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

Primary Purpose

Cystic Fibrosis

Status
Enrolling by invitation
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
VNZ/TEZ/D-IVA
Sponsored by
Vertex Pharmaceuticals Incorporated
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cystic Fibrosis

Eligibility Criteria

1 Year - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Key Inclusion Criteria: Participants who have completed study drug treatment in the parent study (VX21-121-105; NCT Number: NCT05422222) Key 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 Other protocol defined Inclusion/Exclusion criteria may apply.

Sites / Locations

  • Children's Hospital of Orange County
  • Children's Hospital of Colorado
  • The Emory Clinic / Children's Healthcare of Atlanta at Egleston
  • Ann & Robert H. Lurie Children's Hospital of Chicago
  • Riley Hospital for Children at Indiana University Health
  • Boston Children's Hospital
  • Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota
  • The Children's Mercy Hospital
  • Washington University School of Medicine / St. Louis Children's Hospital
  • Cohen Children's Medical Center
  • Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
  • Nationwide Children's Hospital
  • UPMC Children's Hospital of Pittsburgh
  • Vermont Lung Center
  • The Royal Childrens Hospital
  • Queensland Children's Hospital
  • CHU Lyon - Hopital Femme Mere-Enfant
  • Hopital Necker, Enfants Malades
  • Charite Paediatric Pulmonology Department
  • Kinderklinik III, Abt. fur Pneumologie
  • Medizinische Hochschule Hannover
  • Erasmus Medical Center / Sophia Children's Hospital
  • Sahlgrenska Universitetssjukhuset
  • Inselspital - Universitaetsspital Bern
  • Kinderspital Zuerich
  • Children and Young Adults Research Unit

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

VNZ/TEZ/D-IVA

Arm Description

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.

Outcomes

Primary Outcome Measures

Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)

Secondary Outcome Measures

All Cohorts: Absolute Change in Sweat Chloride (SwCl)
Cohort 1: Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1)
All Cohorts: Number of Pulmonary Exacerbation (PEx) Related Hospitalizations
All Cohorts: Number of CF- Related Hospitalizations
Cohort 1: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score
Cohorts 1 and 2: Absolute Change in Body Mass Index (BMI)
Cohorts 1 and 2: Absolute Change in BMI-for-age Z-score
Cohort 3: Absolute Change in Weight-for-length
Cohort 3: Absolute Change in Weight-for-length Z-score
All Cohorts: Absolute Change in Weight
All Cohorts: Change in Weight-for-age Z-score
Cohorts 1 and 2: Absolute Change in Height
Cohorts 1 and 2: Absolute Change in Height-for-age Z-score
Cohort 3: Absolute Change in Length
Cohort 3: Absolute Change in Length-for-age Z-score

Full Information

First Posted
April 24, 2023
Last Updated
October 12, 2023
Sponsor
Vertex Pharmaceuticals Incorporated
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1. Study Identification

Unique Protocol Identification Number
NCT05844449
Brief Title
Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and Older
Official Title
A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 1 Year of Age and Older
Study Type
Interventional

2. Study Status

Record Verification Date
April 2023
Overall Recruitment Status
Enrolling by invitation
Study Start Date
August 11, 2023 (Actual)
Primary Completion Date
October 2030 (Anticipated)
Study Completion Date
October 2030 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Vertex Pharmaceuticals Incorporated

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No

5. Study Description

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
180 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
VNZ/TEZ/D-IVA
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
VNZ/TEZ/D-IVA
Other Intervention Name(s)
VX-121/VX-661/VX-561, VX-121/VX-661/CTP-656, vanzacaftor/tezacaftor/deutivacaftor
Intervention Description
Fixed-dose combination tablets for oral administration.
Primary Outcome Measure Information:
Title
Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)
Time Frame
From Day 1 up to Week 100
Secondary Outcome Measure Information:
Title
All Cohorts: Absolute Change in Sweat Chloride (SwCl)
Time Frame
From Baseline Through Week 96
Title
Cohort 1: Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1)
Time Frame
From Baseline Through Week 100
Title
All Cohorts: Number of Pulmonary Exacerbation (PEx) Related Hospitalizations
Time Frame
From Baseline Through Week 100
Title
All Cohorts: Number of CF- Related Hospitalizations
Time Frame
From Baseline Through Week 100
Title
Cohort 1: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score
Time Frame
From Baseline Through Week 100
Title
Cohorts 1 and 2: Absolute Change in Body Mass Index (BMI)
Time Frame
From Baseline Through Week 100
Title
Cohorts 1 and 2: Absolute Change in BMI-for-age Z-score
Time Frame
From Baseline Through Week 100
Title
Cohort 3: Absolute Change in Weight-for-length
Time Frame
From Baseline Through Week 100
Title
Cohort 3: Absolute Change in Weight-for-length Z-score
Time Frame
From Baseline Through Week 100
Title
All Cohorts: Absolute Change in Weight
Time Frame
From Baseline Through Week 100
Title
All Cohorts: Change in Weight-for-age Z-score
Time Frame
From Baseline Through Week 100
Title
Cohorts 1 and 2: Absolute Change in Height
Time Frame
From Baseline Through Week 100
Title
Cohorts 1 and 2: Absolute Change in Height-for-age Z-score
Time Frame
From Baseline Through Week 100
Title
Cohort 3: Absolute Change in Length
Time Frame
From Baseline Through Week 100
Title
Cohort 3: Absolute Change in Length-for-age Z-score
Time Frame
From Baseline Through Week 100

10. Eligibility

Sex
All
Minimum Age & Unit of Time
1 Year
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria: Participants who have completed study drug treatment in the parent study (VX21-121-105; NCT Number: NCT05422222) Key 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 Other protocol defined Inclusion/Exclusion criteria may apply.
Facility Information:
Facility Name
Children's Hospital of Orange County
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Facility Name
Children's Hospital of Colorado
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
The Emory Clinic / Children's Healthcare of Atlanta at Egleston
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
Ann & Robert H. Lurie Children's Hospital of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
Riley Hospital for Children at Indiana University Health
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
Boston Children's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55404
Country
United States
Facility Name
The Children's Mercy Hospital
City
Kansas City
State/Province
Missouri
ZIP/Postal Code
64108
Country
United States
Facility Name
Washington University School of Medicine / St. Louis Children's Hospital
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
Cohen Children's Medical Center
City
Lake Success
State/Province
New York
ZIP/Postal Code
11042
Country
United States
Facility Name
Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44106
Country
United States
Facility Name
Nationwide Children's Hospital
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43205
Country
United States
Facility Name
UPMC Children's Hospital of Pittsburgh
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15224
Country
United States
Facility Name
Vermont Lung Center
City
Colchester
State/Province
Vermont
ZIP/Postal Code
05446
Country
United States
Facility Name
The Royal Childrens Hospital
City
Parkville
Country
Australia
Facility Name
Queensland Children's Hospital
City
South Brisbane
Country
Australia
Facility Name
CHU Lyon - Hopital Femme Mere-Enfant
City
Bron Cedex
Country
France
Facility Name
Hopital Necker, Enfants Malades
City
Paris Cedex 15
Country
France
Facility Name
Charite Paediatric Pulmonology Department
City
Berlin
Country
Germany
Facility Name
Kinderklinik III, Abt. fur Pneumologie
City
Essen
Country
Germany
Facility Name
Medizinische Hochschule Hannover
City
Hannover
Country
Germany
Facility Name
Erasmus Medical Center / Sophia Children's Hospital
City
Rotterdam
Country
Netherlands
Facility Name
Sahlgrenska Universitetssjukhuset
City
Göteborg
Country
Sweden
Facility Name
Inselspital - Universitaetsspital Bern
City
Bern
Country
Switzerland
Facility Name
Kinderspital Zuerich
City
Zürich
Country
Switzerland
Facility Name
Children and Young Adults Research Unit
City
Cardiff
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
No
IPD Sharing Plan Description
Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing

Learn more about this trial

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

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