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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 a CFTR Gating Mutation

This study is currently recruiting participants.
Verified October 2017 by Vertex Pharmaceuticals Incorporated
Sponsor:
ClinicalTrials.gov Identifier:
NCT02725567
First Posted: April 1, 2016
Last Update Posted: October 13, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated
  Purpose
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 Intervention Phase
Cystic Fibrosis Drug: ivacaftor Phase 3

Study Type: Interventional
Study Design: 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

Resource links provided by NLM:


Further study details as provided by Vertex Pharmaceuticals Incorporated:

Primary Outcome Measures:
  • 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 ]

Secondary Outcome Measures:
  • 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: through Week 24 ]

Estimated Enrollment: 35
Study Start Date: March 2016
Estimated Study Completion Date: June 2020
Estimated Primary Completion Date: June 2020 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

  Eligibility

Information from the National Library of Medicine

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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
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


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

  Show 23 Study Locations
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
  More Information

Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT02725567     History of Changes
Other Study ID Numbers: VX15-770-124
First Submitted: March 14, 2016
First Posted: April 1, 2016
Last Update Posted: October 13, 2017
Last Verified: October 2017

Additional relevant MeSH terms:
Ivacaftor
Fibrosis
Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Chloride Channel Agonists
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action