Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis Participants 12 Months of Age and Older

NCT06460506 · tracked via the Priya Life Science UK tracker

Sponsor

Vertex Pharmaceuticals Incorporated

Phase

Phase 3

Started

2024-11-21

Last updated

2026-06-05

Condition(s) studied

Cystic Fibrosis

Investigational drug(s) / intervention(s)

ELX/TEZ/IVAIVA

ELX/TEZ/IVA: Fixed-dose combination granules for oral administration.

IVA: Granules for oral administration

Study summary

The purpose of the study is to evaluate the long-term safety, tolerability, efficacy, and pharmacodynamics (PD) of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA).

Eligibility

Sex

ALL

Min age

12 Months

Max age

—

Healthy volunteers

Key Inclusion Criteria: * Completed study drug treatment in the parent study VX22-445-122 Part B (NCT05882357) OR had study drug interruption(s) in the parent study but did not permanently discontinue study drug and completed study visits up to the last scheduled visit of the Treatment Period of the parent study Key Exclusion Criteria: * History of drug intolerance in the parent study that would pose an additional risk to the participant * Current participation in an investigational drug trial other than the parent study Other protocol defined Inclusion/Exclusion criteria may apply.

Primary outcome measure(s)

Safety and Tolerability as Assessed by Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) — Day 1 up to Week 100

Trial sites (18)

Facility	City	Region
The Kids Research Institute Australia	Nedlands	Australia
The Royal Children's Hospital	Parkville	Australia
Queensland Children's Hospital	South Brisbane	Australia
The Hospital for Sick Children	Toronto	Canada
British Columbia Children's Hospital	Vancouver	Canada
Juliane Marie Center, Rigshospitalet	Copenhagen	Denmark
Charité Universitätsmedizin BerlinX	Berlin	Germany
Kinderklinik III, Abt. fur Pneumologie	Essen	Germany
Medizinische Hochschule Hannover	Hanover	Germany
Erasmus Medical Center	Rotterdam	Netherlands
Inselspital - Universitaetsspital Bern	Bern	Switzerland
Kinderspital Zurich	Zurich	Switzerland
Children and Young Adults Research Unit	Cardiff	United Kingdom
Leeds General Infirmary	Leeds	United Kingdom
Alder Hey Children's NHS Foundation Trust	Liverpool	United Kingdom
Great Ormond Street Hospital for Children	London	United Kingdom
Royal Brompton Hospital	London	United Kingdom
Southampton General Hospital	Southampton	United Kingdom

More Vertex Pharmaceuticals Incorporated trials in the UK

A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy Active, not recruiting

A Safety, Tolerability, and Efficacy Study of VX-880 and VX-017 in Participants With Type 1 Diabetes Recruiting

Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and Older Enrolling by invitation

Evaluation of Efficacy, Safety, and Tolerability of Povetacicept in Participants With Primary Membranous Nephropathy (pMN) Recruiting

A Phase 2 Study to Evaluate Povetacicept in Adults With Generalized Myasthenia Gravis Recruiting

Other trials for the same condition

A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy Active, not recruiting

A Long-term Study to Monitor the Health Status of People With Cystic Fibrosis Who Took Part in a Previous Study With BI 3720931 (Lenticlair™-ON) Active, not recruiting

Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and Older Enrolling by invitation

A Phase 2 Study Evaluating Safety and Tolerability of RCT2100 (CFTR mRNA) in Healthy Participants and in Participants With CF Active, not recruiting

A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF) Active, not recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06460506 on ClinicalTrials.gov ↗ ← All trials in the UK