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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
Results First Posted : September 7, 2023
Last Update Posted : September 7, 2023
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Study Description
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Brief Summary:
The purpose of this study was to evaluate the safety of ivacaftor treatment, and PK of ivacaftor and metabolites in participants 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: IVA Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 57 participants
Allocation: Non-Randomized
Intervention Model: Parallel 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 : June 2, 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: 3 to <24 months
Participants weighing 5 to less than (<) 7 kilogram (kg) received 25 milligram (mg) IVA, 7 to <14 kg received 50 mg IVA, and those weighing 14 to <25 kg received 75 mg IVA administered every 12 hours (q12h) on Days 1 through 3 and 1 morning dose on Day 4.
 Drug: IVA
Granules in sachet for oral administration.
Other Names:
  • VX-770
  • ivacaftor
Experimental: Part B + A/B:1 to < 24 months
Participants 4 to <6 months of age and weighing greater than or equal to (≥) 5 kg received 25 mg IVA q12h. At 6 months of age and older, participants weighing 5 to <7 kg received 25 mg IVA, 7 to <14 kg received 50 mg IVA, and those weighing 14 to <25 kg received 75 mg IVA q12h for 24 weeks on Part B. For Part A/B, participants 1 to <4 months weighing 3 kg to <5 kg received an initial low dose of 5.7 mg q12h IVA and those weighing ≥5 kg received 11.4 mg q12h IVA for the first 15 days of IVA treatment. Doses were maintained or adjusted upward at Day 15 and based on weight and/or age once they reached 4 months of age.
 Drug: IVA
Granules in sachet for oral administration.
Other Names:
  • VX-770
  • ivacaftor


Outcome Measures
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Primary Outcome Measures :
  1. Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious (TEAEs) [ Time Frame: Day 1 through Day 70 ]
  2. Part A: Observed Plasma Concentration of IVA and Their Metabolites (M1-IVA and M6-IVA) [ Time Frame: Pre-dose, 2-4 hours, 6-8 hours, 24-60 hours post-dose ]
  3. Part B +A/B: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious (TEAEs) [ Time Frame: Day 1 through Week 38 ]
  4. Part A/B: Observed Plasma Concentration of IVA and Their Metabolites (M1-IVA and M6-IVA) [ Time Frame: Day 4 (pre-dose, 2-4 hours, 6-8 hours post-dose); Day 15 (pre-dose); Week 4 (pre-dose); Week 8 (pre-dose, 2-4 hours, 6-8 hours post-dose); Week 12 (pre-dose); Week 18 (pre-dose) and Week 24 (pre-dose) ]

Secondary Outcome Measures :
  1. Part B: Observed Plasma Concentration of IVA and Their Metabolites (M1-IVA and M6-IVA) [ Time Frame: Week 2 (pre-dose, 2-4 hours, 6-8 hours post-dose); Week 8 (pre-dose,1 hour, 4-hour post-dose); Week 24 (pre-dose, 2-4 hours post dose) ]
  2. Part B + A/B: Absolute Change From Baseline in Sweat Chloride [ Time Frame: From Baseline at Week 24 ]
    Sweat samples were collected using an approved collection device.


Eligibility Criteria
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Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   1 Month 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 participant
  • 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
  Study Documents (Full-Text)

Documents provided by Vertex Pharmaceuticals Incorporated:
Study Protocol  [PDF] April 1, 2021
Statistical Analysis Plan  [PDF] December 7, 2022

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
Results First Posted: September 7, 2023
Last Update Posted: September 7, 2023
Last Verified: August 2023
Additional relevant MeSH terms:
Layout table for MeSH terms
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