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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04058366




Registration number
NCT04058366
Ethics application status
Date submitted
14/08/2019
Date registered
15/08/2019

Titles & IDs
Public title
Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy
Scientific title
A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)
Secondary ID [1] 0 0
2019-000833-37
Secondary ID [2] 0 0
VX18-445-110
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(has expanded access)
Description of intervention(s) / exposure
Treatment: Drugs - ELX/TEZ/IVA
Treatment: Drugs - IVA

Experimental: ELX/TEZ/IVA - Part A: Participants received ELX (elexacaftor) 200 milligram (mg) once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the treatment period for 96 weeks.

Part B: Participants from certain countries participated in Part B and continued to receive ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 48 weeks.


Treatment: Drugs: ELX/TEZ/IVA
Fixed-dose combination (FDC) tablet 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
Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Timepoint [1] 0 0
From Baseline up to Week 100
Secondary outcome [1] 0 0
Part A: Absolute Change From Parent Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
Timepoint [1] 0 0
From Baseline at Week 96
Secondary outcome [2] 0 0
Part A: Absolute Change From Parent Study Baseline in Sweat Chloride (SwCl)
Timepoint [2] 0 0
From Baseline at Week 96
Secondary outcome [3] 0 0
Part A: Absolute Change From Parent Study Baseline in Body Mass Index (BMI)
Timepoint [3] 0 0
From Baseline at Week 96
Secondary outcome [4] 0 0
Part A: Absolute Change From Parent Study Baseline in BMI Z-score
Timepoint [4] 0 0
From Baseline at Week 96
Secondary outcome [5] 0 0
Part A: Absolute Change From Parent Study Baseline in Body Weight
Timepoint [5] 0 0
From Baseline at Week 96
Secondary outcome [6] 0 0
Part A: Absolute Change From Parent Study Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score
Timepoint [6] 0 0
From Baseline at Week 96

Eligibility
Key inclusion criteria
Key

* Completed study drug treatment in parent study (VX18-445-104); 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
12 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* History of study drug intolerance in parent study

Other protocol defined Inclusion/Exclusion criteria may apply.

Study design
Purpose of the study
Treatment
Allocation to intervention
NA
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
5/12/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
16/12/2022
Sample size
Target
Accrual to date
Final
251
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
The Prince Charles Hospital - Chermside
Recruitment hospital [2] 0 0
Alfred Hospital - Melbourne
Recruitment hospital [3] 0 0
Telethon Kids Institute - Nedlands
Recruitment hospital [4] 0 0
The Royal Children's Hospital - Parkville
Recruitment hospital [5] 0 0
Mater Adult Hospital - South Brisbane
Recruitment hospital [6] 0 0
Queensland Children's Hospital - South Brisbane
Recruitment hospital [7] 0 0
Westmead Hospital - Westmead
Recruitment postcode(s) [1] 0 0
- Chermside
Recruitment postcode(s) [2] 0 0
- Melbourne
Recruitment postcode(s) [3] 0 0
- Nedlands
Recruitment postcode(s) [4] 0 0
- Parkville
Recruitment postcode(s) [5] 0 0
- South Brisbane
Recruitment postcode(s) [6] 0 0
- Westmead
Recruitment outside Australia
Country [1] 0 0
United States of America
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Arizona
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California
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Colorado
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Florida
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Indiana
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Kansas
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Kentucky
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Massachusetts
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Michigan
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Minnesota
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Missouri
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New York
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North Carolina
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Ohio
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Oklahoma
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Oregon
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Pennsylvania
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Wisconsin
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Brussels
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Gent
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Leuven
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Copenhagen
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Benite Cedex
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Lille
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Marseille
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Montpellier Cedex 5
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France
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Paris Cedex 15
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Paris
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Berlin
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Germany
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Essen
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Germany
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Giessen
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Germany
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Halle
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Germany
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Muenchen
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Dublin
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Ireland
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Limerick
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Italy
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Ancona
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Italy
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Genova
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Italy
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Milano
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Italy
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Rome
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Verona
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Murcia
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Sevilla
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Valencia
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Cambridge
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Exeter
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Leeds
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Liverpool
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London
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Manchester
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United Kingdom
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Southampton

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

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
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

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
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.


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT04058366