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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

Primary Purpose

Cystic Fibrosis

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
LUM/IVA
Sponsored by
Vertex Pharmaceuticals Incorporated
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cystic Fibrosis

Eligibility Criteria

12 Months - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • University of Alabama at Birmingham
  • Arkansas Children's Hospital
  • Children's Hospital Colorado
  • Yale New Haven Hospital
  • Children's Healthcare of Atlanta
  • Ann & Robert H. Lurie Children's Hospital of Chicago
  • Riley Hospital for Children at Indiana University Health
  • Boston Children's Hospital
  • Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota
  • The Children's Mercy Hospital
  • Cardinal Glennon Children's Hospital - St. Louis University
  • University of Rochester Medical Center
  • NC TraCS Institute - CTRC University of North Carolina at Chapel Hill
  • Wake Forest University Baptist Medical Center
  • Cincinnati Children's Hospital Medical Center
  • Children's Medical Center of Dallas
  • Cook Children's Medical Center
  • University of Utah / Primary Children's Medical Center
  • Seattle Children's Hospital
  • University of Wisconsin Hospital and Clinics
  • McGill University Health Centre, Glen Site, Montreal Children's Hospital
  • The Hospital for Sick Children
  • British Columbia's Children's Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

LUM/IVA

Arm Description

Subjects will receive LUM/IVA for 96 weeks.

Outcomes

Primary Outcome Measures

Safety and tolerability as assessed by the number of adverse events (AEs) and serious adverse events (SAEs)

Secondary Outcome Measures

Absolute change in sweat chloride

Full Information

First Posted
January 15, 2020
Last Updated
September 20, 2023
Sponsor
Vertex Pharmaceuticals Incorporated
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1. Study Identification

Unique Protocol Identification Number
NCT04235140
Brief Title
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
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
Study Type
Interventional

2. Study Status

Record Verification Date
September 2023
Overall Recruitment Status
Completed
Study Start Date
February 24, 2020 (Actual)
Primary Completion Date
August 22, 2023 (Actual)
Study Completion Date
August 22, 2023 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Vertex Pharmaceuticals Incorporated

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No

5. 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
52 (Actual)

8. Arms, Groups, and Interventions

Arm Title
LUM/IVA
Arm Type
Experimental
Arm Description
Subjects will receive LUM/IVA for 96 weeks.
Intervention Type
Drug
Intervention Name(s)
LUM/IVA
Other Intervention Name(s)
lumacaftor/ivacaftor, VX-809/VX-770
Intervention Description
LUM/IVA granules for oral administration
Primary Outcome Measure Information:
Title
Safety and tolerability as assessed by the number of adverse events (AEs) and serious adverse events (SAEs)
Time Frame
Up to 120 weeks
Secondary Outcome Measure Information:
Title
Absolute change in sweat chloride
Time Frame
From Baseline at Week 96

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Months
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Facility Information:
Facility Name
University of Alabama at Birmingham
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35233
Country
United States
Facility Name
Arkansas Children's Hospital
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72202
Country
United States
Facility Name
Children's Hospital Colorado
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
Yale New Haven Hospital
City
New Haven
State/Province
Connecticut
ZIP/Postal Code
06510
Country
United States
Facility Name
Children's Healthcare of Atlanta
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30329
Country
United States
Facility Name
Ann & Robert H. Lurie Children's Hospital of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
Riley Hospital for Children at Indiana University Health
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
Boston Children's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55404
Country
United States
Facility Name
The Children's Mercy Hospital
City
Kansas City
State/Province
Missouri
ZIP/Postal Code
64108
Country
United States
Facility Name
Cardinal Glennon Children's Hospital - St. Louis University
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63104
Country
United States
Facility Name
University of Rochester Medical Center
City
Rochester
State/Province
New York
ZIP/Postal Code
14642
Country
United States
Facility Name
NC TraCS Institute - CTRC University of North Carolina at Chapel Hill
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27599
Country
United States
Facility Name
Wake Forest University Baptist Medical Center
City
Winston-Salem
State/Province
North Carolina
ZIP/Postal Code
27157
Country
United States
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
Children's Medical Center of Dallas
City
Dallas
State/Province
Texas
ZIP/Postal Code
75235
Country
United States
Facility Name
Cook Children's Medical Center
City
Fort Worth
State/Province
Texas
ZIP/Postal Code
76104
Country
United States
Facility Name
University of Utah / Primary Children's Medical Center
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84132
Country
United States
Facility Name
Seattle Children's Hospital
City
Seattle
State/Province
Washington
ZIP/Postal Code
98105
Country
United States
Facility Name
University of Wisconsin Hospital and Clinics
City
Madison
State/Province
Wisconsin
ZIP/Postal Code
53792
Country
United States
Facility Name
McGill University Health Centre, Glen Site, Montreal Children's Hospital
City
Montreal
Country
Canada
Facility Name
The Hospital for Sick Children
City
Toronto
Country
Canada
Facility Name
British Columbia's Children's Hospital
City
Vancouver
Country
Canada

12. IPD Sharing Statement

Plan to Share IPD
No
IPD Sharing 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.
Citations:
PubMed Identifier
33331662
Citation
Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3.
Results Reference
derived

Learn more about this 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

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