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

NCT ID: NCT05422222

Last Updated: 2025-11-06

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

210 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-06-21

Study Completion Date

2030-06-30

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Cystic Fibrosis

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

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

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

Group Type EXPERIMENTAL

VX-121/TEZ/D-IVA

Intervention Type DRUG

Fixed-dose combination for oral administration.

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.

Group Type EXPERIMENTAL

VX-121/TEZ/D-IVA

Intervention Type DRUG

Fixed-dose combination for oral administration.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

VX-121/TEZ/D-IVA

Fixed-dose combination for oral administration.

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

VX-121/VX-661/VX-561 VX-121/VX-661/CTP-656 VX-121/tezacaftor/deutivacaftor

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

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

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
Minimum Eligible Age

1 Year

Maximum Eligible Age

11 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Vertex Pharmaceuticals Incorporated

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Children's Hospital of Orange County

Orange, California, United States

Site Status RECRUITING

Stanford University Clinical and Translational Research Unit

Palo Alto, California, United States

Site Status RECRUITING

Children's Hospital of Colorado

Aurora, Colorado, United States

Site Status RECRUITING

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

Atlanta, Georgia, United States

Site Status RECRUITING

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

Chicago, Illinois, United States

Site Status RECRUITING

Riley Hospital for Children at Indiana University Health

Indianapolis, Indiana, United States

Site Status RECRUITING

Boston Children's Hospital

Boston, Massachusetts, United States

Site Status RECRUITING

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

Minneapolis, Minnesota, United States

Site Status RECRUITING

The Children's Mercy Hospital

Kansas City, Missouri, United States

Site Status RECRUITING

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

St Louis, Missouri, United States

Site Status RECRUITING

Cohen Children's Medical Center

Lake Success, New York, United States

Site Status COMPLETED

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status RECRUITING

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

Cleveland, Ohio, United States

Site Status RECRUITING

Nationwide Children's Hospital

Columbus, Ohio, United States

Site Status RECRUITING

Oregon Health & Science University

Portland, Oregon, United States

Site Status COMPLETED

UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States

Site Status RECRUITING

Texas Children's Hospital - Wallace Tower

Houston, Texas, United States

Site Status RECRUITING

Vermont Lung Center

Colchester, Vermont, United States

Site Status RECRUITING

American Family Childrens Hospital

Madison, Wisconsin, United States

Site Status ACTIVE_NOT_RECRUITING

The Kids Research Institute Australia

Nedlands, , Australia

Site Status RECRUITING

Women's and Children's Hospital

North Adelaide, , Australia

Site Status RECRUITING

The Royal Children's Hospital

Parkville, , Australia

Site Status RECRUITING

Queensland Children's Hospital

South Brisbane, , Australia

Site Status RECRUITING

The Hospital for Sick Children

Toronto, , Canada

Site Status RECRUITING

British Columbia Children's Hospital

Vancouver, , Canada

Site Status RECRUITING

CHU Lyon - Hopital Femme Mere-Enfant

Bron, , France

Site Status RECRUITING

Hopital Necker, Enfants Malades

Paris, , France

Site Status RECRUITING

Charité Universitätsmedizin BerlinX

Berlin, , Germany

Site Status RECRUITING

Kinderklinik III, Abt. fur Pneumologie

Essen, , Germany

Site Status RECRUITING

Medizinische Hochschule Hannover

Hanover, , Germany

Site Status RECRUITING

Erasmus Medical Center / Sophia Children's Hospital

Rotterdam, , Netherlands

Site Status RECRUITING

Starship Children's Hospital

Grafton, , New Zealand

Site Status RECRUITING

Sahlgrenska Universitetssjukhuset

Gothenburg, , Sweden

Site Status COMPLETED

Karolinska Universitetssjukhuset, Huddinge

Stockholm, , Sweden

Site Status RECRUITING

Inselspital - Universitaetsspital Bern

Bern, , Switzerland

Site Status COMPLETED

Kinderspital Zurich

Zurich, , Switzerland

Site Status RECRUITING

Children and Young Adults Research Unit

Cardiff, , United Kingdom

Site Status RECRUITING

Great Ormond Street Hospital for Children

London, , United Kingdom

Site Status RECRUITING

Countries

Review the countries where the study has at least one active or historical site.

United States Australia Canada France Germany Netherlands New Zealand Sweden Switzerland United Kingdom

Central Contacts

Reach out to these primary contacts for questions about participation or study logistics.

Medical Information

Role: CONTACT

Phone: 617-341-6777

Email: [email protected]

References

Explore related publications, articles, or registry entries linked to this study.

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.

Reference Type DERIVED
PMID: 39756425 (View on PubMed)

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

2024-513754-29-00

Identifier Type: OTHER

Identifier Source: secondary_id

VX21-121-105

Identifier Type: -

Identifier Source: org_study_id