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)
Sponsor
Vertex Pharmaceuticals Incorporated (Industry)
Overall Status
Recruiting
CT.gov ID
NCT05033080
Collaborator
(none)
400
129
2
27.6
3.1
0.1
Study Details
Study Description
Brief Summary
The purpose of this study is to evaluate the efficacy and safety of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants who are heterozygous for F508del and a minimal function mutation (F/MF participants).
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 3 |
Study Design
Study Type:
Interventional
Anticipated Enrollment
:
400 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose:
Treatment
Official Title:
A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-121 Combination Therapy in Subjects With Cystic Fibrosis (CF) Who Are Heterozygous for F508del and a Minimal Function Mutation (F/MF)
Actual Study Start Date
:
Sep 14, 2021
Anticipated Primary Completion Date
:
Jun 1, 2023
Anticipated Study Completion Date
:
Jan 1, 2024
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: VX-121/TEZ/D-IVA Participants will receive VX-121/TEZ/D-IVA in the morning. |
Drug: VX-121/TEZ/D-IVA
Fixed-dose combination tablets for oral administration.
Other Names:
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.
|
| Active Comparator: ELX/TEZ/IVA Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening. |
Drug: ELX/TEZ/IVA
Fixed-dose combination tablets for oral administration.
Other Names:
Drug: IVA
Tablet for oral administration.
Other Names:
Drug: Placebo (matched to VX-121/TEZ/D-IVA)
Placebo matched to VX-121/TEZ/D-IVA for oral administration.
|
Outcome Measures
Primary Outcome Measures
- Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) Through Week 24 [From Baseline Through Week 24]
Secondary Outcome Measures
- Absolute Change From Baseline in Sweat Chloride (SwCl) Through Week 24 [From Baseline Through Week 24]
- Proportion of Participants With SwCl <60 Millimole per Liter (mmol/L) Through Week 24 (Pooled With Data From Study VX20-121-103) [From Baseline Through Week 24]
- Proportion of Participants With SwCl <30 mmol/L Through Week 24 (Pooled With Data From Study VX20-121-103) [From Baseline Through Week 24]
Eligibility Criteria
Criteria
Ages Eligible for Study:
12 Years
and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
Key Inclusion Criteria:
-
Heterozygous for F508del and a minimal function mutation (F/MF genotype)
-
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
Key Exclusion Criteria:
-
History of solid organ or hematological transplantation
-
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
Other protocol defined Inclusion/Exclusion criteria may apply
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | University of Alabama at Birmingham | Birmingham | Alabama | United States | 35233 |
| 2 | Banner University of Arizona Medical Center | Tucson | Arizona | United States | 85724 |
| 3 | Arkansas Children's Hospital | Little Rock | Arkansas | United States | 72202 |
| 4 | Miller Children's Hospital / Long Beach Memorial | Long Beach | California | United States | 90806 |
| 5 | Children's Hospital Los Angeles | Los Angeles | California | United States | 90027 |
| 6 | Kaiser Permanente | Oakland | California | United States | 94611 |
| 7 | University of California Davis Medical Center | Sacramento | California | United States | 95817 |
| 8 | University of California San Francisco, Lung Transplant Program | San Francisco | California | United States | 94143 |
| 9 | Children's Hospital of Colorado | Aurora | Colorado | United States | 80045 |
| 10 | National Jewish Health | Denver | Colorado | United States | 80206 |
| 11 | University of Florida, Shands Hospital | Gainesville | Florida | United States | 32610 |
| 12 | Joe DiMaggio Cystic Fibrosis & Pulmonary Center | Hollywood | Florida | United States | 33021 |
| 13 | Central Florida Pulmonary Group, PA | Orlando | Florida | United States | 32803 |
| 14 | Nemours Children's Hospital | Orlando | Florida | United States | 32827 |
| 15 | Johns Hopkins All Children's Hospital Outpatient Care Center | Saint Petersburg | Florida | United States | 33701 |
| 16 | The Emory Clinic at Chantilly | Atlanta | Georgia | United States | 30324 |
| 17 | Augusta University | Augusta | Georgia | United States | 30912 |
| 18 | St. Luke's Cystic Fibrosis Center of Idaho | Boise | Idaho | United States | 83702 |
| 19 | Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | United States | 60611 |
| 20 | Northwestern Memorial Hospital | Chicago | Illinois | United States | 60611 |
| 21 | Indiana University | Indianapolis | Indiana | United States | 46202 |
| 22 | Riley Hospital for Children at Indiana University Health | Indianapolis | Indiana | United States | 46202 |
| 23 | University of Kansas Medical Center | Kansas City | Kansas | United States | 66160 |
| 24 | Tulane Medical Center | New Orleans | Louisiana | United States | 70112 |
| 25 | Massachusetts General Hospital Cystic Fibrosis Center Clinical Rsearch Center | Boston | Massachusetts | United States | 02114 |
| 26 | Boston Children's Hospital | Boston | Massachusetts | United States | 02115 |
| 27 | UMass Memorial Medical Center | Worcester | Massachusetts | United States | 01655 |
| 28 | Michigan Medicine | Ann Arbor | Michigan | United States | 48109-5212 |
| 29 | Helen DeVos Children's Hospital CF Center | Grand Rapids | Michigan | United States | 49503 |
| 30 | University of Minnesota | Minneapolis | Minnesota | United States | 55455 |
| 31 | University of Mississippi Medical Center | Jackson | Mississippi | United States | 39216 |
| 32 | Childrens Hospital University of Missouri Health Sciences Center | Columbia | Missouri | United States | 65212 |
| 33 | The Children's Mercy Hospital | Kansas City | Missouri | United States | 64108 |
| 34 | Cardinal Glennon Children's Hospital - St. Louis University | Saint Louis | Missouri | United States | 63104 |
| 35 | Washington University School of Medicine / St. Louis Children's Hospital | Saint Louis | Missouri | United States | 63110 |
| 36 | Billings Clinic | Billings | Montana | United States | 59101 |
| 37 | Nebraska Medical Center | Omaha | Nebraska | United States | 68198 |
| 38 | Dartmouth Hitchcock, Manchester | Manchester | New Hampshire | United States | 03104 |
| 39 | Morristown Medical Center | Morristown | New Jersey | United States | 07960 |
| 40 | Albany Medical College | Albany | New York | United States | 12208 |
| 41 | CF Therapeutics Development Center of Western New York | Buffalo | New York | United States | 14203 |
| 42 | Northwell Health- Long Island Jewish Medical Center | New Hyde Park | New York | United States | 11040 |
| 43 | Columbia University Medical Center | New York | New York | United States | 10032 |
| 44 | New York Medical College | Valhalla | New York | United States | 10595 |
| 45 | Atrium Health Levine Children's Hospital | Charlotte | North Carolina | United States | 28203 |
| 46 | Wake Forest Baptist Health | Winston-Salem | North Carolina | United States | 27104 |
| 47 | Cleveland Clinic CF | Cleveland | Ohio | United States | 44195 |
| 48 | Nationwide Children's Hospital | Columbus | Ohio | United States | 43205 |
| 49 | Dayton Children's Hospital | Dayton | Ohio | United States | 45404 |
| 50 | Oregon Health & Science University | Portland | Oregon | United States | 97239 |
| 51 | Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | United States | 19104 |
| 52 | UPMC Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | United States | 15224 |
| 53 | Sanford Children's Speciality Clinic | Sioux Falls | South Dakota | United States | 57105 |
| 54 | University of Tennessee Medical Center | Knoxville | Tennessee | United States | 37920 |
| 55 | Children's Foundation Research Center / Le Bonheur Children's Hospital | Memphis | Tennessee | United States | 38105 |
| 56 | Vanderbilt University Medical Center | Nashville | Tennessee | United States | 37232 |
| 57 | Driscoll Children's Hospital | Corpus Christi | Texas | United States | 78411 |
| 58 | The University of Texas Southwestern Medical Center | Dallas | Texas | United States | 75390-8558 |
| 59 | Cook Children's Health Care System | Fort Worth | Texas | United States | 76104 |
| 60 | Texas Children's Hospital - Baylor College of Medicine | Houston | Texas | United States | 77030 |
| 61 | University of Utah - Primary Children's Medical Center | Salt Lake City | Utah | United States | 84132 |
| 62 | Vermont Lung Center | Colchester | Vermont | United States | 05446 |
| 63 | University of Virginia Health System | Charlottesville | Virginia | United States | 22908 |
| 64 | Inova Fairfax | Falls Church | Virginia | United States | 22042 |
| 65 | University of Washington Medical Center | Seattle | Washington | United States | 98195 |
| 66 | University of Wisconsin Hospital and Clinics | Madison | Wisconsin | United States | 53792 |
| 67 | Royal Prince Alfred Hospital | Camperdown | Australia | ||
| 68 | The Prince Charles Hospital | Chermside | Australia | ||
| 69 | Alfred Hospital | Melbourne, VIC | Australia | ||
| 70 | Mater Adult Hospital | South Brisbane | Australia | ||
| 71 | Queensland Children's Hospital | South Brisbane | Australia | ||
| 72 | Westmead Hospital | Westmead | Australia | ||
| 73 | Klinika Detskych Infekcnich Nemoci | Brno | Czechia | ||
| 74 | Fakultni nemocnice v Motole | Praha 5 | Czechia | ||
| 75 | Charite Paediatric Pulmonology Department | Berlin | Germany | ||
| 76 | St. Josef-Hospital | Bochum | Germany | ||
| 77 | Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen | Essen | Germany | ||
| 78 | Universitatsklinikum Essen (AoR), Kinderklinik III, Abt. fur Pneumologie | Essen | Germany | ||
| 79 | Johann Wolfgang Goethe University | Frankfurt | Germany | ||
| 80 | Hannover Medical School | Hannover | Germany | ||
| 81 | Medizinische Hochschule Hannover | Hannover | Germany | ||
| 82 | Mukoviszidose-Zentrum am Universitätsklinikum Jena, Klinik für Kinder- und Jugendmedizin | Jena | Germany | ||
| 83 | Johannes Gutenberg-Universitaet | Mainz | Germany | ||
| 84 | Pneumologisches Studienzentrum Muenchen-West | Muenchen | Germany | ||
| 85 | Klinikum Innenstadt, University of Munich | München | Germany | ||
| 86 | Klinikum Westbrandenburg (CF) | Potsdam | Germany | ||
| 87 | Universitätsklinikum Würzburg | Würzburg | Germany | ||
| 88 | Cork University Hospital | Cork | Ireland | ||
| 89 | Children's Health Ireland at Crumlin | Dublin | Ireland | ||
| 90 | Children's Health Ireland at Tallaght | Dublin | Ireland | ||
| 91 | Children's Health Ireland at Temple Street | Dublin | Ireland | ||
| 92 | St. Vincent's University Hospital | Dublin | Ireland | ||
| 93 | University Hospital Limerick (Adults) | Limerick | Ireland | ||
| 94 | University Hospital Limerick (Pediatrics) | Limerick | Ireland | ||
| 95 | Hadassah University Hospital Mount Scopus | Jerusalem | Israel | ||
| 96 | Schneider Children's Medical Center of Israel | Petach Tikvah | Israel | ||
| 97 | Sheba Medical Center - The Edmond and Lili Safra Children's Hospital | Tel HaShomer | Israel | ||
| 98 | Greenlane Clinical Centre | Auckland | New Zealand | ||
| 99 | Starship Children's Hospital | Auckland | New Zealand | ||
| 100 | Canterbury Respiratory Research Group | Christchurch Hospital | New Zealand | ||
| 101 | Waikato Hospital | Hamilton | New Zealand | ||
| 102 | Hospital de Santa Maria | Lisbon | Portugal | ||
| 103 | Hospital Sao Joao | Porto | Portugal | ||
| 104 | Hospital Saint Joan de Deu | Barcelona | Spain | ||
| 105 | Hospital Universitari Vall d Hebron | Barcelona | Spain | ||
| 106 | Hospital Universitario 12 de Octubre | Madrid | Spain | ||
| 107 | Hospital Universitario Ramon y Cajal | Madrid | Spain | ||
| 108 | Hospital Virgen de la Arrixaca | Murcia | Spain | ||
| 109 | Corporacio Sanitaria Parc Tauli - Sabadell Hospital Universitari | Sabadell | Spain | ||
| 110 | Hospital Universitario Virgen del Rocio | Sevilla | Spain | ||
| 111 | Hospital Universitario y Politecnico La Fe | Valencia | Spain | ||
| 112 | Sahlgrenska Universitetssjukhuset | Göteborg | Sweden | ||
| 113 | Lund University Skanes Universitetssjukhus | Malmoe | Sweden | ||
| 114 | Karolinska Universitetssjukhuset, Huddinge | Stockholm | Sweden | ||
| 115 | Birmingham Heartlands Hospital | Birmingham | United Kingdom | ||
| 116 | University Hospitals Bristol and Weston NHS Foundation Trust, Bristol Royal Hospital | Bristol | United Kingdom | ||
| 117 | Royal Papworth Hospital NHS Foundation Trust | Cambridge | United Kingdom | ||
| 118 | Royal Hospital for Sick Children | Edinburgh | United Kingdom | ||
| 119 | Western General Hospital | Edinburgh | United Kingdom | ||
| 120 | Royal Devon and Exeter Hospital | Exeter | United Kingdom | ||
| 121 | Clinical Research Facility, Queen Elizabeth University Hospital | Glasgow | United Kingdom | ||
| 122 | St. James University Hospital | Leeds | United Kingdom | ||
| 123 | King's College Hospital | London | United Kingdom | ||
| 124 | Royal Brompton Hospital | London | United Kingdom | ||
| 125 | St. Bartholomew's Hospital | London | United Kingdom | ||
| 126 | Wythenshawe Hospital | Manchester | United Kingdom | ||
| 127 | Clinical Research Facility | Newcastle Upon Tyne | United Kingdom | ||
| 128 | All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough | Penarth | United Kingdom | ||
| 129 | Southampton General Hospital | Southampton | United Kingdom |
Sponsors and Collaborators
- Vertex Pharmaceuticals Incorporated
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.Responsible Party:
Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier:
NCT05033080
Other Study ID Numbers:
- VX20-121-102
- 2021-000712-31
First Posted:
Sep 2, 2021
Last Update Posted:
Aug 25, 2022
Last Verified:
Apr 1, 2022
Individual Participant Data (IPD) Sharing Statement:
No
Plan to Share IPD:
No
Studies a U.S. FDA-regulated Drug Product:
Yes
Studies a U.S. FDA-regulated Device Product:
No
Additional relevant MeSH terms: