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

NCT ID: NCT05844449

Last Updated: 2025-12-17

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

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Recruitment Status

ENROLLING_BY_INVITATION

Clinical Phase

PHASE3

Total Enrollment

174 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-08-11

Study Completion Date

2029-07-30

Brief Summary

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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).

Detailed Description

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Conditions

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Cystic Fibrosis

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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VNZ/TEZ/D-IVA

Part 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.

Group Type EXPERIMENTAL

VNZ/TEZ/D-IVA

Intervention Type DRUG

Fixed-dose combination tablets or granules for oral administration.

Interventions

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VNZ/TEZ/D-IVA

Fixed-dose combination tablets or granules for oral administration.

Intervention Type DRUG

Other Intervention Names

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

Eligibility Criteria

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Inclusion Criteria

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

Exclusion Criteria

* 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
Minimum Eligible Age

1 Year

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Vertex Pharmaceuticals Incorporated

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Children's Hospital of Orange County

Orange, California, United States

Site Status

Stanford University - Palo Alto - Pulmonology

Palo Alto, California, United States

Site Status

Children's Hospital of Colorado - Pulmonology

Aurora, Colorado, United States

Site Status

The Emory Clinic - Clifton Road - Pulmonology

Atlanta, Georgia, United States

Site Status

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

Chicago, Illinois, United States

Site Status

JW Riley Hospital for Children - Pulmonology

Indianapolis, Indiana, United States

Site Status

Boston Children's Hospital

Boston, Massachusetts, United States

Site Status

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

Minneapolis, Minnesota, United States

Site Status

The Children's Mercy Hospital

Kansas City, Missouri, United States

Site Status

St. Louis Children's Hospital - Pulmonology

St Louis, Missouri, United States

Site Status

Cohen Children's Medical Center - Lakeville Road

Lake Success, New York, United States

Site Status

Cincinnati Children's Hospital Medical Center - Pulmonology

Cincinnati, Ohio, United States

Site Status

UH Cleveland Medical Center - Pulmonology

Cleveland, Ohio, United States

Site Status

Nationwide Children's Hospital

Columbus, Ohio, United States

Site Status

Children's Hospital of Pittsburgh - Pulmonology

Pittsburgh, Pennsylvania, United States

Site Status

Texas Children's Hospital - Wallace Tower - Pulmonology

Houston, Texas, United States

Site Status

Vermont Lung Center

Colchester, Vermont, United States

Site Status

American Family Childrens Hospital

Madison, Wisconsin, United States

Site Status

The Kids Research Institute Australia

Nedlands, , Australia

Site Status

Women & Children's Hospital

North Adelaide, , Australia

Site Status

The Royal Children's Hospital Melbourne

Parkville, , Australia

Site Status

Children's Health Queensland Hospital and Health Service

South Brisbane, , Australia

Site Status

Hospital for Sick Children - Pulmonology

Toronto, , Canada

Site Status

British Columbia Children's Hospital

Vancouver, , Canada

Site Status

Hopital Femme Mere-Enfant

Bron, , France

Site Status

Hopital Necker Enfants Malades - Pulmonology

Paris, , France

Site Status

Charite Paediatric Pulmonology Department

Berlin, , Germany

Site Status

Universitatsklinikum Essen

Essen, , Germany

Site Status

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

Hanover, , Germany

Site Status

Sophia Children's Hospital

Rotterdam, , Netherlands

Site Status

Sahlgrenska Universitetssjukhuset - Göteborg CF-center

Gothenburg, , Sweden

Site Status

Karolinska University Hospital - Pulmonology

Stockholm, , Sweden

Site Status

Inselspital Bern

Bern, , Switzerland

Site Status

Kinderspital Zurich - Abteilung Pneumologie

Zurich, , Switzerland

Site Status

Noah's Ark Children's Hospital for Wales

Cardiff, , United Kingdom

Site Status

Great Ormond Street Hospital for Children

London, , United Kingdom

Site Status

Countries

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United States Australia Canada France Germany Netherlands Sweden Switzerland United Kingdom

Other Identifiers

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VX22-121-106

Identifier Type: -

Identifier Source: org_study_id

2022-503081-74-00

Identifier Type: OTHER

Identifier Source: secondary_id