A Phase 1/2 Dose Escalation Study Evaluating the Safety, and Tolerability and Efficacy of VX-522 in Subjects 18 Years of Age and Older With Cystic Fibrosis and a CFTR Genotype Not Responsive to CFTR Modulator Therapy
Status: Recruiting
Location: See all (42) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1/Phase 2
SUMMARY
The purpose of this study is to evaluate the safety, and tolerability and efficacy of VX-522 in participants 18 years of age and older with cystic fibrosis and a cystic fibrosis transmembrane conductance regulator (CFTR) genotype not responsive to CFTR modulator therapy.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 65
Healthy Volunteers: f
View:
• Body mass index is less than (\<) 30.0 kilograms per meter square (kg/m\^2)
• A total body weight greater than (\>) 50 kg
• Stable CF disease
• CFTR gene mutations on both alleles that are not responsive to CFTR modulator therapy
• o Example mutations include but are not limited to, mutations that do not produce CFTR protein (i.e., Class I): nonsense mutations (e.g., G542X, W1282X) and canonical splice mutations (e.g., 621+1G-\>T)
• Forced expiratory volume in 1 second (FEV1) value for SAD: greater than or equal to (≥)40 percent (%), MAD: ≥ 40% to less than or equal to (≤) 90%
Locations
United States
Alabama
Child Health Research Unit
ACTIVE_NOT_RECRUITING
Birmingham
California
Memorial Health Services on behalf of Long Beach Memorial Medical Center d/b/a Miller Children's Hospital Long Beach
ACTIVE_NOT_RECRUITING
Long Beach
Stanford University - Palo Alto - Pulmonology
ACTIVE_NOT_RECRUITING
Palo Alto
Colorado
National Jewish Health
ACTIVE_NOT_RECRUITING
Denver
Florida
Shands Hospital - Pulmonology
ACTIVE_NOT_RECRUITING
Gainesville
Illinois
Northwestern Memorial Hospital - Feinberg Pavilion
ACTIVE_NOT_RECRUITING
Chicago
Kansas
Clinical & Translational Science Unit (CTSU) - Pulmonology
ACTIVE_NOT_RECRUITING
Kansas City
Massachusetts
Boston Children's Hospital
ACTIVE_NOT_RECRUITING
Boston
MGH - MGfC Pediatric Cystic Fibrosis Center
ACTIVE_NOT_RECRUITING
Boston
Maryland
PAREXEL International - Baltimore
COMPLETED
Baltimore
The Johns Hopkins University - Johns Hopkins Hospital - Pulmonology
ACTIVE_NOT_RECRUITING
Baltimore
Minnesota
University of Minnesota -Pulmonology
RECRUITING
Minneapolis
Missouri
St. Louis Children's Hospital - Pulmonology
COMPLETED
St Louis
New York
Medicine at East 85th Street
ACTIVE_NOT_RECRUITING
New York
Presbyterian Hospital - Pulmonology
ACTIVE_NOT_RECRUITING
New York
Ohio
UC Health Holmes Hospital
ACTIVE_NOT_RECRUITING
Cincinnati
UH Cleveland Medical Center - Pulmonology
ACTIVE_NOT_RECRUITING
Cleveland
Nationwide Children's Hospital
ACTIVE_NOT_RECRUITING
Columbus
Pennsylvania
Children's Hospital of Pittsburgh - Pulmonology
ACTIVE_NOT_RECRUITING
Pittsburgh
South Carolina
Medical University of South Carolina - Pulmonology
ACTIVE_NOT_RECRUITING
Charleston
Tennessee
Vanderbilt Interventional Pulmonology
ACTIVE_NOT_RECRUITING
Nashville
Texas
Texas Children's Hospital - Wallace Tower - Pulmonology
ACTIVE_NOT_RECRUITING
Houston
Utah
University of Utah Hospital - Pulmonology
ACTIVE_NOT_RECRUITING
Salt Lake City
Wisconsin
University of Wisconsin Hospital and Clinics
ACTIVE_NOT_RECRUITING
Madison
Other Locations
Australia
The Alfred Hospital - Pulmonology
ACTIVE_NOT_RECRUITING
Melbourne
Belgium
Universitair Ziekenhuis Gent
ACTIVE_NOT_RECRUITING
Ghent
Canada
University of Calgary Medical Clinic of the Foothills Medical Centre
ACTIVE_NOT_RECRUITING
Calgary
IUCPQ Pavillon Recherche U-1771
ACTIVE_NOT_RECRUITING
Québec
Germany
Ruhrlandklinik
ACTIVE_NOT_RECRUITING
Essen
Italy
IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico - Pulmonology
ACTIVE_NOT_RECRUITING
Genova
Centro Ricerche Cliniche di Verona S.r.l.
ACTIVE_NOT_RECRUITING
Verona
Netherlands
UMCU - Department of Pulmonology and Tuberculosis
WITHDRAWN
Utrecht
Spain
Hospital Universitari Vall d´Hebron Servicio de Broncoscopia
ACTIVE_NOT_RECRUITING
Barcelona
Hospital Virgen del Rocio - Pulmonology
ACTIVE_NOT_RECRUITING
Seville
Hospital Universitario y Politecnico La Fe - Pulmonology
ACTIVE_NOT_RECRUITING
Valencia
Sweden
Karolinska University Hospital - Pulmonology
ACTIVE_NOT_RECRUITING
Stockholm
United Kingdom
Papworth Hospital NHS Foundation Trust
COMPLETED
Cambridge
Queen Elizabeth University Hospital - Pulmonology
COMPLETED
Glasgow
Royal Brompton Hospital
ACTIVE_NOT_RECRUITING
London
Wythenshawe Hospital - OPD
ACTIVE_NOT_RECRUITING
Manchester
All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough
RECRUITING
Penarth
University Hospital Southampton NHS Fountion - Southampton General Hospital
ACTIVE_NOT_RECRUITING
Southampton
Contact Information
Primary
Medical Information
medicalinfo@vrtx.com
617-341-6777
Time Frame
Start Date: 2023-02-27
Estimated Completion Date: 2027-05-12
Participants
Target number of participants: 39
Treatments
Experimental: Single Ascending Dose (SAD)
Participants grouped into different cohorts will receive a single ascending dose of VX-522.
Experimental: Multiple Ascending Dose (MAD) Cohort 1: VX-522
Participants will receive multiple ascending doses of VX-522.
Experimental: MAD Cohort 1: VX-522+ IVA
Following run-in period with ivacaftor (IVA), participants will receive multiple ascending doses of VX-522 with IVA.
Experimental: MAD Cohort 2: VX-522+ IVA
Following run-in period with ivacaftor (IVA), participants will receive multiple ascending doses of VX-522 with IVA.
Related Therapeutic Areas
Sponsors
Leads: Vertex Pharmaceuticals Incorporated
Collaborators: Moderna, Inc