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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03559062




Registration number
NCT03559062
Ethics application status
Date submitted
5/06/2018
Date registered
15/06/2018
Date last updated
11/02/2020

Titles & IDs
Public title
A Study to Evaluate Efficacy and Safety of TEZ/IVA in Subjects Aged 6 Through 11 Years With Cystic Fibrosis
Scientific title
A Phase 3, Double-blind, Parallel-group Study to Evaluate the Efficacy and Safety of Tezacaftor in Combination With Ivacaftor in Subjects Aged 6 Through 11 Years With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation
Secondary ID [1] 0 0
2016-004479-35
Secondary ID [2] 0 0
VX16-661-115
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 - TEZ/IVA
Treatment: Drugs - IVA
Treatment: Drugs - Placebo
Treatment: Drugs - Placebo

Other: Placebo - Participants with genotype F/F received placebo matched to TEZ/IVA fixed dose combination (FDC) in the morning and placebo matched to IVA in the evening for 8 weeks.

Experimental: TEZ/IVA - Participants with genotype F/F received TEZ/IVA FDC in the morning and IVA in the evening for 8 weeks. Participants with genotype F/RF received TEZ/IVA FDC and placebo matched to IVA in the morning and IVA in the evening for 8 weeks.

Experimental: Ivacaftor - Participants with genotype F/RF received placebo matched to TEZ/IVA FDC in the morning and IVA in morning and evening for 8 weeks.


Treatment: Drugs: TEZ/IVA
Participants weighing <40 kg received TEZ 50 mg/IVA 75 mg FDC tablet and those weighing =40 kg received TEZ 100 mg/IVA 150 mg FDC tablet.

Treatment: Drugs: IVA
Participants weighing <40 kg IVA 75 mg tablet and those weighing =40 kg received IVA 150 mg tablet.

Treatment: Drugs: Placebo
Placebo matched to TEZ/IVA FDC

Treatment: Drugs: Placebo
Placebo matched to IVA

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Absolute Change in Lung Clearance Index 2.5 (LCI2.5) Through Week 8 - LCI2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.
Timepoint [1] 0 0
From baseline through Week 8
Secondary outcome [1] 0 0
Absolute Change in Sweat Chloride At Week 8 - Sweat samples were collected using an approved collection device.
Timepoint [1] 0 0
From baseline at Week 8
Secondary outcome [2] 0 0
Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 8 - 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.
Timepoint [2] 0 0
From baseline through Week 8
Secondary outcome [3] 0 0
Safety and Tolerability as Assessed Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) up to Safety Follow-up Visit
Timepoint [3] 0 0
From first dose of study drug up to safety follow-up visit (up to Week 12)

Eligibility
Key inclusion criteria
Key

- Homozygous for F508del or heterozygous for F508del and an RF mutation (as defined in
the protocol).

- Participants with ppFEV1 of =70 percentage points adjusted for age, sex, height.

- Participants with a screening LCI2.5 result =7.5.

- Participants who are able to swallow tablets.

Key
Minimum age
6 Years
Maximum age
11 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Clinically significant cirrhosis with or without portal hypertension.

- Colonization 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
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Hunter Medical Research Institute (HMRI) - New Lambton Heights
Recruitment hospital [2] 0 0
Princess Margaret Hospital for Children - Perth
Recruitment hospital [3] 0 0
Lady Cilento Children's Hospital - South Brisbane
Recruitment hospital [4] 0 0
The Children's Hospital at Westmead - Westmead
Recruitment postcode(s) [1] 0 0
- New Lambton Heights
Recruitment postcode(s) [2] 0 0
- Perth
Recruitment postcode(s) [3] 0 0
- South Brisbane
Recruitment postcode(s) [4] 0 0
- Westmead
Recruitment outside Australia
Country [1] 0 0
Belgium
State/province [1] 0 0
Brussels
Country [2] 0 0
Belgium
State/province [2] 0 0
Leuven
Country [3] 0 0
Denmark
State/province [3] 0 0
Copenhagen
Country [4] 0 0
France
State/province [4] 0 0
Bordeaux Cedex
Country [5] 0 0
France
State/province [5] 0 0
Paris
Country [6] 0 0
Germany
State/province [6] 0 0
Essen
Country [7] 0 0
Germany
State/province [7] 0 0
Frankfurt
Country [8] 0 0
Germany
State/province [8] 0 0
Giessen
Country [9] 0 0
Germany
State/province [9] 0 0
Hannover
Country [10] 0 0
Germany
State/province [10] 0 0
Heidelberg
Country [11] 0 0
Germany
State/province [11] 0 0
Jena
Country [12] 0 0
Germany
State/province [12] 0 0
Koeln
Country [13] 0 0
Germany
State/province [13] 0 0
Tuebingen
Country [14] 0 0
Ireland
State/province [14] 0 0
Dublin
Country [15] 0 0
Ireland
State/province [15] 0 0
Limerick
Country [16] 0 0
Poland
State/province [16] 0 0
Dziekanow Lesny
Country [17] 0 0
Switzerland
State/province [17] 0 0
Bern
Country [18] 0 0
Switzerland
State/province [18] 0 0
Zuerich
Country [19] 0 0
United Kingdom
State/province [19] 0 0
Edinburgh
Country [20] 0 0
United Kingdom
State/province [20] 0 0
Leeds
Country [21] 0 0
United Kingdom
State/province [21] 0 0
London
Country [22] 0 0
United Kingdom
State/province [22] 0 0
Nottingham
Country [23] 0 0
United Kingdom
State/province [23] 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 of tezacaftor in combination with ivacaftor (TEZ/IVA)
in participants with cystic fibrosis (CF) aged 6 through 11 years, who are homozygous for the
F508del mutation (F/F) or heterozygous for F508del with an eligible residual function
mutation (F/RF).
Trial website
https://clinicaltrials.gov/show/NCT03559062
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
Other publications