EUCTR2017-001379-21-DE
Active, not recruiting
Phase 1
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
ConditionsCystic FibrosisMedDRA version: 20.0Level: PTClassification code 10011762Term: Cystic fibrosisSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic 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
- 2 years ago
Overview
Brief Summary
No summary available.
Investigators
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.
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
Outcomes
Primary Outcomes
Not specified
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