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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT02725567




Registration number
NCT02725567
Ethics application status
Date submitted
14/03/2016
Date registered
1/04/2016
Date last updated
22/12/2020

Titles & IDs
Public title
A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age and Have an Ivacaftor-Responsive CFTR Mutation
Scientific title
A Phase 3, 2 Part, Open-Label Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age and Have an Ivacaftor-Responsive CFTR Mutation
Secondary ID [1] 0 0
2015-001997-16
Secondary ID [2] 0 0
VX15-770-124
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: Part A - Group 1: Participants 12 to < 24 months
Group 2: Participants 6 to < 12 months (enrollment begins after an assessment of data from Group 1)
Group 3: Participants 3 to < 6 months (enrollment begins after an assessment of data from Group 2)

Experimental: Part B - Group 5: Participants 12 to < 24 months (enrollment begins after an assessment of data from Part A, Group 1)
Group 6: Participants 6 to < 12 months (enrollment begins after an assessment of data from Part A, Group 2)
Group 7: Participants 4 to < 6 months (enrollment begins after an assessment of data from Part A, Group 3)

Experimental: Part A/B - - Group 8: Participants 1 to < 4 months of age


Treatment: Drugs: ivacaftor


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

Outcomes
Primary outcome [1] 0 0
Part A: Safety, as determined by number of subjects with adverse events (AEs), clinically relevant abnormal laboratory values (serum chemistry and hematology), standard 12 lead electrocardiograms (ECGs), vital signs, and ophthalmologic examinations
Timepoint [1] 0 0
Day 1 up to Day 70
Primary outcome [2] 0 0
Part B: Safety, as determined by number of subjects with adverse events (AEs), clinically relevant abnormal laboratory values (serum chemistry and hematology), standard 12 lead electrocardiograms (ECGs), vital signs, and ophthalmologic examinations
Timepoint [2] 0 0
Day 1 up to Week 24
Primary outcome [3] 0 0
Part A: Peak concentrations (C3-6h) of ivacaftor, M1 ivacaftor, and M6 ivacaftor
Timepoint [3] 0 0
after 4 days of ivacaftor treatment
Primary outcome [4] 0 0
Part A: Trough concentrations (Ctrough) of ivacaftor, M1 ivacaftor, and M6 ivacaftor
Timepoint [4] 0 0
after 4 days of ivacaftor treatment
Primary outcome [5] 0 0
Part A/B: Safety, as determined by number of subjects with adverse events (AEs), clinically relevant abnormal laboratory values (serum chemistry and hematology), standard 12 lead electrocardiograms (ECGs), vital signs, and ophthalmologic examinations
Timepoint [5] 0 0
Day 1 up to Week 24
Primary outcome [6] 0 0
Part A/B: Trough concentrations (Ctrough) of ivacaftor, M1 ivacaftor, and M6 ivacaftor
Timepoint [6] 0 0
after 4 days of ivacaftor treatment
Secondary outcome [1] 0 0
Part B: Peak concentrations (C3-6h) of ivacaftor, M1 ivacaftor, and M6 ivacaftor
Timepoint [1] 0 0
through Week 24
Secondary outcome [2] 0 0
Part B: Trough concentrations (Ctrough) of ivacaftor, M1 ivacaftor, and M6 ivacaftor
Timepoint [2] 0 0
through Week 24
Secondary outcome [3] 0 0
Part B: Absolute change from baseline in sweat chloride using quantitative pilocarpine iontophoresis
Timepoint [3] 0 0
up to Week 24
Secondary outcome [4] 0 0
Part A/B: Absolute change from baseline in sweat chloride using quantitative pilocarpine iontophoresis
Timepoint [4] 0 0
up to Week 24

Eligibility
Key inclusion criteria
- Confirmed diagnosis of CF by sweat chloride value or CF mutation criteria.

- Have 1 of the following 10 CFTR mutations on at least 1 allele: G551D, G178R, S549N,
S549R, G551S, G1244E, S1251N, S1255P, G1349D or R117H (eligible in regions where
ivacaftor is approved for use). Part A/B group may also have other
ivacaftor-responsive mutations.

- Hematology, serum chemistry, and vital signs results at screening with no clinically
significant abnormalities that would interfere with the study assessments, as judged
by the investigator.
Minimum age
No limit
Maximum age
24 Months
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- 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 study
drug to the subject

- Colonization with organisms associated with a more rapid decline in pulmonary status
at screening (Only for Parts A and B)

- History of abnormal liver function or abnormal liver function at screening

- History of solid organ or hematological transplantation

- Use of any moderate or strong inducers or inhibitors of cytochrome P450 (CYP) 3A
within 2 weeks before Day 1

- Participation in a clinical study involving administration of either an
investigational or a marketed drug within 30 days or 5 terminal half-lives before
screening

- Hemoglobin (Hgb) <9.5 g/dL at screening

- Chronic kidney disease of Stage 3 or above

- Presence of a non-congenital or progressive lens opacity or cataract at Screening

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
Single group
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)
VIC
Recruitment hospital [1] 0 0
- Parkville
Recruitment hospital [2] 0 0
- South Brisbane
Recruitment hospital [3] 0 0
- Westmead
Recruitment postcode(s) [1] 0 0
- Parkville
Recruitment postcode(s) [2] 0 0
- South Brisbane
Recruitment postcode(s) [3] 0 0
- Westmead
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
Georgia
Country [4] 0 0
United States of America
State/province [4] 0 0
Illinois
Country [5] 0 0
United States of America
State/province [5] 0 0
Indiana
Country [6] 0 0
United States of America
State/province [6] 0 0
Maryland
Country [7] 0 0
United States of America
State/province [7] 0 0
Massachusetts
Country [8] 0 0
United States of America
State/province [8] 0 0
Missouri
Country [9] 0 0
United States of America
State/province [9] 0 0
Ohio
Country [10] 0 0
United States of America
State/province [10] 0 0
Pennsylvania
Country [11] 0 0
United States of America
State/province [11] 0 0
Texas
Country [12] 0 0
United States of America
State/province [12] 0 0
Washington
Country [13] 0 0
United States of America
State/province [13] 0 0
Wisconsin
Country [14] 0 0
Canada
State/province [14] 0 0
Toronto
Country [15] 0 0
Ireland
State/province [15] 0 0
Dublin
Country [16] 0 0
United Kingdom
State/province [16] 0 0
Edinburgh
Country [17] 0 0
United Kingdom
State/province [17] 0 0
Leicester
Country [18] 0 0
United Kingdom
State/province [18] 0 0
London
Country [19] 0 0
United Kingdom
State/province [19] 0 0
Manchester
Country [20] 0 0
United Kingdom
State/province [20] 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
The purpose of this study is to evaluate the safety of ivacaftor treatment, and PK of
ivacaftor and metabolites in subjects with cystic fibrosis (CF) who are <24 months of age at
treatment initiation and have an ivacaftor-responsive CF transmembrane conductance regulator
(CFTR) gene mutation.
Trial website
https://clinicaltrials.gov/show/NCT02725567
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
Medical Information
Address 0 0
Country 0 0
Phone 0 0
617-341-6777
Fax 0 0
Email 0 0
medicalinfo@vrtx.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT02725567