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

Cystic Fibrosis Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03277196
• Other study ID #: VX15-770-126
• Secondary ID: 2017-001379-21
• Status: Completed
• Phase: Phase 3
• Start date: August 16, 2017
• Est. completion date: October 2, 2023

Study information

• Verified date: December 2023
• Source: Vertex Pharmaceuticals Incorporated
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 86
• Est. completion date: October 2, 2023
• Est. primary completion date: October 2, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 0 Months to 24 Months

Eligibility

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.

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• Ivacaftor
Ivacaftor will be administered every 12 hours (q12h) from Day 1 through the morning dose of the Week 96 Visit.

Locations

Country	Name	City	State
Australia	Queensland Children's Hospital	South Brisbane
Canada	The Hospital for Sick Children	Toronto	Ontario
Germany	Universitaetsklinikum Heidelberg, Zenter fuer Kinder-und Jugendmedizin	Heidelberg
Ireland	Children's Health Ireland at Crumlin	Dublin
Ireland	Children's University Hospital Temple Street	Dublin
Ireland	University Hospital Limerick	Limerick
United Kingdom	Paediatric Clinical Research Facility	Edinburgh
United Kingdom	Alder Hey Children's Alder Hey Children's NHS Foundation Trust	Liverpool
United Kingdom	Great Ormond Street Hospital for Sick Children	London
United Kingdom	Royal Brompton & Harefield NHS Founcation Trust, Royal BromptonHospital	London
United Kingdom	Royal Manchester Children's Hospital	Manchester
United Kingdom	Oxford University Hospitals NHS Trust, John Radcliffe Hospital	Oxford
United States	Center for Advanced Pediatrics	Atlanta	Georgia
United States	John Hopkins Hospital	Baltimore	Maryland
United States	Billings Clinic Hospital	Billings	Montana
United States	University of Alabama at Birmingham	Birmingham	Alabama
United States	Boston Children's Hospital	Boston	Massachusetts
United States	Northwestern University	Chicago	Illinois
United States	Nationwide Children's Hospital	Columbus	Ohio
United States	Texas Children's Hospital	Houston	Texas
United States	Riley Hospital for Children at Indiana University Health	Indianapolis	Indiana
United States	Children's Mercy Hospital	Kansas City	Missouri
United States	University of Wisconsin	Madison	Wisconsin
United States	Childrens's Hospitals and Clinics of Minnnesota	Minneapolis	Minnesota
United States	Nemours Children's Hospital	Orlando	Florida
United States	Stanford University	Palo Alto	California
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	Seattle Children's Hospital	Seattle	Washington
United States	Alfred I DuPont Hospital for Children	Wilmington	Delaware

Sponsors (1)

Lead Sponsor	Collaborator
Vertex Pharmaceuticals Incorporated

Countries where clinical trial is conducted

United States,  Australia,  Canada,  Germany,  Ireland,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	from baseline through safety follow-up (up to 24 weeks after last dose)
Secondary	Absolute change in sweat chloride		from baseline through Week 96