Cystic Fibrosis MedDRA version: 20.0 Level: PT Classification code 10011762 Term: Cystic fibrosis System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: VX-445 / TEZ / IVA FDC
Product Code: VX-445 / TEZ / IVA
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: IVACAFTOR
Sponsors
Vertex Pharmaceuticals Incorporated
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Subject (or his or her legally appointed and authorized representative) will sign and date an ICF, and, when appropriate, an assent form. 2. Willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines, and other study procedures. 3. Did not withdraw consent from the parent study. 4. Meets at least 1 of the following criteria: - Completed study drug treatment in the parent study. - Had study drug interruption(s) in the parent study, but did not permanently discontinue study drug, and completed study visits up to the last scheduled visit of the Treatment Period of the parent study. 5. Willing to remain on a stable CF treatment regimen (as defined in Section 9.5 of the protocol) through completion of study participation. Are the trial subjects under 18? yes Number of subjects for this age range: 56 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
Exclusion criteria: 1. History of any comorbidity 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. 2. Pregnant or breast-feeding females. Female subjects must have a negative pregnancy test at the Day 1 Visit before receiving the first dose of study drug. 3. History of drug intolerance in the parent study that would pose an additional risk to the subject in the opinion of the investigator (e.g., subjects with a history of allergy or hypersensitivity to the study drug). 4. Current participation in an investigational drug study other than the parent study. Participation in a noninterventional study and screening for another Vertex study is permitted.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the long-term safety and tolerability of VX-445/tezacaftor (TEZ)/ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are 6 years of age and older; Secondary Objective: To evaluate the long-term efficacy and pharmacodynamics (PD) of VX-445/TEZ/IVA ;Primary end point(s): Safety and tolerability assessments based on adverse events (AEs), clinical laboratory values, ECGs, vital signs, pulse oximetry, and ophthalmologic examinations;Timepoint(s) of evaluation of this end point: From Signing of ICF until the safety FU visit | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Secondary Endpoint Absolute change in percent predicted forced expiratory volume in 1 second (ppFEV1) Absolute change in sweat chloride (SwCl) Absolute change in CFQ-R respiratory domain (RD) score Absolute change in body mass index (BMI) and BMI-for-age z-score Number of pulmonary exacerbations (PEx) and CF-related hospitalizations Absolute change in lung clearance index2.5 (LCI2.5) Absolute change in weight and weight-for-age z-score Absolute change in height and height-for-age z-score Other endpoints Absolute change in fecal elastase-1 (FE-1) levels Absolute change in serum levels of immunoreactive trypsinogen (IRT) ;Timepoint(s) of evaluation of this end point: From Signing of ICF until the safety FU visit | — |
Countries
Australia, Canada, Ireland, United Kingdom, United States
Contacts
Public ContactClinical Trials and Medical Info
Vertex Pharmaceuticals Incorporated
Outcome results
None listed