Vanderbilt Clinical Trials

A Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Who Are Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive (TCR) CFTR Mutation and No F508del Mutation

Purpose

The purpose of this study is to evaluate the efficacy and safety of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants who are homozygous for F508del, heterozygous for F508del and a gating (F/G) or residual function (F/RF) mutation, or have at least 1 other TCR CF transmembrane conductance regulator (CFTR) gene mutation and no F508del mutation.

Condition

Cystic Fibrosis

Eligibility

Eligible Ages: Over 12 Years
Eligible Sex: All
Accepts Healthy Volunteers: No

Inclusion Criteria

Participant has one of the following genotypes: - Homozygous for F508del; - Heterozygous for F508del and a gating (F/G) mutation; - Heterozygous for F508del and a residual function (F/RF) mutation; - At least 1 other TCR CFTR gene mutation identified as responsive to ELX/TEZ/IVA and no F508del mutation - Forced expiratory volume in 1 second (FEV1) value >=40% and <=90% of predicted mean for age, sex, and height for participants currently receiving CFTR protein modulator therapy; FEV1 >=40% and <=80% for participants not currently receiving CFTR protein modulator therapy

Exclusion Criteria

History of solid organ or hematological transplantation - Hepatic cirrhosis with portal hypertension, moderate hepatic impairment (Child Pugh Score 7 to 9), or severe hepatic impairment (Child Pugh Score 10 to 15) - Lung infection with organisms associated with a more rapid decline in pulmonary status - Pregnant or breast-feeding females 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

Arm	Description	Assigned Intervention
Active Comparator ELX/TEZ/IVA	Following elexacftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) run-in period of 4 weeks, participants received ELX 200 milligram (mg) once daily (qd) /TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the treatment period for 52 weeks.	Drug: ELX/TEZ/IVA Fixed-dose combination tablets for oral administration. Other names: VX-445/VX-661/VX-770 elexacaftor/tezacaftor/ivacaftor Drug: IVA Tablet for oral administration. Other names: VX-770 ivacaftor Drug: Placebo (matched to VX-121/TEZ/D-IVA) Placebo matched to VX-121/TEZ/D-IVA for oral administration.
Experimental VX-121/TEZ/D-IVA	Following ELX/TEZ/IVA run-in period of 4 weeks, participants received VX-121 20 mg qd/TEZ 100 mg qd/D-IVA 250 mg qd in the treatment period for 52 weeks.	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 Drug: Placebo (matched to ELX/TEZ/IVA) Placebo matched to ELX/TEZ/IVA for oral administration. Drug: Placebo (matched to IVA) Placebo matched to IVA for oral administration.

More Details

Status: Completed
Sponsor: Vertex Pharmaceuticals Incorporated

Study Contact

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.