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Long-term Safety of Lumacaftor/Ivacaftor in Subjects With Cystic Fibrosis Who Are Homozygous for F508del and 12 to <24 Months of Age at Treatment Initiation

Sponsor
Vertex Pharmaceuticals Incorporated (Industry)
Overall Status
Active, not recruiting
CT.gov ID
NCT04235140
Collaborator
(none)
52
Enrollment
23
Locations
1
Arm
47.2
Anticipated Duration (Months)
2.3
Patients Per Site
0
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This is a Phase 3, multicenter, open-label and roll-over study in subjects who are 12 to <24 months of age at initiation of Lumacaftor/Ivacaftor (LUM/IVA) treatment.

Condition or Disease Intervention/Treatment Phase
Phase 3

Study Design

Study Type:
Interventional
Actual Enrollment :
52 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
A Phase 3, Open-label, and Rollover Study to Evaluate the Long-term Safety and Tolerability of Lumacaftor/Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Homozygous for F508del and 12 to <24 Months of Age at Treatment Initiation
Actual Study Start Date :
Feb 24, 2020
Anticipated Primary Completion Date :
Feb 1, 2024
Anticipated Study Completion Date :
Feb 1, 2024

Arms and Interventions

Arm Intervention/Treatment
Experimental: LUM/IVA

Subjects will receive LUM/IVA for 96 weeks.

Drug: LUM/IVA
LUM/IVA granules for oral administration
Other Names:
  • lumacaftor/ivacaftor
  • VX-809/VX-770

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Safety and tolerability as assessed by the number of adverse events (AEs) and serious adverse events (SAEs) [Up to 120 weeks]

    Secondary Outcome Measures

    1. Absolute change in sweat chloride [From Baseline at Week 96]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    12 Months and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Key Inclusion Criteria:

    • Subjects From Study VX16-809-122 Part B (Study 122)

    • Completed the 24-week Treatment Period and the Safety Follow-up Visit in Study 122B

    • Subjects Not From Study 122

    • Subjects will be 1 to less than 2 years of age

    • Homozygous for the F508del mutation (F/F)

    Key Exclusion Criteria:

    • Any clinically significant laboratory abnormalities that would interfere with the study assessments or pose an undue risk for the subject

    • Solid organ or hematological transplantation

    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 Arkansas Children's Hospital Little Rock Arkansas United States 72202
    3 Children's Hospital Colorado Aurora Colorado United States 80045
    4 Yale New Haven Hospital New Haven Connecticut United States 06510
    5 Children's Healthcare of Atlanta Atlanta Georgia United States 30329
    6 Ann & Robert H. Lurie Children's Hospital of Chicago Chicago Illinois United States 60611
    7 Riley Hospital for Children at Indiana University Health Indianapolis Indiana United States 46202
    8 Boston Children's Hospital Boston Massachusetts United States 02115
    9 Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota Minneapolis Minnesota United States 55404
    10 The Children's Mercy Hospital Kansas City Missouri United States 64108
    11 Cardinal Glennon Children's Hospital - St. Louis University Saint Louis Missouri United States 63104
    12 University of Rochester Medical Center Rochester New York United States 14642
    13 NC TraCS Institute - CTRC University of North Carolina at Chapel Hill Chapel Hill North Carolina United States 27599
    14 Wake Forest University Baptist Medical Center Winston-Salem North Carolina United States 27157
    15 Cincinnati Children's Hospital Medical Center Cincinnati Ohio United States 45229
    16 Children's Medical Center of Dallas Dallas Texas United States 75235
    17 Cook Children's Medical Center Fort Worth Texas United States 76104
    18 University of Utah / Primary Children's Medical Center Salt Lake City Utah United States 84132
    19 Seattle Children's Hospital Seattle Washington United States 98105
    20 University of Wisconsin Hospital and Clinics Madison Wisconsin United States 53792
    21 McGill University Health Centre, Glen Site, Montreal Children's Hospital Montreal Canada
    22 The Hospital for Sick Children Toronto Canada
    23 British Columbia's Children's Hospital Vancouver Canada

    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:
    NCT04235140
    Other Study ID Numbers:
    • VX19-809-124
    First Posted:
    Jan 21, 2020
    Last Update Posted:
    Nov 29, 2021
    Last Verified:
    Nov 1, 2021
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    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 Nov 29, 2021