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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT05668741
Recruitment Status : Recruiting
First Posted : December 30, 2022
Last Update Posted : November 1, 2023
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: VX-522 mRNA therapy Phase 1

Detailed Description:
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.).

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 9 participants
Allocation: N/A
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : February 27, 2023
Estimated Primary Completion Date : January 2024
Estimated Study Completion Date : January 2024

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Arm Intervention/treatment
Experimental: VX-522
Participants will receive a single dose of different dose levels of VX-522.
Drug: VX-522 mRNA therapy
Oral inhalation using nebulizer.

Primary Outcome Measures :
  1. 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 (Week 24) ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT05668741

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Contact: Medical Information 617-341-6777

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Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
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Responsible Party: Vertex Pharmaceuticals Incorporated Identifier: NCT05668741    
Other Study ID Numbers: VX21-522-001
2022-000726-25 ( EudraCT Number )
First Posted: December 30, 2022    Key Record Dates
Last Update Posted: November 1, 2023
Last Verified: June 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: Details on Vertex data sharing criteria and process for requesting access can be found at:

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
Additional relevant MeSH terms:
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Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases