Actively Recruiting

Phase 3
Age: 1Year - 11Years
All Genders
NCT05422222

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

Led by Vertex Pharmaceuticals Incorporated · Updated on 2026-03-30

210

Participants Needed

38

Research Sites

418 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

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

Official Title

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

Who Can Participate

Age: 1Year - 11Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Participants must have stable cystic fibrosis.
  • Must have at least one triple combination responsive mutation in the CFTR gene, including F508del.
  • Age between 1 and 11 years old.
Not Eligible

You will not qualify if you...

  • History of solid organ, hematological transplantation, or cancer.
  • Presence of hepatic cirrhosis with portal hypertension.
  • Moderate hepatic impairment (Child Pugh Score 7 to 9).
  • Severe hepatic impairment (Child Pugh Score 10 to 15).
  • Current lung infection with organisms linked to rapid decline in lung function.
  • Other protocol-defined exclusion criteria may apply.

AI-Screening

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Trial Site Locations

Total: 38 locations

1

Children's Hospital of Orange County

Orange, California, United States, 92868

Completed

2

Stanford University Clinical and Translational Research Unit

Palo Alto, California, United States, 94304

Completed

3

Children's Hospital of Colorado

Aurora, Colorado, United States, 80045

Not Yet Recruiting

4

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

Atlanta, Georgia, United States, 30322

Completed

5

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

Chicago, Illinois, United States, 60611

Completed

6

Riley Hospital for Children at Indiana University Health

Indianapolis, Indiana, United States, 46202

Not Yet Recruiting

7

Boston Children's Hospital

Boston, Massachusetts, United States, 02115

Not Yet Recruiting

8

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

Minneapolis, Minnesota, United States, 55404

Completed

9

The Children's Mercy Hospital

Kansas City, Missouri, United States, 64108

Completed

10

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

St Louis, Missouri, United States, 63110

Completed

11

Cohen Children's Medical Center

Lake Success, New York, United States, 11042

Completed

12

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States, 45229

Completed

13

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

Cleveland, Ohio, United States, 44106

Completed

14

Nationwide Children's Hospital

Columbus, Ohio, United States, 43205

Completed

15

Oregon Health & Science University

Portland, Oregon, United States, 97239

Completed

16

UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States, 15224

Completed

17

Texas Children's Hospital - Wallace Tower

Houston, Texas, United States, 77030

Completed

18

Vermont Lung Center

Colchester, Vermont, United States, 05446

Completed

19

American Family Childrens Hospital

Madison, Wisconsin, United States, 53792

Completed

20

The Kids Research Institute Australia

Nedlands, Australia

Not Yet Recruiting

21

Women's and Children's Hospital

North Adelaide, Australia

Not Yet Recruiting

22

The Royal Children's Hospital

Parkville, Australia

Not Yet Recruiting

23

Queensland Children's Hospital

South Brisbane, Australia

Not Yet Recruiting

24

The Hospital for Sick Children

Toronto, Canada

Not Yet Recruiting

25

British Columbia Children's Hospital

Vancouver, Canada

Not Yet Recruiting

26

CHU Lyon - Hopital Femme Mere-Enfant

Bron, France

Not Yet Recruiting

27

Hopital Necker, Enfants Malades

Paris, France

Not Yet Recruiting

28

Charité - Paediatric Pulmonology Department

Berlin, Germany

Not Yet Recruiting

29

Universitatsklinikum Essen

Essen, Germany

Not Yet Recruiting

30

Medizinische Hochschule Hannover

Hanover, Germany

Not Yet Recruiting

31

Erasmus Medical Center / Sophia Children's Hospital

Rotterdam, Netherlands

Not Yet Recruiting

32

Starship Children's Hospital

Grafton, New Zealand

Completed

33

Sahlgrenska Universitetssjukhuset

Gothenburg, Sweden

Completed

34

Karolinska University Hospital - Pulmonology

Stockholm, Sweden

Not Yet Recruiting

35

Inselspital - Universitaetsspital Bern

Bern, Switzerland

Actively Recruiting

36

Kinderspital Zurich

Zurich, Switzerland

Not Yet Recruiting

37

Children and Young Adults Research Unit

Cardiff, United Kingdom

Not Yet Recruiting

38

Great Ormond Street Hospital for Children

London, United Kingdom

Not Yet Recruiting

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Research Team

M

Medical Information

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NON_RANDOMIZED

Model

SEQUENTIAL

Primary Purpose

TREATMENT

Number of Arms

2

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