Actively Recruiting
A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)
Led by Vertex Pharmaceuticals Incorporated · Updated on 2026-03-25
39
Participants Needed
43
Research Sites
219 weeks
Total Duration
On this page
Sponsors
V
Vertex Pharmaceuticals Incorporated
Lead Sponsor
M
Moderna, Inc
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Body mass index is less than 30.0 kilograms per meter squared (kg/m²)
- Weighs more than 50 kilograms
- Has stable cystic fibrosis disease
- Has CFTR gene mutations on both alleles not responsive to CFTR modulator therapy, such as nonsense mutations (e.g., G542X, W1282X) or canonical splice mutations (e.g., 621+1G->T)
- Forced expiratory volume in 1 second (FEV1) value of at least 40% for single ascending dose (SAD) and between 50% and 90% for multiple ascending dose (MAD)
You will not qualify if you...
- History of uncontrolled asthma within one year before screening
- History of solid organ or blood 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)
- Arterial oxygen saturation on room air less than 94% at screening
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 43 locations
1
University of Alabama at Birmingham - Child Health Research Unit
Birmingham, Alabama, United States, 35233
Actively Recruiting
2
Memorial Health Services on behalf of Long Beach Memorial Medical Center d/b/a Miller Children's Hospital Long Beach
Long Beach, California, United States, 90806
Actively Recruiting
3
Stanford University - Palo Alto - Pulmonology
Palo Alto, California, United States, 94304
Actively Recruiting
4
National Jewish Health
Denver, Colorado, United States, 80206
Actively Recruiting
5
Shands Hospital - Pulmonology
Gainesville, Florida, United States, 32610
Actively Recruiting
6
Northwestern Memorial Hospital - Feinberg Pavilion
Chicago, Illinois, United States, 60611
Actively Recruiting
7
Clinical & Translational Science Unit (CTSU) - Pulmonology
Kansas City, Kansas, United States, 66160
Actively Recruiting
8
PAREXEL International - Baltimore
Baltimore, Maryland, United States, 21225
Completed
9
The Johns Hopkins University - Johns Hopkins Hospital - Pulmonology
Baltimore, Maryland, United States, 21287
Active, Not Recruiting
10
MGH - MGfC Pediatric Cystic Fibrosis Center
Boston, Massachusetts, United States, 02114
Actively Recruiting
11
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
12
University of Minnesota -Pulmonology
Minneapolis, Minnesota, United States, 55455
Actively Recruiting
13
St. Louis Children's Hospital - Pulmonology
St Louis, Missouri, United States, 63110
Completed
14
Medicine at East 85th Street
New York, New York, United States, 10028
Actively Recruiting
15
Presbyterian Hospital - Pulmonology
New York, New York, United States, 10032
Actively Recruiting
16
UC Health Holmes Hospital
Cincinnati, Ohio, United States, 45220
Actively Recruiting
17
UH Cleveland Medical Center - Pulmonology
Cleveland, Ohio, United States, 44106
Active, Not Recruiting
18
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Actively Recruiting
19
Children's Hospital of Pittsburgh - Pulmonology
Pittsburgh, Pennsylvania, United States, 15224
Actively Recruiting
20
Medical University of South Carolina - Pulmonology
Charleston, South Carolina, United States, 29425
Actively Recruiting
21
Vanderbilt Interventional Pulmonology
Nashville, Tennessee, United States, 37232
Actively Recruiting
22
Texas Children's Hospital - Wallace Tower - Pulmonology
Houston, Texas, United States, 77030
Active, Not Recruiting
23
University of Utah Hospital - Pulmonology
Salt Lake City, Utah, United States, 84132
Active, Not Recruiting
24
University of Wisconsin Hospital and Clinics
Madison, Wisconsin, United States, 53792
Actively Recruiting
25
The Alfred Hospital - Pulmonology
Melbourne, Australia
Actively Recruiting
26
Universitair Ziekenhuis Gent
Ghent, Belgium
Actively Recruiting
27
University of Calgary Medical Clinic of the Foothills Medical Centre
Calgary, Canada
Actively Recruiting
28
IUCPQ Pavillon Recherche U-1771
Québec, Canada
Actively Recruiting
29
Ruhrlandklinik
Essen, Germany
Actively Recruiting
30
IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico - Pulmonology
Genova, Italy
Actively Recruiting
31
IRCSS Ospedale Pediatrico Bambino Gesu - Pulmonology
Rome, Italy
Actively Recruiting
32
Centro Ricerche Cliniche di Verona S.r.l.
Verona, Italy
Actively Recruiting
33
UMCU - Department of Pulmonology and Tuberculosis
Utrecht, Netherlands
Withdrawn
34
Hospital Universitari Vall d´Hebron Servicio de Broncoscopia
Barcelona, Spain
Actively Recruiting
35
Hospital Virgen del Rocio - Pulmonology
Seville, Spain
Actively Recruiting
36
Hospital Universitario y Politecnico La Fe - Pulmonology
Valencia, Spain
Actively Recruiting
37
Karolinska University Hospital - Pulmonology
Stockholm, Sweden
Actively Recruiting
38
Papworth Hospital NHS Foundation Trust
Cambridge, United Kingdom
Completed
39
Queen Elizabeth University Hospital - Pulmonology
Glasgow, United Kingdom
Completed
40
Royal Brompton Hospital
London, United Kingdom
Actively Recruiting
41
Wythenshawe Hospital - OPD
Manchester, United Kingdom
Actively Recruiting
42
All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough
Penarth, United Kingdom
Actively Recruiting
43
University Hospital Southampton NHS Fountion - Southampton General Hospital
Southampton, United Kingdom
Actively Recruiting
Research Team
M
Medical Information
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
4
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