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

NCT05844449 | Enrolling By Invitation Phase 3 FDA Drug

• 2 other identifiers: VX22-121-1062022-503081-74-00
• Study Type: interventional
• Target: 174
• Locations: 10 countries
• Sites: 37

Brief Summary

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).

Conditions

Cystic Fibrosis

Outcome Measures

Primary Outcomes (2)

• Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)

  From Baseline up to Week 100

• Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)

  From Baseline up to Week 196

Secondary Outcomes (30)

• Part A (All Cohorts): Absolute Change in Sweat Chloride (SwCl)

  From Baseline Through Week 96

• Part B: Absolute Change in Sweat Chloride (SwCl)

  From Baseline Through Week 192

• Part A (Cohort 1): Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1)

  From Baseline Through Week 100

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

  From Baseline Through Week 196

• Part A (All Cohorts): Number of Pulmonary Exacerbation (PEx)

  From Baseline Through Week 100

Study Arms (1)

VNZ/TEZ/D-IVA

EXPERIMENTAL

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.

Drug: VNZ/TEZ/D-IVA

Interventions

VNZ/TEZ/D-IVA DRUG

Fixed-dose combination tablets or granules for oral administration.

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

VNZ/TEZ/D-IVA

Eligibility Criteria

• Age: 1 Year+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Vertex Pharmaceuticals Incorporated: lead

Study Sites (37)

Children's Hospital of Orange County

Orange, California, 92868, United States

Location

Stanford University - Palo Alto - Pulmonology

Palo Alto, California, 94304, United States

Location

Children's Hospital of Colorado - Pulmonology

Aurora, Colorado, 80045, United States

Location

The Emory Clinic - Clifton Road - Pulmonology

Atlanta, Georgia, 30322, United States

Location

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

Chicago, Illinois, 60611, United States

Location

JW Riley Hospital for Children - Pulmonology

Indianapolis, Indiana, 46202, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

Children's Health Care d/b/a Children's Hospitals and Clinics of Minnesota

Minneapolis, Minnesota, 55404, United States

Location

The Children's Mercy Hospital

Kansas City, Missouri, 64108, United States

Location

St. Louis Children's Hospital - Pulmonology

St Louis, Missouri, 63110, United States

Location

Cohen Children's Medical Center - Lakeville Road

Lake Success, New York, 11042, United States

Location

Cincinnati Children's Hospital Medical Center - Pulmonology

Cincinnati, Ohio, 45229, United States

Location

UH Cleveland Medical Center - Pulmonology

Cleveland, Ohio, 44106, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

Children's Hospital of Pittsburgh - Pulmonology

Pittsburgh, Pennsylvania, 15224, United States

Location

Texas Children's Hospital - Wallace Tower - Pulmonology

Houston, Texas, 77030, United States

Location

Vermont Lung Center

Colchester, Vermont, 05446, United States

Location

American Family Childrens Hospital

Madison, Wisconsin, 53792, United States

Location

The Kids Research Institute Australia

Nedlands, Australia

Location

Women's & Children's Hospital

North Adelaide, Australia

Location

The Royal Children's Hospital Melbourne

Parkville, Australia

Location

Children's Health Queensland Hospital and Health Service

South Brisbane, Australia

Location

Hospital for Sick Children - Pulmonology

Toronto, Canada

Location

British Columbia Children's Hospital

Vancouver, Canada

Location

Hopital Femme Mere-Enfant

Bron, France

Location

Hopital Necker Enfants Malades - Pulmonology

Paris, France

Location

Charite Paediatric Pulmonology Department

Berlin, Germany

Location

Universitatsklinikum Essen

Essen, Germany

Location

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

Hanover, Germany

Location

Sophia Children's Hospital

Rotterdam, Netherlands

Location

Starship Child Health

Grafton, New Zealand

Location

Sahlgrenska Universitetssjukhuset - Göteborg CF-center

Gothenburg, Sweden

Location

Karolinska University Hospital - Pulmonology

Stockholm, Sweden

Location

Inselspital Bern

Bern, Switzerland

Location

Kinderspital Zurich - Abteilung Pneumologie

Zurich, Switzerland

Location

Noah's Ark Children's Hospital for Wales

Cardiff, United Kingdom

Location

Great Ormond Street Hospital for Children

London, United Kingdom

Location

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

deutivacaftor, tezacaftor , vanzacaftor

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 24, 2023
• First Posted: May 6, 2023
• Study Start: August 11, 2023
• Primary Completion (Estimated): July 30, 2029
• Study Completion (Estimated): July 30, 2029
• Last Updated: April 29, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

NZ (1); CA (2); AU (4); GB (2); US (18); SE (2); DE (3); NL (1); CH (2); FR (2)