A Phase 3 Study of VX-445 Combination Therapy in Cystic Fibrosis (CF) Subjects Heterozygous for F508del and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)

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

  • Cystic Fibrosis

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