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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03277196




Registration number
NCT03277196
Ethics application status
Date submitted
6/09/2017
Date registered
8/09/2017
Date last updated
21/01/2020

Titles & IDs
Public title
A Study to Evaluate the Safety of Long-term Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive Mutation
Scientific title
A Phase 3, 2-Arm, Open-label Study to Evaluate the Safety and Pharmacodynamics of Long-term Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive Mutation
Secondary ID [1] 0 0
2017-001379-21
Secondary ID [2] 0 0
VX15-770-126
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 - Ivacaftor

Experimental: Ivacaftor Arm -

No Intervention: Observational Arm -


Treatment: Drugs: Ivacaftor
Ivacaftor will be administered every 12 hours (q12h) from Day 1 through the morning dose of the Week 96 Visit.

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

Outcomes
Primary outcome [1] 0 0
Safety assessments based on the number of subjects with adverse events (AEs) and serious adverse events (SAEs) - Number of subjects with AEs and SAEs will be reported.
Timepoint [1] 0 0
from baseline through safety follow-up (up to 24 weeks after last dose)
Secondary outcome [1] 0 0
Absolute change in sweat chloride
Timepoint [1] 0 0
from baseline through Week 96

Eligibility
Key inclusion criteria
Ivacaftor Arm: Subjects From Study 124 (NCT02725567 ) Part B:

- Subjects transitioning from Study 124 Part B must have completed the last study visit
of Study 124 Part B.

- As judged by the investigator, parent or legal guardian must be able to understand
protocol requirements, restrictions, and instructions; and must sign the informed
consent form (ICF).

Ivacaftor Arm: Subjects Not From Study 124 Part B:

- Confirmed diagnosis of CF, or 2 CF-causing mutations.

- An ivacaftor- responsive CFTR mutation on at least 1 allele. Subjects will be eligible
in countries/regions where ivacaftor is approved for use in subjects 2 years of age
and older.

- As judged by the investigator, parent or legal guardian must be able to understand
protocol requirements, restrictions, and instructions; and must sign the ICF.

Observational Arm:

- Received ivacaftor treatment in Study 124 Part B and elected not to enroll or
ineligible to enroll in the ivacaftor arm of Study 126.
Minimum age
No limit
Maximum age
24 Months
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Ivacaftor Arm: Subjects From Study 124 Part B:

- History of any illness or condition that, in the opinion of the investigator, might
confound the results of the study or pose an additional risk in administering
ivacaftor to the subject.

- Subjects receiving commercially available ivacaftor treatment

Ivacaftor Arm: Subjects Not From Study 124 Part B:

- History of any illness or condition that, in the opinion of the investigator, might
confound the results of the study or pose an additional risk in administering
ivacaftor to the subject

- An acute upper or lower respiratory infection, or pulmonary exacerbation, or changes
in therapy for pulmonary disease within 4 weeks of Day 1

- Abnormal liver function at screening

- Hemoglobin <9.5 g/dL at screening

- History of solid organ or hematological transplantation

- Use of any moderate or strong inducers or inhibitors of CYP3A within 2 weeks of Day 1

Observational Arm:

- Receiving ivacaftor treatment

Other protocol defined Inclusion/Exclusion criteria may apply.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised 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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
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
Lady Cilento Children's Hospital - South Brisbane
Recruitment postcode(s) [1] 0 0
- South Brisbane
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Delaware
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Georgia
Country [6] 0 0
United States of America
State/province [6] 0 0
Illinois
Country [7] 0 0
United States of America
State/province [7] 0 0
Indiana
Country [8] 0 0
United States of America
State/province [8] 0 0
Maryland
Country [9] 0 0
United States of America
State/province [9] 0 0
Massachusetts
Country [10] 0 0
United States of America
State/province [10] 0 0
Minnesota
Country [11] 0 0
United States of America
State/province [11] 0 0
Missouri
Country [12] 0 0
United States of America
State/province [12] 0 0
Montana
Country [13] 0 0
United States of America
State/province [13] 0 0
Ohio
Country [14] 0 0
United States of America
State/province [14] 0 0
Pennsylvania
Country [15] 0 0
United States of America
State/province [15] 0 0
Texas
Country [16] 0 0
United States of America
State/province [16] 0 0
Washington
Country [17] 0 0
United States of America
State/province [17] 0 0
Wisconsin
Country [18] 0 0
Canada
State/province [18] 0 0
Ontario
Country [19] 0 0
Germany
State/province [19] 0 0
Heidelberg
Country [20] 0 0
Ireland
State/province [20] 0 0
Dublin
Country [21] 0 0
Ireland
State/province [21] 0 0
Limerick
Country [22] 0 0
United Kingdom
State/province [22] 0 0
Edinburgh
Country [23] 0 0
United Kingdom
State/province [23] 0 0
Liverpool
Country [24] 0 0
United Kingdom
State/province [24] 0 0
London
Country [25] 0 0
United Kingdom
State/province [25] 0 0
Manchester
Country [26] 0 0
United Kingdom
State/province [26] 0 0
Oxford

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

Ethics approval
Ethics application status

Summary
Brief summary
This is a Phase 3, 2-arm, multicenter study with an open-label ivacaftor arm and an
observational arm to evaluate the safety and efficacy of long-term ivacaftor treatment in
subjects with cystic fibrosis (CF) who are <24 months of age at treatment initiation and have
an approved Ivacaftor-Responsive mutation
Trial website
https://clinicaltrials.gov/show/NCT03277196
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
For IPD and results data, please see https://clinicaltrials.gov/show/NCT03277196