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A Phase 3 Open-label Study Evaluating the Longterm Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects With Non-F508del CFTR Genotypes

Status
Active, not recruiting
Phases
Phase 3
Study type
Interventional
Source
EU CTIS
Registry ID
CTIS2024-515637-14-00
Acronym
VX21-445-125
Enrollment
295
Registered
2024-10-17
Start date
2022-11-08
Completion date
Unknown
Last updated
2025-11-14

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

Conditions

Cystic Fibrosis

Brief summary

Parts A and B: Safety and tolerability based on adverse events (AEs), clinical laboratory values, ECGs, vital signs, and pulse oximetry

Detailed description

Part A Only: Absolute change from baseline in percent predicted forced expiratory volume in 1 second (ppFEV1), Absolute change from baseline in sweat chloride (SwCl), Absolute change from baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain (RD) score, Absolute change from baseline in body mass index (BMI), Absolute change from baseline in weight, Number of pulmonary exacerbations (PEx)

Interventions

DRUGVX-770 Film-coated tablet
DRUGKaftrio 75 mg/50 mg/100 mg film-coated tablets
DRUGKalydeco 150 mg film-coated tablets
DRUGVX-445/VX-661/VX-770 film-coated fixed-dose combination tablet
DRUGKalydeco 75 mg film-coated tablets
DRUGVX-445/VX-661/VX-770 fixed-dose combination tablet
DRUGKaftrio 37.5 mg/25 mg/50 mg film-coated tablets

Sponsors

Vertex Pharmaceuticals Inc.
Lead SponsorINDUSTRY

Eligibility

Sex/Gender
All
Age
0 Years to 64 Years

Design outcomes

Primary

MeasureTime frame
Parts A and B: Safety and tolerability based on adverse events (AEs), clinical laboratory values, ECGs, vital signs, and pulse oximetry—

Secondary

MeasureTime frame
Part A Only: Absolute change from baseline in percent predicted forced expiratory volume in 1 second (ppFEV1), Absolute change from baseline in sweat chloride (SwCl), Absolute change from baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain (RD) score, Absolute change from baseline in body mass index (BMI), Absolute change from baseline in weight, Number of pulmonary exacerbations (PEx)—

Countries

Austria, Belgium, Czechia, France, Germany, Hungary, Italy, Netherlands, Norway, Poland, Portugal, Spain, Sweden

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026