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Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis (CF) Participants 2 Years and Older

Primary Purpose

Cystic Fibrosis

Status
Enrolling by invitation
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
ELX/TEZ/IVA
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

2 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

key Inclusion Criteria:

  • Completed study drug treatment in the parent study (VX20-445-111 Part B, NCT04537793), or had study drug interruption(s) in the parent study, but did not permanently discontinue study drug, and completed study visits up to the last scheduled visit of the Treatment Period of the parent study

Key Exclusion Criteria:

  • History of study drug intolerance in the parent study
  • History of poor compliance with ELX/TEZ/IVA and/or procedures in the parent study

Other protocol defined Inclusion/Exclusion criteria may apply

Sites / Locations

  • Banner University of Arizona Medical Center
  • Stanford University
  • Children's Hospital of Colorado
  • 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
  • NC TraCS Institute - CTRC University of North Carolina at Chapel Hill
  • Oregon Health & Science University
  • UPMC Children's Hospital of Pittsburgh
  • Seattle Children's Hospital
  • Telethon Kids Institute
  • The Royal Children's Hospital
  • Queensland Children's Hospital
  • The Hospital for Sick Children
  • British Columbia Children's Hospital
  • Charite Paediatric Pulmonology Department
  • Universitatsklinikum Essen (AoR), Kinderklinik III, Abt. fur Pneumologie
  • Alder Hey Children's NHS Foundation Trust
  • Royal Brompton Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

ELX/TEZ/IVA

Arm Description

Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening.

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

Absolute Change in Sweat Chloride (SwCl)
Absolute Change in Lung Clearance Index (LCI) 2.5

Full Information

First Posted
November 29, 2021
Last Updated
March 15, 2023
Sponsor
Vertex Pharmaceuticals Incorporated
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1. Study Identification

Unique Protocol Identification Number
NCT05153317
Brief Title
Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis (CF) Participants 2 Years and Older
Official Title
A Phase 3 Open-label Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 2 Years and Older
Study Type
Interventional

2. Study Status

Record Verification Date
March 2023
Overall Recruitment Status
Enrolling by invitation
Study Start Date
January 17, 2022 (Actual)
Primary Completion Date
April 2026 (Anticipated)
Study Completion Date
April 2026 (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, efficacy and pharmacodynamics of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) in CF participants 2 years of age and older.

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
70 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
ELX/TEZ/IVA
Arm Type
Experimental
Arm Description
Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening.
Intervention Type
Drug
Intervention Name(s)
ELX/TEZ/IVA
Other Intervention Name(s)
VX-445/VX-661/VX-770, elexacaftor/tezacaftor/ivacaftor
Intervention Description
Fixed-dose combination granules or tablets for oral administration.
Intervention Type
Drug
Intervention Name(s)
IVA
Other Intervention Name(s)
VX-770, ivacaftor
Intervention Description
Granules or tablets for oral administration.
Primary Outcome Measure Information:
Title
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 196
Secondary Outcome Measure Information:
Title
Absolute Change in Sweat Chloride (SwCl)
Time Frame
From Baseline up to Week 192
Title
Absolute Change in Lung Clearance Index (LCI) 2.5
Time Frame
From Baseline up to Week 192

10. Eligibility

Sex
All
Minimum Age & Unit of Time
2 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
key Inclusion Criteria: Completed study drug treatment in the parent study (VX20-445-111 Part B, NCT04537793), or had study drug interruption(s) in the parent study, but did not permanently discontinue study drug, and completed study visits up to the last scheduled visit of the Treatment Period of the parent study Key Exclusion Criteria: History of study drug intolerance in the parent study History of poor compliance with ELX/TEZ/IVA and/or procedures in the parent study Other protocol defined Inclusion/Exclusion criteria may apply
Facility Information:
Facility Name
Banner University of Arizona Medical Center
City
Tucson
State/Province
Arizona
ZIP/Postal Code
85724
Country
United States
Facility Name
Stanford University
City
Palo Alto
State/Province
California
ZIP/Postal Code
94304
Country
United States
Facility Name
Children's Hospital of Colorado
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
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
NC TraCS Institute - CTRC University of North Carolina at Chapel Hill
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27599
Country
United States
Facility Name
Oregon Health & Science University
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
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
Seattle Children's Hospital
City
Seattle
State/Province
Washington
ZIP/Postal Code
98105
Country
United States
Facility Name
Telethon Kids Institute
City
Nedlands
Country
Australia
Facility Name
The Royal Children's Hospital
City
Parkville
Country
Australia
Facility Name
Queensland Children's Hospital
City
South Brisbane
Country
Australia
Facility Name
The Hospital for Sick Children
City
Toronto
Country
Canada
Facility Name
British Columbia Children's Hospital
City
Vancouver
Country
Canada
Facility Name
Charite Paediatric Pulmonology Department
City
Berlin
Country
Germany
Facility Name
Universitatsklinikum Essen (AoR), Kinderklinik III, Abt. fur Pneumologie
City
Essen
Country
Germany
Facility Name
Alder Hey Children's NHS Foundation Trust
City
Liverpool
Country
United Kingdom
Facility Name
Royal Brompton Hospital
City
London
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 ELX/TEZ/IVA in Cystic Fibrosis (CF) Participants 2 Years and Older

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