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Cystic FibrosisPhase 3NCT05422222

Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of Age

A Phase 3 study testing VX-121/TEZ/D-IVA in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 38 sites in 10 countries.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
Search all trials

Trial summary

The purpose of this study is to evaluate the pharmacokinetics, safety, tolerability and efficacy of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants with at least 1 triple combination responsive (TCR) mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Read more about this study

Who is it for?
Ages 1 year to 11 years
Conditions the study is for
Cystic Fibrosis
What is being tested?
VX-121/TEZ/D-IVA
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
210 participants
Where
38 sitesin 10 countries
Study dates
21 Jun 2022to 30 Jun 2030
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 to 11 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • Participants with stable CF and at least 1 TCR mutation (including F508del) in the CFTR gene

Who cannot join

  • History of solid organ, hematological transplantation, or cancer
  • Hepatic cirrhosis with portal hypertension, moderate hepatic impairment (Child Pugh Score 7 to 9), or severe hepatic impairment (Child Pugh Score 10 to 15)
  • Lung infection with organisms associated with a more rapid decline in pulmonary status
  • Other protocol defined Inclusion/Exclusion criteria may apply.

Trial locations and contacts

38 sites in 10 countries.

38 sites in 10 countries

United States19 sites

  • The Emory Clinic / Children's Healthcare of Atlanta at Egleston

    Atlanta, Georgia, 30322

  • Children's Hospital of Colorado

    Aurora, Colorado, 80045

  • Boston Children's Hospital

    Boston, Massachusetts, 02115

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

    Chicago, Illinois, 60611

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229

  • Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center

    Cleveland, Ohio, 44106

  • Vermont Lung Center

    Colchester, Vermont, 05446

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205

  • Texas Children's Hospital - Wallace Tower

    Houston, Texas, 77030

  • Riley Hospital for Children at Indiana University Health

    Indianapolis, Indiana, 46202

  • The Children's Mercy Hospital

    Kansas City, Missouri, 64108

  • Cohen Children's Medical Center

    Lake Success, New York, 11042

  • American Family Childrens Hospital

    Madison, Wisconsin, 53792

  • Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota

    Minneapolis, Minnesota, 55404

  • Children's Hospital of Orange County

    Orange, California, 92868

  • Stanford University Clinical and Translational Research Unit

    Palo Alto, California, 94304

  • UPMC Children's Hospital of Pittsburgh

    Pittsburgh, Pennsylvania, 15224

  • Oregon Health & Science University

    Portland, Oregon, 97239

  • Washington University School of Medicine / St. Louis Children's Hospital

    St Louis, Missouri, 63110

Other trials in United States

Australia4 sites

  • The Kids Research Institute Australia

    Nedlands

  • Women's and Children's Hospital

    North Adelaide

  • The Royal Children's Hospital

    Parkville

  • Queensland Children's Hospital

    South Brisbane

Other trials in Australia

Germany3 sites

  • Charité - Paediatric Pulmonology Department

    Berlin

  • Universitatsklinikum Essen

    Essen

  • Medizinische Hochschule Hannover

    Hanover

Other trials in Germany

Canada2 sites

  • The Hospital for Sick Children

    Toronto

  • British Columbia Children's Hospital

    Vancouver

Other trials in Canada

France2 sites

  • CHU Lyon - Hopital Femme Mere-Enfant

    Bron

  • Hopital Necker, Enfants Malades

    Paris

Other trials in France

Sweden2 sites

  • Sahlgrenska Universitetssjukhuset

    Gothenburg

  • Karolinska University Hospital - Pulmonology

    Stockholm

Other trials in Sweden

Switzerland2 sites

  • Inselspital - Universitaetsspital Bern

    Bern

  • Kinderspital Zurich

    Zurich

Other trials in Switzerland

United Kingdom2 sites

  • Children and Young Adults Research Unit

    Cardiff

  • Great Ormond Street Hospital for Children

    London

Other trials in United Kingdom

Netherlands1 site

  • Erasmus Medical Center / Sophia Children's Hospital

    Rotterdam

Other trials in Netherlands

New Zealand1 site

  • Starship Children's Hospital

    Grafton

Other trials in New Zealand

About this study

Official title: A Phase 3 Study Evaluating the Pharmacokinetics, Safety, and Tolerability of VX-121/Tezacaftor/Deutivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 1 Through 11 Years of Age

The purpose of this study is to evaluate the pharmacokinetics, safety, tolerability and efficacy of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants with at least 1 triple combination responsive (TCR) mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Conditions
Cystic Fibrosis
Being tested
VX-121/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.

  • VX-121/TEZ/D-IVADrug

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

    Fixed-dose combination for oral administration.

Study groups in NCT05422222
Study groupRoleWhat they receive
Part A: VX-121/TEZ/D-IVAExperimentalParticipants will receive VX-121/TEZ/D-IVA in the morning.
Receives: Drug: VX-121/TEZ/D-IVA
Part B: VX-121/TEZ/D-IVAExperimentalParticipants will receive VX-121/TEZ/D-IVA in the morning with the dose(s) to be based on the outcome of Part A.
Receives: Drug: VX-121/TEZ/D-IVA

What is being measured

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

  1. Part A: Observed Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ, D-IVA, and Relevant Metabolites

    Measured: From Day 1 up to Day 22

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

    Measured: From Day 1 up to Day 50

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

    Measured: From Day 1 up to Week 28

Secondary outcomes (19)

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

    Measured: From Baseline Through Week 24

  2. Part B: Observed Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ, D-IVA, and Relevant Metabolites

    Measured: From Day 1 up to Week 16

  3. Part B: Drug Acceptability Assessment Using Modified Facial Hedonic Scale

    Measured: At Day 1 and Week 24

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

    Measured: From Baseline Through Week 24

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

    Measured: From Baseline Through Week 24

  6. Part B: Number of CF-Related Hospitalizations

    Measured: From Baseline Through Week 24

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

    Measured: From Baseline Through Week 24

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

    Measured: From Baseline at Week 24

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

    Measured: From Baseline at Week 24

  10. Part B: Absolute Change in Weight

    Measured: From Baseline at Week 24

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

    Measured: From Baseline at Week 24

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

    Measured: From Baseline at Week 24

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

    Measured: From Baseline at Week 24

  14. Part B: Absolute Change in Height

    Measured: From Baseline at Week 24

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

    Measured: From Baseline at Week 24

  16. Part B: Absolute Change in Length

    Measured: From Baseline at Week 24

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

    Measured: From Baseline at Week 24

  18. Part B: Proportion of Participants With SwCl <60 millimole per liter (mmol/L)

    Measured: From Baseline Through Week 24

  19. Part B: Proportion of Participants With SwCl <30 mmol/L

    Measured: From Baseline Through Week 24

Timeline

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

  1. First posted16 Jun 2022
  2. Study start21 Jun 2022
  3. Primary completion30 Jun 2030 (estimated)
  4. Study completion30 Jun 2030 (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
Non-randomised
Intervention model
Sequential
Masking
None (open label)
Enrolment
210 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05422222
Sponsor's protocol ID
VX21-121-105
Other IDs
2024-513754-29-00
Status last verified
May 2026
First submitted
13 Jun 2022
Last updated
28 May 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/

Publications

  • Rayment JH, Davies G, Gaffin JM, Hoppe JE, Kasi AS, Mall MA, McKone EF, Ramsey B, Taylor JL, Tullis E, McDougall E, Pottackal R, Zahigian R, Krone K, Jennings M, Chmiel JF; 121-105 Study Group. Safety and efficacy of vanzacaftor-tezacaftor-deutivacaftor in children with cystic fibrosis aged 2-5 years (TIMBERLINE Trial VX21-121-105): a phase 3, open-label study. Lancet Respir Med. 2026 Sep 8:S2213-2600(26)00257-2. doi: 10.1016/S2213-2600(26)00257-2. Online ahead of print.

    PubMed 42710511

  • Hoppe JE, Kasi AS, Pittman JE, Jensen R, Thia LP, Robinson P, Tirakitsoontorn P, Ramsey B, Mall MA, Taylor-Cousar JL, McKone EF, Tullis E, Salinas DB, Zhu J, Chen YC, Rodriguez-Romero V, Sosnay PR, Davies G; VX21-121-105 Study Group. Vanzacaftor-tezacaftor-deutivacaftor for children aged 6-11 years with cystic fibrosis (RIDGELINE Trial VX21-121-105): an analysis from a single-arm, phase 3 trial. Lancet Respir Med. 2025 Mar;13(3):244-255. doi: 10.1016/S2213-2600(24)00407-7. Epub 2025 Jan 2.

    PubMed 39756425

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing VX-121/TEZ/D-IVA in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 38 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 1 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: under way, not looking for participants, as of the sponsor's last update on 28 May 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 38 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: Observed Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ, D-IVA, and Relevant Metabolites (time frame: From Day 1 up to Day 22). The study lists 3 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 NCT05422222, which also lists 2 linked publications.

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 28 May 2026.

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Clinical Trial NCT05422222 | AzTrials