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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03525574




Registration number
NCT03525574
Ethics application status
Date submitted
2/05/2018
Date registered
15/05/2018
Date last updated
8/05/2024

Titles & IDs
Public title
A 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 Homozygous or Heterozygous for the F508del Mutation
Secondary ID [1] 0 0
2018-000185-11
Secondary ID [2] 0 0
VX17-445-105
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 - Treatment Period: Participants received ELX 200 milligram (mg) once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hrs (q12h) in the treatment period for 192 weeks.

Extension Period: Participants from certain countries participated in extension period and continued to receive ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the extension 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
Treatment Period: 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 Day 1 up to Week 196
Secondary outcome [1] 0 0
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 102/105 Efficacy Set
Timepoint [1] 0 0
From Baseline at Week 192
Secondary outcome [2] 0 0
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 103/105 Efficacy Set
Timepoint [2] 0 0
From Baseline at Week 192
Secondary outcome [3] 0 0
Absolute Change in Sweat Chloride (SwCl) for 102/105 Efficacy Set
Timepoint [3] 0 0
From Baseline at Week 192
Secondary outcome [4] 0 0
Absolute Change in Sweat Chloride (SwCl) for 103/105 Efficacy Set
Timepoint [4] 0 0
From Baseline at Week 192
Secondary outcome [5] 0 0
Number of Pulmonary Exacerbations (PEx) for 102/105 Efficacy Set
Timepoint [5] 0 0
From Baseline at Week 192
Secondary outcome [6] 0 0
Number of Pulmonary Exacerbations (PEx) for 103/105 Efficacy Set
Timepoint [6] 0 0
From Baseline at Week 192
Secondary outcome [7] 0 0
Time to First PEx for 102/105 Efficacy Set
Timepoint [7] 0 0
From Baseline at Week 192
Secondary outcome [8] 0 0
Time to First PEx for 103/105 Efficacy Set
Timepoint [8] 0 0
From Baseline at Week 192
Secondary outcome [9] 0 0
Absolute Change in Body Mass Index (BMI) for 102/105 Efficacy Set
Timepoint [9] 0 0
From Baseline at Week 192
Secondary outcome [10] 0 0
Absolute Change in Body Mass Index (BMI) for 103/105 Efficacy Set
Timepoint [10] 0 0
From Baseline at Week 192
Secondary outcome [11] 0 0
Absolute Change in BMI Z-score for 102/105 Efficacy Set
Timepoint [11] 0 0
From Baseline at Week 192
Secondary outcome [12] 0 0
Absolute Change in BMI Z-score for 103/105 Efficacy Set
Timepoint [12] 0 0
From Baseline at Week 192
Secondary outcome [13] 0 0
Absolute Change in Body Weight for 102/105 Efficacy Set
Timepoint [13] 0 0
From Baseline at Week 192
Secondary outcome [14] 0 0
Absolute Change in Body Weight for 103/105 Efficacy Set
Timepoint [14] 0 0
From Baseline at Week 192
Secondary outcome [15] 0 0
Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for 102/105 Efficacy Set
Timepoint [15] 0 0
From Baseline at Week 192
Secondary outcome [16] 0 0
Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for 103/105 Efficacy Set
Timepoint [16] 0 0
From Baseline at Week 192

Eligibility
Key inclusion criteria
* Completed study drug treatment in a parent study i.e.VX17-445-102 (NCT03525444) and VX17-445-103 (NCT03525548); or had study drug interruption(s) in a parent study but completed study visits up to the last scheduled visit of the Treatment Period in the parent study.
Minimum age
12 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* History of drug intolerance in a parent study that would pose an additional risk to the subject in the opinion of the investigator.
* Current participation in an investigational drug trial (other than a 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
9/10/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
9/01/2023
Sample size
Target
Accrual to date
Final
507
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Women & Children's Hospital - North Adelaide
Recruitment hospital [2] 0 0
The Royal Children's Hospital - Parkville
Recruitment hospital [3] 0 0
Mater Misericordiae Ltd - South Brisbane
Recruitment hospital [4] 0 0
The Children's Hospital at Westmead - Westmead
Recruitment hospital [5] 0 0
Westmead Hospital - Westmead
Recruitment postcode(s) [1] 0 0
- North Adelaide
Recruitment postcode(s) [2] 0 0
- Parkville
Recruitment postcode(s) [3] 0 0
- South Brisbane
Recruitment postcode(s) [4] 0 0
- Westmead
Recruitment outside Australia
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Tuebingen
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Germany
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Wurzburg
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Greece
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Maroussi
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Ancona
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Firenze
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Genova
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Messina
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Potenza
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Den Haag
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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
The study evaluates the long-term safety and tolerability of VX-445 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous or heterozygous for the F508del mutation
Trial website
https://clinicaltrials.gov/study/NCT03525574
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/NCT03525574