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: 100-mg VX-445 / 50-mg TEZ / 75-mg IVA FDC
Product Code: VX-445/TEZ/IVA
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: TEZACAFTOR
CAS Number: 1152311-62-0
Current Sponsor co
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 (informed consent form), 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 without prematurely discontinuing study drug in the parent study 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 through completion of study participation. Are the trial subjects under 18? yes Number of subjects for this age range: 75 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 175 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 illness or any clinical condition 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 and breast-feeding females. All female subjects, regardless of childbearing potential status, 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 non-interventional 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 VX-445/tezacaftor (TEZ)/ivacaftor (IVA) in subjects with cystic fibrosis who are heterozygous for the F508del mutation and a gating (F/G) or residual function (F/RF) mutation;Secondary Objective: - To evaluate the long-term efficacy of VX-445/TEZ/IVA - To evaluate the pharmacodynamics (PD) of VX-445/TEZ/IVA;Primary end point(s): Safety and tolerability of long-term treatment with VX-445/TEZ/IVA based on adverse events (AEs), clinical laboratory values, ECGs, vital signs, and pulse oximetry.;Timepoint(s) of evaluation of this end point: Safety and tolerability assessments: a. AEs: Continuous (signing of ICF until completion of study participation) b. clinical laboratory values, vital signs, pulse oximetry: day 1; day 15; weeks 4, 8, 16, 24, 36, 48, 60, 72, 84, 96; Early Termination of Treatment (ETT) visit (if applicable); Safety Follow-up (SFU) visit (if applicable) c. ECGs: day 1; day 15; weeks 4, 8, 24, 48, 72, 96; ETT visit (if applicable); SFU visit (if applicable) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - Absolute change in percent predicted forced expiratory volume in 1 second (ppFEV1) - Absolute change in sweat chloride (SwCl) - Absolute change in body mass index (BMI) - Absolute change in BMI z-score - Absolute change in body weight - Absolute change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain (RD) score;Timepoint(s) of evaluation of this end point: - ppFEV1: day 1; day 15; weeks 4, 8, 16, 24, 36, 48, 60, 72, 84, 96; ETT visit (if applicable); SFU visit (if applicable) - SwCl: day 1; day 15; weeks 4, 8, 24, 48, 72, 96, ETT visit (if applicable) - BMI: day 1; day 15; weeks 4, 8, 16, 24, 36, 48, 60, 72, 84, 96; ETT visit (if applicable); SFU visit (if applicable) - BMI z-score: day 1; day 15; weeks 4, 8, 16, 24, 36, 48, 60, 72, 84, 96; ETT visit (if applicable); SFU visit (if applicable) - body weight: day 1; day 15; weeks 4, 8, 16, 24, 36, 48, 60, 72, 84, 96; ETT visit (if applicable); SFU visit (if applicable) - CFQ-R RD score: day 1; weeks 4, 8, 24, 48, 72, 96; ETT visit (if applicable); SFU visit (if applicable) | — |
Countries
Australia, Belgium, Canada, Denmark, France, Germany, Ireland, Italy, Netherlands, Spain, United Kingdom, United States
Contacts
Public ContactClinical Trials and Medical Info
Vertex Pharmaceuticals Incorporated
Outcome results
None listed