Evaluation of VX-659/TEZ/IVA in Cystic Fibrosis Subjects 6 Through 11 Years of Age

Cystic Fibrosis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT03633526
• Other study ID #: VX18-659-106
• Secondary ID: 2018-001711-67
• Status: Terminated
• Phase: Phase 3
• Start date: August 3, 2018
• Est. completion date: January 18, 2019

Study information

• Verified date: January 2020
• Source: Vertex Pharmaceuticals Incorporated
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 18
• Est. completion date: January 18, 2019
• Est. primary completion date: January 18, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years to 11 Years

Eligibility

Key Inclusion Criteria:

• 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.

Key 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.

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• VX-659/TEZ/IVA
VX-659/TEZ/IVA FDC tablet.
• IVA
IVA mono tablet.

Locations

Country	Name	City	State
United States	Children's Hospital Colorado	Aurora	Colorado
United States	Clinical Research of Charlotte	Charlotte	North Carolina
United States	Ann & Robert Lurie Children's Hospital of Chicago	Chicago	Illinois
United States	Texas Children's Hospital	Houston	Texas
United States	The Children's Mercy Hospital	Kansas City	Missouri
United States	Oregon Health & Science University	Portland	Oregon

Sponsors (1)

Lead Sponsor	Collaborator
Vertex Pharmaceuticals Incorporated

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Maximum Observed Concentration (Cmax) of VX-659, TEZ, and IVA		Day 1 and Day 15
Primary	Observed Pre-Dose Concentration (Ctrough) of VX-659, TEZ, and IVA		Day 8 and Day 15
Primary	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	Maximum Observed Concentration (Cmax) of TEZ Metabolite (M1-TEZ), and IVA Metabolite (M1-IVA)		Day 1 and Day 15
Secondary	Observed Pre-Dose Concentration (Ctrough) of TEZ Metabolite (M1-TEZ), and IVA Metabolite (M1-IVA)		Day 8 and Day 15
Secondary	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
Secondary	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)