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

Arm | Intervention/treatment |
---|---|
Experimental: Part A
|
Drug: ivacaftor
Other Name: Kalydeco |
Experimental: Part B
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Drug: ivacaftor
Other Name: Kalydeco |
Experimental: Part A/B
- Group 8: Participants 1 to < 4 months of age
|
Drug: ivacaftor
Other Name: Kalydeco |
- 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 ]
- 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 ]
- Part A: Peak concentrations (C3-6h) of ivacaftor, M1 ivacaftor, and M6 ivacaftor [ Time Frame: after 4 days of ivacaftor treatment ]
- Part A: Trough concentrations (Ctrough) of ivacaftor, M1 ivacaftor, and M6 ivacaftor [ Time Frame: after 4 days of ivacaftor treatment ]
- 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 ]
- Part A/B: Trough concentrations (Ctrough) of ivacaftor, M1 ivacaftor, and M6 ivacaftor [ Time Frame: after 4 days of ivacaftor treatment ]
- Part B: Peak concentrations (C3-6h) of ivacaftor, M1 ivacaftor, and M6 ivacaftor [ Time Frame: through Week 24 ]
- Part B: Trough concentrations (Ctrough) of ivacaftor, M1 ivacaftor, and M6 ivacaftor [ Time Frame: through Week 24 ]
- Part B: Absolute change from baseline in sweat chloride using quantitative pilocarpine iontophoresis [ Time Frame: up to Week 24 ]
- Part A/B: Absolute change from baseline in sweat chloride using quantitative pilocarpine iontophoresis [ Time Frame: up to Week 24 ]

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

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
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 |
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 |
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 |