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NCT05331183Phase 3ACTIVE NOT RECRUITING

Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Long-term Safety and Efficacy in Subjects Without F508del

Study Summary

This study will evaluate the long-term safety, efficacy and pharmacodynamics of ELX/TEZ/IVA in participants with cystic fibrosis (CF) with at least 1 non-F508del ELX/TEZ/IVA-responsive CF transmembrane conductance regulator (CFTR) gene mutation.

Conditions
Cystic Fibrosis
Interventions / Drugs
ELX/TEZ/IVAIVA
View on ClinicalTrials.gov ↗
Sponsor
VRTX
Vertex Pharmaceuticals
Gene Therapy
F22/100
Study Details
Enrollment297 participants
Primary Completion—
Lead SponsorVertex Pharmaceuticals Incorporated
Data Retrieved2026-04-11 02:55:58.035116+00:00
What This Trial Means for Biotech Investors
Trial Phase Context

Phase 3 trials are pivotal studies with large patient populations (hundreds to thousands) designed to prove efficacy and safety for FDA submission. A successful Phase 3 readout is typically the last major hurdle before a New Drug Application (NDA) or BLA filing.

Status & Expected Data

Enrollment is complete. The trial is in its follow-up or data collection phase — a readout may occur on or near the primary completion date.

Enrolled: 297 participants
Sponsor Catalyst Profile

Vertex Pharmaceuticals (VRTX) is the sponsoring company for this trial. BiotechSign currently grades this company F (22/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.

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