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

Primary Purpose

Cystic Fibrosis

Status
Recruiting
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
VX-121/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 - 11 Years (Child)All SexesDoes not accept healthy volunteers

Key Inclusion Criteria:

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

Key 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

Other protocol defined Inclusion/Exclusion criteria may apply.

Sites / Locations

  • Children's Hospital of Orange CountyRecruiting
  • Stanford UniversityRecruiting
  • Children's Hospital of ColoradoRecruiting
  • The Emory Clinic / Children's Healthcare of Atlanta at EglestonRecruiting
  • Ann & Robert H. Lurie Children's Hospital of ChicagoRecruiting
  • Riley Hospital for Children at Indiana University HealthRecruiting
  • Boston Children's HospitalRecruiting
  • Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of MinnesotaRecruiting
  • The Children's Mercy HospitalRecruiting
  • Washington University School of Medicine / St. Louis Children's HospitalRecruiting
  • Cohen Children's Medical CenterRecruiting
  • Cincinnati Children's Hospital Medical CenterRecruiting
  • Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical CenterRecruiting
  • Nationwide Children's HospitalRecruiting
  • Oregon Health & Science University
  • UPMC Children's Hospital of PittsburghRecruiting
  • Texas Children's Hospital - Baylor College of MedicineRecruiting
  • Vermont Lung CenterRecruiting
  • University of Wisconsin Hospital and ClinicsRecruiting
  • Telethon Kids InstituteRecruiting
  • Women & Children's HospitalRecruiting
  • The Royal Children's HospitalRecruiting
  • Queensland Children's HospitalRecruiting
  • CHU Lyon - Hopital Femme Mere-EnfantRecruiting
  • Hopital Necker, Enfants MaladesRecruiting
  • Charite Paediatric Pulmonology DepartmentRecruiting
  • Kinderklinik III, Abt. fur PneumologieRecruiting
  • Medizinische Hochschule HannoverRecruiting
  • Erasmus Medical Center / Sophia Children's HospitalRecruiting
  • Sahlgrenska UniversitetssjukhusetRecruiting
  • Inselspital - Universitaetsspital BernRecruiting
  • Kinderspital ZuerichRecruiting
  • Children and Young Adults Research UnitRecruiting
  • Great Ormond Street Hospital for ChildrenRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

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

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

Arm Description

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

Participants will receive VX-121/TEZ/D-IVA in the morning with the dose(s) to be based on the outcome of Part A.

Outcomes

Primary Outcome Measures

Part A: Observed Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ, D-IVA, and Relevant Metabolites
Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

Secondary Outcome Measures

Part B: Absolute Change in Sweat Chloride (SwCl)
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ, D-IVA, and Relevant Metabolites
Part B: Drug Acceptability Assessment Using Modified Facial Hedonic Scale
Part B: Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1)
Part B: Number of Pulmonary Exacerbation (PEx)
Part B: Number of CF-Related Hospitalizations
Part B: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score
Part B: Absolute Change in Body Mass Index (BMI)
Part B: Absolute Change in BMI-for-age Z-score
Part B: Absolute Change in Weight
Part B: Absolute Change in Weight-for-age Z-score
Part B: Absolute Change in Weight-for-length
Part B: Absolute Change in Weight-for-length Z-score
Part B: Absolute Change in Height
Part B: Absolute Change in Height-for-age Z-score
Part B: Absolute Change in Length
Part B: Absolute Change in Length-for-age Z-score
Part B: Proportion of Participants With SwCl <60 millimole per liter (mmol/L)
Part B: Proportion of Participants With SwCl <30 mmol/L

Full Information

First Posted
June 13, 2022
Last Updated
September 29, 2023
Sponsor
Vertex Pharmaceuticals Incorporated
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1. Study Identification

Unique Protocol Identification Number
NCT05422222
Brief Title
Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of Age
Official Title
A Phase 3 Study Evaluating the Pharmacokinetics, Safety, and Tolerability of VX-121/Tezacaftor/Deutivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 1 Through 11 Years of Age
Study Type
Interventional

2. Study Status

Record Verification Date
June 2023
Overall Recruitment Status
Recruiting
Study Start Date
June 21, 2022 (Actual)
Primary Completion Date
June 2030 (Anticipated)
Study Completion Date
June 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 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.

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
Sequential Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
210 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Part A: VX-121/TEZ/D-IVA
Arm Type
Experimental
Arm Description
Participants will receive VX-121/TEZ/D-IVA in the morning.
Arm Title
Part B: VX-121/TEZ/D-IVA
Arm Type
Experimental
Arm Description
Participants will receive VX-121/TEZ/D-IVA in the morning with the dose(s) to be based on the outcome of Part A.
Intervention Type
Drug
Intervention Name(s)
VX-121/TEZ/D-IVA
Other Intervention Name(s)
VX-121/VX-661/VX-561, VX-121/VX-661/CTP-656, VX-121/tezacaftor/deutivacaftor
Intervention Description
Fixed-dose combination for oral administration.
Primary Outcome Measure Information:
Title
Part A: Observed Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ, D-IVA, and Relevant Metabolites
Time Frame
From Day 1 up to Day 22
Title
Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time Frame
From Day 1 up to Day 50
Title
Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time Frame
From Day 1 up to Week 28
Secondary Outcome Measure Information:
Title
Part B: Absolute Change in Sweat Chloride (SwCl)
Time Frame
From Baseline Through Week 24
Title
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ, D-IVA, and Relevant Metabolites
Time Frame
From Day 1 up to Week 16
Title
Part B: Drug Acceptability Assessment Using Modified Facial Hedonic Scale
Time Frame
At Day 1 and Week 24
Title
Part B: Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1)
Time Frame
From Baseline Through Week 24
Title
Part B: Number of Pulmonary Exacerbation (PEx)
Time Frame
From Baseline Through Week 24
Title
Part B: Number of CF-Related Hospitalizations
Time Frame
From Baseline Through Week 24
Title
Part B: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score
Time Frame
From Baseline Through Week 24
Title
Part B: Absolute Change in Body Mass Index (BMI)
Time Frame
From Baseline at Week 24
Title
Part B: Absolute Change in BMI-for-age Z-score
Time Frame
From Baseline at Week 24
Title
Part B: Absolute Change in Weight
Time Frame
From Baseline at Week 24
Title
Part B: Absolute Change in Weight-for-age Z-score
Time Frame
From Baseline at Week 24
Title
Part B: Absolute Change in Weight-for-length
Time Frame
From Baseline at Week 24
Title
Part B: Absolute Change in Weight-for-length Z-score
Time Frame
From Baseline at Week 24
Title
Part B: Absolute Change in Height
Time Frame
From Baseline at Week 24
Title
Part B: Absolute Change in Height-for-age Z-score
Time Frame
From Baseline at Week 24
Title
Part B: Absolute Change in Length
Time Frame
From Baseline at Week 24
Title
Part B: Absolute Change in Length-for-age Z-score
Time Frame
From Baseline at Week 24
Title
Part B: Proportion of Participants With SwCl <60 millimole per liter (mmol/L)
Time Frame
From Baseline Through Week 24
Title
Part B: Proportion of Participants With SwCl <30 mmol/L
Time Frame
From Baseline Through Week 24

10. Eligibility

Sex
All
Minimum Age & Unit of Time
1 Year
Maximum Age & Unit of Time
11 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria: Participants with stable CF and at least 1 TCR mutation (including F508del) in the CFTR gene Key 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 Other protocol defined Inclusion/Exclusion criteria may apply.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Medical Information
Phone
617-341-6777
Email
medicalinfo@vrtx.com
Facility Information:
Facility Name
Children's Hospital of Orange County
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Individual Site Status
Recruiting
Facility Name
Stanford University
City
Palo Alto
State/Province
California
ZIP/Postal Code
94304
Country
United States
Individual Site Status
Recruiting
Facility Name
Children's Hospital of Colorado
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Individual Site Status
Recruiting
Facility Name
The Emory Clinic / Children's Healthcare of Atlanta at Egleston
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Individual Site Status
Recruiting
Facility Name
Ann & Robert H. Lurie Children's Hospital of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Individual Site Status
Recruiting
Facility Name
Riley Hospital for Children at Indiana University Health
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Individual Site Status
Recruiting
Facility Name
Boston Children's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Individual Site Status
Recruiting
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
Individual Site Status
Recruiting
Facility Name
The Children's Mercy Hospital
City
Kansas City
State/Province
Missouri
ZIP/Postal Code
64108
Country
United States
Individual Site Status
Recruiting
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
Individual Site Status
Recruiting
Facility Name
Cohen Children's Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
11042
Country
United States
Individual Site Status
Recruiting
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Individual Site Status
Recruiting
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
Individual Site Status
Recruiting
Facility Name
Nationwide Children's Hospital
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43205
Country
United States
Individual Site Status
Recruiting
Facility Name
Oregon Health & Science University
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Individual Site Status
Completed
Facility Name
UPMC Children's Hospital of Pittsburgh
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15224
Country
United States
Individual Site Status
Recruiting
Facility Name
Texas Children's Hospital - Baylor College of Medicine
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Individual Site Status
Recruiting
Facility Name
Vermont Lung Center
City
Colchester
State/Province
Vermont
ZIP/Postal Code
05446
Country
United States
Individual Site Status
Recruiting
Facility Name
University of Wisconsin Hospital and Clinics
City
Madison
State/Province
Wisconsin
ZIP/Postal Code
53792
Country
United States
Individual Site Status
Recruiting
Facility Name
Telethon Kids Institute
City
Nedlands
Country
Australia
Individual Site Status
Recruiting
Facility Name
Women & Children's Hospital
City
North Adelaide
Country
Australia
Individual Site Status
Recruiting
Facility Name
The Royal Children's Hospital
City
Parkville
Country
Australia
Individual Site Status
Recruiting
Facility Name
Queensland Children's Hospital
City
South Brisbane
Country
Australia
Individual Site Status
Recruiting
Facility Name
CHU Lyon - Hopital Femme Mere-Enfant
City
Bron Cedex
Country
France
Individual Site Status
Recruiting
Facility Name
Hopital Necker, Enfants Malades
City
Paris Cedex 15
Country
France
Individual Site Status
Recruiting
Facility Name
Charite Paediatric Pulmonology Department
City
Berlin
Country
Germany
Individual Site Status
Recruiting
Facility Name
Kinderklinik III, Abt. fur Pneumologie
City
Essen
Country
Germany
Individual Site Status
Recruiting
Facility Name
Medizinische Hochschule Hannover
City
Hannover
Country
Germany
Individual Site Status
Recruiting
Facility Name
Erasmus Medical Center / Sophia Children's Hospital
City
Rotterdam
Country
Netherlands
Individual Site Status
Recruiting
Facility Name
Sahlgrenska Universitetssjukhuset
City
Göteborg
Country
Sweden
Individual Site Status
Recruiting
Facility Name
Inselspital - Universitaetsspital Bern
City
Bern
Country
Switzerland
Individual Site Status
Recruiting
Facility Name
Kinderspital Zuerich
City
Zürich
Country
Switzerland
Individual Site Status
Recruiting
Facility Name
Children and Young Adults Research Unit
City
Cardiff
Country
United Kingdom
Individual Site Status
Recruiting
Facility Name
Great Ormond Street Hospital for Children
City
London
Country
United Kingdom
Individual Site Status
Recruiting

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

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