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
Trade Name: Kaftrio
Product Name: 100mg ELX/50mg TEZ /75mg IVA
Product Code: VX-445/TEZ/IVA
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: Elexacaftor
CAS Number: 2216712-66-0
Current S
Sponsors
Vertex Pharmaceuticals Incorporated
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Subject (or the subject’s legally appointed and authorized representative) will sign and date an informed consent form (ICF) and, when appropriate, an assent form. 2. Willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines (as applicable), and other study procedures. - For subjects =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. History of any illness or any clinical condition that might confound the results of the study or pose an additional risk in administering study drug(s) to the subject. 2. History of drug intolerance in a parent study that would pose an additional risk to the subject. (e.g., subjects with a history of allergy or hypersensitivity to the study drug). 3. Pregnant and nursing females. Females of childbearing potential (Section 11.5.6.1) must have a negative pregnancy test at the Day 1 Visit before receiving the first dose of study drug. 4. Current participation in an investigational drug trial (other than a parent study). Participation in a noninterventional study (including observational studies, registry studies, and studies requiring blood collections without administration of study drug) 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 elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) ;Secondary Objective: To evaluate the long-term efficacy and pharmacodynamics (PD) of ELX/TEZ/IVA ;Primary end point(s): Safety and tolerability based on AEs, clinical laboratory values, ECGs, vital signs, and pulse oximetry;Timepoint(s) of evaluation of this end point: From signing of ICF until safety Follow up visit. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - Absolute change from baseline in percent predicted forced expiratory volume in 1 second (ppFEV1) - Absolute change from baseline in SwCl - Absolute change from baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score. - Absolute change from baseline in body mass index (BMI) - Absolute change from baseline in weight - Number of pulmonary exacerbations (PEx);Timepoint(s) of evaluation of this end point: From signing of ICF until safety Follow up visit. | — |
Countries
Austria, Belgium, Canada, Czechia, Czech Republic, France, Germany, Hungary, Italy, Netherlands, Norway, Poland, Portugal, Spain, Sweden, Switzerland
Contacts
Public ContactClinical Trials and Medical Info
Vertex Pharmaceuticals Incorporated
Outcome results
None listed