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)

PHASE3CompletedINTERVENTIONAL
Enrollment

435

Participants

Timeline

Start Date

September 14, 2021

Primary Completion Date

May 12, 2023

Study Completion Date

November 21, 2023

Conditions
Cystic Fibrosis
Interventions
DRUG

VX-121/TEZ/D-IVA

Fixed-dose combination tablets for oral administration.

DRUG

ELX/TEZ/IVA

Fixed-dose combination tablets for oral administration.

DRUG

IVA

Tablet for oral administration.

DRUG

Placebo (matched to VX-121/TEZ/D-IVA)

Placebo matched to VX-121/TEZ/D-IVA for oral administration.

DRUG

Placebo (matched to ELX/TEZ/IVA)

Placebo matched to ELX/TEZ/IVA for oral administration.

DRUG

Placebo (matched to IVA)

Placebo matched to IVA for oral administration.

Trial Locations (139)

10032

Columbia University Medical Center, New York

10595

New York Medical College, Valhalla

11040

Northwell Health- Long Island Jewish Medical Center, New Hyde Park

12208

Albany Medical College, Albany

14203

CF Therapeutics Development Center of Western New York, Buffalo

15224

UPMC Children's Hospital of Pittsburgh, Pittsburgh

19104

Children's Hospital of Philadelphia, Philadelphia

22042

Inova Fairfax, Falls Church

22908

University of Virginia Health System, Charlottesville

27104

Wake Forest Baptist Health, Winston-Salem

28203

Atrium Health Levine Children's Hospital, Charlotte

30324

The Emory Clinic at Chantilly, Atlanta

30912

Augusta University, Augusta

32610

University of Florida, Shands Hospital, Gainesville

32803

Central Florida Pulmonary Group, PA, Orlando

32827

Nemours Children's Hospital, Orlando

33021

Joe DiMaggio Cystic Fibrosis & Pulmonary Center, Hollywood

33701

Johns Hopkins All Children's Hospital Outpatient Care Center, St. Petersburg

35233

University of Alabama at Birmingham, Birmingham

37232

Vanderbilt University Medical Center, Nashville

37920

University of Tennessee Medical Center, Knoxville

38105

Children's Foundation Research Center / Le Bonheur Children's Hospital, Memphis

39216

University of Mississippi Medical Center, Jackson

40202

Kosair Charities Pediatric Clinical Research Unit, Louisville

40536

University of Kentucky, Lexington

43205

Nationwide Children's Hospital, Columbus

43606

ProMedica Toledo Hospital/Toledo Children's Hospital/Pediatric Pulmonary & Cystic Fibrosis Center, Toledo

44195

Cleveland Clinic CF, Cleveland

45404

Dayton Children's Hospital, Dayton

46202

Indiana University, Indianapolis

Riley Hospital for Children at Indiana University Health, Indianapolis

49503

Helen DeVos Children's Hospital CF Center, Grand Rapids

53792

University of Wisconsin Hospital and Clinics, Madison

55455

University of Minnesota, Minneapolis

57105

Sanford Children's Speciality Clinic, Sioux Falls

59101

Billings Clinic, Billings

60611

Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago

Northwestern Memorial Hospital, Chicago

63104

Cardinal Glennon Children's Hospital - St. Louis University, St Louis

63110

Washington University School of Medicine / St. Louis Children's Hospital, St Louis

64108

The Children's Mercy Hospital, Kansas City

65212

Childrens Hospital University of Missouri Health Sciences Center, Columbia

66160

University of Kansas Medical Center, Kansas City

68198

Nebraska Medical Center, Omaha

70112

Tulane Medical Center, New Orleans

72202

Arkansas Children's Hospital, Little Rock

76104

Cook Children's Health Care System, Fort Worth

77030

Texas Children's Hospital - Baylor College of Medicine, Houston

78411

Driscoll Children's Hospital, Corpus Christi

80045

Children's Hospital of Colorado, Aurora

80206

National Jewish Health, Denver

83702

St. Luke's Cystic Fibrosis Center of Idaho, Boise

84132

University of Utah - Primary Children's Medical Center, Salt Lake City

85724

Banner University of Arizona Medical Center, Tucson

90027

Children's Hospital Los Angeles, Los Angeles

90806

Miller Children's Hospital / Long Beach Memorial, Long Beach

94143

University of California San Francisco, Lung Transplant Program, San Francisco

94611

Kaiser Permanente, Oakland

95817

University of California Davis Medical Center, Sacramento

97239

Oregon Health & Science University, Portland

98195

University of Washington Medical Center, Seattle

02114

Massachusetts General Hospital Cystic Fibrosis Center Clinical Rsearch Center, Boston

02115

Boston Children's Hospital, Boston

01655

UMass Memorial Medical Center, Worcester

48109-5212

Michigan Medicine, Ann Arbor

03104

Dartmouth Hitchcock, Manchester, Manchester

07960

Morristown Medical Center, Morristown

75390-8558

The University of Texas Southwestern Medical Center, Dallas

05446

Vermont Lung Center, Colchester

Unknown

Royal Prince Alfred Hospital, Camperdown

The Prince Charles Hospital, Chermside

Alfred Hospital, Melbourne, VIC

Mater Adult Hospital, South Brisbane

Queensland Children's Hospital, South Brisbane

Westmead Hospital, Westmead

Klinika Detskych Infekcnich Nemoci, Brno

Fakultni nemocnice v Motole, Prague

Charite Paediatric Pulmonology Department, Berlin

St. Josef-Hospital, Bochum

Friedrich-Alexander University of Erlangen-Nuremberg, University Children's Hospital, Erlangen

Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen, Essen

Universitatsklinikum Essen (AoR), Kinderklinik III, Abt. fur Pneumologie, Essen

Johann Wolfgang Goethe University, Frankfurt

Hannover Medical School, Hanover

Medizinische Hochschule Hannover, Hanover

Mukoviszidose-Zentrum am Universitätsklinikum Jena, Klinik für Kinder- und Jugendmedizin, Jena

Johannes Gutenberg-Universitaet, Mainz

Klinikum Innenstadt, University of Munich, München

Pneumologisches Studienzentrum Muenchen-West, München

Klinikum Westbrandenburg (CF), Potsdam

Universitätsklinikum Ulm, Klinik für Kinder- und Jugendmedizin (CF), Ulm

Universitätsklinikum Würzburg, Würzburg

National Koranyi Institute for TBC and Pulmonology, Budapest

Pulmonology Institute Torokbalint, Törökbálint

Cork University Hospital, Cork

Children's Health Ireland at Crumlin, Dublin

Children's Health Ireland at Tallaght, Dublin

Children's Health Ireland at Temple Street, Dublin

St. Vincent's University Hospital, Dublin

University Hospital Limerick (Adults), Limerick

University Hospital Limerick (Pediatrics), Limerick

Hadassah University Hospital Mount Scopus, Jerusalem

Schneider Children's Medical Center of Israel, Petach Tikvah

Sheba Medical Center - The Edmond and Lili Safra Children's Hospital, Tel Litwinsky

Greenlane Clinical Centre, Auckland

Starship Children's Hospital, Auckland

Canterbury Respiratory Research Group, Christchurch Hospital

Waikato Hospital, Hamilton

Hospital de Santa Maria, Lisbon

CHP - Hospital de Santo Antonio, Porto

Hospital Sao Joao, Porto

Hospital Saint Joan de Deu, Barcelona

Hospital Universitari Vall d Hebron, Barcelona

Hospital Infantil Universitario Nino Jesus, Madrid

Hospital Universitario 12 de Octubre, Madrid

Hospital Universitario Ramon y Cajal, Madrid

Hospital Virgen de la Arrixaca, Murcia

Corporacio Sanitaria Parc Tauli - Sabadell Hospital Universitari, Sabadell

Hospital Universitario Virgen del Rocio, Seville

Hospital Universitario y Politecnico La Fe, Valencia

Sahlgrenska Universitetssjukhuset, Gothenburg

Lund University Skanes Universitetssjukhus, Malmo

Karolinska Universitetssjukhuset, Huddinge, Stockholm

Birmingham Heartlands Hospital, Birmingham

University Hospitals Bristol and Weston NHS Foundation Trust, Bristol Royal Hospital, Bristol

Royal Papworth Hospital NHS Foundation Trust, Cambridge

Royal Hospital for Sick Children, Edinburgh

Western General Hospital, Edinburgh

Royal Devon and Exeter Hospital, Exeter

Clinical Research Facility, Queen Elizabeth University Hospital, Glasgow

St. James University Hospital, Leeds

Liverpool Heart and Chest Hospital, Liverpool

King's College Hospital, London

Royal Brompton Hospital, London

St. Bartholomew's Hospital, London

Wythenshawe Hospital, Manchester

Clinical Research Facility, Newcastle upon Tyne

All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough, Penarth

Southampton General Hospital, Southampton

All Listed Sponsors
lead

Vertex Pharmaceuticals Incorporated

INDUSTRY

NCT05033080 - 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) | Biotech Hunter | Biotech Hunter