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Cystic FibrosisPhase 3NCT05844449

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

A Phase 3 study testing VNZ/TEZ/D-IVA in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 37 sites in 10 countries.

Enrolling by invitation

Enrolling by invitation. The study team selects and invites participants, usually from an existing group of patients.

Sites and contacts
Official record
Search all trials

Trial summary

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). Read more about this study

Who is it for?
Ages 1 year and older
Conditions the study is for
Cystic Fibrosis
What is being tested?
VNZ/TEZ/D-IVA
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
174 participants
Where
37 sitesin 10 countries
Study dates
11 Aug 2023to 30 Jul 2029
Phase
Phase 3
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 1 year and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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.

Trial locations and contacts

37 sites in 10 countries. Contact the study team or a site near you to ask about taking part.

37 sites in 10 countries

United States18 sites

  • The Emory Clinic - Clifton Road - Pulmonology

    Atlanta, Georgia, 30322

  • Children's Hospital of Colorado - Pulmonology

    Aurora, Colorado, 80045

  • Boston Children's Hospital

    Boston, Massachusetts, 02115

  • Ann & Robert H. Lurie Children's Hospital of Chicago - Hematology

    Chicago, Illinois, 60611

  • Cincinnati Children's Hospital Medical Center - Pulmonology

    Cincinnati, Ohio, 45229

  • UH Cleveland Medical Center - Pulmonology

    Cleveland, Ohio, 44106

  • Vermont Lung Center

    Colchester, Vermont, 05446

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205

  • Texas Children's Hospital - Wallace Tower - Pulmonology

    Houston, Texas, 77030

  • JW Riley Hospital for Children - Pulmonology

    Indianapolis, Indiana, 46202

  • The Children's Mercy Hospital

    Kansas City, Missouri, 64108

  • Cohen Children's Medical Center - Lakeville Road

    Lake Success, New York, 11042

  • American Family Childrens Hospital

    Madison, Wisconsin, 53792

  • Children's Health Care d/b/a Children's Hospitals and Clinics of Minnesota

    Minneapolis, Minnesota, 55404

  • Children's Hospital of Orange County

    Orange, California, 92868

  • Stanford University - Palo Alto - Pulmonology

    Palo Alto, California, 94304

  • Children's Hospital of Pittsburgh - Pulmonology

    Pittsburgh, Pennsylvania, 15224

  • St. Louis Children's Hospital - Pulmonology

    St Louis, Missouri, 63110

Other trials in United States

Australia4 sites

  • The Kids Research Institute Australia

    Nedlands

  • Women's & Children's Hospital

    North Adelaide

  • The Royal Children's Hospital Melbourne

    Parkville

  • Children's Health Queensland Hospital and Health Service

    South Brisbane

Other trials in Australia

Germany3 sites

  • Charite Paediatric Pulmonology Department

    Berlin

  • Universitatsklinikum Essen

    Essen

  • Medizinische Hochschule Hannover - Clinic for Pediatric Pneumology, Allergology and Neonatology, CF-Centre

    Hanover

Other trials in Germany

Canada2 sites

  • Hospital for Sick Children - Pulmonology

    Toronto

  • British Columbia Children's Hospital

    Vancouver

Other trials in Canada

France2 sites

  • Hopital Femme Mere-Enfant

    Bron

  • Hopital Necker Enfants Malades - Pulmonology

    Paris

Other trials in France

Sweden2 sites

  • Sahlgrenska Universitetssjukhuset - Göteborg CF-center

    Gothenburg

  • Karolinska University Hospital - Pulmonology

    Stockholm

Other trials in Sweden

Switzerland2 sites

  • Inselspital Bern

    Bern

  • Kinderspital Zurich - Abteilung Pneumologie

    Zurich

Other trials in Switzerland

United Kingdom2 sites

  • Noah's Ark Children's Hospital for Wales

    Cardiff

  • Great Ormond Street Hospital for Children

    London

Other trials in United Kingdom

Netherlands1 site

  • Sophia Children's Hospital

    Rotterdam

Other trials in Netherlands

New Zealand1 site

  • Starship Child Health

    Grafton

Other trials in New Zealand

About this study

Official title: 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).

Conditions
Cystic Fibrosis
Being tested
VNZ/TEZ/D-IVA
Medical subject headings
Cystic Fibrosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • VNZ/TEZ/D-IVADrug

    Also called VX-121/VX-661/VX-561, VX-121/VX-661/CTP-656, vanzacaftor/tezacaftor/deutivacaftor

    Fixed-dose combination tablets or granules for oral administration.

Study groups in NCT05844449
Study groupRoleWhat they receive
VNZ/TEZ/D-IVAExperimentalPart 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.
Receives: Drug: VNZ/TEZ/D-IVA

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)

    Measured: From Baseline up to Week 100

  2. Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)

    Measured: From Baseline up to Week 196

Secondary outcomes (30)

  1. Part A (All Cohorts): Absolute Change in Sweat Chloride (SwCl)

    Measured: From Baseline Through Week 96

  2. Part B: Absolute Change in Sweat Chloride (SwCl)

    Measured: From Baseline Through Week 192

  3. Part A (Cohort 1): Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1)

    Measured: From Baseline Through Week 100

  4. Part B: Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1)

    Measured: From Baseline Through Week 196

  5. Part A (All Cohorts): Number of Pulmonary Exacerbation (PEx)

    Measured: From Baseline Through Week 100

  6. Part B: Number of Pulmonary Exacerbation (PEx)

    Measured: From Baseline Through Week 196

  7. Part A (All Cohorts): Number of CF- Related Hospitalizations

    Measured: From Baseline Through Week 100

  8. Part B: Number of CF- Related Hospitalizations

    Measured: From Baseline Through Week 196

  9. Part A (Cohort 1): Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score

    Measured: From Baseline Through Week 100

  10. Part B: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score

    Measured: From Baseline Through Week 196

  11. Part A (Cohorts 1 and 2): Absolute Change in Body Mass Index (BMI)

    Measured: From Baseline Through Week 100

  12. Part B: Absolute Change in Body Mass Index (BMI)

    Measured: From Baseline Through Week 196

  13. Part A (Cohorts 1 and 2): Absolute Change in BMI-for-age Z-score

    Measured: From Baseline Through Week 100

  14. Part B: Absolute Change in BMI-for-age Z-score

    Measured: From Baseline Through Week 196

  15. Part A (Cohort 3): Absolute Change in Weight-for-length

    Measured: From Baseline Through Week 100

  16. Part B: Absolute Change in Weight-for-length

    Measured: From Baseline Through Week 196

  17. Part A (Cohort 3): Absolute Change in Weight-for-length Z-score

    Measured: From Baseline Through Week 100

  18. Part B: Absolute Change in Weight-for-length Z-score

    Measured: From Baseline Through Week 196

  19. Part A (All Cohorts): Absolute Change in Weight

    Measured: From Baseline Through Week 100

  20. Part B: Absolute Change in Weight

    Measured: From Baseline Through Week 196

  21. Part A (All Cohorts): Change in Weight-for-age Z-score

    Measured: From Baseline Through Week 100

  22. Part B: Change in Weight-for-age Z-score

    Measured: From Baseline Through Week 196

  23. Part A (Cohorts 1 and 2): Absolute Change in Height

    Measured: From Baseline Through Week 100

  24. Part B: Absolute Change in Height

    Measured: From Baseline Through Week 196

  25. Part A (Cohorts 1 and 2): Absolute Change in Height-for-age Z-score

    Measured: From Baseline Through Week 100

  26. Part B: Absolute Change in Height-for-age Z-score

    Measured: From Baseline Through Week 196

  27. Part A (Cohort 3): Absolute Change in Length

    Measured: From Baseline Through Week 100

  28. Part B: Absolute Change in Length

    Measured: From Baseline Through Week 196

  29. Part A (Cohort 3): Absolute Change in Length-for-age Z-score

    Measured: From Baseline Through Week 100

  30. Part B: Absolute Change in Length-for-age Z-score

    Measured: From Baseline Through Week 196

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted6 May 2023
  2. Study start11 Aug 2023
  3. Primary completion30 Jul 2029 (estimated)
  4. Study completion30 Jul 2029 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
174 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05844449
Sponsor's protocol ID
VX22-121-106
Other IDs
2022-503081-74-00
Status last verified
Apr 2026
First submitted
24 Apr 2023
Last updated
29 Apr 2026
Results posted
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No: 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/

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing VNZ/TEZ/D-IVA in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 37 sites in 10 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: enrolling by invitation, as of the sponsor's last update on 29 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 37 sites in United States, Australia, Germany, Canada, France and 5 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) (time frame: From Baseline up to Week 100). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05844449.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Apr 2026.

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