will receive placebo matched to ELX/TEZ/IVA in the morning and placebo matched to IVA in the evening.


Treatment: Drugs: ELX/TEZ/IVA
Fixed-dose combination (FDC) tablet.

Treatment: Drugs: IVA
Mono tablet.

Other interventions: Placebo
Placebo matched to ELX/TEZ/IVA.

Other interventions: Placebo
Placebo matched to IVA.

Intervention code [1] 0 0
Treatment: Drugs
Intervention code [2] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Absolute change in lung clearance index 2.5 (LCI2.5)
Timepoint [1] 0 0
From baseline through Week 24
Secondary outcome [1] 0 0
Absolute change in sweat chloride (SwCl)
Timepoint [1] 0 0
From baseline through Week 24
Secondary outcome [2] 0 0
Safety and tolerability as assessed by number of subjects with adverse events (AEs) and serious adverse events (SAEs)
Timepoint [2] 0 0
From Day 1 up to Week 28

Eligibility
Key inclusion criteria
Key

- Heterozygous for the F508del mutation (F/MF)

- Forced expiratory volume in 1 second (FEV1) value greater than equal to(=) 70%

Key
Minimum age
6 Years
Maximum age
11 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Clinically significant cirrhosis with or without portal hypertension

- Lung infection with organisms associated with a more rapid decline in pulmonary status

- Solid organ or hematological transplantation

Other protocol defined Inclusion/Exclusion criteria may apply

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Telethon Kids Institute - Nedlands
Recruitment hospital [2] 0 0
Queensland Children's Hospital - South Brisbane
Recruitment hospital [3] 0 0
The Children's Hospital at Westmead - Westmead
Recruitment postcode(s) [1] 0 0
- Nedlands
Recruitment postcode(s) [2] 0 0
- South Brisbane
Recruitment postcode(s) [3] 0 0
- Westmead
Recruitment outside Australia
Country [1] 0 0
Canada
State/province [1] 0 0
Montréal
Country [2] 0 0
Canada
State/province [2] 0 0
Toronto
Country [3] 0 0
Canada
State/province [3] 0 0
Vancouver
Country [4] 0 0
Denmark
State/province [4] 0 0
Copenhagen
Country [5] 0 0
France
State/province [5] 0 0
Bordeaux cedex
Country [6] 0 0
France
State/province [6] 0 0
Bron Cedex
Country [7] 0 0
France
State/province [7] 0 0
Paris Cedex 15
Country [8] 0 0
France
State/province [8] 0 0
Paris
Country [9] 0 0
France
State/province [9] 0 0
Roscoff cedex
Country [10] 0 0
Germany
State/province [10] 0 0
Berlin
Country [11] 0 0
Germany
State/province [11] 0 0
Essen
Country [12] 0 0
Germany
State/province [12] 0 0
Frankfurt
Country [13] 0 0
Germany
State/province [13] 0 0
Gießen
Country [14] 0 0
Germany
State/province [14] 0 0
Hannover
Country [15] 0 0
Germany
State/province [15] 0 0
Heidelberg
Country [16] 0 0
Germany
State/province [16] 0 0
Koeln
Country [17] 0 0
Israel
State/province [17] 0 0
Jerusalem
Country [18] 0 0
Israel
State/province [18] 0 0
Petah Tikva
Country [19] 0 0
Netherlands
State/province [19] 0 0
Groningen
Country [20] 0 0
Netherlands
State/province [20] 0 0
Rotterdam
Country [21] 0 0
Spain
State/province [21] 0 0
Barcelona
Country [22] 0 0
Spain
State/province [22] 0 0
Murcia
Country [23] 0 0
Switzerland
State/province [23] 0 0
Bern
Country [24] 0 0
Switzerland
State/province [24] 0 0
Zurich
Country [25] 0 0
United Kingdom
State/province [25] 0 0
Bristol
Country [26] 0 0
United Kingdom
State/province [26] 0 0
Cardiff
Country [27] 0 0
United Kingdom
State/province [27] 0 0
Edinburgh
Country [28] 0 0
United Kingdom
State/province [28] 0 0
Liverpool
Country [29] 0 0
United Kingdom
State/province [29] 0 0
London
Country [30] 0 0
United Kingdom
State/province [30] 0 0
Southampton

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Vertex Pharmaceuticals Incorporated
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This study will evaluate the efficacy and safety of elexacaftor (ELX) / tezacaftor (TEZ) /
ivacaftor (IVA) triple combination (TC) in subjects 6 through 11 years of age with cystic
fibrosis (CF) who are heterozygous for F508del and a minimal function (MF) mutation (F/MF
genotypes).
Trial website
https://clinicaltrials.gov/show/NCT04353817
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
Other publications