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A Study to Evaluate the Safety of Long-term Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive Mutation

Sponsor
Vertex Pharmaceuticals Incorporated (Industry)
Overall Status
Enrolling by invitation
CT.gov ID
NCT03277196
Collaborator
(none)
75
Enrollment
29
Locations
2
Arms
82.6
Anticipated Duration (Months)
2.6
Patients Per Site
0
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This is a Phase 3, 2-arm, multicenter study with an open-label ivacaftor arm and an observational arm to evaluate the safety and efficacy of long-term ivacaftor treatment in subjects with cystic fibrosis (CF) who are <24 months of age at treatment initiation and have an approved Ivacaftor-Responsive mutation

Condition or Disease Intervention/Treatment Phase
Phase 3

Study Design

Study Type:
Interventional
Anticipated Enrollment :
75 participants
Allocation:
Non-Randomized
Intervention Model:
Parallel Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
A Phase 3, 2-Arm, Open-label Study to Evaluate the Safety and Pharmacodynamics of Long-term Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive Mutation
Actual Study Start Date :
Aug 16, 2017
Anticipated Primary Completion Date :
Jul 3, 2024
Anticipated Study Completion Date :
Jul 3, 2024

Arms and Interventions

Arm Intervention/Treatment
Experimental: Ivacaftor Arm

Drug: Ivacaftor
Ivacaftor will be administered every 12 hours (q12h) from Day 1 through the morning dose of the Week 96 Visit.
Other Names:
  • VX-770

    		•
    No Intervention: Observational Arm

    Outcome Measures

    Primary Outcome Measures

    1. Safety assessments based on the number of subjects with adverse events (AEs) and serious adverse events (SAEs) [from baseline through safety follow-up (up to 24 weeks after last dose)]

      Number of subjects with AEs and SAEs will be reported.

    Secondary Outcome Measures

    1. Absolute change in sweat chloride [from baseline through Week 96]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    0 Months to 24 Months
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:
    Ivacaftor Arm: Subjects From Study 124 (NCT02725567 ) Part B:

    • Subjects transitioning from Study 124 Part B must have completed the last study visit of Study 124 Part B.

    • As judged by the investigator, parent or legal guardian must be able to understand protocol requirements, restrictions, and instructions; and must sign the informed consent form (ICF).

    Ivacaftor Arm: Subjects Not From Study 124 Part B:

    • Confirmed diagnosis of CF, or 2 CF-causing mutations.

    • An ivacaftor- responsive CFTR mutation on at least 1 allele. Subjects will be eligible in countries/regions where ivacaftor is approved for use in subjects 2 years of age and older.

    • As judged by the investigator, parent or legal guardian must be able to understand protocol requirements, restrictions, and instructions; and must sign the ICF.

    Observational Arm:
    • Received ivacaftor treatment in Study 124 Part B and elected not to enroll or ineligible to enroll in the ivacaftor arm of Study 126.
    Exclusion Criteria:
    Ivacaftor Arm: Subjects From Study 124 Part B:

    • 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 ivacaftor to the subject.

    • Subjects receiving commercially available ivacaftor treatment

    Ivacaftor Arm: Subjects Not From Study 124 Part B:

    • 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 ivacaftor to the subject

    • An acute upper or lower respiratory infection, or pulmonary exacerbation, or changes in therapy for pulmonary disease within 4 weeks of Day 1

    • Abnormal liver function at screening

    • Hemoglobin <9.5 g/dL at screening

    • History of solid organ or hematological transplantation

    • Use of any moderate or strong inducers or inhibitors of CYP3A within 2 weeks of Day 1

    Observational Arm:
    • Receiving ivacaftor treatment

    Other protocol defined Inclusion/Exclusion criteria may apply.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 University of Alabama at Birmingham Birmingham Alabama United States 35233
    2 Stanford University Palo Alto California United States 94034
    3 Alfred I DuPont Hospital for Children Wilmington Delaware United States 19803
    4 Nemours Children's Hospital Orlando Florida United States 32827
    5 Center for Advanced Pediatrics Atlanta Georgia United States 30329
    6 Northwestern University Chicago Illinois United States 60611
    7 Riley Hospital for Children at Indiana University Health Indianapolis Indiana United States 46202
    8 John Hopkins Hospital Baltimore Maryland United States 21287
    9 Boston Children's Hospital Boston Massachusetts United States 02115
    10 Childrens's Hospitals and Clinics of Minnnesota Minneapolis Minnesota United States 55404
    11 Children's Mercy Hospital Kansas City Missouri United States 64108
    12 Billings Clinic Hospital Billings Montana United States 59101
    13 Nationwide Children's Hospital Columbus Ohio United States 43205
    14 Children's Hospital of Philadelphia Philadelphia Pennsylvania United States 19104
    15 Texas Children's Hospital Houston Texas United States 77030
    16 Seattle Children's Hospital Seattle Washington United States 98105
    17 University of Wisconsin Madison Wisconsin United States 53792
    18 Queensland Children's Hospital South Brisbane Australia
    19 The Hospital for Sick Children Toronto Ontario Canada
    20 Universitaetsklinikum Heidelberg, Zenter fuer Kinder-und Jugendmedizin Heidelberg Germany
    21 Children's Health Ireland at Crumlin Dublin Ireland
    22 Children's University Hospital Temple Street Dublin Ireland
    23 University Hospital Limerick Limerick Ireland
    24 Paediatric Clinical Research Facility Edinburgh United Kingdom
    25 Alder Hey Children's Alder Hey Children's NHS Foundation Trust Liverpool United Kingdom
    26 Great Ormond Street Hospital for Sick Children London United Kingdom
    27 Royal Brompton & Harefield NHS Founcation Trust, Royal BromptonHospital London United Kingdom
    28 Royal Manchester Children's Hospital Manchester United Kingdom
    29 Oxford University Hospitals NHS Trust, John Radcliffe Hospital 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:
    NCT03277196
    Other Study ID Numbers:
    • VX15-770-126
    • 2017-001379-21
    First Posted:
    Sep 8, 2017
    Last Update Posted:
    Jun 21, 2022
    Last Verified:
    Jan 1, 2022
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Additional relevant MeSH terms:
    Cystic Fibrosis
    Fibrosis
    Ivacaftor

    Study Results

    No Results Posted as of Jun 21, 2022