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: VX-445
CAS Number: 2216712-66-0
Current Sponsor code: VX-
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 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, and other study procedures. 3. Did not withdraw consent from a parent study. 4. Meets at least 1 of the following criteria: Completed study drug treatment in a parent study. Had study drug interruption(s) in a parent study, but completed study visits up to the last scheduled visit of the Treatment Period of a parent study. 5. Willing to remain on a stable CF treatment regimen (as defined in Section 9.5) through completion of study participation. Are the trial subjects under 18? yes Number of subjects for this age range: 90 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 370 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 and nursing females. Females of childbearing potential 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 a 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 trial (other than a 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 in TC with TEZ and IVA in subjects with CF who are homozygous or heterozygous for the F508del mutation;Secondary Objective: To evaluate the long-term efficacy of VX-445 in TC with TEZ and IVA To evaluate the pharmacodynamics (PD) of VX-445 in TC with TEZ and IVA;Primary end point(s): Safety and tolerability of long-term treatment with VX-445 in TC with TEZ and IVA based on adverse events (AEs), clinical laboratory values, ECGs, vital signs, and pulse oximetry.;Timepoint(s) of evaluation of this end point: from baseline through safety follow-up (up to 196 weeks) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Secondary Endpoints Absolute change from baseline in ppFEV1 Absolute change in sweat chloride (SwCl) Number of pulmonary exacerbations (PEx) Time-to-first PEx Absolute change in body mass index (BMI) Absolute change in BMI z-score Absolute change in body weight Absolute change from baseline in CFQ-R respiratory domain score Additional Endpoints Absolute change in CFQ-R non-respiratory domain scores Changes in inflammatory mediators Changes in microbiology analysis;Timepoint(s) of evaluation of this end point: from baseline through last dose of study drug (up to 192 weeks) | — |
Countries
Australia, Austria, Belgium, Canada, Czechia, Czech Republic, France, Germany, Greece, Italy, Netherlands, Sweden, United Kingdom, United States
Contacts
Public ContactClinical Trials and Medical Info
Vertex Pharmaceuticals Incorporated
Outcome results
None listed