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

Status

Active, not recruiting

Phases

Phase 1Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT05668741

Enrollment

Registered

2022-12-30

Start date

2023-02-27

Completion date

2026-09-09

Last updated

2026-09-08

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Cystic Fibrosis

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.

Interventions

DRUGVX-522 mRNA therapy

Oral inhalation using nebulizer.

DRUGIVA

Tablet for oral administration.

Study design

Allocation

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to 65 Years

Healthy volunteers

Inclusion criteria

Key Inclusion Criteria: * 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% Key

Exclusion criteria

* 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/

Design outcomes

Primary

Measure	Time frame
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

Measure	Time frame
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]

Countries

Australia, Belgium, Canada, Germany, Sweden, United Kingdom, United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Sep 9, 2026