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Active, not recruitingNCT05422222Updated May 28, 2026

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

A Phase 3 interventional study of VX-121/TEZ/D-IVA in Cystic Fibrosis, sponsored by Vertex Pharmaceuticals Incorporated. Active, not recruiting at 38 sites in 10 countries. Open to participants aged 1 Year to 11 Years. Per ClinicalTrials.gov, last updated 2026-05-28.

Sponsored by Vertex Pharmaceuticals Incorporated · Phase 3, Interventional, and Treatment

Phase
Phase 3
Study type
Interventional
Enrollment
210
Allocation
Non-randomized
Ages
1 Year to 11 Years
Sex
All
01

Study 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.

02

Conditions studied

  • Cystic Fibrosis

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03

In context

Cystic Fibrosis

1,581 studies on the registry are indexed under Cystic Fibrosis; 190 are open to participants now.

This study's planned enrollment of 210 is above the median of 36 across 1,034 interventional studies indexed under Cystic Fibrosis.

Browse Cystic Fibrosis studies →

Lead sponsor

Vertex Pharmaceuticals Incorporated is the lead sponsor of 243 studies on the registry; 19 are open to participants now.

Of its 102 completed or terminated interventional studies of FDA-regulated products, 49 (48%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
1 Year to 11 Years
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility criteria

Key Inclusion Criteria:

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

Key Exclusion Criteria:

  • 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.

05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Sequential assignment
Masking
None (open label)
Enrollment
210 participants (estimated)

Study arms

  • Experimental
    Part A: VX-121/TEZ/D-IVA

    Participants will receive VX-121/TEZ/D-IVA in the morning.

    Drug: VX-121/TEZ/D-IVA

  • Experimental
    Part B: VX-121/TEZ/D-IVA

    Participants will receive VX-121/TEZ/D-IVA in the morning with the dose(s) to be based on the outcome of Part A.

    Drug: VX-121/TEZ/D-IVA

Interventions

  • DrugVX-121/TEZ/D-IVA

    Fixed-dose combination for oral administration.

    Also known as: VX-121/VX-661/VX-561, VX-121/VX-661/CTP-656, VX-121/tezacaftor/deutivacaftor

06

What researchers measure

Primary outcomes

  1. 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

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

    Time frame: 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)

    Time frame: From Day 1 up to Week 28

Secondary outcomes

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

    Time frame: From Baseline Through Week 24

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

    Time frame: From Day 1 up to Week 16

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

    Time frame: At Day 1 and Week 24

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

    Time frame: From Baseline Through Week 24

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

    Time frame: From Baseline Through Week 24

  6. Part B: Number of CF-Related Hospitalizations

    Time frame: From Baseline Through Week 24

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

    Time frame: From Baseline Through Week 24

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

    Time frame: From Baseline at Week 24

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

    Time frame: From Baseline at Week 24

  10. Part B: Absolute Change in Weight

    Time frame: From Baseline at Week 24

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

    Time frame: From Baseline at Week 24

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

    Time frame: From Baseline at Week 24

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

    Time frame: From Baseline at Week 24

  14. Part B: Absolute Change in Height

    Time frame: From Baseline at Week 24

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

    Time frame: From Baseline at Week 24

  16. Part B: Absolute Change in Length

    Time frame: From Baseline at Week 24

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

    Time frame: From Baseline at Week 24

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

    Time frame: From Baseline Through Week 24

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

    Time frame: From Baseline Through Week 24

07

Study locations

38 sites
  • Children's Hospital of Orange County
    Orange, California 92868, United States
  • Stanford University Clinical and Translational Research Unit
    Palo Alto, California 94304, United States
  • Children's Hospital of Colorado
    Aurora, Colorado 80045, United States
  • The Emory Clinic / Children's Healthcare of Atlanta at Egleston
    Atlanta, Georgia 30322, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago
    Chicago, Illinois 60611, United States
  • Riley Hospital for Children at Indiana University Health
    Indianapolis, Indiana 46202, United States
  • Boston Children's Hospital
    Boston, Massachusetts 02115, United States
  • Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota
    Minneapolis, Minnesota 55404, United States
  • The Children's Mercy Hospital
    Kansas City, Missouri 64108, United States
  • Washington University School of Medicine / St. Louis Children's Hospital
    St Louis, Missouri 63110, United States
  • Cohen Children's Medical Center
    Lake Success, New York 11042, United States
  • Cincinnati Children's Hospital Medical Center
    Cincinnati, Ohio 45229, United States
  • Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
    Cleveland, Ohio 44106, United States
  • Nationwide Children's Hospital
    Columbus, Ohio 43205, United States
  • Oregon Health & Science University
    Portland, Oregon 97239, United States
  • UPMC Children's Hospital of Pittsburgh
    Pittsburgh, Pennsylvania 15224, United States
  • Texas Children's Hospital - Wallace Tower
    Houston, Texas 77030, United States
  • Vermont Lung Center
    Colchester, Vermont 05446, United States
  • American Family Childrens Hospital
    Madison, Wisconsin 53792, United States
  • The Kids Research Institute Australia
    Nedlands, Australia
  • Women's and Children's Hospital
    North Adelaide, 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
  • CHU Lyon - Hopital Femme Mere-Enfant
    Bron, France
  • Hopital Necker, Enfants Malades
    Paris, France
  • Charité - Paediatric Pulmonology Department
    Berlin, Germany
  • Universitatsklinikum Essen
    Essen, Germany
  • Medizinische Hochschule Hannover
    Hanover, Germany
  • Erasmus Medical Center / Sophia Children's Hospital
    Rotterdam, Netherlands
  • Starship Children's Hospital
    Grafton, New Zealand
  • Sahlgrenska Universitetssjukhuset
    Gothenburg, Sweden
  • Karolinska University Hospital - Pulmonology
    Stockholm, Sweden
  • Inselspital - Universitaetsspital Bern
    Bern, Switzerland
  • Kinderspital Zurich
    Zurich, Switzerland
  • Children and Young Adults Research Unit
    Cardiff, United Kingdom
  • Great Ormond Street Hospital for Children
    London, United Kingdom
08

References and documents

Publications

  • 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 ↗

Individual participant data

Plan to share: 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/

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Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on May 28, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT05422222
Lead sponsor
Vertex Pharmaceuticals Incorporated
Responsible party
Sponsor
First posted
Jun 16, 2022
Start date
Jun 21, 2022
Primary completion
Jun 30, 2030 (estimated)
Completion
Jun 30, 2030 (estimated)
Last update
May 28, 2026

Oversight

FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is active, not recruiting, as verified in May 2026. You cannot join it, but the record below documents what was studied.

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