A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis (CF) Who Are Homozygous or Heterozygous for the F508del Mutation

A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis (CF) Who Are Homozygous or Heterozygous for the F508del Mutation - Phase3 Open-label: VX-445 Combination Therapy in Subjects With CF(F/F&F/MF)

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

NL-OMON

Registry ID

NL-OMON52615

Enrollment

Registered

2018-09-19

Start date

2018-11-29

Completion date

Unknown

Last updated

2025-08-18

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Cystic fibrosis

Interventions

Study drug refers to VX-445/TEZ/IVA. Study drugs will be orally administered as 2 fixed-dose combination (FDC) film-coated tablets (VX-445/TEZ/IVA) in the morning and as 1film-coated IVA tablet in t

VX-661), and IVA (ivacaftor

VX-770) Activity:CFTR corrector, CFTR corrector, and CFTR potentiator (increased Cl* secretion) Strength:100-mg VX-445/50mg TEZ/75mg IVA FDC tablet Active substance: IVA (ivacaftor

VX-770) Activity: CFTR potentiator (increased Cl- secretion) Strength: 150-mg tablet

Eligibility

Age

12 Years to 64 Years

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.

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
To evaluate the long-term safety and tolerability of VX-445 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA)in subjects with cystic fibrosis (CF) who are homozygous or heterozygous for the F508del mutation	—

Secondary

Measure	Time frame
Secondary Objectives: 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	—

Countries

Netherlands

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)