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

Primary Purpose

Cystic Fibrosis

Status
Terminated
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
VX-659/TEZ/IVA
IVA
Sponsored by
Vertex Pharmaceuticals Incorporated
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cystic Fibrosis

Eligibility Criteria

6 Years - 11 Years (Child)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Children's Hospital Colorado
  • Ann & Robert Lurie Children's Hospital of Chicago
  • The Children's Mercy Hospital
  • Clinical Research of Charlotte
  • Oregon Health & Science University
  • Texas Children's Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

VX-659/TEZ/IVA

Arm Description

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.

Outcomes

Primary Outcome Measures

Maximum Observed Concentration (Cmax) of VX-659, TEZ, and IVA
Observed Pre-Dose Concentration (Ctrough) of VX-659, TEZ, and IVA
Area Under the Concentration Versus Time Curve From Time 0 to 6 Hours (AUC0-6h) of VX-659, TEZ, and IVA

Secondary Outcome Measures

Maximum Observed Concentration (Cmax) of TEZ Metabolite (M1-TEZ), and IVA Metabolite (M1-IVA)
Observed Pre-Dose Concentration (Ctrough) of TEZ Metabolite (M1-TEZ), and IVA Metabolite (M1-IVA)
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)
Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

Full Information

First Posted
July 27, 2018
Last Updated
January 17, 2020
Sponsor
Vertex Pharmaceuticals Incorporated
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1. Study Identification

Unique Protocol Identification Number
NCT03633526
Brief Title
Evaluation of VX-659/TEZ/IVA in Cystic Fibrosis Subjects 6 Through 11 Years of Age
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
Study Type
Interventional

2. Study Status

Record Verification Date
January 2020
Overall Recruitment Status
Terminated
Why Stopped
Study discontinued after Part A due to Sponsor's discretion.
Study Start Date
August 3, 2018 (Actual)
Primary Completion Date
January 18, 2019 (Actual)
Study Completion Date
January 18, 2019 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Vertex Pharmaceuticals Incorporated

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Sequential Assignment
Masking
None (Open Label)
Enrollment
18 (Actual)

8. Arms, Groups, and Interventions

Arm Title
VX-659/TEZ/IVA
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
VX-659/TEZ/IVA
Other Intervention Name(s)
VX-659/VX-661/VX-770, VX-659/tezacaftor/ivacaftor
Intervention Description
VX-659/TEZ/IVA FDC tablet.
Intervention Type
Drug
Intervention Name(s)
IVA
Other Intervention Name(s)
VX-770, ivacaftor
Intervention Description
IVA mono tablet.
Primary Outcome Measure Information:
Title
Maximum Observed Concentration (Cmax) of VX-659, TEZ, and IVA
Time Frame
Day 1 and Day 15
Title
Observed Pre-Dose Concentration (Ctrough) of VX-659, TEZ, and IVA
Time Frame
Day 8 and Day 15
Title
Area Under the Concentration Versus Time Curve From Time 0 to 6 Hours (AUC0-6h) of VX-659, TEZ, and IVA
Time Frame
Day 1 and Day 15
Secondary Outcome Measure Information:
Title
Maximum Observed Concentration (Cmax) of TEZ Metabolite (M1-TEZ), and IVA Metabolite (M1-IVA)
Time Frame
Day 1 and Day 15
Title
Observed Pre-Dose Concentration (Ctrough) of TEZ Metabolite (M1-TEZ), and IVA Metabolite (M1-IVA)
Time Frame
Day 8 and Day 15
Title
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)
Time Frame
Day 1 and Day 15
Title
Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time Frame
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)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
11 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Facility Information:
Facility Name
Children's Hospital Colorado
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
Ann & Robert Lurie Children's Hospital of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
The Children's Mercy Hospital
City
Kansas City
State/Province
Missouri
ZIP/Postal Code
64108
Country
United States
Facility Name
Clinical Research of Charlotte
City
Charlotte
State/Province
North Carolina
ZIP/Postal Code
28277
Country
United States
Facility Name
Oregon Health & Science University
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
Texas Children's Hospital
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States

12. IPD Sharing Statement

Citations:
PubMed Identifier
33331662
Citation
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. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3.
Results Reference
derived

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

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