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

September 20, 2023 updated by: Vertex Pharmaceuticals Incorporated

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

52

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
      • Montreal, Canada
        • McGill University Health Centre, Glen Site, Montreal Children's Hospital
      • Toronto, Canada
        • The Hospital for Sick Children
      • Vancouver, Canada
        • British Columbia's Children's Hospital
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35233
        • University of Alabama at Birmingham
    • Arkansas
      • Little Rock, Arkansas, United States, 72202
        • Arkansas Children's Hospital
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Children's Hospital Colorado
    • Connecticut
      • New Haven, Connecticut, United States, 06510
        • Yale New Haven Hospital
    • Georgia
      • Atlanta, Georgia, United States, 30329
        • Children's Healthcare of Atlanta
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Ann & Robert H. Lurie Children's Hospital of Chicago
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Riley Hospital for Children at Indiana University Health
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Boston Children's Hospital
    • Minnesota
      • Minneapolis, Minnesota, United States, 55404
        • Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota
    • Missouri
      • Kansas City, Missouri, United States, 64108
        • The Children's Mercy Hospital
      • Saint Louis, Missouri, United States, 63104
        • Cardinal Glennon Children's Hospital - St. Louis University
    • New York
      • Rochester, New York, United States, 14642
        • University of Rochester Medical Center
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27599
        • NC TraCS Institute - CTRC University of North Carolina at Chapel Hill
      • Winston-Salem, North Carolina, United States, 27157
        • Wake Forest University Baptist Medical Center
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Children's Hospital Medical Center
    • Texas
      • Dallas, Texas, United States, 75235
        • Children's Medical Center of Dallas
      • Fort Worth, Texas, United States, 76104
        • Cook Children's Medical Center
    • Utah
      • Salt Lake City, Utah, United States, 84132
        • University of Utah / Primary Children's Medical Center
    • Washington
      • Seattle, Washington, United States, 98105
        • Seattle Children's Hospital
    • Wisconsin
      • Madison, Wisconsin, United States, 53792
        • University of Wisconsin Hospital and Clinics

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Safety and tolerability as assessed by the number of adverse events (AEs) and serious adverse events (SAEs)
Time Frame: Up to 120 weeks
Up to 120 weeks

Secondary Outcome Measures

Outcome Measure
Time Frame
Absolute change in sweat chloride
Time Frame: From Baseline at Week 96
From Baseline at Week 96

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Vertex Pharmaceuticals Incorporated

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

February 24, 2020

Primary Completion (Actual)

August 22, 2023

Study Completion (Actual)

August 22, 2023

Study Registration Dates

First Submitted

January 15, 2020

First Submitted That Met QC Criteria

January 15, 2020

First Posted (Actual)

January 21, 2020

Study Record Updates

Last Update Posted (Actual)

September 21, 2023

Last Update Submitted That Met QC Criteria

September 20, 2023

Last Verified

September 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VX19-809-124

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent research/clinical-trial-data-sharing.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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