Clincosm LogoSign in Get a demo

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

Sponsor
Vertex Pharmaceuticals Incorporated (Industry)
Overall Status
Not yet recruiting
CT.gov ID
NCT05444257
Collaborator
(none)
850
Enrollment
1
Arm
35
Anticipated Duration (Months)

Study Details

Study Description

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.

Condition or Disease Intervention/Treatment Phase
Phase 3

Study Design

Study Type:
Interventional
Anticipated Enrollment :
850 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy in Subjects With Cystic Fibrosis
Anticipated Study Start Date :
Nov 1, 2022
Anticipated Primary Completion Date :
Oct 1, 2025
Anticipated Study Completion Date :
Oct 1, 2025

Arms and Interventions

Arm Intervention/Treatment
Experimental: VX-121/TEZ/D-IVA

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

Drug: VX-121/TEZ/D-IVA
Fixed-dose combination tablets for oral administration.
Other Names:
  • VX-121/VX-661/CTP-656
  • VX-121/VX-661/VX-561
  • VX-121/tezacaftor/deutivacaftor

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [From Day 1 up to Week 100]

    Secondary Outcome Measures

    1. Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) [From Baseline up to Week 96]

    2. Absolute Change From Baseline in Sweat Chloride (SwCl) [From Baseline up to Week 96]

    3. Number of Pulmonary Exacerbations (PEx) [From Baseline up to Week 96]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    12 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Key Inclusion Criteria:
    • 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
    Key Exclusion Criteria:

    • History of drug intolerance in a parent study

    • Pregnant or breast-feeding females

    Other protocol defined Inclusion/Exclusion criteria may apply.

    Contacts and Locations

    Locations

    No locations specified.

    Sponsors and Collaborators

    • Vertex Pharmaceuticals Incorporated

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Vertex Pharmaceuticals Incorporated
    ClinicalTrials.gov Identifier:
    NCT05444257
    Other Study ID Numbers:
    • VX20-121-104
    • 2021-000713-17
    First Posted:
    Jul 5, 2022
    Last Update Posted:
    Jul 5, 2022
    Last Verified:
    May 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Additional relevant MeSH terms:
    Cystic Fibrosis
    Fibrosis

    Study Results

    No Results Posted as of Jul 5, 2022