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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04545515




Registration number
NCT04545515
Ethics application status
Date submitted
3/09/2020
Date registered
11/09/2020
Date last updated
8/05/2024

Titles & IDs
Public title
A Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis (CF) Particpants 6 Years and Older and F/MF Genotypes
Scientific title
A Phase 3b Open-label Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor Combination Therapy in Cystic Fibrosis Subjects Ages 6 Years and Older Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
Secondary ID [1] 0 0
2020-001404-42
Secondary ID [2] 0 0
VX20-445-119
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

Experimental: ELX/TEZ/IVA - Participants 6 to less than \<12 year of age and weighing \<30 kilogram (kg) at Day 1 received ELX 100 milligram (mg)/TEZ 50 mg /IVA 75 mg as fixed dose combination (FDC) tablets in the morning and IVA as mono tablet in the evening and those weighing more than or equal to (=) 30 kg at Day 1 received ELX 200 mg/TEZ 100 mg /IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 96 weeks. Doses were adjusted upward with subsequent changes in weight. Participants =12 years age at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 96 weeks.


Treatment: Drugs: ELX/TEZ/IVA
Fixed dose combination (FDC) tablets for oral administration.

Treatment: Drugs: IVA
Tablet for oral administration.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Timepoint [1] 0 0
From Baseline up to Week 100
Secondary outcome [1] 0 0
Absolute Change From Parent Study Baseline in Sweat Chloride (SwCl)
Timepoint [1] 0 0
From Parent Study Baseline to Week 96
Secondary outcome [2] 0 0
Absolute Change From Parent Study Baseline in Lung Clearance Index 2.5 (LCI2.5)
Timepoint [2] 0 0
From Parent Study Baseline to Week 96

Eligibility
Key inclusion criteria
Key

* Completed study drug treatment in parent study (VX19-445-116, NCT04353817), or had study drug interruption(s) in parent study but completed study visits up to the last scheduled visit of the treatment period in the parent study

Key
Minimum age
6 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* History of study drug intolerance in the parent study

Other protocol defined Inclusion/Exclusion criteria may apply

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
11/01/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
24/03/2023
Sample size
Target
Accrual to date
Final
120
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
Montreal
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
Petach Tikvah
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
The study evaluates the long-term safety and efficacy of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) triple combination (TC) in participants with CF who are 6 years of age and older with F/MF genotypes.
Trial website
https://clinicaltrials.gov/study/NCT04545515
Trial related presentations / publications
Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3.
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

For IPD and results data, please see https://clinicaltrials.gov/study/NCT04545515