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Genetic Therapy Closed to Enrollment

Study to evaluate VX-522 in adults 18 years and older with cystic fibrosis. (Vertex VX21-522-001 SAD)

This study will evaluate the safety and tolerability of VX-522, an investigational inhaled messenger RNA (mRNA), in adults with CF whose mutations are not responsive to CFTR modulator therapy. 

 This study is open label, meaning all participants will receive a dose of VX-522. Study participants will receive one dose of the study drug and then have follow-up study visits for 24 weeks. Different doses of VX-522 are being studied. The study drug will be administered through inhalation. In this study, researchers will assess for safety and tolerability by monitoring for any changes in a participant’s health.This study may require blood draws, lung function testing, electrocardiograms, and/or other measurements.For additional information about the study, visit the sponsor-developed website. This website is not managed or operated by the CF Foundation, and the Foundation is not responsible for the contents of the website.

Eligibility

See other primary eligibility criteria for more information.

  • Age:

    18 Years to 65 Years

  • Mutation(s):

    No Copies F508del

  • FEV1% Predicted:

    40% or greater

For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.

Other Primary Eligibility Criteria

Potential participants must have 2 CFTR mutations that are not eligible to receive CFTR modulators. Potential participants who have participated in other studies of nucleic acid therapies (e.g. RNA-base therapies) must not have used those therapies within 12 months and must have completed all required follow-up prior to participating in this study. 

Study Design

  • Study Type: ?more info

    Interventional

  • Randomized Study: ?more info

    No

  • Placebo Controlled: ?more info

    No

  • Length of Participation:

    6 months

  • Number of Study Visits:

    8

Additional Information

  • Phase: ?more info

    Phase One

  • Study Sponsor: ?more info

    Vertex

  • Study Drugs:

    VX-522 mRNA

Study Sites

  • Closed to Enrollment

    Alabama

    University of Alabama at Birmingham, Birmingham, AL 35233

  • Closed to Enrollment

    California

    Stanford University Medical Center, Palo Alto, CA 94304

  • Closed to Enrollment

    California

    Long Beach Memorial Medical Center, Long Beach, CA 90806

  • Closed to Enrollment

    Colorado

    National Jewish Health, Denver, CO 80206

  • Closed to Enrollment

    Florida

    University of Florida, Gainesville, FL 32610

  • Closed to Enrollment

    Illinois

    Northwestern University, Chicago, IL 60611

  • Closed to Enrollment

    Iowa

    University of Iowa, Iowa City, IA 52242

  • Closed to Enrollment

    Kansas

    University of Kansas Medical Center, Kansas City, KS 66160

  • Closed to Enrollment

    Maryland

    Johns Hopkins University, Baltimore, MD 21205

  • Closed to Enrollment

    Massachusetts

    Massachusetts General Hospital, Boston, MA 02114

  • Closed to Enrollment

    Massachusetts

    Boston Children's Hospital, Brigham & Women's Hospital, Boston, MA 02115

  • Closed to Enrollment

    Minnesota

    The Minnesota Cystic Fibrosis Center, Minneapolis, MN 55455

  • Closed to Enrollment

    Missouri

    Washington University School of Medicine, St. Louis, MO 63110

  • Closed to Enrollment

    New York

    Columbia University Cystic Fibrosis Program, New York, NY 10032

  • Closed to Enrollment

    Ohio

    University of Cincinnati Medical Center, Cincinnati, OH 45267

  • Closed to Enrollment

    Ohio

    Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center, Cleveland, OH 44106

  • Closed to Enrollment

    Ohio

    Nationwide Children's Hospital, Columbus, OH 43205

  • Closed to Enrollment

    Pennsylvania

    University of Pittsburgh Medical Center, Pittsburgh, PA 15224

  • Closed to Enrollment

    South Carolina

    Medical University of South Carolina, Charleston, SC 29425

  • Closed to Enrollment

    Tennessee

    Vanderbilt University Medical Center, Nashville, TN 37232

  • Closed to Enrollment

    Texas

    Baylor College of Medicine, Houston, TX 77030

  • Closed to Enrollment

    Wisconsin

    University of Wisconsin, Madison, WI 53792

Eligibility

See other primary eligibility criteria for more information.

  • Age:

    18 Years to 65 Years

  • Mutation(s):

    No Copies F508del

  • FEV1% Predicted:

    40% or greater

For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.

Other Primary Eligibility Criteria

Potential participants must have 2 CFTR mutations that are not eligible to receive CFTR modulators. Potential participants who have participated in other studies of nucleic acid therapies (e.g. RNA-base therapies) must not have used those therapies within 12 months and must have completed all required follow-up prior to participating in this study. 

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