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

April 23, 2026 updated by: Vertex Pharmaceuticals Incorporated

A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 1 Year of Age and Older

The purpose of this study is to evaluate the long-term safety, tolerability, and efficacy of vanzacaftor/tezacaftor/deutivacaftor (VNZ/TEZ/D-IVA) in participants with cystic fibrosis (CF).

Study Overview

Status

Enrolling by invitation

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

174

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
      • Nedlands, Australia
        • The Kids Research Institute Australia
      • North Adelaide, Australia
        • Women's & Children's Hospital
      • Parkville, Australia
        • The Royal Children's Hospital Melbourne
      • South Brisbane, Australia
        • Children's Health Queensland Hospital and Health Service
  • Canada
      • Toronto, Canada
        • Hospital for Sick Children - Pulmonology
      • Vancouver, Canada
        • British Columbia Children's Hospital
  • France
      • Bron, France
        • Hopital Femme Mere-Enfant
      • Paris, France
        • Hopital Necker Enfants Malades - Pulmonology
  • Germany
      • Berlin, Germany
        • Charite Paediatric Pulmonology Department
      • Essen, Germany
        • Universitätsklinikum Essen
      • Hanover, Germany
        • Medizinische Hochschule Hannover - Clinic for Pediatric Pneumology, Allergology and Neonatology, CF-Centre
  • Netherlands
      • Rotterdam, Netherlands
        • Sophia Children's Hospital
  • New Zealand
      • Grafton, New Zealand
        • Starship Child Health
  • Sweden
      • Gothenburg, Sweden
        • Sahlgrenska Universitetssjukhuset - Göteborg CF-center
      • Stockholm, Sweden
        • Karolinska University Hospital - Pulmonology
  • Switzerland
      • Bern, Switzerland
        • Inselspital Bern
      • Zurich, Switzerland
        • Kinderspital Zurich - Abteilung Pneumologie
  • United Kingdom
      • Cardiff, United Kingdom
        • Noah's Ark Children's Hospital for Wales
      • London, United Kingdom
        • Great Ormond Street Hospital for Children
  • United States
    • California
      • Orange, California, United States, 92868
        • Children's Hospital of Orange County
      • Palo Alto, California, United States, 94304
        • Stanford University - Palo Alto - Pulmonology
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Children's Hospital of Colorado - Pulmonology
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • The Emory Clinic - Clifton Road - Pulmonology
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Ann & Robert H. Lurie Children's Hospital of Chicago - Hematology
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • JW Riley Hospital for Children - Pulmonology
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Boston Children's Hospital
    • Minnesota
      • Minneapolis, Minnesota, United States, 55404
        • Children's Health Care d/b/a Children's Hospitals and Clinics of Minnesota
    • Missouri
      • Kansas City, Missouri, United States, 64108
        • The Children's Mercy Hospital
      • St Louis, Missouri, United States, 63110
        • St. Louis Children's Hospital - Pulmonology
    • New York
      • Lake Success, New York, United States, 11042
        • Cohen Children's Medical Center - Lakeville Road
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Children's Hospital Medical Center - Pulmonology
      • Cleveland, Ohio, United States, 44106
        • UH Cleveland Medical Center - Pulmonology
      • Columbus, Ohio, United States, 43205
        • Nationwide Children's Hospital
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15224
        • Children's Hospital of Pittsburgh - Pulmonology
    • Texas
      • Houston, Texas, United States, 77030
        • Texas Children's Hospital - Wallace Tower - Pulmonology
    • Vermont
      • Colchester, Vermont, United States, 05446
        • Vermont Lung Center
    • Wisconsin
      • Madison, Wisconsin, United States, 53792
        • American Family Childrens Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Key Inclusion Criteria:

Parts A and B:

• Participants who have completed study drug treatment in the parent study (VX21-121-105; NCT Number: NCT05422222)

Part B:

-Meets at least 1 of the following criteria:

  • Completed study drug treatment in Part A
  • Had study drug interruption(s) in Part A, but did not permanently discontinue study drug and completed study visits up to the last scheduled visit of the treatment period of Part A

Key Exclusion Criteria:

  • Hepatic cirrhosis with portal hypertension, moderate hepatic impairment, or severe hepatic impairment that might pose an additional risk in administering study drug
  • History of solid organ, hematological transplantation, or cancer
  • History of drug intolerance in the parent study

Other protocol defined Inclusion/Exclusion criteria may apply.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: VNZ/TEZ/D-IVA

Part A: Participants (Cohort 1: 6 through 11 years of age (inclusive); Cohort 2: 2 through 5 years of age (inclusive) and Cohort 3: 1 to less than (<) 2 years of age) will receive VNZ/TEZ/D-IVA once daily at doses determined based on their age and weight. The age range is based on date of informed consent in parent study.

Part B: Participants in Cohort 1 who meet the eligibility criteria will receive VNZ/TEZ/D-IVA for an additional 96 weeks.
Drug: VNZ/TEZ/D-IVA
Fixed-dose combination tablets or granules for oral administration.
Other Names:
  • VX-121/VX-661/CTP-656
  • VX-121/VX-661/VX-561
  • vanzacaftor/tezacaftor/deutivacaftor

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)
Time Frame: From Baseline up to Week 100
From Baseline up to Week 100
Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)
Time Frame: From Baseline up to Week 196
From Baseline up to Week 196

Secondary Outcome Measures

Outcome Measure
Time Frame
Part A (All Cohorts): Absolute Change in Sweat Chloride (SwCl)
Time Frame: From Baseline Through Week 96
From Baseline Through Week 96
Part B: Absolute Change in Sweat Chloride (SwCl)
Time Frame: From Baseline Through Week 192
From Baseline Through Week 192
Part A (Cohort 1): Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1)
Time Frame: From Baseline Through Week 100
From Baseline Through Week 100
Part B: Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1)
Time Frame: From Baseline Through Week 196
From Baseline Through Week 196
Part A (All Cohorts): Number of Pulmonary Exacerbation (PEx)
Time Frame: From Baseline Through Week 100
From Baseline Through Week 100
Part B: Number of Pulmonary Exacerbation (PEx)
Time Frame: From Baseline Through Week 196
From Baseline Through Week 196
Part A (All Cohorts): Number of CF- Related Hospitalizations
Time Frame: From Baseline Through Week 100
From Baseline Through Week 100
Part B: Number of CF- Related Hospitalizations
Time Frame: From Baseline Through Week 196
From Baseline Through Week 196
Part A (Cohort 1): Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score
Time Frame: From Baseline Through Week 100
From Baseline Through Week 100
Part B: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score
Time Frame: From Baseline Through Week 196
From Baseline Through Week 196
Part A (Cohorts 1 and 2): Absolute Change in Body Mass Index (BMI)
Time Frame: From Baseline Through Week 100
From Baseline Through Week 100
Part B: Absolute Change in Body Mass Index (BMI)
Time Frame: From Baseline Through Week 196
From Baseline Through Week 196
Part A (Cohorts 1 and 2): Absolute Change in BMI-for-age Z-score
Time Frame: From Baseline Through Week 100
From Baseline Through Week 100
Part B: Absolute Change in BMI-for-age Z-score
Time Frame: From Baseline Through Week 196
From Baseline Through Week 196
Part A (Cohort 3): Absolute Change in Weight-for-length
Time Frame: From Baseline Through Week 100
From Baseline Through Week 100
Part B: Absolute Change in Weight-for-length
Time Frame: From Baseline Through Week 196
From Baseline Through Week 196
Part A (Cohort 3): Absolute Change in Weight-for-length Z-score
Time Frame: From Baseline Through Week 100
From Baseline Through Week 100
Part B: Absolute Change in Weight-for-length Z-score
Time Frame: From Baseline Through Week 196
From Baseline Through Week 196
Part A (All Cohorts): Absolute Change in Weight
Time Frame: From Baseline Through Week 100
From Baseline Through Week 100
Part B: Absolute Change in Weight
Time Frame: From Baseline Through Week 196
From Baseline Through Week 196
Part A (All Cohorts): Change in Weight-for-age Z-score
Time Frame: From Baseline Through Week 100
From Baseline Through Week 100
Part B: Change in Weight-for-age Z-score
Time Frame: From Baseline Through Week 196
From Baseline Through Week 196
Part A (Cohorts 1 and 2): Absolute Change in Height
Time Frame: From Baseline Through Week 100
From Baseline Through Week 100
Part B: Absolute Change in Height
Time Frame: From Baseline Through Week 196
From Baseline Through Week 196
Part A (Cohorts 1 and 2): Absolute Change in Height-for-age Z-score
Time Frame: From Baseline Through Week 100
From Baseline Through Week 100
Part B: Absolute Change in Height-for-age Z-score
Time Frame: From Baseline Through Week 196
From Baseline Through Week 196
Part A (Cohort 3): Absolute Change in Length
Time Frame: From Baseline Through Week 100
From Baseline Through Week 100
Part B: Absolute Change in Length
Time Frame: From Baseline Through Week 196
From Baseline Through Week 196
Part A (Cohort 3): Absolute Change in Length-for-age Z-score
Time Frame: From Baseline Through Week 100
From Baseline Through Week 100
Part B: Absolute Change in Length-for-age Z-score
Time Frame: From Baseline Through Week 196
From Baseline Through Week 196

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Vertex Pharmaceuticals Incorporated

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 11, 2023

Primary Completion (Estimated)

July 30, 2029

Study Completion (Estimated)

July 30, 2029

Study Registration Dates

First Submitted

April 24, 2023

First Submitted That Met QC Criteria

April 24, 2023

First Posted (Actual)

May 6, 2023

Study Record Updates

Last Update Posted (Actual)

April 29, 2026

Last Update Submitted That Met QC Criteria

April 23, 2026

Last Verified

April 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VX22-121-106
  • 2022-503081-74-00 (Other Identifier: EU CT)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

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

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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