NCT04058366

Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy

A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 251 (actual)
• Sponsor: Vertex Pharmaceuticals Incorporated · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

This study evaluated the long-term safety, efficacy, and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for the F508del mutation and a gating or residual function mutation (F/G and F/RF genotypes).

Conditions

• Cystic Fibrosis

Interventions

Type	Name	Description
DRUG	ELX/TEZ/IVA	Fixed-dose combination (FDC) tablet for oral administration.
DRUG	IVA	Tablet for oral administration.

Timeline

• Start date: 2019-12-05
• Primary completion: 2022-12-16
• Completion: 2022-12-16
• First posted: 2019-08-15
• Last updated: 2024-01-16
• Results posted: 2024-01-16

Locations

84 sites across 12 countries: United States, Australia, Belgium, Canada, Denmark, France, Germany, Ireland, Italy, Netherlands, Spain, United Kingdom

Regulatory

• FDA-regulated drug study