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.
LOGIN
CREATE ACCOUNT
LOGIN
CREATE ACCOUNT
MY TRIALS
REGISTER TRIAL
FAQs
HINTS AND TIPS
DEFINITIONS
Trial Review
The ANZCTR website will be unavailable from 1pm until 3pm (AEDT) on Wednesday the 30th of October for website maintenance. Please be sure to log out of the system in order to avoid any loss of data.
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/NCT03525444
Registration number
NCT03525444
Ethics application status
Date submitted
2/05/2018
Date registered
15/05/2018
Date last updated
19/05/2020
Titles & IDs
Public title
A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
Query!
Scientific title
A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
Query!
Secondary ID [1]
0
0
2018-000183-28
Query!
Secondary ID [2]
0
0
VX17-445-102
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
Query!
Description of intervention(s) / exposure
Treatment: Drugs - VX-445/TEZ/IVA
Treatment: Drugs - IVA
Treatment: Drugs - Placebo
Placebo comparator: Placebo - Participants who received placebo matched to VX-445/TEZ/IVA for 24 weeks in the TC treatment period.
Experimental: VX-445/TEZ/IVA TC - Participants who received VX-445 200 mg/TEZ 100 mg/IVA150 mg as fixed-dose combination (FDC) tablets in the morning and IVA 150 mg as mono tablet in the evening for 24 weeks in the TC treatment period.
Treatment: Drugs: VX-445/TEZ/IVA
Participants received VX-445/TEZ/IVA orally once daily in the morning.
Treatment: Drugs: IVA
Participants received IVA orally once daily in the evening
Treatment: Drugs: Placebo
Participants received placebo matched VX-445/TEZ/IVA orally once daily in the morning and placebo matched to IVA orally once daily in the evening.
Query!
Intervention code [1]
0
0
Treatment: Drugs
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary 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 at Week 4
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 through Week 24
Query!
Secondary outcome [2]
0
0
Number of Pulmonary Exacerbations (PEx)
Query!
Assessment method [2]
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 [2]
0
0
From Baseline through Week 24
Query!
Secondary outcome [3]
0
0
Absolute Change in Sweat Chloride (SwCl)
Query!
Assessment method [3]
0
0
Sweat samples were collected using an approved collection device.
Query!
Timepoint [3]
0
0
From Baseline through Week 24
Query!
Secondary outcome [4]
0
0
Absolute Change in Cystic Fibrosis Questionnaire Revised (CFQ-R) Respiratory Domain Score
Query!
Assessment method [4]
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 [4]
0
0
From Baseline through Week 24
Query!
Secondary outcome [5]
0
0
Absolute Change in Body Mass Index (BMI)
Query!
Assessment method [5]
0
0
BMI was defined as weight in kilogram (kg) divided by height in square meter (m\^2).
Query!
Timepoint [5]
0
0
From Baseline at Week 24
Query!
Secondary outcome [6]
0
0
Absolute Change in Sweat Chloride
Query!
Assessment method [6]
0
0
Sweat samples were collected using an approved collection device.
Query!
Timepoint [6]
0
0
From Baseline at Week 4
Query!
Secondary outcome [7]
0
0
Absolute Change in Cystic Fibrosis Questionnaire Revised (CFQ-R) Respiratory Domain Score
Query!
Assessment method [7]
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 [7]
0
0
From Baseline at Week 4
Query!
Secondary outcome [8]
0
0
Time-to-first Pulmonary Exacerbation (PEx)
Query!
Assessment method [8]
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 [8]
0
0
From Baseline through Week 24
Query!
Secondary outcome [9]
0
0
Absolute Change in BMI Z-score for Participants <=20 Years of Age at Baseline
Query!
Assessment method [9]
0
0
BMI was defined as weight in kg divided by height in m\^2. Z-score is a statistical measure to describe whether a mean was above or below the standard. BMI, adjusted for age and sex, was analyzed as BMI-for-age z-score. 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. Higher values are indicative of higher BMI.
Query!
Timepoint [9]
0
0
From Baseline at Week 24
Query!
Secondary outcome [10]
0
0
Absolute Change in Body Weight
Query!
Assessment method [10]
0
0
Query!
Timepoint [10]
0
0
From Baseline at Week 24
Query!
Secondary outcome [11]
0
0
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Query!
Assessment method [11]
0
0
Query!
Timepoint [11]
0
0
From first dose of study drug in TC treatment period up to 28 days after last dose of study drug or to the completion of study participation date, whichever occurs first (up to 28 weeks)
Query!
Secondary outcome [12]
0
0
Observed Pre-dose Concentration (Ctrough) of VX-445, TEZ, M1-TEZ, and IVA
Query!
Assessment method [12]
0
0
Query!
Timepoint [12]
0
0
Pre-dose on Week 4, 8, 12, and 16
Query!
Eligibility
Key inclusion criteria
Key
* Heterozygous for the F508del mutation (F/MF)
* Forced expiratory volume in 1 second (FEV1) value =40% and =90% of predicted mean for age, sex, and height
Key
Query!
Minimum age
12
Years
Query!
Query!
Maximum age
No limit
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
No
Query!
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
Query!
Study design
Purpose of the study
Treatment
Query!
Allocation to intervention
Randomised controlled trial
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
Blinded (masking used)
Query!
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
Query!
Query!
Query!
Query!
Intervention assignment
Parallel
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
15/06/2018
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
Query!
Actual
24/04/2019
Query!
Sample size
Target
Query!
Accrual to date
Query!
Final
405
Query!
Recruitment in Australia
Recruitment state(s)
Query!
Recruitment hospital [1]
0
0
Women & Children's Hospital - North Adelaide
Query!
Recruitment hospital [2]
0
0
The Royal Children's Hospital - Parkville
Query!
Recruitment hospital [3]
0
0
Mater Adult Hospital - South Brisbane
Query!
Recruitment hospital [4]
0
0
The Children's Hospital at Westmead - Westmead
Query!
Recruitment hospital [5]
0
0
Westmead Hospital - Westmead
Query!
Recruitment postcode(s) [1]
0
0
- North Adelaide
Query!
Recruitment postcode(s) [2]
0
0
- Parkville
Query!
Recruitment postcode(s) [3]
0
0
- South Brisbane
Query!
Recruitment postcode(s) [4]
0
0
- Westmead
Query!
Recruitment outside Australia
Country [1]
0
0
United States of America
Query!
State/province [1]
0
0
Arizona
Query!
Country [2]
0
0
United States of America
Query!
State/province [2]
0
0
Arkansas
Query!
Country [3]
0
0
United States of America
Query!
State/province [3]
0
0
California
Query!
Country [4]
0
0
United States of America
Query!
State/province [4]
0
0
Colorado
Query!
Country [5]
0
0
United States of America
Query!
State/province [5]
0
0
Florida
Query!
Country [6]
0
0
United States of America
Query!
State/province [6]
0
0
Georgia
Query!
Country [7]
0
0
United States of America
Query!
State/province [7]
0
0
Illinois
Query!
Country [8]
0
0
United States of America
Query!
State/province [8]
0
0
Kansas
Query!
Country [9]
0
0
United States of America
Query!
State/province [9]
0
0
Louisiana
Query!
Country [10]
0
0
United States of America
Query!
State/province [10]
0
0
Maine
Query!
Country [11]
0
0
United States of America
Query!
State/province [11]
0
0
Massachusetts
Query!
Country [12]
0
0
United States of America
Query!
State/province [12]
0
0
Michigan
Query!
Country [13]
0
0
United States of America
Query!
State/province [13]
0
0
Minnesota
Query!
Country [14]
0
0
United States of America
Query!
State/province [14]
0
0
Montana
Query!
Country [15]
0
0
United States of America
Query!
State/province [15]
0
0
Nebraska
Query!
Country [16]
0
0
United States of America
Query!
State/province [16]
0
0
New Jersey
Query!
Country [17]
0
0
United States of America
Query!
State/province [17]
0
0
New Mexico
Query!
Country [18]
0
0
United States of America
Query!
State/province [18]
0
0
New York
Query!
Country [19]
0
0
United States of America
Query!
State/province [19]
0
0
North Carolina
Query!
Country [20]
0
0
United States of America
Query!
State/province [20]
0
0
Ohio
Query!
Country [21]
0
0
United States of America
Query!
State/province [21]
0
0
Oklahoma
Query!
Country [22]
0
0
United States of America
Query!
State/province [22]
0
0
Pennsylvania
Query!
Country [23]
0
0
United States of America
Query!
State/province [23]
0
0
South Carolina
Query!
Country [24]
0
0
United States of America
Query!
State/province [24]
0
0
Texas
Query!
Country [25]
0
0
United States of America
Query!
State/province [25]
0
0
Vermont
Query!
Country [26]
0
0
United States of America
Query!
State/province [26]
0
0
Virginia
Query!
Country [27]
0
0
United States of America
Query!
State/province [27]
0
0
Washington
Query!
Country [28]
0
0
United States of America
Query!
State/province [28]
0
0
West Virginia
Query!
Country [29]
0
0
United States of America
Query!
State/province [29]
0
0
Wisconsin
Query!
Country [30]
0
0
Austria
Query!
State/province [30]
0
0
Graz
Query!
Country [31]
0
0
Austria
Query!
State/province [31]
0
0
Innsbruck
Query!
Country [32]
0
0
Austria
Query!
State/province [32]
0
0
Salzburg
Query!
Country [33]
0
0
Austria
Query!
State/province [33]
0
0
Vienna
Query!
Country [34]
0
0
Belgium
Query!
State/province [34]
0
0
Brussels
Query!
Country [35]
0
0
Belgium
Query!
State/province [35]
0
0
Edegem
Query!
Country [36]
0
0
Belgium
Query!
State/province [36]
0
0
Gent
Query!
Country [37]
0
0
Belgium
Query!
State/province [37]
0
0
Leuven
Query!
Country [38]
0
0
Canada
Query!
State/province [38]
0
0
British Columbia
Query!
Country [39]
0
0
Canada
Query!
State/province [39]
0
0
Quebec
Query!
Country [40]
0
0
Canada
Query!
State/province [40]
0
0
Calgary
Query!
Country [41]
0
0
Canada
Query!
State/province [41]
0
0
Qubec
Query!
Country [42]
0
0
Canada
Query!
State/province [42]
0
0
Saint John
Query!
Country [43]
0
0
Canada
Query!
State/province [43]
0
0
Toronto
Query!
Country [44]
0
0
Canada
Query!
State/province [44]
0
0
Victoria
Query!
Country [45]
0
0
Czechia
Query!
State/province [45]
0
0
Brno
Query!
Country [46]
0
0
Czechia
Query!
State/province [46]
0
0
Praha 5
Query!
Country [47]
0
0
France
Query!
State/province [47]
0
0
Benite Cedex
Query!
Country [48]
0
0
France
Query!
State/province [48]
0
0
Bordeaux Cedex
Query!
Country [49]
0
0
France
Query!
State/province [49]
0
0
Marseille
Query!
Country [50]
0
0
France
Query!
State/province [50]
0
0
Nice
Query!
Country [51]
0
0
France
Query!
State/province [51]
0
0
Paris
Query!
Country [52]
0
0
France
Query!
State/province [52]
0
0
Rouen Cedex, Seine Maritime
Query!
Country [53]
0
0
France
Query!
State/province [53]
0
0
Suresnes
Query!
Country [54]
0
0
Germany
Query!
State/province [54]
0
0
Erlangen
Query!
Country [55]
0
0
Germany
Query!
State/province [55]
0
0
Giessen
Query!
Country [56]
0
0
Germany
Query!
State/province [56]
0
0
Hannover
Query!
Country [57]
0
0
Germany
Query!
State/province [57]
0
0
Heidelberg
Query!
Country [58]
0
0
Germany
Query!
State/province [58]
0
0
Mainz
Query!
Country [59]
0
0
Germany
Query!
State/province [59]
0
0
München
Query!
Country [60]
0
0
Germany
Query!
State/province [60]
0
0
Tuebingen
Query!
Country [61]
0
0
Germany
Query!
State/province [61]
0
0
Wuerzburg
Query!
Country [62]
0
0
Greece
Query!
State/province [62]
0
0
Maroúsi
Query!
Country [63]
0
0
Italy
Query!
State/province [63]
0
0
Ancona
Query!
Country [64]
0
0
Italy
Query!
State/province [64]
0
0
Firenze
Query!
Country [65]
0
0
Italy
Query!
State/province [65]
0
0
Genova
Query!
Country [66]
0
0
Italy
Query!
State/province [66]
0
0
Messina
Query!
Country [67]
0
0
Italy
Query!
State/province [67]
0
0
Milano
Query!
Country [68]
0
0
Italy
Query!
State/province [68]
0
0
Potenza
Query!
Country [69]
0
0
Italy
Query!
State/province [69]
0
0
Verona
Query!
Country [70]
0
0
Netherlands
Query!
State/province [70]
0
0
Amsterdam
Query!
Country [71]
0
0
Netherlands
Query!
State/province [71]
0
0
Den Haag
Query!
Country [72]
0
0
Netherlands
Query!
State/province [72]
0
0
Heidelberglaan
Query!
Country [73]
0
0
Netherlands
Query!
State/province [73]
0
0
Nijmegen
Query!
Country [74]
0
0
Netherlands
Query!
State/province [74]
0
0
Rotterdam
Query!
Country [75]
0
0
Sweden
Query!
State/province [75]
0
0
Stockholm
Query!
Country [76]
0
0
United Kingdom
Query!
State/province [76]
0
0
Belfast
Query!
Country [77]
0
0
United Kingdom
Query!
State/province [77]
0
0
Brimingham
Query!
Country [78]
0
0
United Kingdom
Query!
State/province [78]
0
0
Edinburgh
Query!
Country [79]
0
0
United Kingdom
Query!
State/province [79]
0
0
Exeter
Query!
Country [80]
0
0
United Kingdom
Query!
State/province [80]
0
0
Leeds
Query!
Country [81]
0
0
United Kingdom
Query!
State/province [81]
0
0
London
Query!
Country [82]
0
0
United Kingdom
Query!
State/province [82]
0
0
Manchester
Query!
Country [83]
0
0
United Kingdom
Query!
State/province [83]
0
0
Southampton
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
This study will evaluate the efficacy of VX-445 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function mutation (F/MF subjects).
Query!
Trial website
https://clinicaltrials.gov/study/NCT03525444
Query!
Trial related presentations / publications
Middleton PG, Mall MA, Drevinek P, Lands LC, McKone EF, Polineni D, Ramsey BW, Taylor-Cousar JL, Tullis E, Vermeulen F, Marigowda G, McKee CM, Moskowitz SM, Nair N, Savage J, Simard C, Tian S, Waltz D, Xuan F, Rowe SM, Jain R; VX17-445-102 Study Group. Elexacaftor-Tezacaftor-Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele. N Engl J Med. 2019 Nov 7;381(19):1809-1819. doi: 10.1056/NEJMoa1908639. Epub 2019 Oct 31.
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
No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Type
Other Details
Attachment
Study protocol
https://cdn.clinicaltrials.gov/large-docs/44/NCT03525444/Prot_000.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/44/NCT03525444/SAP_001.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT03525444
Download to PDF