Back to results

A Study of VX-445 in Healthy Subjects and Subjects With Cystic Fibrosis

Primary Purpose

Cystic Fibrosis

Status

Completed

Phase

Phase 1

Locations

International

Study Type

Interventional

Intervention

IVA

TEZ/IVA

VX-445

Matched Placebo

TEZ

VX-561

VX-445

About this trial

This is an interventional treatment trial for Cystic Fibrosis

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesAccepts Healthy Volunteers

Key Inclusion Criteria:

Parts A, B, and C:

Female subjects must be of non-childbearing potential.
Between the ages of 18 and 55 years, inclusive.
Body mass index (BMI) of 18.0 to 32.0 kg/m2, inclusive, and a total body weight >50 kg

Parts D, E, and F:

Body weight ≥35 kg.
Subjects must have an eligible CFTR genotype:
- Parts D and F: Heterozygous for F508del and an MF mutation (F/MF)
- Part E: Homozygous for F508del (F/F)
FEV1 value ≥40% and ≤90% of predicted mean for age, sex, and height.

Key Exclusion Criteria:

Parts A, B, and C:

Any condition possibly affecting drug absorption.
History of febrile illness within 14 days before the first study drug dose.
Glucose-6-phosphate dehydrogenase (G6PD) deficiency.

Parts D, E, and F:

History of clinically significant cirrhosis with or without portal hypertension.
Glucose-6-phosphate dehydrogenase (G6PD) deficiency.
Lung infection with organisms associated with a more rapid decline in pulmonary status.
History of solid organ or hematological transplantation.

Other protocol defined Inclusion/Exclusion criteria may apply.

Sites / Locations

Banner University of Arizona Medical Center
University of Arkansas for Medical Sciences
Valley Children's Healthcare
(Kaiser Permanente) Oakland Medical Center
National Jewish Health
Central Florida Pulmonary Group
Tampa General Hospital Cardiac and Lung Transplant Clinic
Children's Specialty Services at North Druid Hills
Northwestern Memorial Hospital
Covance Clinical Research Unit Inc., Evansville Clinic [Parts A, B, C only]
University of Kansas Medical Center
Tulane Medical Center
Harper University Hospital
Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minn
Morristown Medical Center
University of New Mexico School of Medicine
UC Health Office of Clinical Research
University Hospitals Cleveland Medical Center/Rainbow Babies and Children's Hospital
Nationwide Children's Hospital
Austin Children's Chest Associates
The University of Vermont
University of Virginia Health System
West Virginia University Hospitals
Children's Hospital of the King's Daughters
Virginia Commonwealth University
Medical College of Wisconsin
Monash Medical Center
The Alfred Hospital
The Royal Children's Hospital Melbourne
Mater Adult Hospital
Royal Prince Alfred Hospital
Westmead Hospital
Antwerp University Hospital
Universitair Ziekenhuis Gent
Academic Medical Centre
HagaZiekenhuis van den Haag
Radboud UMC
Erasmus Medical Center

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm 5

Arm 6

Arm 7

Arm 8

Arm 9

Arm 10

Arm 11

Arm 12

Arm 13

Arm 14

Arm 15

Arm Type

Placebo Comparator

Experimental

Placebo Comparator

Experimental

Placebo Comparator

Experimental

Placebo Comparator

Experimental

Active Comparator

Experimental

Placebo Comparator

Experimental

Arm Label

Part A: Pooled Placebo (Except Cohort A7)

Part A: VX-445 (Except Cohort A7)

Part A: VX-445 (Cohort A7)

Part B: Pooled Placebo (Cohort B1 to B4)

Part B: VX-445 (Cohort B1 to B4)

Part C: Pooled Placebo (Cohort C1 to C3)

Part C: VX-445/TEZ/IVA TC (Cohort C1 to C3)

Part D: Placebo

Part D: VX-445/TEZ/IVA TC - Low Dose

Part D: VX-445/TEZ/IVA TC - Medium Dose

Part D: VX-445/TEZ/IVA TC - High Dose

Part E: TEZ/IVA

Part E: VX-445/TEZ/IVA TC

Part F: Placebo

Part F: VX-445/TEZ/VX-561 TC

Arm Description

Participants without CF who received single dose of placebo matched to VX-445 in Cohort A1 to A5.

Participants without CF who received single ascending dose of VX-445 tablet starting from 20 milligrams (mg) to 360 mg in Cohort A1 to A5.

Participants without CF who received single dose of VX-445 100 mg tablet on Day 1 in fasted state and on Day 7 in fed state, followed by VX-445 20 mg intravenous (IV) injection on Day 13 in fed state in Cohort A7.

Participants without CF who received multiple doses of placebo matched to VX-445 once daily (qd) for 10 days in Cohort B1 to B4.

Participants without CF who received VX-445 tablet qd for 10 days in Cohort B1 (60 mg), B2 (120 mg), B3 (240 mg) and B4 (340 mg).

Participants without CF who received placebo matched to VX-445/TEZ/IVA triple combination (TC) qd in the morning and placebo matched to IVA in the evening for 14 days.

Participants without CF who received VX-445 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in Cohort C1; VX-445 280 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in Cohort C2 and VX-445 100 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in Cohort C3 for 14 days.

Participants with CF, F/MF genotype who received placebo matched to VX-445/TEZ/IVA TC qd in the morning and placebo matched to IVA qd in the evening for 4 weeks in the TC treatment period.

Participants with CF, F/MF genotype who received VX-445 50 mg qd/TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks in the TC treatment period.

Participants with CF, F/MF genotype who received VX-445 100 mg qd/TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks in the TC treatment period.

Participants with CF, F/MF genotype who received VX-445 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks in the TC treatment period.

Following run-in period of 4 weeks with TEZ/IVA, participants with CF, F/F genotype who received TEZ 100 mg qd/IVA 150 mg q12h and placebo matched to VX-445 for 4 weeks in the TC treatment period.

Following run-in period of 4 weeks with TEZ/IVA, participants with CF, F/F genotype who received VX-445 200 mg qd/TEZ 100 mg qd /IVA 150 mg q12h for 4 weeks in the TC treatment period.

Participants with CF, F/MF genotype who received placebo matched to VX-445/TEZ/VX-561 for 4 weeks in the TC treatment period.

Participants with CF, F/MF genotype who received VX-445 200 mg qd/TEZ 100 mg qd/VX-561 150 mg qd for 4 weeks in the TC treatment period.

Outcomes

Primary Outcome Measures

Parts A, B and C: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

Parts D, E and F: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

Part D: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)

FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

Part E: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)

FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

Part F: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)

FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

Secondary Outcome Measures

Part A: Maximum Observed Concentration (Cmax) of VX-445

Part A: Area Under Plasma Concentration Time Curve From Time of Dosing to Last Measurable Concentration (AUC0-tlast) of VX-445

Part B: Maximum Observed Concentration (Cmax) of VX-445

Part B: Area Under Plasma Concentration Time Curve From Time of Dosing to Last Measurable Concentration (AUC0-tlast) of VX-445

Part B: Observed Pre-dose Plasma Concentration (Ctrough) of VX-445

Part C: Maximum Observed Concentration (Cmax) of VX-445, TEZ and Its Metabolites (M1-TEZ and M2-TEZ)

Part C: Area Under Plasma Concentration Time Curve From Time of Dosing to Last Measurable Concentration (AUC0-tlast) of VX-445, TEZ and Its Metabolites (M1-TEZ and M2-TEZ)

Part C: Maximum Observed Concentration (Cmax) of IVA and Its Metabolites (M1-IVA and M6-IVA)

Part C: Area Under Plasma Concentration Time Curve From Time of Dosing to Last Measurable Concentration (AUC0-tlast) of IVA and Its Metabolites (M1-IVA and M6-IVA)

Part C: Observed Pre-dose Concentration (Ctrough) of VX-445, TEZ and Its Metabolites (M1-TEZ and M2-TEZ)

Part C: Observed Pre-dose Concentration (Ctrough) of IVA and Its Metabolites (M1-IVA and M6-IVA)

Part D: Observed Pre-dose Plasma Concentration (Ctrough) of VX-445, TEZ and Its Metabolite (M1-TEZ), IVA and Its Metabolite (M1-IVA)

Part E: Observed Pre-dose Concentration (Ctrough) of VX-445, TEZ and Its Metabolite (M1-TEZ) and IVA and Its Metabolite (M1-IVA)

Part F: Observed Pre-dose Concentration (Ctrough) of VX-445, TEZ and Its Metabolite (M1-TEZ) and VX-561

Part D: Absolute Change in Sweat Chloride Concentration

Sweat samples were collected using an approved collection device.

Part E: Absolute Change in Sweat Chloride Concentration

Sweat samples were collected using an approved collection device.

Part F: Absolute Change in Sweat Chloride Concentration

Sweat samples were collected using an approved collection device.

Part D: Relative Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)

FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

Part E: Relative Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)

FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

Part F: Relative Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)

FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

Part D: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score

The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.

Part E: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score

Part F: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score

Full Information

NCT ID

NCT03227471

First Posted

July 18, 2017

Last Updated

December 16, 2021

Sponsor

Vertex Pharmaceuticals Incorporated

1. Study Identification

Unique Protocol Identification Number

NCT03227471

Brief Title

A Study of VX-445 in Healthy Subjects and Subjects With Cystic Fibrosis

Official Title

A Phase 1/2 Study of VX-445 in Healthy Subjects and Subjects With Cystic Fibrosis

Study Type

Interventional

2. Study Status

Record Verification Date

December 2021

Overall Recruitment Status

Completed

Study Start Date

January 23, 2017 (Actual)

Primary Completion Date

March 27, 2018 (Actual)

Study Completion Date

March 27, 2018 (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

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

This is a first-in-human and proof-of-concept study of VX-445. The study includes 6 parts. Parts A, B, and C were conducted in healthy subjects. Parts D, E, and F were conducted in subjects with Cystic Fibrosis (CF) who are homozygous for the F508del mutation of the CF transmembrane conductance regulator (CFTR) gene (F/F genotype), or who are heterozygous for the F508del mutation and a minimal function (MF) CFTR mutation not likely to respond to TEZ, IVA, or TEZ/IVA (F/MF genotypes).

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 1, Phase 2

Interventional Study Model

Parallel Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

225 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Part A: Pooled Placebo (Except Cohort A7)

Arm Type

Placebo Comparator

Arm Description

Participants without CF who received single dose of placebo matched to VX-445 in Cohort A1 to A5.

Arm Title

Part A: VX-445 (Except Cohort A7)

Arm Type

Experimental

Arm Description

Participants without CF who received single ascending dose of VX-445 tablet starting from 20 milligrams (mg) to 360 mg in Cohort A1 to A5.

Arm Title

Part A: VX-445 (Cohort A7)

Arm Type

Experimental

Arm Description

Arm Title

Part B: Pooled Placebo (Cohort B1 to B4)

Arm Type

Placebo Comparator

Arm Description

Participants without CF who received multiple doses of placebo matched to VX-445 once daily (qd) for 10 days in Cohort B1 to B4.

Arm Title

Part B: VX-445 (Cohort B1 to B4)

Arm Type

Experimental

Arm Description

Participants without CF who received VX-445 tablet qd for 10 days in Cohort B1 (60 mg), B2 (120 mg), B3 (240 mg) and B4 (340 mg).

Arm Title

Part C: Pooled Placebo (Cohort C1 to C3)

Arm Type

Placebo Comparator

Arm Description

Participants without CF who received placebo matched to VX-445/TEZ/IVA triple combination (TC) qd in the morning and placebo matched to IVA in the evening for 14 days.

Arm Title

Part C: VX-445/TEZ/IVA TC (Cohort C1 to C3)

Arm Type

Experimental

Arm Description

Arm Title

Part D: Placebo

Arm Type

Placebo Comparator

Arm Description

Participants with CF, F/MF genotype who received placebo matched to VX-445/TEZ/IVA TC qd in the morning and placebo matched to IVA qd in the evening for 4 weeks in the TC treatment period.

Arm Title

Part D: VX-445/TEZ/IVA TC - Low Dose

Arm Type

Experimental

Arm Description

Participants with CF, F/MF genotype who received VX-445 50 mg qd/TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks in the TC treatment period.

Arm Title

Part D: VX-445/TEZ/IVA TC - Medium Dose

Arm Type

Experimental

Arm Description

Participants with CF, F/MF genotype who received VX-445 100 mg qd/TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks in the TC treatment period.

Arm Title

Part D: VX-445/TEZ/IVA TC - High Dose

Arm Type

Experimental

Arm Description

Participants with CF, F/MF genotype who received VX-445 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks in the TC treatment period.

Arm Title

Part E: TEZ/IVA

Arm Type

Active Comparator

Arm Description

Following run-in period of 4 weeks with TEZ/IVA, participants with CF, F/F genotype who received TEZ 100 mg qd/IVA 150 mg q12h and placebo matched to VX-445 for 4 weeks in the TC treatment period.

Arm Title

Part E: VX-445/TEZ/IVA TC

Arm Type

Experimental

Arm Description

Following run-in period of 4 weeks with TEZ/IVA, participants with CF, F/F genotype who received VX-445 200 mg qd/TEZ 100 mg qd /IVA 150 mg q12h for 4 weeks in the TC treatment period.

Arm Title

Part F: Placebo

Arm Type

Placebo Comparator

Arm Description

Participants with CF, F/MF genotype who received placebo matched to VX-445/TEZ/VX-561 for 4 weeks in the TC treatment period.

Arm Title

Part F: VX-445/TEZ/VX-561 TC

Arm Type

Experimental

Arm Description

Participants with CF, F/MF genotype who received VX-445 200 mg qd/TEZ 100 mg qd/VX-561 150 mg qd for 4 weeks in the TC treatment period.

Intervention Type

Drug

Intervention Name(s)

IVA

Other Intervention Name(s)

VX-770, ivacaftor

Intervention Description

IVA tablet for oral administration

Intervention Type

Drug

Intervention Name(s)

TEZ/IVA

Other Intervention Name(s)

VX-661/VX-770, tezacaftor/ivacaftor

Intervention Description

TEZ/IVA fixed-dose combination for oral administration.

Intervention Type

Drug

Intervention Name(s)

VX-445

Other Intervention Name(s)

ELX, elexacaftor

Intervention Description

VX-445 tablet for oral administration.

Intervention Type

Drug

Intervention Name(s)

Matched Placebo

Intervention Description

Matched placebo.

Intervention Type

Drug

Intervention Name(s)

TEZ

Other Intervention Name(s)

VX-661, tezacaftor

Intervention Description

Tablet for oral administration.

Intervention Type

Drug

Intervention Name(s)

VX-561

Other Intervention Name(s)

CTP-656

Intervention Description

Tablet for oral administration.

Intervention Type

Drug

Intervention Name(s)

VX-445

Other Intervention Name(s)

ELX, elexacaftor

Intervention Description

VX-445 IV injection

Primary Outcome Measure Information:

Title

Parts A, B and C: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

Time Frame

From first dose of study drug in treatment period up to safety follow-up (up to 28 days)

Title

Parts D, E and F: Number of Participants With 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 (up to 5 weeks)

Title

Part D: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)

Description

FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

Time Frame

From Baseline through Day 29

Title

Part E: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)

Description

FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

Time Frame

From Baseline through Day 29

Title

Part F: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)

Description

FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

Time Frame

From Baseline through Day 29

Secondary Outcome Measure Information:

Title

Part A: Maximum Observed Concentration (Cmax) of VX-445

Time Frame

Cohort A1-A5: Pre-dose to 96 hours post-dose; Cohort A7: Pre-dose to 120 hours post-dose

Title

Part A: Area Under Plasma Concentration Time Curve From Time of Dosing to Last Measurable Concentration (AUC0-tlast) of VX-445

Time Frame

Cohort A1-5: Pre-dose to 96 hours post-dose; Cohort A7: Pre-dose to 120 hours post-dose

Title

Part B: Maximum Observed Concentration (Cmax) of VX-445

Time Frame

Pre-dose to 96 hours post-dose on Day 1 and Day 10

Title

Part B: Area Under Plasma Concentration Time Curve From Time of Dosing to Last Measurable Concentration (AUC0-tlast) of VX-445

Time Frame

Pre-dose to 96 hours post-dose on Day 1 and Day 10

Title

Part B: Observed Pre-dose Plasma Concentration (Ctrough) of VX-445

Time Frame

Pre-dose on Day 10

Title

Part C: Maximum Observed Concentration (Cmax) of VX-445, TEZ and Its Metabolites (M1-TEZ and M2-TEZ)

Time Frame

Pre-dose to 96 hours post-dose on Day 1, Day 7 and Day 14

Title

Part C: Area Under Plasma Concentration Time Curve From Time of Dosing to Last Measurable Concentration (AUC0-tlast) of VX-445, TEZ and Its Metabolites (M1-TEZ and M2-TEZ)

Time Frame

Pre-dose to 96 hours post-dose on Day 1, Day 7 and Day 14

Title

Part C: Maximum Observed Concentration (Cmax) of IVA and Its Metabolites (M1-IVA and M6-IVA)

Time Frame

Pre-dose to 96 hours post-dose on Day 1, Day 7 and Day 14

Title

Part C: Area Under Plasma Concentration Time Curve From Time of Dosing to Last Measurable Concentration (AUC0-tlast) of IVA and Its Metabolites (M1-IVA and M6-IVA)

Time Frame

Pre-dose to 96 hours post-dose on Day 1, Day 7 and Day 14

Title

Part C: Observed Pre-dose Concentration (Ctrough) of VX-445, TEZ and Its Metabolites (M1-TEZ and M2-TEZ)

Time Frame

Pre-dose on Day 7 and Day 14

Title

Part C: Observed Pre-dose Concentration (Ctrough) of IVA and Its Metabolites (M1-IVA and M6-IVA)

Time Frame

Pre-dose on Day 7 and Day 14

Title

Part D: Observed Pre-dose Plasma Concentration (Ctrough) of VX-445, TEZ and Its Metabolite (M1-TEZ), IVA and Its Metabolite (M1-IVA)

Time Frame

Pre-dose on Day 15 and Day 29

Title

Part E: Observed Pre-dose Concentration (Ctrough) of VX-445, TEZ and Its Metabolite (M1-TEZ) and IVA and Its Metabolite (M1-IVA)

Time Frame

Pre-dose on Day 15 and Day 29

Title

Part F: Observed Pre-dose Concentration (Ctrough) of VX-445, TEZ and Its Metabolite (M1-TEZ) and VX-561

Time Frame

Pre-dose on Day 15 and Day 29

Title

Part D: Absolute Change in Sweat Chloride Concentration

Description

Sweat samples were collected using an approved collection device.

Time Frame

From Baseline through Day 29

Title

Part E: Absolute Change in Sweat Chloride Concentration

Description

Sweat samples were collected using an approved collection device.

Time Frame

From Baseline through Day 29

Title

Part F: Absolute Change in Sweat Chloride Concentration

Description

Sweat samples were collected using an approved collection device.

Time Frame

From Baseline through Day 29

Title

Part D: Relative Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)

Description

FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

Time Frame

From Baseline through Day 29

Title

Part E: Relative Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)

Description

FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

Time Frame

From Baseline through Day 29

Title

Part F: Relative Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)

Description

FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

Time Frame

From Baseline through Day 29

Title

Part D: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score

Description

Time Frame

From Baseline through Day 29

Title

Part E: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score

Description

Time Frame

From Baseline through Day 29

Title

Part F: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score

Description

Time Frame

From Baseline through Day 29

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

Key Inclusion Criteria: Parts A, B, and C: Female subjects must be of non-childbearing potential. Between the ages of 18 and 55 years, inclusive. Body mass index (BMI) of 18.0 to 32.0 kg/m2, inclusive, and a total body weight >50 kg Parts D, E, and F: Body weight ≥35 kg. Subjects must have an eligible CFTR genotype: Parts D and F: Heterozygous for F508del and an MF mutation (F/MF) Part E: Homozygous for F508del (F/F) FEV1 value ≥40% and ≤90% of predicted mean for age, sex, and height. Key Exclusion Criteria: Parts A, B, and C: Any condition possibly affecting drug absorption. History of febrile illness within 14 days before the first study drug dose. Glucose-6-phosphate dehydrogenase (G6PD) deficiency. Parts D, E, and F: History of clinically significant cirrhosis with or without portal hypertension. Glucose-6-phosphate dehydrogenase (G6PD) deficiency. Lung infection with organisms associated with a more rapid decline in pulmonary status. History of solid organ or hematological transplantation. Other protocol defined Inclusion/Exclusion criteria may apply.

Facility Information:

Facility Name

Banner University of Arizona Medical Center

City

Tucson

State/Province

Arizona

ZIP/Postal Code

85724

Country

United States

Facility Name

University of Arkansas for Medical Sciences

City

Little Rock

State/Province

Arkansas

ZIP/Postal Code

72205

Country

United States

Facility Name

Valley Children's Healthcare

City

Madera

State/Province

California

ZIP/Postal Code

93636

Country

United States

Facility Name

(Kaiser Permanente) Oakland Medical Center

City

Oakland

State/Province

California

ZIP/Postal Code

96411

Country

United States

Facility Name

National Jewish Health

City

Denver

State/Province

Colorado

ZIP/Postal Code

80206

Country

United States

Facility Name

Central Florida Pulmonary Group

City

Orlando

State/Province

Florida

ZIP/Postal Code

32803

Country

United States

Facility Name

Tampa General Hospital Cardiac and Lung Transplant Clinic

City

Tampa

State/Province

Florida

ZIP/Postal Code

33606

Country

United States

Facility Name

Children's Specialty Services at North Druid Hills

City

Atlanta

State/Province

Georgia

ZIP/Postal Code

30324

Country

United States

Facility Name

Northwestern Memorial Hospital

City

Chicago

State/Province

Illinois

ZIP/Postal Code

60611

Country

United States

Facility Name

Covance Clinical Research Unit Inc., Evansville Clinic [Parts A, B, C only]

City

Evansville

State/Province

Indiana

ZIP/Postal Code

47710

Country

United States

Facility Name

University of Kansas Medical Center

City

Kansas City

State/Province

Kansas

ZIP/Postal Code

66160

Country

United States

Facility Name

Tulane Medical Center

City

New Orleans

State/Province

Louisiana

ZIP/Postal Code

70112

Country

United States

Facility Name

Harper University Hospital

City

Detroit

State/Province

Michigan

ZIP/Postal Code

48201

Country

United States

Facility Name

Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minn

City

Minneapolis

State/Province

Minnesota

ZIP/Postal Code

55404

Country

United States

Facility Name

Morristown Medical Center

City

Morristown

State/Province

New Jersey

ZIP/Postal Code

07960

Country

United States

Facility Name

University of New Mexico School of Medicine

City

Albuquerque

State/Province

New Mexico

ZIP/Postal Code

87131

Country

United States

Facility Name

UC Health Office of Clinical Research

City

Cincinnati

State/Province

Ohio

ZIP/Postal Code

45267

Country

United States

Facility Name

University Hospitals Cleveland Medical Center/Rainbow Babies and Children's Hospital

City

Cleveland

State/Province

Ohio

ZIP/Postal Code

44106

Country

United States

Facility Name

Nationwide Children's Hospital

City

Columbus

State/Province

Ohio

ZIP/Postal Code

43205

Country

United States

Facility Name

Austin Children's Chest Associates

City

Austin

State/Province

Texas

ZIP/Postal Code

78723

Country

United States

Facility Name

The University of Vermont

City

Burlington

State/Province

Vermont

ZIP/Postal Code

05401

Country

United States

Facility Name

University of Virginia Health System

City

Charlottesville

State/Province

Virginia

ZIP/Postal Code

22908

Country

United States

Facility Name

West Virginia University Hospitals

City

Morgantown

State/Province

Virginia

ZIP/Postal Code

26506

Country

United States

Facility Name

Children's Hospital of the King's Daughters

City

Norfolk

State/Province

Virginia

ZIP/Postal Code

23507

Country

United States

Facility Name

Virginia Commonwealth University

City

Richmond

State/Province

Virginia

ZIP/Postal Code

23298

Country

United States

Facility Name

Medical College of Wisconsin

City

Milwaukee

State/Province

Wisconsin

ZIP/Postal Code

53226

Country

United States

Facility Name

Monash Medical Center

City

Clayton

State/Province

Victoria

Country

Australia

Facility Name

The Alfred Hospital

City

Melbourne

State/Province

Victoria

Country

Australia

Facility Name

The Royal Children's Hospital Melbourne

City

Parkville

State/Province

Victoria

Country

Australia

Facility Name

Mater Adult Hospital

City

Brisbane

Country

Australia

Facility Name

Royal Prince Alfred Hospital

City

Sydney

Country

Australia

Facility Name

Westmead Hospital

City

Sydney

Country

Australia

Facility Name

Antwerp University Hospital

City

Edegem

Country

Belgium

Facility Name

Universitair Ziekenhuis Gent

City

Gent

Country

Belgium

Facility Name

Academic Medical Centre

City

Amsterdam

Country

Netherlands

Facility Name

HagaZiekenhuis van den Haag

City

Den Haag

Country

Netherlands

Facility Name

Radboud UMC

City

Nijmegen

Country

Netherlands

Facility Name

Erasmus Medical Center

City

Rotterdam

Country

Netherlands

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

PubMed Identifier

30334692

Citation

Keating D, Marigowda G, Burr L, Daines C, Mall MA, McKone EF, Ramsey BW, Rowe SM, Sass LA, Tullis E, McKee CM, Moskowitz SM, Robertson S, Savage J, Simard C, Van Goor F, Waltz D, Xuan F, Young T, Taylor-Cousar JL; VX16-445-001 Study Group. VX-445-Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles. N Engl J Med. 2018 Oct 25;379(17):1612-1620. doi: 10.1056/NEJMoa1807120. Epub 2018 Oct 18.

Results Reference

derived

Learn more about this trial

A Study of VX-445 in Healthy Subjects and Subjects With Cystic Fibrosis

We'll reach out to this number within 24 hrs