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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT04353817
Ethics application status
Date submitted
16/04/2020
Date registered
20/04/2020
Date last updated
26/07/2022

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 (matched to ELX/TEZ/IVA)
Other interventions - Placebo (matched to IVA)

Placebo comparator: Placebo - Participants received placebo matched to ELX/TEZ/IVA and placebo matched to IVA in the treatment period for 24 weeks.

Experimental: ELX/TEZ/IVA - Participants weighing less than (\<) 30 kilograms (kg) at screening received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg every 12 hours (q12h) and participants weighing greater than equals to (\>=) 30 kg at screening received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 24 weeks.


Treatment: Drugs: ELX/TEZ/IVA
Fixed-dose combination tablet for oral administration qd in the morning.

Treatment: Drugs: IVA
Tablet for oral administration qd in the evening.

Other interventions: Placebo (matched to ELX/TEZ/IVA)
Placebo matched to ELX/TEZ/IVA for oral administration once daily (qd) in the morning.

Other interventions: Placebo (matched to IVA)
Placebo matched to IVA for oral administration qd in the evening.
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 Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Timepoint [2] 0 0
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
Sex
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
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
19/06/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
17/05/2021
Sample size
Target
Accrual to date
Final
121
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/study/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

For IPD and results data, please see https://clinicaltrials.gov/study/NCT04353817