Cystic FibrosisPhase 1/2NCT05668741
A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)
A Phase 1/2 study testing VX-522 mRNA therapy and IVA in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 26 sites in 7 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- VX-522 mRNA therapy and IVA
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 26 participants
- Where
- 26 sitesin 7 countries
- Study dates
- 27 Feb 2023to 9 Sep 2026
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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%
Who cannot join
- 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
- Other protocol defined Inclusion/Exclusion criteria may apply.
Trial locations and contacts
26 sites in 7 countries.
26 sites in 7 countries
United States14 sites
PAREXEL International - Baltimore
Baltimore, Maryland, 21225
The Johns Hopkins University - Johns Hopkins Hospital - Pulmonology
Baltimore, Maryland, 21287
University of Alabama at Birmingham - Child Health Research Unit
Birmingham, Alabama, 35233
MGH - MGfC Pediatric Cystic Fibrosis Center
Boston, Massachusetts, 02114
Boston Children's Hospital
Boston, Massachusetts, 02115
Medical University of South Carolina - Pulmonology
Charleston, South Carolina, 29425
UC Health Holmes Hospital
Cincinnati, Ohio, 45220
National Jewish Health
Denver, Colorado, 80206
Clinical & Translational Science Unit (CTSU) - Pulmonology
Kansas City, Kansas, 66160
Memorial Health Services on behalf of Long Beach Memorial Medical Center d/b/a Miller Children's Hospital Long Beach
Long Beach, California, 90806
University of Minnesota -Pulmonology
Minneapolis, Minnesota, 55455
Stanford University - Palo Alto - Pulmonology
Palo Alto, California, 94304
University of Utah Hospital - Pulmonology
Salt Lake City, Utah, 84132
St. Louis Children's Hospital - Pulmonology
St Louis, Missouri, 63110
United Kingdom6 sites
Papworth Hospital NHS Foundation Trust
Cambridge
Queen Elizabeth University Hospital - Pulmonology
Glasgow
Royal Brompton Hospital
London
Wythenshawe Hospital - OPD
Manchester
All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough
Penarth
University Hospital Southampton NHS Fountion - Southampton General Hospital
Southampton
Canada2 sites
University of Calgary Medical Clinic of the Foothills Medical Centre
Calgary
IUCPQ Pavillon Recherche U-1771
Québec
About this study
Official title: 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
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
- Cystic Fibrosis
- Being tested
- VX-522 mRNA therapy, IVA
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VX-522 mRNA therapyDrug
Oral inhalation using nebulizer.
IVADrug
Also called ivacaftor, VX-770
Tablet for oral administration.
| Study group | Role | What they receive |
|---|---|---|
| Single Ascending Dose (SAD) | Experimental | Participants grouped into different cohorts will receive a single ascending dose of VX-522. Receives: Drug: VX-522 mRNA therapy |
| Multiple Ascending Dose (MAD) Cohort 1: VX-522 | Experimental | Participants will receive multiple ascending doses of VX-522. Receives: Drug: VX-522 mRNA therapy |
| MAD Cohort 1: VX-522+ IVA | Experimental | Following run-in period with ivacaftor (IVA), participants will receive multiple ascending doses of VX-522 with IVA. Receives: Drug: VX-522 mRNA therapy; Drug: IVA |
| MAD Cohort 2: VX-522+ IVA | Experimental | Following run-in period with ivacaftor (IVA), participants will receive multiple ascending doses of VX-522 with IVA. Receives: Drug: VX-522 mRNA therapy; Drug: IVA |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Measured: 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)
Measured: From Baseline at Day 29
MAD: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Measured: From Day 1 Through Safety Follow-up Visit [up to Week 28]
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Dec 2022
- Study start27 Feb 2023
- Primary completion21 Apr 2026
- Study completion9 Sep 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by Vertex Pharmaceuticals IncorporatedThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 26 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Moderna, Inc
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05668741
- Sponsor's protocol ID
- VX21-522-001
- Other IDs
- 2022-000726-25, 2023-504786-23-00, 2023
- Status last verified
- Oct 2026
- First submitted
- 19 Dec 2022
- Last updated
- 9 Oct 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: 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/
Study at a glance
From the registry record, last updated by the sponsor on 9 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT05668741
- Acronym / sponsor ID
- VX21-522-001
- Conditions
- Cystic Fibrosis
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 26 participants (actual)
- Lead sponsor
- Vertex Pharmaceuticals IncorporatedIndustry (pharma, biotech, devices)
- Collaborators
- Moderna, Inc
- Study start
- 27 Feb 2023 (actual)
- Primary completion
- 21 Apr 2026 (actual)
- Study completion
- 9 Sep 2026 (actual)
- First posted
- 30 Dec 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Single Ascending Dose (SAD) | Experimental | Drug: VX-522 mRNA therapyParticipants grouped into different cohorts will receive a single ascending dose of VX-522. |
| Multiple Ascending Dose (MAD) Cohort 1: VX-522 | Experimental | Drug: VX-522 mRNA therapyParticipants will receive multiple ascending doses of VX-522. |
| MAD Cohort 1: VX-522+ IVA | Experimental | Drug: VX-522 mRNA therapy, Drug: IVAFollowing run-in period with ivacaftor (IVA), participants will receive multiple ascending doses of VX-522 with IVA. |
| MAD Cohort 2: VX-522+ IVA | Experimental | Drug: VX-522 mRNA therapy, Drug: IVAFollowing run-in period with ivacaftor (IVA), participants will receive multiple ascending doses of VX-522 with IVA. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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)
Key exclusion criteria (5 listed)
- 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
- Other protocol defined Inclusion/Exclusion criteria may apply.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VX-522 mRNA therapyOral inhalation using nebulizer. | Drug | MAD Cohort 1: VX-522+ IVA, MAD Cohort 2: VX-522+ IVA, Multiple Ascending Dose (MAD) Cohort 1: VX-522, Single Ascending Dose (SAD) |
| IVAAlso: ivacaftor, VX-770Tablet for oral administration. | Drug | MAD Cohort 1: VX-522+ IVA, MAD Cohort 2: VX-522+ IVA |
Primary outcome
Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: From Day 1 Through Week 8 [SAD and MAD]
2 secondary outcomes
MAD: Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
Time frame: From Baseline at Day 29
MAD: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: From Day 1 Through Safety Follow-up Visit [up to Week 28]
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Oct 2026.
- Individual participant dataSharing planned: No. 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/
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
26 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing VX-522 mRNA therapy and IVA in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 26 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 9 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 26 sites in United States, United Kingdom, Canada, Australia, Belgium and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) (time frame: From Day 1 Through Week 8 [SAD and MAD]). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Oct 2026.