A Phase 2 Study to Evaluate Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis
A Phase 2, Randomized, Double-blind Study to Evaluate the Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis
2 other identifiers
interventional
77
7 countries
48
Brief Summary
The purpose of this study is to evaluate the efficacy, safety, pharmacodynamic (PD) and pharmacokinetic (PK) effect of VX-561.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_2
Started Apr 2019
Shorter than P25 for phase_2
48 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
April 9, 2019
CompletedFirst Posted
Study publicly available on registry
April 11, 2019
CompletedStudy Start
First participant enrolled
April 17, 2019
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 20, 2020
CompletedStudy Completion
Last participant's last visit for all outcomes
August 20, 2020
CompletedResults Posted
Study results publicly available
January 25, 2022
CompletedJanuary 25, 2022
December 1, 2021
1.3 years
April 9, 2019
November 9, 2021
December 31, 2021
Conditions
Outcome Measures
Primary Outcomes (1)
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 at Week 12
Secondary Outcomes (3)
Absolute Change in Sweat Chloride (SwCl)
From Baseline at Week 12
Observed Pre-Dose Concentration (Ctrough) of VX-561 and Its Metabolites (M1-VX-561 and M6-VX-561) and IVA and Its Metabolites (M1-IVA and M6-IVA)
At Week 4
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Baseline up to Week 16
Study Arms (5)
Ivacaftor
ACTIVE COMPARATORParticipants received IVA 150 milligrams (mg) orally every 12 hours (q12h) in the treatment period for 12 weeks.
VX-561: 25 mg
EXPERIMENTALParticipants received VX-561 25 mg orally daily (qd) in the treatment period for 12 weeks.
VX-561: 50 mg
EXPERIMENTALParticipants received VX-561 50 mg orally qd in the treatment period for 12 weeks.
VX-561: 150 mg
EXPERIMENTALParticipants received VX-561 150 mg orally qd in the treatment period for 12 weeks.
VX-561: 250 mg
EXPERIMENTALParticipants received VX-561 250 mg orally qd in the treatment period for 12 weeks.
Interventions
VX-561 tablets for oral administration.
Eligibility Criteria
You may qualify if:
- Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D
- On ivacaftor therapy
- FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height
You may not qualify if:
- History of clinically significant cirrhosis with or without portal hypertension
- History of solid organ or hematological transplantation
- Lung infection with organisms associated with a more rapid decline in pulmonary status
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (48)
University of Alabama at Birmingham
Birmingham, Alabama, 35233, United States
Banner University of Arizona Medical Center
Tucson, Arizona, 85724, United States
Miller Children's Hospital / Long Beach Memorial
Long Beach, California, 90806, United States
UCSF Gateway Medical Center
San Francisco, California, 94143, United States
National Jewish Health
Denver, Colorado, 80206, United States
University of Miami Miller School of Medicine
Miami, Florida, 33136, United States
Central Florida Pulmonary Group, PA
Orlando, Florida, 32803, United States
Indiana University
Indianapolis, Indiana, 46202, United States
University of Kansas Medical Center
Kansas City, Kansas, 66160, United States
University of Kentucky
Lexington, Kentucky, 40536, United States
Johns Hopkins Hospital
Baltimore, Maryland, 21287, United States
Massachusetts General Hospital Cystic Fibrosis Center Clinical Research Center
Boston, Massachusetts, 02114, United States
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
Michigan Medicine
Ann Arbor, Michigan, 48109-5212, United States
University of Minnesota
Minneapolis, Minnesota, 55455, United States
Cardinal Glennon Children's Hospital
St Louis, Missouri, 63104, United States
Washington University School of Medicine / St. Louis Children's Hospital
St Louis, Missouri, 63110, United States
Dartmouth Hitchcock Medical Center
Lebanon, New Hampshire, 03756, United States
Columbia University Medical Center
New York, New York, 10032, United States
University of North Carolina Hospitals
Chapel Hill, North Carolina, 27514, United States
UC Health Holmes
Cincinnati, Ohio, 45220, United States
ProMedica Toledo Hospital/Toledo Children's Hospital/Pediatric Pulmonary & Cystic Fibrosis Center
Toledo, Ohio, 43606, United States
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, 73104, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224, United States
Medical University of South Carolina
Charleston, South Carolina, 29425, United States
Dell Children's Medical Group
Austin, Texas, 78723, United States
The University of Texas Southwestern Medical Center
Dallas, Texas, 75390, United States
University of Utah / Primary Children's Medical Center
Salt Lake City, Utah, 84132, United States
University of Wisconsin Hospital and Clinics
Madison, Wisconsin, 53792, United States
The Prince Charles Hospital
Chermside, Australia
Alfred Hospital
Melbourne, VIC, Australia
Mater Adult Hospital
South Brisbane, Australia
Westmead Hospital
Westmead, Australia
Universitair Ziekenhuis Gent
Ghent, Belgium
Friedrich-Alexander University of Erlangen-Nuremberg, University Children's Hospital
Erlangen, Germany
Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen
Essen, Germany
Universitätsklinikum Halle (Saale) / Universitätsklinik und Poliklinik für Innere Medizin, Schwerpunkt Pneumologie
Halle, Germany
Pneumologisches Studienzentrum Muenchen-West
München, Germany
University Hospital Wuerzburg
Würzburg, Germany
Beaumont Hospital
Dublin, Ireland
Cork University Hospital
Dublin, Ireland
St. Vincent's University Hospital
Dublin, Ireland
University Hospital Limerick (Adults)
Limerick, Ireland
UMC St. Radboud
Nijmegen, Netherlands
Clinical Research Facility, Queen Elizabeth University Hospital
Glasgow, United Kingdom
St. James University Hospital
Leeds, United Kingdom
Liverpool Heart and Chest Hospital
Liverpool, United Kingdom
Related Publications (1)
Uluer AZ, MacGregor G, Azevedo P, Indihar V, Keating C, Mall MA, McKone EF, Ramsey BW, Rowe SM, Rubenstein RC, Taylor-Cousar JL, Tullis E, Yonker LM, Chu C, Lam AP, Nair N, Sosnay PR, Tian S, Van Goor F, Viswanathan L, Waltz D, Wang LT, Xi Y, Billings J, Horsley A; VX18-121-101; VX18-561-101 Study Groups. Safety and efficacy of vanzacaftor-tezacaftor-deutivacaftor in adults with cystic fibrosis: randomised, double-blind, controlled, phase 2 trials. Lancet Respir Med. 2023 Jun;11(6):550-562. doi: 10.1016/S2213-2600(22)00504-5. Epub 2023 Feb 23.
PMID: 36842446DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Medical Monitor
- Organization
- Vertex Pharmaceuticals Incorporated
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
April 9, 2019
First Posted
April 11, 2019
Study Start
April 17, 2019
Primary Completion
August 20, 2020
Study Completion
August 20, 2020
Last Updated
January 25, 2022
Results First Posted
January 25, 2022
Record last verified: 2021-12
Data Sharing
- IPD Sharing
- Will not share
Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing