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 a CFTR Gating Mutation

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 a CF transmembrane conductance regulator (CFTR) gene gating mutation

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

Study Design

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Study Type :	Interventional (Clinical Trial)
Estimated Enrollment :	35 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 a CFTR Gating Mutation
Study Start Date :	March 2016
Estimated Primary Completion Date :	June 2020
Estimated Study Completion Date :	June 2020

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); Group 4: Participants 0 to < 3 months (enrollment begins after an assessment of data from Group 3)	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 0 to < 6 months (enrollment begins after an assessment of data from Part A, Group 3, and also enrollment for subjects < 3 months begins after review of data from Part A Group 4)	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 ]

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: through Week 24 ]

Eligibility Criteria

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Ages Eligible for Study:	up 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.
• Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D.
• 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
• 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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Contacts

Contact: Medical Information	617-341-6777	medicalinfo@vrtx.com

  Show 23 Study Locations

Sponsors and Collaborators

Vertex Pharmaceuticals Incorporated

More Information

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Responsible Party:	Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier:	NCT02725567 History of Changes
Other Study ID Numbers:	VX15-770-124
First Posted:	April 1, 2016
Last Update Posted:	October 13, 2017
Last Verified:	October 2017

Additional relevant MeSH terms:

Fibrosis; Cystic 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