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NCT04235140 Clinical Trial

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

Cystic Fibrosis Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data
NCT number NCT04235140
Other study ID # VX19-809-124
Secondary ID
Status Completed
Phase Phase 3
First received
Last updated
Start date February 24, 2020
Est. completion date August 22, 2023

Study information
Verified date September 2023
Source Vertex Pharmaceuticals Incorporated
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.

Recruitment information / eligibility
Status Completed
Enrollment 52
Est. completion date August 22, 2023
Est. primary completion date August 22, 2023
Accepts healthy volunteers No
Gender All
Age group 12 Months and older
Eligibility 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 Design

Related Conditions & MeSH terms

Intervention

Drug:
• LUM/IVA
LUM/IVA granules for oral administration

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

Sponsors (1)
Lead Sponsor Collaborator
Vertex Pharmaceuticals Incorporated

Countries where clinical trial is conducted

United States,  Canada, 

Outcome
Type Measure Description Time frame Safety issue
Primary Safety and tolerability as assessed by the number of adverse events (AEs) and serious adverse events (SAEs) Up to 120 weeks
Secondary Absolute change in sweat chloride From Baseline at Week 96
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