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

Vertex Pharmaceuticals Incorporated0 sites75 target enrollmentJuly 20, 2017

ConditionsCystic Fibrosis MedDRA version: 20.0Level: PTClassification code 10011762Term: Cystic fibrosisSystem Organ Class: 10010331 - Congenital, familial and genetic disorders Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

DrugsKalydeco

Overview

Phase: Phase 1
Intervention: Not specified
Conditions: Cystic Fibrosis
Sponsor: Vertex Pharmaceuticals Incorporated
Enrollment: 75
Status: Active, not recruiting
Last Updated: 5 years ago

Overview Investigators Eligibility Criteria Outcomes Similar Trials

Overview

Brief Summary

No summary available.

Registry

who.int

Start Date

July 20, 2017

End Date

TBD

Last Updated

5 years ago

Study Type

Interventional clinical trial of medicinal product

Sex

All

Investigators

Sponsor

Vertex Pharmaceuticals Incorporated

Eligibility Criteria

Inclusion Criteria

•Ivacaftor Arm: Subjects From Study 124 Part B
•1\. Subjects transitioning from Study 124 Part B must have completed the last study visit of Study 124 Part B
•Added guidance:
•\- Subjects who had a study drug interruption at the last scheduled visit of Study 124 Part B, subjects who required study drug interruption that was to be continued or initiated at Day 1 in Study 126, or subjects who resumed study drug in Study 124 Part B after a study drug interruption due to elevated transaminases but who did not complete at least 4 weeks of rechallenge with study drug (due to the timing of the rechallenge versus the time remaining in Study 124 Part B) must meet eligibility criteria and have received approval from the Vertex medical monitor.
•2\. For Gap Transition subjects: hematology and serum chemistry results at baseline with no clinically significant abnormalities that would confound the study assessments or pose an additional risk to administering ivacaftor to the study subject, as judged by the investigator.
•3\. 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
•1\. Age \<24 months at the Day 1 Visit
•2\. Confirmed diagnosis of CF, defined as a sweat chloride value \=60 mmol/L by quantitative pilocarpine iontophoresis or 2 CF\-causing mutations
•\- If the results of the sweat chloride and/or the genotype test are documented in the subject’s medical record, and the historic genotype result is approved by the Vertex medical monitor, the tests do not need to be performed at screening. A sweat chloride test must be performed if the sweat chloride value is not available in the subject’s medical records and the value is needed to establish eligibility.

Exclusion Criteria

•Ivacaftor Arm: Subjects From Study 124 Part B
•1\. 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.
•Examples of subjects who may not be eligible include
•\- subjects with a history of allergy or hypersensitivity to the study drug; and
•\- subjects with severe or life\-threatening reactions to the study drug in Study 124\.
•2\. Subjects receiving commercially available ivacaftor treatment
•Ivacaftor Arm: Subjects Not From Study 124 Part B
•1\. 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
•2\. An acute upper or lower respiratory infection, or pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 4 weeks of Day 1
•3\. Known colonization with organisms associated with a more rapid decline in pulmonary status (e.g., Burkholderia cenocepacia, Burkholderia dolosa, and Mycobacterium abscessus) at screening. A subject is excluded if they have a positive culture of one of these organisms.