Completed

A Phase 3, 2-Part, Open-Label Study to Evaluate the Safety, Pharmacokinetics and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are 2 Through 5 Years of Age and Have a CFTR Gating Mutation

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What is being tested

Ivacaftor

Drug
Who is being recruted

Digestive System Diseases+5

+ Infant, Newborn, Diseases

+ Lung Diseases

From 2 to 5 Years
+11 Eligibility Criteria
See all eligibility criteria
How is the trial designed

Treatment Study

Phase 3
Interventional
Study Start: January 2013
See protocol details

Summary

Principal SponsorVertex Pharmaceuticals Incorporated
Last updated: April 5, 2016
Sourced from a government-validated database.Claim as a partner

Study start date: January 1, 2013

Actual date on which the first participant was enrolled.

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.

NCT01705145
Principal SponsorVertex Pharmaceuticals Incorporated
Last updated: April 5, 2016
Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.
Design Details

35 patients to be enrolled

Total number of participants that the clinical trial aims to recruit.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.
Conditions
Criteria

Any sex

Biological sex of participants that are eligible to enroll.

From 2 to 5 Years

Range of ages for which participants are eligible to join.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

Digestive System DiseasesInfant, Newborn, DiseasesLung DiseasesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesPancreatic DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCystic Fibrosis

Criteria

5 inclusion criteria required to participate
Aged 2 through 5 years at screening and Day 1
Hematology, serum chemistry, coagulation, and vital signs results at screening with no clinically significant abnormalities that would interfere with the study assessments, as judged by the investigator
Male or female with confirmed diagnosis of CF
Must have a CFTR gating mutation in at least 1 allele
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6 exclusion criteria prevent from participating
Abnormal liver function, at screening
An acute upper or lower respiratory infection, or pulmonary exacerbation, or changes in therapy for pulmonary disease within 4 weeks before Day 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 study drug to the participant
History of solid organ or hematological transplantation
Show More Criteria

Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.
Treatment Groups
Study Objectives

One single intervention group is designated in this study

This study does not include a placebo group 

Treatment Groups

Group I

Experimental
Part A: Ivacaftor 50 milligram (mg) (for participants weighing less than [<] 14 kilograms [kg]) or 75 mg (for participants weighing greater than or equal to [>=] 14 kg) every 12 hours (q12h) from Day 1 through Day 3 and 1 morning dose on Day 4 during Part A of the study.. Part B: Ivacaftor 50 mg (for participants weighing <14 kg) or 75 mg (for participants weighing >=14 kg) q12h for 24 weeks during Part B of the study.

Study Objectives

Primary Objectives

Secondary Objectives

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.

This study has 20 locations

Birmingham, United StatesOpen in Google Maps

Aurora, United States

Atlanta, United States

Indianapolis, United States
Completed20 Study Centers