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A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy

Phase 3
Active, not recruiting
Conditions
Cystic Fibrosis
Interventions
Registration Number
NCT05444257
Lead Sponsor
Vertex Pharmaceuticals Incorporated
Brief Summary

The purpose of this study is to evaluate the long-term safety, tolerability, and efficacy of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in participants with cystic fibrosis.

Detailed Description

Not available

Recruitment & Eligibility

Status
ACTIVE_NOT_RECRUITING
Sex
All
Target Recruitment
822
Inclusion Criteria
  • Part A: Completed study drug treatment in a parent study VX20-121-102 (NCT05033080) and VX20-121-103 (NCT05076149); or had study drug interruption(s) in a parent study but did not permanently discontinue study drug, and completed study visits up to the last scheduled visit of the Treatment Period in the parent study
  • Part B: Completed study drug treatment in Part A; or 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

Key

Exclusion Criteria
  • History of drug intolerance in a parent study
  • Pregnant or breast-feeding females at the time of enrollment in Part A

Other protocol defined Inclusion/Exclusion criteria may apply.

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
VX-121/TEZ/D-IVAVX-121/TEZ/D-IVAPart A: Participants will receive VX-121/TEZ/D-IVA once daily for 96 weeks. Part B: Participants will receive VX-121/TEZ/D-IVA once daily for an additional 48 weeks.
Primary Outcome Measures
NameTimeMethod
Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)From Baseline up to Week 148
Secondary Outcome Measures
NameTimeMethod
Absolute Change From Baseline in Sweat Chloride (SwCl)From Baseline up to Week 144
Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)From Baseline up to Week 144
Number of Pulmonary Exacerbations (PEx)From Baseline up to Week 144

Trial Locations

Locations (195)

University of Alabama at Birmingham

🇺🇸

Birmingham, Alabama, United States

Banner University of Arizona Medical Center

🇺🇸

Tucson, Arizona, United States

University of Arkansas for Medical Sciences

🇺🇸

Little Rock, Arkansas, United States

Miller Children's Hospital / Long Beach Memorial

🇺🇸

Long Beach, California, United States

Children's Hospital Los Angeles

🇺🇸

Los Angeles, California, United States

Kaiser Permanente

🇺🇸

Oakland, California, United States

University of California Davis Medical Center

🇺🇸

Sacramento, California, United States

University of California San Francisco, Lung Transplant Program

🇺🇸

San Francisco, California, United States

Children's Hospital of Colorado

🇺🇸

Aurora, Colorado, United States

National Jewish Health

🇺🇸

Denver, Colorado, United States

Scroll for more (185 remaining)
University of Alabama at Birmingham
🇺🇸Birmingham, Alabama, United States

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