A study to assess the efficacy and safety of ivacaftor in children aged 3 to 5 with Cystic Fibrosis (a rare hereditary disease that affects the lungs, digestive system and other organs).

Phase 1

Conditions: Cystic Fibrosis
MedDRA version: 18.1Level: PTClassification code 10011762Term: Cystic fibrosisSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Registration Number: EUCTR2015-001267-39-FR

Lead Sponsor: Vertex Pharmaceuticals Incorporated

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: A

Sex: All

Target Recruitment: 50

Inclusion Criteria

1. Male or female with confirmed diagnosis of CF, defined as:
- a sweat chloride value =60 mmol/L by quantitative pilocarpine iontophoresis
OR
2 CF causing mutations (all as documented in the subject's medical record)
AND
- Clinical manifestations of CF
2. Must have 1 of the following CFTR gating mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D.
3. Subjects will be between the ages of 3 and 5 years, inclusive, at Screening and Day 1
4. Weight =8 kg to <25 kg at Screening and Day 1
5. Hematology, serum chemistry, and coagulation at Screening with no clinically significant abnormalities or concomitant diagnosis that would interfere with the LCI and CT scan study assessments, as judged by the investigator

Are the trial subjects under 18? yes
Number of subjects for this age range: 50
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

1. History of any illness or condition that, in the opinion of the investigator, might confound the results of the study, impact use of the LCI or CT scan as assessments or pose an additional risk in administering study drug to the subject
2. An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 4 weeks before Day 1
3. Any clinically significant laboratory abnormalities at the Screening Visit that would interfere with the study assessments or pose an undue risk for the subject (in the opinion of the investigator)
4. Abnormal liver function, at Screening, defined as 3 × upper limit of normal (ULN), of any 3 or more of the following: serum aspartate transaminase (AST), serum alanine transaminase (ALT), gamma-glutamyl transpeptidase (GGT), serum alkaline phosphatase (ALP), and total bilirubin
5. History of solid organ or hematological transplantation
6. Any clinically significant non-CF-related illness within 2 weeks before Day 1. Illness is defined as an acute (serious or nonserious) condition (e.g., gastroenteritis)
7. Use of any moderate or strong inducers or inhibitors of cytochrome P450 (CYP) 3A within 2 weeks before Day 1
8. Participation in a clinical study involving administration of either an investigational or a marketed drug within 30 days or 5 terminal half-lives (whichever is longer or as determined by the local requirements) 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 efficacy of ivacaftor treatment in subjects with cystic fibrosis (CF) who have a specified CF transmembrane conductance regulator (CFTR) gating mutation, and who are 3 through 5 years of age at the start of the study, as measured by lung clearance index (LCI);Secondary Objective: • To evaluate disease progression as measured by changes in computed tomography (CT) scan<br>• To evaluate disease progression as measured by changes in pancreatic function<br>• To evaluate the safety of ivacaftor treatment, as measured by clinical laboratory measures (including liver function tests [LFTs]), ophthalmologic examinations, and adverse events (AEs)<br>;Primary end point(s): Part 1<br>Absolute change from baseline in LCI2.5 through 8 weeks of treatment;Timepoint(s) of evaluation of this end point: 8 weeks

Secondary Outcome Measures

Name	Time	Method

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