A Phase 1 Study of VX-522 in Participants With Cystic Fibrosis (CF)

Study Overview
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Brief Summary
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The purpose of this study is to evaluate the safety and tolerability 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.

Detailed Description
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This clinical trial information was submitted voluntarily under the applicable law and, therefore, certain submission deadlines may not apply. (That is, clinical trial information for this applicable clinical trial was submitted under section 402(j)(4) (A) of the Public Health Service Act and 42 CFR 11.60 and is not subject to the deadlines established by sections 402(j)(2) and (3) of the Public Health Service Act or 42 CFR 11.24 and 11.44.).

Official Title
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A Phase 1 Single Dose Escalation Study Evaluating the Safety and Tolerability of VX-522 in Subjects 18 Years of Age and Older With Cystic Fibrosis and a CFTR Genotype Not Responsive to CFTR Modulator Therapy

Conditions
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Cystic Fibrosis

Intervention / Treatment
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* Drug: VX-522 mRNA therapy


Participation Criteria
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Eligibility Criteria
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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, percent of predicted mean for age, sex, and height (equations of the Global Lung Function Initiative [GLI])18 ≥40% 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/Exclusion criteria may apply.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 65 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Sequential Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: VX-522<br>Participants will receive a single dose of different dose levels of VX-522. | Drug: VX-522 mRNA therapy<br>* Oral inhalation using nebulizer.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 (Week 24) |