A study in children aged 2 to 5 with Cystic Fibrosis (a rare hereditary pulmonary disease) to assess the safety, pharmacokinetics and pharmacodynamics of ivacaftor

Phase 1

Conditions: cystic fibrosis
MedDRA version: 14.1 Level: PT Classification code 10011762 Term: Cystic fibrosis System Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Registration Number: EUCTR2012-000204-15-GB

Lead Sponsor: Vertex Pharmaceuticals Incorporated

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: Not specified

Target Recruitment: 35

Inclusion Criteria

•Male or female with confirmed diagnosis of CF
•Must have a CFTR gating mutation in at least 1 allele
•Aged 2 through 5 years at screening and Day 1
•Weight =8 kg 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

Are the trial subjects under 18? yes
Number of subjects for this age range: 35
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

•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 subject
•An acute upper or lower respiratory infection, or pulmonary exacerbation, or changes in therapy for pulmonary disease within 4 weeks before Day 1
•Abnormal liver function, at screening
•History of solid organ or hematological transplantation
•Use of any inducers or inhibitors of cytochrome P450 (CYP) 3A within 2 weeks before Day 1
•Participation in a clinical study involving administration of either an investigational or a marketed drug within 30 days or 5 terminal half-lives before screening

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate the safety of ivacaftor treatment in subjects with CF who are 2 through 5 years of age and have a CFTR gating mutation ;Secondary Objective: To evaluate the PK of ivacaftor and metabolites M1 and M6 in subjects with CF who are 2 through 5 years of age and have a CFTR gating mutation ;<br> Primary end point(s): Safety, as determined by adverse events, clinical laboratory values (serum chemistry, hematology, and coagulation studies), ECGs, vital signs, and ophthalmologic examinations<br> ;Timepoint(s) of evaluation of this end point: Through week 24

Secondary Outcome Measures

Name	Time	Method
<br> Secondary end point(s): •PK parameter estimation of ivacaftor and metabolites M1 and M6<br> •Absolute change from baseline in sweat chloride through 24 weeks of ivacaftor treatment <br> •Absolute change from baseline in weight at 24 weeks of ivacaftor treatment<br> •Absolute change from baseline in stature at 24 weeks of ivacaftor treatment<br> •Absolute change from baseline in body mass index (BMI) at 24 weeks of ivacaftor treatment<br> ;Timepoint(s) of evaluation of this end point: Through 24 weeks

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A study in children aged 2 to 5 with Cystic Fibrosis (a rare hereditary pulmonary disease) to assess the safety, pharmacokinetics and pharmacodynamics of ivacaftor

Recruitment & Eligibility

Study & Design

Related Trials

A study to assess the safety, pharmacokinetics, and pharmacodynamics of ivacaftor in children less than 24 months of age with cystic fibrosis (a rare hereditary disease that affects the lungs, digestive system and other organs).

A study to assess the safety, pharmacokinetics, and pharmacodynamics of ivacaftor in children less than 24 months of age with cystic fibrosis (a rare hereditary disease that affects the lungs, digestive system and other organs).

A study to assess the safety, pharmacokinetics, and pharmacodynamics of ivacaftor in children less than 24 months of age with cystic fibrosis (a rare hereditary disease that affects the lungs, digestive system and other organs).

A study to assess the safety and pharmacodynamics of long-term ivacaftor treatment in children less than 24 months of age with cystic fibrosis (a rare hereditary disease that affects the lungs, digestive system and other organs).

A study to assess the safety and pharmacodynamics of long-term ivacaftor treatment in children less than 24 months of age with cystic fibrosis (a rare hereditary disease that affects the lungs, digestive system and other organs).

A study to assess the safety and pharmacodynamics of long-term ivacaftor treatment in children less than 24 months of age with cystic fibrosis (a rare hereditary disease that affects the lungs, digestive system and other organs).

Study to Evaluate the Long-Term Efficacy and Safety of the Investigational Drug LIB003 for the Reduction of Cholesterol in Patients with Homozygous and Heterozygous Familial Hypercholesterolemia, Cardiovascular Disease, or at High Risk for Cardiovascular Disease.

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