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

Phase 3

Terminated

• Conditions: Cystic Fibrosis
• Interventions: Drug: VX-659/TEZ/IVA; Drug: IVA

• Registration Number: NCT03633526
• Lead Sponsor: Vertex Pharmaceuticals Incorporated

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2018-07-27
• Study Start: 2018-08-03
• Study First Submitted QC: 2018-08-13
• Study First Posted: 2018-08-16
• Primary Completion: 2019-01-18
• Study Completion: 2019-01-18
• Status Verified: 2020-01
• Results First Submitted: 2020-01-17
• Results First Submitted QC: 2020-01-17
• Last Update Submitted: 2020-01-17
• Results First Posted: 2020-02-05
• Last Update Posted: 2020-02-05

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 18

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
VX-659/TEZ/IVA	VX-659/TEZ/IVA	Participants 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.
VX-659/TEZ/IVA	IVA	Participants 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.

Primary Outcome Measures

Name	Time	Method
Observed Pre-Dose Concentration (Ctrough) of VX-659, TEZ, and IVA	Day 8 and Day 15
Maximum Observed Concentration (Cmax) of VX-659, TEZ, and IVA	Day 1 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 Outcome Measures

Name	Time	Method
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)
Observed Pre-Dose Concentration (Ctrough) of TEZ Metabolite (M1-TEZ), and IVA Metabolite (M1-IVA)	Day 8 and Day 15
Maximum Observed Concentration (Cmax) of TEZ Metabolite (M1-TEZ), and IVA Metabolite (M1-IVA)	Day 1 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

Trial Locations

Locations (6)

Children's Hospital Colorado; 🇺🇸 Aurora, Colorado, United States

Ann & Robert Lurie Children's Hospital of Chicago; 🇺🇸 Chicago, Illinois, United States

Clinical Research of Charlotte; 🇺🇸 Charlotte, North Carolina, 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, United States