A Study to Evaluate the Safety and Efficacy of VX-121 Combination Therapy in Subjects With Cystic Fibrosis

Cystic Fibrosis Clinical Trial

Official title:

A Phase 2, Randomized, Double-blind, Controlled Study to Evaluate the Safety and Efficacy of VX-121 Combination Therapy in Subjects Aged 18 Years and Older With Cystic Fibrosis

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03912233
• Other study ID #: VX18-121-101
• Status: Completed
• Phase: Phase 2
• Start date: April 30, 2019
• Est. completion date: December 10, 2019

Study information

• Verified date: March 2023
• Source: Vertex Pharmaceuticals Incorporated
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to evaluate the safety, tolerability and efficacy of VX-121 combination therapy in subjects with cystic fibrosis (CF).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 87
• Est. completion date: December 10, 2019
• Est. primary completion date: December 10, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Key Inclusion Criteria:

• Part 1: Heterozygous for F508del and an MF mutation (F/MF)

• Part 2: Homozygous for F508del (F/F)

• FEV1 value =40% and =90% of the predicted mean for age, sex, and height

Key Exclusion Criteria:

• History of clinically significant cirrhosis with or without portal hypertension

• 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

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• VX-121
Tablets for oral administration.
• TEZ
TEZ tablet for oral administration.
• VX-561
Tablets for oral administration.
• TEZ/IVA
Fixed-dose combination tablets for oral administration.
• IVA
Tablets for oral administration.
• Placebo
Placebos matched to VX-121, TEZ, and VX-561 for oral administration.

Locations

Country	Name	City	State
Germany	Charite Paediatric Pulmonology Department	Berlin
Germany	Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen	Essen
Germany	Pneumologisches Studienzentrum Muenchen-West	Muenchen
Netherlands	Academic Medical Center	Amsterdam
Netherlands	HagaZiekenhuis van den Haag	Den Haag
Netherlands	University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis	Heidelberglaan
Netherlands	UMC St. Radboud	Nijmegen
Netherlands	Erasmus Medical Center	Rotterdam
Portugal	Hospital de Santa Maria	Lisbon
United Kingdom	University Hospitals Birmingham NHS Foundation Trust	Birmingham
United Kingdom	Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital	London
United Kingdom	Wythenshawe Hospital	Manchester
United Kingdom	The Newcastle upon Tyne Hospitals NHS Foundation Trust, The Royal Victoria Infirmary	Newcastle Upon Tyne
United Kingdom	All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough	Penarth
United States	Boston Children's Hospital	Boston	Massachusetts
United States	Nationwide Children's Hospital	Columbus	Ohio
United States	University of Kentucky.	Lexington	Kentucky
United States	Keck Medical Center of University of Southern California	Los Angeles	California
United States	University of Minnesota	Minneapolis	Minnesota
United States	Vanderbilt University Medical Center	Nashville	Tennessee
United States	Tulane Medical Center	New Orleans	Louisiana
United States	Columbia University Medical Center	New York	New York
United States	Kaiser Permanente	Oakland	California
United States	Santiago Reyes, M.D.	Oklahoma City	Oklahoma
United States	University of Texas Health Science Center at San Antonio	San Antonio	Texas
United States	Wake Forest University Baptist Medical Center	Winston-Salem	North Carolina

Sponsors (1)

Lead Sponsor	Collaborator
Vertex Pharmaceuticals Incorporated

Countries where clinical trial is conducted

United States,  Germany,  Netherlands,  Portugal,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)		From Day 1 Through Safety Follow-up (up to Day 75 for Part 1 and up to Day 85 for Part 2)
Primary	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.	From Baseline Through Day 29
Secondary	Absolute Change in Sweat Chloride (SwCl) Concentrations	Sweat samples were collected using an approved collection device.	From Baseline Through Day 29
Secondary	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.	From Baseline at Day 29
Secondary	Observed Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ and Its Metabolite (M1-TEZ) and, VX-561 and Its Metabolites (M1-VX-561 and M6-VX-561)		Pre-dose at Day 15 and Day 29