Study Stopped
Study discontinued after Part A due to Sponsor's discretion.
Evaluation of VX-659/TEZ/IVA in Cystic Fibrosis Subjects 6 Through 11 Years of Age
A Phase 3 Study Evaluating the Pharmacokinetics, Safety, and Tolerability of VX-659/TEZ/IVA Triple Combination Therapy in Cystic Fibrosis Subjects 6 Through 11 Years of Age
2 other identifiers
interventional
18
1 country
6
Brief Summary
This study will evaluate the pharmacokinetics (PK), safety, tolerability, efficacy, and pharmacodynamic effect of VX-659, tezacaftor (TEZ), and ivacaftor (IVA) when dosed in triple combination (TC) in Cystic Fibrosis (CF) subjects with F/F or F/MF genotypes. The study was discontinued after completion of Part A due to Sponsor's discretion.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Aug 2018
Shorter than P25 for phase_3
6 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
July 27, 2018
CompletedStudy Start
First participant enrolled
August 3, 2018
CompletedFirst Posted
Study publicly available on registry
August 16, 2018
CompletedPrimary Completion
Last participant's last visit for primary outcome
January 18, 2019
CompletedStudy Completion
Last participant's last visit for all outcomes
January 18, 2019
CompletedResults Posted
Study results publicly available
February 5, 2020
CompletedFebruary 5, 2020
January 1, 2020
6 months
July 27, 2018
January 17, 2020
January 17, 2020
Conditions
Outcome Measures
Primary Outcomes (3)
Maximum Observed Concentration (Cmax) of VX-659, TEZ, and IVA
Day 1 and Day 15
Observed Pre-Dose Concentration (Ctrough) of VX-659, TEZ, and IVA
Day 8 and Day 15
Area Under the Concentration Versus Time Curve From Time 0 to 6 Hours (AUC0-6h) of VX-659, TEZ, and IVA
Day 1 and Day 15
Secondary Outcomes (4)
Maximum Observed Concentration (Cmax) of TEZ Metabolite (M1-TEZ), and IVA Metabolite (M1-IVA)
Day 1 and Day 15
Observed Pre-Dose Concentration (Ctrough) of TEZ Metabolite (M1-TEZ), and IVA Metabolite (M1-IVA)
Day 8 and Day 15
Area Under the Concentration Versus Time Curve From Time 0 to 6 Hours (AUC0-6h) of TEZ Metabolite (M1-TEZ), and IVA Metabolite (M1-IVA)
Day 1 and Day 15
Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
From first dose of study drug in TC treatment period up to 28 days after last dose of study drug or to the completion of study participation date, whichever occurs first (up to 6 weeks)
Study Arms (1)
VX-659/TEZ/IVA
EXPERIMENTALParticipants who received VX-659 120 milligram (mg)/TEZ 50 mg/ IVA 75 mg as fixed-dose combination (FDC) in the morning and IVA 75 mg as a mono tablet in the evening in the triple combination (TC) treatment period.
Interventions
VX-659/TEZ/IVA FDC tablet.
Eligibility Criteria
You may qualify if:
- Homozygous or heterozygous for F508del mutation (F/F or F/MF genotypes)
- Forced expiratory volume in 1 second (FEV1) value ≥40% of predicted mean for age, sex, and height.
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (6)
Children's Hospital Colorado
Aurora, Colorado, 80045, United States
Ann & Robert Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
The Children's Mercy Hospital
Kansas City, Missouri, 64108, United States
Clinical Research of Charlotte
Charlotte, North Carolina, 28277, United States
Oregon Health & Science University
Portland, Oregon, 97239, United States
Texas Children's Hospital
Houston, Texas, 77030, United States
Related Publications (2)
Heneghan M, Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2023 Nov 20;11(11):CD010966. doi: 10.1002/14651858.CD010966.pub4.
PMID: 37983082DERIVEDSouthern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3.
PMID: 33331662DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Limitations and Caveats
The study was terminated before start of Part B due to sponsor discretion.
Results Point of Contact
- Title
- Medical Monitor
- Organization
- Vertex Pharmaceuticals Incorporated
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SEQUENTIAL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
July 27, 2018
First Posted
August 16, 2018
Study Start
August 3, 2018
Primary Completion
January 18, 2019
Study Completion
January 18, 2019
Last Updated
February 5, 2020
Results First Posted
February 5, 2020
Record last verified: 2020-01