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Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Subjects 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del

Sponsor
Vertex Pharmaceuticals Incorporated (Industry)
Overall Status
Completed
CT.gov ID
NCT03601637
Collaborator
(none)
60
Enrollment
27
Locations
3
Arms
38.2
Actual Duration (Months)
2.2
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This study will evaluate the safety and pharmacokinetics (PK) of lumacaftor (LUM) and ivacaftor (IVA) in subjects 1 to less than 2 years of age with cystic fibrosis (CF), homozygous for F508del (F/F).

Condition or Disease Intervention/Treatment Phase
Phase 3

Study Design

Study Type:
Interventional
Actual Enrollment :
60 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 and Pharmacokinetics of Lumacaftor/Ivacaftor in Subjects 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del
Actual Study Start Date :
Aug 23, 2018
Actual Primary Completion Date :
Oct 29, 2021
Actual Study Completion Date :
Oct 29, 2021

Arms and Interventions

Arm Intervention/Treatment
Experimental: Part A Cohort 1 [aged 18 to <24 months]

Subjects will receive LUM/IVA as FDC granules dependent upon weight at Day 1.

Drug: LUM
Fixed Dose Combination (FDC) granule (LUM/IVA)
Other Names:
  • lumacaftor
  • VX-809

    • Drug: IVA
    FDC granule (LUM/IVA)
    Other Names:
  • ivacaftor
  • VX-770

    		•
    Experimental: Part A Cohort 2 [12 to <18months]

    Subjects will receive LUM/IVA as FDC granules dependent upon weight at Day 1.

    Drug: LUM
    Fixed Dose Combination (FDC) granule (LUM/IVA)
    Other Names:
  • lumacaftor
  • VX-809

    • Drug: IVA
    FDC granule (LUM/IVA)
    Other Names:
  • ivacaftor
  • VX-770

    		•
    Experimental: Part B

    Subjects will receive LUM/IVA as FDC granules dependent upon weight at Day 1.

    Drug: LUM
    Fixed Dose Combination (FDC) granule (LUM/IVA)
    Other Names:
  • lumacaftor
  • VX-809

    • Drug: IVA
    FDC granule (LUM/IVA)
    Other Names:
  • ivacaftor
  • VX-770

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Part A: Area under the concentration versus time curve during a dosing interval (AUCtau) of LUM and IVA [from baseline through Day 15]

    2. Part B: Safety and tolerability as assessed by number of subjects with adverse events (AEs) and serious adverse events (SAEs) [from baseline through safety follow-up (up to 2 weeks after the last dose)]

    Secondary Outcome Measures

    1. Part A: Safety and tolerability as assessed by number of subjects with adverse events (AEs) and serious adverse events (SAEs) [from baseline through safety follow-up (up to 10 days after the last dose)]

    2. Part A: Average observed pre-dose concentrations (Ctrough) of LUM and IVA metabolites [from baseline through Day 15]

    3. Part B: Absolute change in sweat chloride [from baseline at Week 24]

    4. Part B: Average observed pre-dose concentration (Ctrough) of LUM, IVA, and their respective metabolites [from baseline through Week 24]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    12 Months to 23 Months
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Key Inclusion Criteria:

    • Subjects will be 1 to less than 2 years of age on Day 1 of the relevant part of the study.

    • Homozygous for F508del (F/F).

    Key Exclusion Criteria:

    • Any clinically significant laboratory abnormalities at the Screening Visit 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 Stanford University Palo Alto California United States 94304
    4 Children's Hospital Colorado Aurora Colorado United States 80045
    5 Yale New Haven Medical Center New Haven Connecticut United States 06511
    6 Nemours / Alfred I. duPont Hospital for Children Wilmington Delaware United States 19803
    7 Children's Healthcare of Atlanta Atlanta Georgia United States 30329
    8 Ann & Robert Lurie Children's Hospital of Chicago Chicago Illinois United States 60611
    9 Riley Hospital for Children at Indiana University Health Indianapolis Indiana United States 46202
    10 Johns Hopkins Hospital Baltimore Maryland United States 21287
    11 Boston Children's Hospital Boston Massachusetts United States 02115
    12 Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota Minneapolis Minnesota United States 55404
    13 The Children's Mercy Hospital Kansas City Missouri United States 64108
    14 Cardinal Glennon Children's Hospital - St. Louis University Saint Louis Missouri United States 63104
    15 University of Rochester Medical Center Rochester New York United States 14642
    16 University of North Carolina Hospitals Chapel Hill North Carolina United States 27514
    17 Wake Forest University School of Medicine - Brenner Children's Hospital Winston-Salem North Carolina United States 27157
    18 Cincinnati Children's Hospital Medical Center Cincinnati Ohio United States 45229
    19 Children's Hospital of Philadelphia Philadelphia Pennsylvania United States 19104
    20 Children's Medical Center of Dallas Dallas Texas United States 75235
    21 Cook Children's Medical Center Fort Worth Texas United States 76104
    22 University of Utah / Primary Children's Medical Center Salt Lake City Utah United States 84132
    23 Seattle Children's Hospital Seattle Washington United States 98105
    24 University of Wisconsin Hospital and Clinics Madison Wisconsin United States 53792
    25 McGill University Health Centre, Glen Site, Montreal Children's Hospital Montreal Canada
    26 The Hospital for Sick Children Toronto Canada
    27 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:
    NCT03601637
    Other Study ID Numbers:
    • VX16-809-122
    • 2017-004794-13
    First Posted:
    Jul 26, 2018
    Last Update Posted:
    Nov 29, 2021
    Last Verified:
    Nov 1, 2021
    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