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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/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
Timepoint [1] 0 0
From baseline through Week 8
Secondary outcome [1] 0 0
Absolute Change in Sweat Chloride At Week 8
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
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
Sex
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
17/05/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
21/12/2018
Sample size
Target
Accrual to date
Final
67
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
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

No information has been provided regarding IPD availability


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/NCT03559062