Clinical trial NCT02742519: A Study to Evaluate Efficacy and Safety of Ivacaftor in Subjects With Cystic Fibrosis Aged 3 Through 5 Years Who Have a Specified CFTR Gating Mutation
Official title: A Phase 3b, 2-part, Randomized, Double-blind, Placebo-controlled Crossover Study With a Long-term Open-label Period to Investigate Ivacaftor in Subjects With Cystic Fibrosis Aged 3 Through 5 Years Who Have a Specified CFTR Gating Mutation
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Cystic Fibrosis
Lead sponsor: Vertex Pharmaceuticals Incorporated
Sponsor class: INDUSTRY
First posted: 2016-04-19
Enrollment: 14
Sex: ALL
Minimum age (years): 3
Maximum age (years): 5
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

Brief summary: To evaluate the efficacy of ivacaftor treatment, as measured by lung clearance index (LCI), in subjects with cystic fibrosis (CF) who have a specified CF transmembrane conductance regulator (CFTR) gating mutation


Eligibility criteria: Inclusion Criteria:

* Male or female with confirmed diagnosis of CF.
* Must have 1 of the following CFTR gating mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D.
* 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.

Exclusion Criteria:

* An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 4 weeks before Day 1
* 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)
* 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
* History of solid organ or hematological transplantation
* Any clinically significant "non-CF-related" illness within 2 weeks before Day 1
* Use of any moderate or strong 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 (whichever is longer or as determined by the local requirements) before Screening

Primary outcomes:
measure: Absolute Change From Baseline in Lung Clearance Index (LCI2.5) Through 8 Weeks of Treatment (Average of Week 4 and Week 8 LCI2.5)
timeFrame: Baseline Through Week 8 for each treatment period, Up to 24 Weeks
Secondary outcomes:
measure: Absolute Change From Baseline in Immunoreactive Trypsinogen Levels at Week 8
timeFrame: Baseline and Week 8 of each treatment period, Up to 24 Weeks
measure: Absolute Change From Baseline in Fecal Elastase-1 Levels at Week 8
timeFrame: Baseline and Week 8 of each treatment period, Up to 24 Weeks
measure: Absolute Change From Baseline in Weight at Week 8
timeFrame: Baseline and Week 8 of each treatment period, Up to 24 Weeks
measure: Absolute Change From Baseline in Body Mass Index (BMI) at Week 8
timeFrame: Baseline and Week 8 of each treatment period, Up to 24 Weeks
measure: Number of Subjects With Adverse Events (AEs) and Serious Adverse Events (SAEs)
timeFrame: Baseline up to Month 15
Other outcomes:
Locations:
city: Parkville
country: Australia
state: Victoria
city: South Brisbane
country: Australia
city: Subiaco
country: Australia
city: Westmead
country: Australia
city: Toronto
country: Canada
state: Ontario
city: London
country: United Kingdom
Study officials:
References: