NCT06460506

Brief 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).

Trial Health

82
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
50

participants targeted

Target at below P25 for phase_3

Timeline
17mo left

Started Nov 2024

Typical duration for phase_3

Geographic Reach
7 countries

18 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress51%
Nov 2024Sep 2027

First Submitted

Initial submission to the registry

June 10, 2024

Completed
4 days until next milestone

First Posted

Study publicly available on registry

June 14, 2024

Completed
5 months until next milestone

Study Start

First participant enrolled

November 21, 2024

Completed
2.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 30, 2027

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 30, 2027

Last Updated

October 6, 2025

Status Verified

June 1, 2025

Enrollment Period

2.9 years

First QC Date

June 10, 2024

Last Update Submit

October 3, 2025

Conditions

Cystic Fibrosis

Outcome Measures

Primary Outcomes (1)

  • Safety and Tolerability as Assessed by Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Day 1 up to Week 100

Secondary Outcomes (1)

  • Absolute Change in Sweat Chloride (SwCl)

    From Baseline through Week 96

Study Arms (1)

ELX/TEZ/IVA

EXPERIMENTAL

Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening.

Drug: ELX/TEZ/IVADrug: IVA

Interventions

ELX/TEZ/IVADRUG

Fixed-dose combination granules for oral administration.

Also known as: VX-445/VX-661/VX-770, elexacaftor/tezacaftor/ivacaftor
ELX/TEZ/IVA
IVADRUG

Granules for oral administration

Also known as: VX-770, ivacaftor
ELX/TEZ/IVA

Eligibility Criteria

Age12 Months+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (18)

The Kids Research Institute Australia

Nedlands, Australia

Location

The Royal Children's Hospital

Parkville, Australia

Location

Queensland Children's Hospital

South Brisbane, Australia

Location

The Hospital for Sick Children

Toronto, Canada

Location

British Columbia Children's Hospital

Vancouver, Canada

Location

Juliane Marie Center, Rigshospitalet

Copenhagen, Denmark

Location

Charité Universitätsmedizin BerlinX

Berlin, Germany

Location

Kinderklinik III, Abt. fur Pneumologie

Essen, Germany

Location

Medizinische Hochschule Hannover

Hanover, Germany

Location

Erasmus Medical Center

Rotterdam, Netherlands

Location

Inselspital - Universitaetsspital Bern

Bern, Switzerland

Location

Kinderspital Zurich

Zurich, Switzerland

Location

Children and Young Adults Research Unit

Cardiff, United Kingdom

Location

Leeds General Infirmary

Leeds, United Kingdom

Location

Alder Hey Children's NHS Foundation Trust

Liverpool, United Kingdom

Location

Great Ormond Street Hospital for Children

London, United Kingdom

Location

Royal Brompton Hospital

London, United Kingdom

Location

Southampton General Hospital

Southampton, United Kingdom

Location

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

elexacaftor, ivacaftor, tezacaftor drug combinationivacaftor

Condition Hierarchy (Ancestors)

Pancreatic DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, Diseases

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 10, 2024

First Posted

June 14, 2024

Study Start

November 21, 2024

Primary Completion (Estimated)

September 30, 2027

Study Completion (Estimated)

September 30, 2027

Last Updated

October 6, 2025

Record last verified: 2025-06

Data Sharing

IPD Sharing
Will not share

Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent research/clinical-trial-data-sharing

Locations

AU(3)CA(2)DK(1)DE(3)NL(1)CH(2)GB(6)