MedTrace Logo

Study of Ivacaftor in Cystic Fibrosis Subjects 2 Through 5 Years of Age With a CFTR Gating Mutation

NCT01705145 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 35

Last updated 2016-04-05

Study results available
· View outcomes & findings →

Summary

The purpose of this study is to evaluate the safety, pharmacokinetics (PK), and pharmacodynamics (PD), of ivacaftor in children with cystic fibrosis (CF) who are 2 through 5 years of age and have a CF Transmembrane Conductance Regulator (CFTR) gating mutation in at least 1 allele.

Part A is designed to evaluate the safety and PK of multiple-dose administration of ivacaftor in participants 2 through 5 years of age and to confirm the doses for Part B. Part B is designed to evaluate the safety, PK, PD, and efficacy of ivacaftor in participants 2 through 5 years of age.

Conditions

Interventions

DRUG

Ivacaftor

Sponsors & Collaborators

Principal Investigators

  • Margaret Rosenfeld, MD · Seattle Children's Hospital

  • Jane Davies, MD · Imperial College London

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Max Age
5 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2013-01-31
Primary Completion
2014-03-31
Completion
2014-03-31

Countries

  • United States
  • Canada
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01705145 on ClinicalTrials.gov