Print

Purpose

This study will evaluate the efficacy, safety and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and a gating or residual function mutation (F/G and F/RF genotypes).

Condition

Eligibility

Eligible Ages
Over 12 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • Subject has a confirmed diagnosis of CF and is heterozygous for F508del and either a gating or residual function mutation (F/G and F/RF genotypes) - Forced expiratory volume in 1 second (FEV1) value ≥40% and ≤90% of predicted mean for age, sex, and height

Exclusion Criteria

  • Clinically significant cirrhosis with or without portal hypertension - Lung infection with organisms associated with a more rapid decline in pulmonary status - Solid organ or hematological transplantation Other protocol defined Inclusion/Exclusion criteria may apply

Study Design

Phase
Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
Active Comparator
Control: IVA or TEZ/IVA 		Following an IVA or TEZ/IVA run-in period of 4 weeks, participants either received IVA 150 milligrams (mg) every 12 hours (q12h) or TEZ 100 mg once daily (qd)/IVA 150 mg q12h in the treatment period for 8 weeks.
  • Drug: IVA
    Mono-tablet for oral administration.
    Other names:
    • VX-770
    • ivacaftor
  • Drug: TEZ/IVA
    Fixed-dose combination (FDC) tablet for oral administration.
    Other names:
    • VX-661/VX-770
    • tezacaftor/ivacaftor
Experimental
TC: ELX/TEZ/IVA 		Following an IVA or TEZ/IVA run-in period of 4 weeks, participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 8 weeks.
  • Drug: ELX/TEZ/IVA
    FDC tablet for oral administration.
    Other names:
    • VX-445/VX-661/VX-770
    • elexacaftor/tezacaftor/ivacaftor
  • Drug: IVA
    Mono-tablet for oral administration.
    Other names:
    • VX-770
    • ivacaftor

Recruiting Locations

More Details

NCT ID
NCT04058353
Status
Completed
Sponsor
Vertex Pharmaceuticals Incorporated

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.