A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)
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
3 other identifiers
interventional
39
10 countries
43
Brief 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_1
Started Feb 2023
Longer than P75 for phase_1
43 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
December 19, 2022
CompletedFirst Posted
Study publicly available on registry
December 30, 2022
CompletedStudy Start
First participant enrolled
February 27, 2023
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 23, 2026
ExpectedStudy Completion
Last participant's last visit for all outcomes
May 12, 2027
March 25, 2026
September 1, 2025
3.8 years
December 19, 2022
March 24, 2026
Conditions
Outcome Measures
Primary Outcomes (1)
Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
From Day 1 Through Week 8 [SAD and MAD]
Secondary Outcomes (2)
MAD: Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
From Baseline at Day 29
MAD: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
From Day 1 Through Safety Follow-up Visit [up to Week 28]
Study Arms (4)
Single Ascending Dose (SAD)
EXPERIMENTALParticipants grouped into different cohorts will receive a single ascending dose of VX-522.
Multiple Ascending Dose (MAD) Cohort 1: VX-522
EXPERIMENTALParticipants will receive multiple ascending doses of VX-522.
MAD Cohort 1: VX-522+ IVA
EXPERIMENTALFollowing run-in period with ivacaftor (IVA), participants will receive multiple ascending doses of VX-522 with IVA.
MAD Cohort 2: VX-522+ IVA
EXPERIMENTALFollowing run-in period with ivacaftor (IVA), participants will receive multiple ascending doses of VX-522 with IVA.
Interventions
Oral inhalation using nebulizer.
Tablet for oral administration.
Eligibility Criteria
You may qualify if:
- 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: ≥ 50% to less than or equal to (≤) 90%
You may not qualify if:
- History of uncontrolled asthma within a year prior to screening
- 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)
- Arterial oxygen saturation on room air less than (\<) 94% at screening
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Moderna, Inccollaborator
- Vertex Pharmaceuticals Incorporatedlead
Study Sites (43)
University of Alabama at Birmingham - Child Health Research Unit
Birmingham, Alabama, 35233, United States
Memorial Health Services on behalf of Long Beach Memorial Medical Center d/b/a Miller Children's Hospital Long Beach
Long Beach, California, 90806, United States
Stanford University - Palo Alto - Pulmonology
Palo Alto, California, 94304, United States
National Jewish Health
Denver, Colorado, 80206, United States
Shands Hospital - Pulmonology
Gainesville, Florida, 32610, United States
Northwestern Memorial Hospital - Feinberg Pavilion
Chicago, Illinois, 60611, United States
Clinical & Translational Science Unit (CTSU) - Pulmonology
Kansas City, Kansas, 66160, United States
PAREXEL International - Baltimore
Baltimore, Maryland, 21225, United States
The Johns Hopkins University - Johns Hopkins Hospital - Pulmonology
Baltimore, Maryland, 21287, United States
MGH - MGfC Pediatric Cystic Fibrosis Center
Boston, Massachusetts, 02114, United States
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
University of Minnesota -Pulmonology
Minneapolis, Minnesota, 55455, United States
St. Louis Children's Hospital - Pulmonology
St Louis, Missouri, 63110, United States
Medicine at East 85th Street
New York, New York, 10028, United States
Presbyterian Hospital - Pulmonology
New York, New York, 10032, United States
UC Health Holmes Hospital
Cincinnati, Ohio, 45220, United States
UH Cleveland Medical Center - Pulmonology
Cleveland, Ohio, 44106, United States
Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
Children's Hospital of Pittsburgh - Pulmonology
Pittsburgh, Pennsylvania, 15224, United States
Medical University of South Carolina - Pulmonology
Charleston, South Carolina, 29425, United States
Vanderbilt Interventional Pulmonology
Nashville, Tennessee, 37232, United States
Texas Children's Hospital - Wallace Tower - Pulmonology
Houston, Texas, 77030, United States
University of Utah Hospital - Pulmonology
Salt Lake City, Utah, 84132, United States
University of Wisconsin Hospital and Clinics
Madison, Wisconsin, 53792, United States
The Alfred Hospital - Pulmonology
Melbourne, Australia
Universitair Ziekenhuis Gent
Ghent, Belgium
University of Calgary Medical Clinic of the Foothills Medical Centre
Calgary, Canada
IUCPQ Pavillon Recherche U-1771
Québec, Canada
Ruhrlandklinik
Essen, Germany
IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico - Pulmonology
Genova, Italy
IRCSS Ospedale Pediatrico Bambino Gesu - Pulmonology
Rome, Italy
Centro Ricerche Cliniche di Verona S.r.l.
Verona, Italy
UMCU - Department of Pulmonology and Tuberculosis
Utrecht, Netherlands
Hospital Universitari Vall d´Hebron Servicio de Broncoscopia
Barcelona, Spain
Hospital Virgen del Rocio - Pulmonology
Seville, Spain
Hospital Universitario y Politecnico La Fe - Pulmonology
Valencia, Spain
Karolinska University Hospital - Pulmonology
Stockholm, Sweden
Papworth Hospital NHS Foundation Trust
Cambridge, United Kingdom
Queen Elizabeth University Hospital - Pulmonology
Glasgow, United Kingdom
Royal Brompton Hospital
London, United Kingdom
Wythenshawe Hospital - OPD
Manchester, United Kingdom
All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough
Penarth, United Kingdom
University Hospital Southampton NHS Fountion - Southampton General Hospital
Southampton, United Kingdom
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SEQUENTIAL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
December 19, 2022
First Posted
December 30, 2022
Study Start
February 27, 2023
Primary Completion (Estimated)
December 23, 2026
Study Completion (Estimated)
May 12, 2027
Last Updated
March 25, 2026
Record last verified: 2025-09
Data Sharing
- IPD Sharing
- Will not share
Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/our-science/clinical-trials-data-sharing/