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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

Sponsor
Vertex Pharmaceuticals Incorporated (Industry)
Overall Status
Active, not recruiting
CT.gov ID
NCT02725567
Collaborator
(none)
56
Enrollment
23
Locations
3
Arms
76
Anticipated Duration (Months)
2.4
Patients Per Site
0
Patients Per Site Per Month

Study Details

Study Description

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
Phase 3

Study Design

Study Type:
Interventional
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 :
Mar 1, 2016
Anticipated Primary Completion Date :
Jul 1, 2022
Anticipated Study Completion Date :
Jul 1, 2022

Arms and Interventions

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 Names:
  • 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 Names:
  • Kalydeco

    		•
    Experimental: Part A/B

    - Group 8: Participants 1 to < 4 months of age

    Drug: ivacaftor
    Other Names:
  • Kalydeco

    		•

    Outcome Measures

    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 [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 [Day 1 up to Week 24]

    3. Part A: Peak concentrations (C3-6h) of ivacaftor, M1 ivacaftor, and M6 ivacaftor [after 4 days of ivacaftor treatment]

    4. Part A: Trough concentrations (Ctrough) of ivacaftor, M1 ivacaftor, and M6 ivacaftor [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 [Day 1 up to Week 24]

    6. Part A/B: Trough concentrations (Ctrough) of ivacaftor, M1 ivacaftor, and M6 ivacaftor [after 4 days of ivacaftor treatment]

    Secondary Outcome Measures

    1. Part B: Peak concentrations (C3-6h) of ivacaftor, M1 ivacaftor, and M6 ivacaftor [through Week 24]

    2. Part B: Trough concentrations (Ctrough) of ivacaftor, M1 ivacaftor, and M6 ivacaftor [through Week 24]

    3. Part B: Absolute change from baseline in sweat chloride using quantitative pilocarpine iontophoresis [up to Week 24]

    4. Part A/B: Absolute change from baseline in sweat chloride using quantitative pilocarpine iontophoresis [up to Week 24]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    0 Months to 24 Months
    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.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Birmingham Alabama United States
    2 Palo Alto California United States
    3 Atlanta Georgia United States
    4 Chicago Illinois United States
    5 Indianapolis Indiana United States
    6 Baltimore Maryland United States
    7 Boston Massachusetts United States
    8 Kansas City Missouri United States
    9 Columbus Ohio United States
    10 Philadelphia Pennsylvania United States
    11 Houston Texas United States
    12 Seattle Washington United States
    13 Madison Wisconsin United States
    14 Parkville Victoria Australia
    15 South Brisbane Australia
    16 Westmead Australia
    17 Toronto Canada
    18 Dublin Ireland
    19 Edinburgh United Kingdom
    20 Leicester United Kingdom
    21 London United Kingdom
    22 Manchester United Kingdom
    23 Oxford United Kingdom

    Sponsors and Collaborators

    • Vertex Pharmaceuticals Incorporated

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Vertex Pharmaceuticals Incorporated
    ClinicalTrials.gov Identifier:
    NCT02725567
    Other Study ID Numbers:
    • VX15-770-124
    • 2015-001997-16
    First Posted:
    Apr 1, 2016
    Last Update Posted:
    Feb 7, 2022
    Last Verified:
    Dec 1, 2021
    Additional relevant MeSH terms:
    Cystic Fibrosis
    Fibrosis
    Ivacaftor

    Study Results

    No Results Posted as of Feb 7, 2022