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A Study to Evaluate the Safety of Long-term Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive Mutation

Primary Purpose

Cystic Fibrosis

Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Ivacaftor
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

0 Months - 24 Months (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Ivacaftor Arm: Subjects From Study 124 (NCT02725567 ) Part B:

  • Subjects transitioning from Study 124 Part B must have completed the last study visit of Study 124 Part B.
  • As judged by the investigator, parent or legal guardian must be able to understand protocol requirements, restrictions, and instructions; and must sign the informed consent form (ICF).

Ivacaftor Arm: Subjects Not From Study 124 Part B:

  • Confirmed diagnosis of CF, or 2 CF-causing mutations.
  • An ivacaftor- responsive CFTR mutation on at least 1 allele. Subjects will be eligible in countries/regions where ivacaftor is approved for use in subjects 2 years of age and older.
  • As judged by the investigator, parent or legal guardian must be able to understand protocol requirements, restrictions, and instructions; and must sign the ICF.

Observational Arm:

  • Received ivacaftor treatment in Study 124 Part B and elected not to enroll or ineligible to enroll in the ivacaftor arm of Study 126.

Exclusion Criteria:

Ivacaftor Arm: Subjects From Study 124 Part B:

  • History of any illness or condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering ivacaftor to the subject.
  • Subjects receiving commercially available ivacaftor treatment

Ivacaftor Arm: Subjects Not From Study 124 Part B:

  • History of any illness or condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering ivacaftor to the subject
  • An acute upper or lower respiratory infection, or pulmonary exacerbation, or changes in therapy for pulmonary disease within 4 weeks of Day 1
  • Abnormal liver function at screening
  • Hemoglobin <9.5 g/dL at screening
  • History of solid organ or hematological transplantation
  • Use of any moderate or strong inducers or inhibitors of CYP3A within 2 weeks of Day 1

Observational Arm:

  • Receiving ivacaftor treatment

Other protocol defined Inclusion/Exclusion criteria may apply.

Sites / Locations

  • University of Alabama at Birmingham
  • Stanford University
  • Alfred I DuPont Hospital for Children
  • Nemours Children's Hospital
  • Center for Advanced Pediatrics
  • Northwestern University
  • Riley Hospital for Children at Indiana University Health
  • John Hopkins Hospital
  • Boston Children's Hospital
  • Childrens's Hospitals and Clinics of Minnnesota
  • Children's Mercy Hospital
  • Billings Clinic Hospital
  • Nationwide Children's Hospital
  • Children's Hospital of Philadelphia
  • Texas Children's Hospital
  • Seattle Children's Hospital
  • University of Wisconsin
  • Queensland Children's Hospital
  • The Hospital for Sick Children
  • Universitaetsklinikum Heidelberg, Zenter fuer Kinder-und Jugendmedizin
  • Children's Health Ireland at Crumlin
  • Children's University Hospital Temple Street
  • University Hospital Limerick
  • Paediatric Clinical Research Facility
  • Alder Hey Children's Alder Hey Children's NHS Foundation Trust
  • Great Ormond Street Hospital for Sick Children
  • Royal Brompton & Harefield NHS Founcation Trust, Royal BromptonHospital
  • Royal Manchester Children's Hospital
  • Oxford University Hospitals NHS Trust, John Radcliffe Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

No Intervention

Arm Label

Ivacaftor Arm

Observational Arm

Arm Description

Outcomes

Primary Outcome Measures

Safety assessments based on the number of subjects with adverse events (AEs) and serious adverse events (SAEs)
Number of subjects with AEs and SAEs will be reported.

Secondary Outcome Measures

Absolute change in sweat chloride

Full Information

First Posted
September 6, 2017
Last Updated
September 5, 2023
Sponsor
Vertex Pharmaceuticals Incorporated
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1. Study Identification

Unique Protocol Identification Number
NCT03277196
Brief Title
A Study to Evaluate the Safety of Long-term Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive Mutation
Official Title
A Phase 3, 2-Arm, Open-label Study to Evaluate the Safety and Pharmacodynamics of Long-term Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive Mutation
Study Type
Interventional

2. Study Status

Record Verification Date
September 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
August 16, 2017 (Actual)
Primary Completion Date
July 3, 2024 (Anticipated)
Study Completion Date
July 3, 2024 (Anticipated)

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
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
This is a Phase 3, 2-arm, multicenter study with an open-label ivacaftor arm and an observational arm to evaluate the safety and efficacy of long-term ivacaftor treatment in subjects with cystic fibrosis (CF) who are <24 months of age at treatment initiation and have an approved Ivacaftor-Responsive mutation

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
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
86 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Ivacaftor Arm
Arm Type
Experimental
Arm Title
Observational Arm
Arm Type
No Intervention
Intervention Type
Drug
Intervention Name(s)
Ivacaftor
Other Intervention Name(s)
VX-770
Intervention Description
Ivacaftor will be administered every 12 hours (q12h) from Day 1 through the morning dose of the Week 96 Visit.
Primary Outcome Measure Information:
Title
Safety assessments based on the number of subjects with adverse events (AEs) and serious adverse events (SAEs)
Description
Number of subjects with AEs and SAEs will be reported.
Time Frame
from baseline through safety follow-up (up to 24 weeks after last dose)
Secondary Outcome Measure Information:
Title
Absolute change in sweat chloride
Time Frame
from baseline through Week 96

10. Eligibility

Sex
All
Minimum Age & Unit of Time
0 Months
Maximum Age & Unit of Time
24 Months
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Ivacaftor Arm: Subjects From Study 124 (NCT02725567 ) Part B: Subjects transitioning from Study 124 Part B must have completed the last study visit of Study 124 Part B. As judged by the investigator, parent or legal guardian must be able to understand protocol requirements, restrictions, and instructions; and must sign the informed consent form (ICF). Ivacaftor Arm: Subjects Not From Study 124 Part B: Confirmed diagnosis of CF, or 2 CF-causing mutations. An ivacaftor- responsive CFTR mutation on at least 1 allele. Subjects will be eligible in countries/regions where ivacaftor is approved for use in subjects 2 years of age and older. As judged by the investigator, parent or legal guardian must be able to understand protocol requirements, restrictions, and instructions; and must sign the ICF. Observational Arm: Received ivacaftor treatment in Study 124 Part B and elected not to enroll or ineligible to enroll in the ivacaftor arm of Study 126. Exclusion Criteria: Ivacaftor Arm: Subjects From Study 124 Part B: History of any illness or condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering ivacaftor to the subject. Subjects receiving commercially available ivacaftor treatment Ivacaftor Arm: Subjects Not From Study 124 Part B: History of any illness or condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering ivacaftor to the subject An acute upper or lower respiratory infection, or pulmonary exacerbation, or changes in therapy for pulmonary disease within 4 weeks of Day 1 Abnormal liver function at screening Hemoglobin <9.5 g/dL at screening History of solid organ or hematological transplantation Use of any moderate or strong inducers or inhibitors of CYP3A within 2 weeks of Day 1 Observational Arm: Receiving ivacaftor treatment 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
Stanford University
City
Palo Alto
State/Province
California
ZIP/Postal Code
94034
Country
United States
Facility Name
Alfred I DuPont Hospital for Children
City
Wilmington
State/Province
Delaware
ZIP/Postal Code
19803
Country
United States
Facility Name
Nemours Children's Hospital
City
Orlando
State/Province
Florida
ZIP/Postal Code
32827
Country
United States
Facility Name
Center for Advanced Pediatrics
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30329
Country
United States
Facility Name
Northwestern University
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
John Hopkins Hospital
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
Facility Name
Boston Children's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Childrens's Hospitals and Clinics of Minnnesota
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55404
Country
United States
Facility Name
Children's Mercy Hospital
City
Kansas City
State/Province
Missouri
ZIP/Postal Code
64108
Country
United States
Facility Name
Billings Clinic Hospital
City
Billings
State/Province
Montana
ZIP/Postal Code
59101
Country
United States
Facility Name
Nationwide Children's Hospital
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43205
Country
United States
Facility Name
Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Texas Children's Hospital
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
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
City
Madison
State/Province
Wisconsin
ZIP/Postal Code
53792
Country
United States
Facility Name
Queensland Children's Hospital
City
South Brisbane
Country
Australia
Facility Name
The Hospital for Sick Children
City
Toronto
State/Province
Ontario
Country
Canada
Facility Name
Universitaetsklinikum Heidelberg, Zenter fuer Kinder-und Jugendmedizin
City
Heidelberg
Country
Germany
Facility Name
Children's Health Ireland at Crumlin
City
Dublin
Country
Ireland
Facility Name
Children's University Hospital Temple Street
City
Dublin
Country
Ireland
Facility Name
University Hospital Limerick
City
Limerick
Country
Ireland
Facility Name
Paediatric Clinical Research Facility
City
Edinburgh
Country
United Kingdom
Facility Name
Alder Hey Children's Alder Hey Children's NHS Foundation Trust
City
Liverpool
Country
United Kingdom
Facility Name
Great Ormond Street Hospital for Sick Children
City
London
Country
United Kingdom
Facility Name
Royal Brompton & Harefield NHS Founcation Trust, Royal BromptonHospital
City
London
Country
United Kingdom
Facility Name
Royal Manchester Children's Hospital
City
Manchester
Country
United Kingdom
Facility Name
Oxford University Hospitals NHS Trust, John Radcliffe Hospital
City
Oxford
Country
United Kingdom

12. IPD Sharing Statement

Learn more about this trial

A Study to Evaluate the Safety of Long-term Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive Mutation

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