Cystic Fibrosis MedDRA version: 18.1 Level: PT Classification code 10011762 Term: Cystic fibrosis System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: Ivacaftor
Product Code: VX-770
Pharmaceutical Form: Granules
INN or Proposed INN: IVACAFTOR
CAS Number: 873054-44-5
Current Sponsor code: VX-770
Concentration unit: mg milligram(s)
Conce
Sponsors
Vertex Pharmaceuticals Incorporated
Eligibility
Sex/Gender
All
Inclusion criteria
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 =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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 • To evaluate disease progression as measured by changes in pancreatic function • To evaluate the safety of ivacaftor treatment, as measured by clinical laboratory measures (including liver function tests [LFTs]), ophthalmologic examinations, and adverse events (AEs) ;Primary end point(s): Part 1 Absolute change from baseline in LCI2.5 through 8 weeks of treatment;Timepoint(s) of evaluation of this end point: 8 weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Part 1 • Absolute change from baseline in serum levels of immunoreactive trypsinogen at 8 weeks of treatment • Absolute change from baseline in fecal elastase-1 at 8 weeks of treatment • Absolute change from baseline in weight at 8 weeks of treatment • Absolute change from baseline in body mass index (BMI) at 8 weeks of treatment • Safety, as determined by AEs, clinical laboratory values (including LFTs), and other relevant assessments Part 2: • Absolute change from baseline in LCI2.5 at Weeks 48, 96, and 144 • Absolute change from baseline in CT scan at Week 96 • Absolute change from baseline in serum levels of immunoreactive trypsinogen at Weeks 48, 96, and 144 • Absolute change from baseline in fecal elastase-1 at Weeks 48, 96, and 144 • Absolute change from baseline in weight-for-age z-score at Weeks 48, 96, and 144 • Absolute change from baseline in height-for-age z-score at Weeks 48, 96, and 144 • Absolute change from baseline in BMI-for-age z-score at Weeks 48, 96, and 144 • Safety, as determined by AEs, clinical laboratory values (including LFTs), and ophthalmologic examinations ;Timepoint(s) of evaluation of this end point: Part 1: 8 weeks Part 2: •Absolute change from baseline in LCI2.5 at Weeks 48, 96, and 144 • Absolute change from baseline in CT scan at Week 96 • Absolute change from baseline in serum levels of immunoreactive trypsinogen at Weeks 48, 96, and 144 • Absolute change from baseline in fecal elastase-1 at Weeks 48, 96, and 144 • Absolute change from baseline in weight-for-age z-score at Weeks 48, 96, and 144 • Absolute change from baseline in height-for-age z-score at Weeks 48, 96, and 144 • Absolute change from baseline in BMI-for-age z-score at Weeks 48, 96, and 144 • Safety: throughout the study | — |
Countries
Australia, Canada, France, United Kingdom
Contacts
Public ContactClinical Trials and Medical Info
Vertex Pharmaceuticals Incorporated
Outcome results
None listed