Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
A database of clinical trials and their results from Australia, New Zealand, and other countries.
account_circle
Log in
to register or update your trial
search
Search for trials
Trial Review
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
Download to PDF
Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT04183790
Registration number
NCT04183790
Ethics application status
Date submitted
28/11/2019
Date registered
3/12/2019
Date last updated
18/05/2025
Titles & IDs
Public title
Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA TC Combination Therapy in Participants With Cystic Fibrosis Who Are 6 Years of Age and Older
Query!
Scientific title
A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of ELX/TEZ/IVA Combination Therapy in Subjects With Cystic Fibrosis Who Are 6 Years of Age and Older
Query!
Secondary ID [1]
0
0
2019-001827-11
Query!
Secondary ID [2]
0
0
VX19-445-107
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis
0
0
Query!
Condition category
Condition code
Human Genetics and Inherited Disorders
0
0
0
0
Query!
Cystic fibrosis
Query!
Respiratory
0
0
0
0
Query!
Other respiratory disorders / diseases
Query!
Oral and Gastrointestinal
0
0
0
0
Query!
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Query!
Inflammatory and Immune System
0
0
0
0
Query!
Connective tissue diseases
Query!
Inflammatory and Immune System
0
0
0
0
Query!
Other inflammatory or immune system disorders
Query!
Intervention/exposure
Study type
Interventional(has expanded access)
Query!
Description of intervention(s) / exposure
Treatment: Drugs - ELX/TEZ/IVA
Treatment: Drugs - IVA
Experimental: ELX/TEZ/IVA - Participants greater than or equal to (=) 6 years and less than (\<) 12 years of age and weighing \<30 kilograms (kg) received ELX (elexacaftor) 100 milligram (mg) once daily (qd) /TEZ (tezacaftor) 50 mg qd/IVA (ivacaftor) 75 mg every 12 hours (q12h) and those weighing (=) 30 kg received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg in the treatment period for up to 192 weeks. Participants =12 years of age received ELX 200 mg qd/ TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for up to 192 weeks.
Treatment: Drugs: ELX/TEZ/IVA
Fixed-dose combination (FDC) tablet for oral administration
Treatment: Drugs: IVA
Mono tablet for oral administration.
Query!
Intervention code [1]
0
0
Treatment: Drugs
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Query!
Assessment method [1]
0
0
Query!
Timepoint [1]
0
0
From Baseline up to Week 196
Query!
Secondary outcome [1]
0
0
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
Query!
Assessment method [1]
0
0
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Query!
Timepoint [1]
0
0
From Baseline up to Week 192
Query!
Secondary outcome [2]
0
0
Absolute Change in Sweat Chloride (SwCl)
Query!
Assessment method [2]
0
0
Sweat samples were collected using an approved collection device.
Query!
Timepoint [2]
0
0
From Baseline up to Week 192
Query!
Secondary outcome [3]
0
0
Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score
Query!
Assessment method [3]
0
0
The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
Query!
Timepoint [3]
0
0
From Baseline up to Week 192
Query!
Secondary outcome [4]
0
0
Absolute Change in Body Mass Index (BMI)
Query!
Assessment method [4]
0
0
BMI was defined as weight in kg divided by squared height in meters (m\^2).
Query!
Timepoint [4]
0
0
From Baseline up to Week 192
Query!
Secondary outcome [5]
0
0
Absolute Change in BMI-for-age Z-score
Query!
Assessment method [5]
0
0
BMI was defined as weight in kg divided by squared height in meters (m\^2). The z-score is a statistical measure to describe whether a mean was above or below the standard. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean.
Query!
Timepoint [5]
0
0
From Baseline up to Week 192
Query!
Secondary outcome [6]
0
0
Number of Participants With Pulmonary Exacerbations (PEx) for 106/107
Query!
Assessment method [6]
0
0
Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms.
Query!
Timepoint [6]
0
0
From Baseline up to Week 192
Query!
Secondary outcome [7]
0
0
Number of CF-related Hospitalizations for 106/107
Query!
Assessment method [7]
0
0
The total number of CF related hospitalization (Planned + Unplanned) events across all participants were reported.
Query!
Timepoint [7]
0
0
From Baseline up to Week 192
Query!
Secondary outcome [8]
0
0
Absolute Change in Lung Clearance Index 2.5 (LCI 2.5)
Query!
Assessment method [8]
0
0
The LCI2.5 index is the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting values and is calculated by dividing the sum of exhaled tidal breaths (cumulative exhaled volume (CEV)) by simultaneously measured functional residual capacity (FRC). An LCI of 7.5 and below is normal; values greater than 7.5 are abnormal. LCI is able to detect abnormalities in lung function earlier than more traditional modalities such as spirometry.
Query!
Timepoint [8]
0
0
From Baseline up to Week 192
Query!
Secondary outcome [9]
0
0
Absolute Change in Weight
Query!
Assessment method [9]
0
0
Query!
Timepoint [9]
0
0
From Baseline up to Week 192
Query!
Secondary outcome [10]
0
0
Absolute Change in Weight-for-age Z-score
Query!
Assessment method [10]
0
0
The z-score is a statistical measure to describe whether a mean was above or below the standard. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean.
Query!
Timepoint [10]
0
0
From Baseline up to Week 192
Query!
Secondary outcome [11]
0
0
Absolute Change in Height
Query!
Assessment method [11]
0
0
Query!
Timepoint [11]
0
0
From Baseline up to Week 192
Query!
Secondary outcome [12]
0
0
Absolute Change in Height-for-age Z-score
Query!
Assessment method [12]
0
0
The z-score is a statistical measure to describe whether a mean was above or below the standard. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean.
Query!
Timepoint [12]
0
0
From Baseline up to Week 192
Query!
Eligibility
Key inclusion criteria
Key
* Completed study drug treatment in parent study (VX18-445-106 Part B, NCT03691779), 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
Query!
Minimum age
6
Years
Query!
Query!
Maximum age
No limit
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
No
Query!
Key exclusion criteria
* History of study drug intolerance in parent study
Other protocol defined Inclusion/Exclusion criteria may apply.
Query!
Study design
Purpose of the study
Treatment
Query!
Allocation to intervention
Not applicable
Query!
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Query!
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Query!
Masking / blinding
Open (masking not used)
Query!
Who is / are masked / blinded?
Query!
Query!
Query!
Query!
Intervention assignment
Single group
Query!
Other design features
Query!
Phase
Phase 3
Query!
Type of endpoint/s
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Completed
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
17/02/2020
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
Query!
Actual
24/02/2024
Query!
Sample size
Target
Query!
Accrual to date
Query!
Final
64
Query!
Recruitment in Australia
Recruitment state(s)
Query!
Recruitment hospital [1]
0
0
Queensland Children's Hospital - South Brisbane
Query!
Recruitment hospital [2]
0
0
The Children's Hospital at Westmead - Westmead
Query!
Recruitment postcode(s) [1]
0
0
- South Brisbane
Query!
Recruitment postcode(s) [2]
0
0
- Westmead
Query!
Recruitment outside Australia
Country [1]
0
0
United States of America
Query!
State/province [1]
0
0
California
Query!
Country [2]
0
0
United States of America
Query!
State/province [2]
0
0
Colorado
Query!
Country [3]
0
0
United States of America
Query!
State/province [3]
0
0
Illinois
Query!
Country [4]
0
0
United States of America
Query!
State/province [4]
0
0
Massachusetts
Query!
Country [5]
0
0
United States of America
Query!
State/province [5]
0
0
Minnesota
Query!
Country [6]
0
0
United States of America
Query!
State/province [6]
0
0
Missouri
Query!
Country [7]
0
0
United States of America
Query!
State/province [7]
0
0
New York
Query!
Country [8]
0
0
United States of America
Query!
State/province [8]
0
0
North Carolina
Query!
Country [9]
0
0
United States of America
Query!
State/province [9]
0
0
Ohio
Query!
Country [10]
0
0
United States of America
Query!
State/province [10]
0
0
Oregon
Query!
Country [11]
0
0
United States of America
Query!
State/province [11]
0
0
Texas
Query!
Country [12]
0
0
United States of America
Query!
State/province [12]
0
0
Washington
Query!
Country [13]
0
0
Canada
Query!
State/province [13]
0
0
Toronto
Query!
Country [14]
0
0
Canada
Query!
State/province [14]
0
0
Vancouver
Query!
Country [15]
0
0
Ireland
Query!
State/province [15]
0
0
Dublin
Query!
Country [16]
0
0
United Kingdom
Query!
State/province [16]
0
0
Birmingham
Query!
Country [17]
0
0
United Kingdom
Query!
State/province [17]
0
0
London
Query!
Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Query!
Name
Vertex Pharmaceuticals Incorporated
Query!
Address
Query!
Country
Query!
Ethics approval
Ethics application status
Query!
Summary
Brief summary
The study evaluates the long-term safety, tolerability, efficacy, and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in participants with cystic fibrosis (CF).
Query!
Trial website
https://clinicaltrials.gov/study/NCT04183790
Query!
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.
Query!
Public notes
Query!
Contacts
Principal investigator
Name
0
0
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
Query!
No/undecided IPD sharing reason/comment
Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent research/clinical-trial-data-sharing
Query!
What supporting documents are/will be available?
No Supporting Document Provided
Type
Other Details
Attachment
Study protocol
https://cdn.clinicaltrials.gov/large-docs/90/NCT04183790/Prot_000.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/90/NCT04183790/SAP_001.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT04183790
Download to PDF