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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

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ClinicalTrials.gov Identifier: NCT02725567
Recruitment Status : Completed
First Posted : April 1, 2016
Last Update Posted : December 8, 2022
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Study Description
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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.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: ivacaftor Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 56 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official 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
Actual Study Start Date : March 2016
Actual Primary Completion Date : June 28, 2022
Actual Study Completion Date : June 28, 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis
Drug Information available for: Ivacaftor

Arms and Interventions
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Arm Intervention/treatment
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)
 Drug: ivacaftor
Other Name: Kalydeco
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)
 Drug: ivacaftor
Other Name: Kalydeco
Experimental: Part A/B
- Group 8: Participants 1 to < 4 months of age
 Drug: ivacaftor
Other Name: Kalydeco


Outcome Measures
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Primary Outcome Measures :
  1. 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 [ Time Frame: Day 1 up to Day 70 ]
  2. 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 [ Time Frame: Day 1 up to Week 24 ]
  3. Part A: Peak concentrations (C3-6h) of ivacaftor, M1 ivacaftor, and M6 ivacaftor [ Time Frame: after 4 days of ivacaftor treatment ]
  4. Part A: Trough concentrations (Ctrough) of ivacaftor, M1 ivacaftor, and M6 ivacaftor [ Time Frame: after 4 days of ivacaftor treatment ]
  5. 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 [ Time Frame: Day 1 up to Week 24 ]
  6. Part A/B: Trough concentrations (Ctrough) of ivacaftor, M1 ivacaftor, and M6 ivacaftor [ Time Frame: after 4 days of ivacaftor treatment ]

Secondary Outcome Measures :
  1. Part B: Peak concentrations (C3-6h) of ivacaftor, M1 ivacaftor, and M6 ivacaftor [ Time Frame: through Week 24 ]
  2. Part B: Trough concentrations (Ctrough) of ivacaftor, M1 ivacaftor, and M6 ivacaftor [ Time Frame: through Week 24 ]
  3. Part B: Absolute change from baseline in sweat chloride using quantitative pilocarpine iontophoresis [ Time Frame: up to Week 24 ]
  4. Part A/B: Absolute change from baseline in sweat chloride using quantitative pilocarpine iontophoresis [ Time Frame: up to Week 24 ]

Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   0 Months to 24 Months   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

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.

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02725567


Locations
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United States, Alabama
Birmingham, Alabama, United States
United States, California
Palo Alto, California, United States
United States, Georgia
Atlanta, Georgia, United States
United States, Illinois
Chicago, Illinois, United States
United States, Indiana
Indianapolis, Indiana, United States
United States, Maryland
Baltimore, Maryland, United States
United States, Massachusetts
Boston, Massachusetts, United States
United States, Missouri
Kansas City, Missouri, United States
United States, Ohio
Columbus, Ohio, United States
United States, Pennsylvania
Philadelphia, Pennsylvania, United States
United States, Texas
Houston, Texas, United States
United States, Washington
Seattle, Washington, United States
United States, Wisconsin
Madison, Wisconsin, United States
Australia, Victoria
Parkville, Victoria, Australia
Australia
South Brisbane, Australia
Westmead, Australia
Canada
Toronto, Canada
Ireland
Dublin, Ireland
United Kingdom
Edinburgh, United Kingdom
Leicester, United Kingdom
London, United Kingdom
Manchester, United Kingdom
Oxford, United Kingdom
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT02725567    
Other Study ID Numbers: VX15-770-124
2015-001997-16 ( EudraCT Number )
First Posted: April 1, 2016    Key Record Dates
Last Update Posted: December 8, 2022
Last Verified: December 2022
Additional relevant MeSH terms:
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Cystic Fibrosis
Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
 Genetic Diseases, Inborn
Infant, Newborn, Diseases
Ivacaftor
Chloride Channel Agonists
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action