A Study Evaluating the Long-term Safety of VX-445 NCT04043806

Clinical Trial Details — Status: Completed

Administrative data
NCT number	NCT04043806
Other study ID #	VX18-445-113
Secondary ID	2018-004652-38
Status	Completed
Phase	Phase 3
First received
Last updated
Start date	August 9, 2019
Est. completion date	December 14, 2022

Study information
Verified date	June 2023
Source	Vertex Pharmaceuticals Incorporated
Contact	n/a
Is FDA regulated	No
Health authority
Study type	Interventional

Clinical Trial Summary

This study evaluated the long-term safety and tolerability of elexacaftor (ELX), tezacaftor (TEZ), and ivacaftor (IVA) triple combination (TC) treatment in participants with cystic fibrosis (CF).

Recruitment information / eligibility
Status	Completed
Enrollment	458
Est. completion date	December 14, 2022
Est. primary completion date	December 14, 2022
Accepts healthy volunteers	No
Gender	All
Age group	12 Years and older
Eligibility	Inclusion Criteria: - Currently participating in study VX17-659-105 (NCT03447262) Exclusion Criteria: - History of drug intolerance in study VX17-659-105 that would pose an additional risk to the participant in the opinion of the investigator - Current participation in an investigational drug trial (other than study VX17-659-105) Other protocol defined Inclusion/Exclusion criteria may apply.

Study Design

Related Conditions & MeSH terms

Intervention

Drug:
• ELX/TEZ/IVA
Fixed-dose combination (FDC) tablet for oral administration.
• IVA
Tablet for oral administration.

Locations
Country	Name	City	State
Australia	Royal Adelaide Hospital	Adelaide
Australia	The Prince Charles Hospital	Chermside
Australia	Alfred Hospital	Melbourne, VIC
Australia	Institute for Respiratory Health	Nedlands
Australia	Perth Children's Hospital	Nedlands
Australia	John Hunter Hospital & Hunter Medical Research Institute and John Hunter Children's Hospital	New Lambton
Australia	Queensland Children's Hospital	South Brisbane
Canada	Stollery Children's Hospital	Edmonton
Canada	Queen Elizabeth II Health Sciences Center	Halifax
Canada	St. Michael's Hospital	Toronto
Denmark	Juliane Marie Center, Rigshospitalet	Copenhagen
Germany	Charite Paediatric Pulmonology Department	Berlin
Germany	Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen	Essen
Germany	Johann Wolfgang Goethe University	Frankfurt
Germany	Hannover Medical School	Hannover
Germany	Universitaetsklinikum Jena, Mukoviszidose-Zentrum	Jena
Germany	Universitaetsklinkum Koeln, CF-Studienzentrum	Koeln
Germany	Universitatsklinikum Schleswig-Holstein, Klinik für Kinder- und Jugendmedizin	Lübeck
Germany	Pneumologisches Studienzentrum Muenchen-West	Muenchen
Germany	Klinikum Innenstadt, University of Munich	München
Ireland	Cork University Hospital	Cork
Ireland	Beaumont Hospital	Dublin
Ireland	Children's Health Ireland at Crumlin	Dublin
Ireland	Children's Health Ireland at Temple Street	Dublin
Ireland	St. Vincent's University Hospital	Dublin
Ireland	University Hospital Galway	Galway
Ireland	University Hospital Limerick (Adults)	Limerick
Israel	Lady Davis Carmel Medical Center	Haifa
Israel	Pediatric Pulmonary Unit Rambam Medical Center	Haifa
Israel	Hadassah Medical Organization	Jerusalem
Israel	Schneider Children's Medical Center	Petah Tikva
Israel	Sheba Medical Center	Tel Hashomer
Poland	Klinika Mukowiscydozy IMD Oddozial Chorob Pluc Szpzoz IM. Dzieci WarszaWY	Lomianki
Spain	Hospital Universitari Vall d Hebron	Barcelona
Spain	Hospital Universitari Vall d´Hebron Servicio de Broncoscopia	Barcelona
Spain	Hospital Universitario 12 de Octubre	Madrid
Spain	Hospital Universitario Infantil La Paz	Madrid
Spain	Parc Tauli Sabadell Hospital Universitari	Sabadell
Spain	Hospital Universitario Virgen del Rocio	Sevilla
Spain	Hospital Universitario y Politecnico La Fe	Valencia
Switzerland	Lindenhofspital - Quartier Bleu	Bern
Switzerland	Kinderspital Zuerich	Zürich
United Kingdom	Papworth Hospital NHS Foundation Trust, Papworth Everard	Cambridge
United Kingdom	NHS Greater Glasgow and Clyde, Gartnavel General Hospital	Glasgow
United Kingdom	St. James University Hospital	Leeds
United Kingdom	Liverpool Heart and Chest Hospital	Liverpool
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	Nottingham University Hospitals NHS Trust, Queens Medical Center	Nottingham
United Kingdom	All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough	Penarth
United States	Albany Medical College, Pulmonary and Critical Care Medicine	Albany	New York
United States	Michigan Medicine	Ann Arbor	Michigan
United States	Children's Hospital Colorado	Aurora	Colorado
United States	Johns Hopkins Hospital	Baltimore	Maryland
United States	University of Alabama at Birmingham	Birmingham	Alabama
United States	St. Luke's CF Center of Idaho	Boise	Idaho
United States	Boston Children's Hospital	Boston	Massachusetts
United States	Lung and Cystic Fibrosis Center at Women and Children's Hospital of Buffalo	Buffalo	New York
United States	Clinical Research of Charlotte	Charlotte	North Carolina
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
United States	Duke University Medical Center	Durham	North Carolina
United States	Cook Children's Medical Center	Fort Worth	Texas
United States	Cystic Fibrosis Institute	Glenview	Illinois
United States	Hartford Hospital	Hartford	Connecticut
United States	Texas Children's Hospital	Houston	Texas
United States	Indiana University	Indianapolis	Indiana
United States	The University of Iowa Hospitals and Clinics	Iowa City	Iowa
United States	University of Mississippi Medical Center	Jackson	Mississippi
United States	The Children's Mercy Hospital	Kansas City	Missouri
United States	University of Tennessee Medical Center	Knoxville	Tennessee
United States	Dartmouth Hitchcock Medical Center	Lebanon	New Hampshire
United States	University of Kentucky	Lexington	Kentucky
United States	Kosair Charities Pediatric Clinical Research Unit	Louisville	Kentucky
United States	Children's Foundation Research Center / Le Bonheur Children's Hospital	Memphis	Tennessee
United States	Nicklaus Children's Hospital	Miami	Florida
United States	University of Miami Miller School of Medicine	Miami	Florida
United States	University of Minnesota	Minneapolis	Minnesota
United States	Vanderbilt University Medical Center	Nashville	Tennessee
United States	Rutgers Robert Wood Johnson Medical School	New Brunswick	New Jersey
United States	Yale New Haven Hospital	New Haven	Connecticut
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	Advocate Children's Hospital - Park Ridge / North Suburban Pulmonary and Critical Care Consultants	Niles	Illinois
United States	Santiago Reyes, M.D.	Oklahoma City	Oklahoma
United States	Arnold Palmer Hospital Pulmonary and Sleep Medicine	Orlando	Florida
United States	Stanford University	Palo Alto	California
United States	Children's Hospital of Pittsburgh of UPMC	Pittsburgh	Pennsylvania
United States	Oregon Health & Science University	Portland	Oregon
United States	Washington University School of Medicine / St. Louis Children's Hospital	Saint Louis	Missouri
United States	Johns Hopkins All Children's Hospital Outpatient Care Center	Saint Petersburg	Florida
United States	University of Utah / Primary Children's Medical Center	Salt Lake City	Utah
United States	Seattle Children's Hospital	Seattle	Washington
United States	Sanford Children's Speciality Clinic	Sioux Falls	South Dakota
United States	Providence Pediatric Pulmonary & Cystic Fibrosis Clinic	Spokane	Washington
United States	SUNY Upstate Medical University	Syracuse	New York
United States	UMass Memorial Medical Center	Worcester	Massachusetts

Sponsors *(1)*
Lead Sponsor	Collaborator
Vertex Pharmaceuticals Incorporated

Countries where clinical trial is conducted

United States, Australia, Canada, Denmark, Germany, Ireland, Israel, Poland, Spain, Switzerland, United Kingdom,

Outcome
Type	Measure	Description	Time frame	Safety issue
Primary	Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)		From Baseline through Week 100

See also
Status	Clinical Trial	Phase
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Completed	`NCT00803205` - Study of Ataluren (PTC124™) in Cystic Fibrosis	Phase 3
Terminated	`NCT04921332` - Bright Light Therapy for Depression Symptoms in Adults With Cystic Fibrosis (CF) and COPD	N/A
Completed	`NCT03601637` - Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Participants 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del	Phase 3
Terminated	`NCT02769637` - Effect of Acid Blockade on Microbiota and Inflammation in Cystic Fibrosis (CF)
Recruiting	`NCT06012084` - The Development and Evaluation of iCF-PWR for Healthy Siblings of Individuals With Cystic Fibrosis	N/A
Recruiting	`NCT06032273` - Lung Transplant READY CF 2: CARING CF Ancillary RCT	N/A
Recruiting	`NCT06030206` - Lung Transplant READY CF 2: A Multi-site RCT	N/A
Recruiting	`NCT05392855` - Symptom Based Performance of Airway Clearance After Starting Highly Effective Modulators for Cystic Fibrosis (SPACE-CF)	N/A
Recruiting	`NCT06088485` - The Effect of Bone Mineral Density in Patients With Adult Cystic Fibrosis
Recruiting	`NCT04039087` - Sildenafil Exercise: Role of PDE5 Inhibition	Phase 2/Phase 3
Recruiting	`NCT04056702` - Impact of Triple Combination CFTR Therapy on Sinus Disease.
Completed	`NCT04058548` - Clinical Utility of the 1-minute Sit to Stand Test as a Measure of Submaximal Exercise Tolerance in Patients With Cystic Fibrosis During Acute Pulmonary Exacerbation	N/A
Completed	`NCT04038710` - Clinical Outcomes of Triple Combination Therapy in Severe Cystic Fibrosis Disease.
Completed	`NCT03637504` - Feasibility of a Mobile Medication Plan Application in CF Patient Care	N/A
Recruiting	`NCT03506061` - Trikafta in Cystic Fibrosis Patients	Phase 2
Completed	`NCT03566550` - Gut Imaging for Function & Transit in Cystic Fibrosis Study 1
Recruiting	`NCT04828382` - Prospective Study of Pregnancy in Women With Cystic Fibrosis
Completed	`NCT04568980` - Assessment of Contraceptive Safety and Effectiveness in Cystic Fibrosis
Recruiting	`NCT04010253` - Impact of Bronchial Drainage Technique by the Medical Device Simeox® on Respiratory Function and Symptoms in Adult Patients With Cystic Fibrosis	N/A

A Study Evaluating the Long-term Safety of VX-445 Combination Therapy

Clinical Trial Details — Status: Completed

Administrative data

Study information

Clinical Trial Summary

Recruitment information / eligibility

Study Design

Related Conditions & MeSH terms

Intervention

Locations

Sponsors (1)

Countries where clinical trial is conducted

Outcome