This study is currently not recruiting participants.

A Phase 3 2-Part Open-label Study to Evaluate the Safety Pharmacokinetics and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have a CFTR Gating Mutation

Investigating the Safety and Effects of an Investigational Medication in Young Children with Cystic Fibrosis

Not Recruiting
100 years or below
All
Phase N/A

Brief description of study.

The purpose of this study is to learn more about the safety and effects ivacaftor has in children with certain forms of cystic fibrosis who are less than 24 months of age when treatment begins.

Detailed description of study

The purpose of this study is to learn more about the safety and effects ivacaftor has in children with certain forms of cystic fibrosis who are less than 24 months of age when treatment begins.

Eligibility of study

You may be eligible for this study if you meet the following criteria:

  • Conditions: cystic fibrosis
  • Age: 100 years or below
  • Gender: All

The purpose of this study is to learn more about the safety and effects an investigational medication has in children with certain forms of cystic fibrosis who are less than 24 months of age when treatment begins. Cystic fibrosis is a genetic disorder that affects the lungs and digestive system, leading to severe respiratory and digestive problems.

Participants in this study will receive either the investigational medication or a placebo. A placebo is an inactive substance that looks like the investigational medication but does not contain any medicine. The study will monitor the safety and effects of the medication in young children.

  • Who can participate: Children less than 24 months of age with certain forms of cystic fibrosis are eligible to participate.
  • Study details: Participants will receive either the investigational medication or a placebo. A placebo is an inactive substance that looks like the investigational medication but does not contain any medicine.
Updated on 19 Feb 2024. Study ID: 1607606687

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