A Phase 3 Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Heterozygous for F508del and a Minimal Function Mutation (F/MF)
NCT ID: NCT05033080
Last Updated: 2024-10-03
Study Results
Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.
View full resultsBasic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
COMPLETED
PHASE3
435 participants
INTERVENTIONAL
2021-09-14
2023-11-21
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
A Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Who Are Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive (TCR) CFTR Mutation and No F508del Mutation
NCT05076149
A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
NCT03525444
A Study to Evaluate the Safety and Efficacy of VX-121 Combination Therapy in Subjects With Cystic Fibrosis
NCT03912233
A Phase 3 Study of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
NCT03447249
A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy
NCT03525574
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
ELX/TEZ/IVA
Following elexacftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) run-in period of 4 weeks, participants received ELX 200 milligram (mg) once daily (qd) /TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the treatment period for 52 weeks.
ELX/TEZ/IVA
Fixed-dose combination tablets for oral administration.
IVA
Tablet for oral administration.
Placebo (matched to VX-121/TEZ/D-IVA)
Placebo matched to VX-121/TEZ/D-IVA for oral administration.
VX-121/TEZ/D-IVA
Following ELX/TEZ/IVA run-in period of 4 weeks, participants received VX-121 20 mg qd/TEZ 100 mg qd/D-IVA 250 mg qd in the treatment period for 52 weeks.
VX-121/TEZ/D-IVA
Fixed-dose combination tablets for oral administration.
Placebo (matched to ELX/TEZ/IVA)
Placebo matched to ELX/TEZ/IVA for oral administration.
Placebo (matched to IVA)
Placebo matched to IVA for oral administration.
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
VX-121/TEZ/D-IVA
Fixed-dose combination tablets for oral administration.
ELX/TEZ/IVA
Fixed-dose combination tablets for oral administration.
IVA
Tablet for oral administration.
Placebo (matched to VX-121/TEZ/D-IVA)
Placebo matched to VX-121/TEZ/D-IVA for oral administration.
Placebo (matched to ELX/TEZ/IVA)
Placebo matched to ELX/TEZ/IVA for oral administration.
Placebo (matched to IVA)
Placebo matched to IVA for oral administration.
Other Intervention Names
Discover alternative or legacy names that may be used to describe the listed interventions across different sources.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* Forced expiratory volume in 1 second (FEV1) value \>=40% and \<=90% of predicted mean for age, sex, and height for participants currently receiving ELX/TEZ/IVA therapy; FEV1 \>=40% and \<=80% for participants not currently receiving ELX/TEZ/IVA
Exclusion Criteria
* Hepatic cirrhosis with portal hypertension, moderate hepatic impairment (Child Pugh Score 7 to 9), or severe hepatic impairment (Child Pugh Score 10 to 15)
* Lung infection with organisms associated with a more rapid decline in pulmonary status
* Pregnant or breast-feeding females
12 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Vertex Pharmaceuticals Incorporated
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
University of Alabama at Birmingham
Birmingham, Alabama, United States
Banner University of Arizona Medical Center
Tucson, Arizona, United States
Arkansas Children's Hospital
Little Rock, Arkansas, United States
Miller Children's Hospital / Long Beach Memorial
Long Beach, California, United States
Children's Hospital Los Angeles
Los Angeles, California, United States
Kaiser Permanente
Oakland, California, United States
University of California Davis Medical Center
Sacramento, California, United States
University of California San Francisco, Lung Transplant Program
San Francisco, California, United States
Children's Hospital of Colorado
Aurora, Colorado, United States
National Jewish Health
Denver, Colorado, United States
University of Florida, Shands Hospital
Gainesville, Florida, United States
Joe DiMaggio Cystic Fibrosis & Pulmonary Center
Hollywood, Florida, United States
Central Florida Pulmonary Group, PA
Orlando, Florida, United States
Nemours Children's Hospital
Orlando, Florida, United States
Johns Hopkins All Children's Hospital Outpatient Care Center
St. Petersburg, Florida, United States
The Emory Clinic at Chantilly
Atlanta, Georgia, United States
Augusta University
Augusta, Georgia, United States
St. Luke's Cystic Fibrosis Center of Idaho
Boise, Idaho, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Northwestern Memorial Hospital
Chicago, Illinois, United States
Indiana University
Indianapolis, Indiana, United States
Riley Hospital for Children at Indiana University Health
Indianapolis, Indiana, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
University of Kentucky
Lexington, Kentucky, United States
Kosair Charities Pediatric Clinical Research Unit
Louisville, Kentucky, United States
Tulane Medical Center
New Orleans, Louisiana, United States
Massachusetts General Hospital Cystic Fibrosis Center Clinical Rsearch Center
Boston, Massachusetts, United States
Boston Children's Hospital
Boston, Massachusetts, United States
UMass Memorial Medical Center
Worcester, Massachusetts, United States
Michigan Medicine
Ann Arbor, Michigan, United States
Helen DeVos Children's Hospital CF Center
Grand Rapids, Michigan, United States
University of Minnesota
Minneapolis, Minnesota, United States
University of Mississippi Medical Center
Jackson, Mississippi, United States
Childrens Hospital University of Missouri Health Sciences Center
Columbia, Missouri, United States
The Children's Mercy Hospital
Kansas City, Missouri, United States
Cardinal Glennon Children's Hospital - St. Louis University
St Louis, Missouri, United States
Washington University School of Medicine / St. Louis Children's Hospital
St Louis, Missouri, United States
Billings Clinic
Billings, Montana, United States
Nebraska Medical Center
Omaha, Nebraska, United States
Dartmouth Hitchcock, Manchester
Manchester, New Hampshire, United States
Morristown Medical Center
Morristown, New Jersey, United States
Albany Medical College
Albany, New York, United States
CF Therapeutics Development Center of Western New York
Buffalo, New York, United States
Northwell Health- Long Island Jewish Medical Center
New Hyde Park, New York, United States
Columbia University Medical Center
New York, New York, United States
New York Medical College
Valhalla, New York, United States
Atrium Health Levine Children's Hospital
Charlotte, North Carolina, United States
Wake Forest Baptist Health
Winston-Salem, North Carolina, United States
Cleveland Clinic CF
Cleveland, Ohio, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Dayton Children's Hospital
Dayton, Ohio, United States
ProMedica Toledo Hospital/Toledo Children's Hospital/Pediatric Pulmonary & Cystic Fibrosis Center
Toledo, Ohio, United States
Oregon Health & Science University
Portland, Oregon, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
Sanford Children's Speciality Clinic
Sioux Falls, South Dakota, United States
University of Tennessee Medical Center
Knoxville, Tennessee, United States
Children's Foundation Research Center / Le Bonheur Children's Hospital
Memphis, Tennessee, United States
Vanderbilt University Medical Center
Nashville, Tennessee, United States
Driscoll Children's Hospital
Corpus Christi, Texas, United States
The University of Texas Southwestern Medical Center
Dallas, Texas, United States
Cook Children's Health Care System
Fort Worth, Texas, United States
Texas Children's Hospital - Baylor College of Medicine
Houston, Texas, United States
University of Utah - Primary Children's Medical Center
Salt Lake City, Utah, United States
Vermont Lung Center
Colchester, Vermont, United States
University of Virginia Health System
Charlottesville, Virginia, United States
Inova Fairfax
Falls Church, Virginia, United States
University of Washington Medical Center
Seattle, Washington, United States
University of Wisconsin Hospital and Clinics
Madison, Wisconsin, United States
Royal Prince Alfred Hospital
Camperdown, , Australia
The Prince Charles Hospital
Chermside, , Australia
Alfred Hospital
Melbourne, VIC, , Australia
Mater Adult Hospital
South Brisbane, , Australia
Queensland Children's Hospital
South Brisbane, , Australia
Westmead Hospital
Westmead, , Australia
Klinika Detskych Infekcnich Nemoci
Brno, , Czechia
Fakultni nemocnice v Motole
Prague, , Czechia
Charite Paediatric Pulmonology Department
Berlin, , Germany
St. Josef-Hospital
Bochum, , Germany
Friedrich-Alexander University of Erlangen-Nuremberg, University Children's Hospital
Erlangen, , Germany
Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen
Essen, , Germany
Universitatsklinikum Essen (AoR), Kinderklinik III, Abt. fur Pneumologie
Essen, , Germany
Johann Wolfgang Goethe University
Frankfurt, , Germany
Hannover Medical School
Hanover, , Germany
Medizinische Hochschule Hannover
Hanover, , Germany
Mukoviszidose-Zentrum am Universitätsklinikum Jena, Klinik für Kinder- und Jugendmedizin
Jena, , Germany
Johannes Gutenberg-Universitaet
Mainz, , Germany
Klinikum Innenstadt, University of Munich
München, , Germany
Pneumologisches Studienzentrum Muenchen-West
München, , Germany
Klinikum Westbrandenburg (CF)
Potsdam, , Germany
Universitätsklinikum Ulm, Klinik für Kinder- und Jugendmedizin (CF)
Ulm, , Germany
Universitätsklinikum Würzburg
Würzburg, , Germany
National Koranyi Institute for TBC and Pulmonology
Budapest, , Hungary
Pulmonology Institute Torokbalint
Törökbálint, , Hungary
Cork University Hospital
Cork, , Ireland
Children's Health Ireland at Crumlin
Dublin, , Ireland
Children's Health Ireland at Tallaght
Dublin, , Ireland
Children's Health Ireland at Temple Street
Dublin, , Ireland
St. Vincent's University Hospital
Dublin, , Ireland
University Hospital Limerick (Adults)
Limerick, , Ireland
University Hospital Limerick (Pediatrics)
Limerick, , Ireland
Hadassah University Hospital Mount Scopus
Jerusalem, , Israel
Schneider Children's Medical Center of Israel
Petach Tikvah, , Israel
Sheba Medical Center - The Edmond and Lili Safra Children's Hospital
Tel Litwinsky, , Israel
Greenlane Clinical Centre
Auckland, , New Zealand
Starship Children's Hospital
Auckland, , New Zealand
Canterbury Respiratory Research Group
Christchurch Hospital, , New Zealand
Waikato Hospital
Hamilton, , New Zealand
Hospital de Santa Maria
Lisbon, , Portugal
CHP - Hospital de Santo Antonio
Porto, , Portugal
Hospital Sao Joao
Porto, , Portugal
Hospital Saint Joan de Deu
Barcelona, , Spain
Hospital Universitari Vall d Hebron
Barcelona, , Spain
Hospital Infantil Universitario Nino Jesus
Madrid, , Spain
Hospital Universitario 12 de Octubre
Madrid, , Spain
Hospital Universitario Ramon y Cajal
Madrid, , Spain
Hospital Virgen de la Arrixaca
Murcia, , Spain
Corporacio Sanitaria Parc Tauli - Sabadell Hospital Universitari
Sabadell, , Spain
Hospital Universitario Virgen del Rocio
Seville, , Spain
Hospital Universitario y Politecnico La Fe
Valencia, , Spain
Sahlgrenska Universitetssjukhuset
Gothenburg, , Sweden
Lund University Skanes Universitetssjukhus
Malmo, , Sweden
Karolinska Universitetssjukhuset, Huddinge
Stockholm, , Sweden
Birmingham Heartlands Hospital
Birmingham, , United Kingdom
University Hospitals Bristol and Weston NHS Foundation Trust, Bristol Royal Hospital
Bristol, , United Kingdom
Royal Papworth Hospital NHS Foundation Trust
Cambridge, , United Kingdom
Royal Hospital for Sick Children
Edinburgh, , United Kingdom
Western General Hospital
Edinburgh, , United Kingdom
Royal Devon and Exeter Hospital
Exeter, , United Kingdom
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
King's College Hospital
London, , United Kingdom
Royal Brompton Hospital
London, , United Kingdom
St. Bartholomew's Hospital
London, , United Kingdom
Wythenshawe Hospital
Manchester, , United Kingdom
Clinical Research Facility
Newcastle upon Tyne, , United Kingdom
All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough
Penarth, , United Kingdom
Southampton General Hospital
Southampton, , United Kingdom
Countries
Review the countries where the study has at least one active or historical site.
References
Explore related publications, articles, or registry entries linked to this study.
Keating C, Yonker LM, Vermeulen F, Prais D, Linnemann RW, Trimble A, Kotsimbos T, Mermis J, Braun AT, O'Carroll M, Sutharsan S, Ramsey B, Mall MA, Taylor-Cousar JL, McKone EF, Tullis E, Floreth T, Michelson P, Sosnay PR, Nair N, Zahigian R, Martin H, Ahluwalia N, Lam A, Horsley A; VX20-121-102 Study Group; VX20-121-103 Study Group. Vanzacaftor-tezacaftor-deutivacaftor versus elexacaftor-tezacaftor-ivacaftor in individuals with cystic fibrosis aged 12 years and older (SKYLINE Trials VX20-121-102 and VX20-121-103): results from two randomised, active-controlled, phase 3 trials. Lancet Respir Med. 2025 Mar;13(3):256-271. doi: 10.1016/S2213-2600(24)00411-9. Epub 2025 Jan 2.
Heneghan M, 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. 2023 Nov 20;11(11):CD010966. doi: 10.1002/14651858.CD010966.pub4.
Provided Documents
Download supplemental materials such as informed consent forms, study protocols, or participant manuals.
Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
2021-000712-31
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
VX20-121-102
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.