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Clinical Trial Finder
Genetic Therapy Enrolling
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.
Email a research coordinator to express interest in participating in this study.
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 a more comprehensive list of eligibility criteria and details on this study, visit ClinicalTrials.gov.
Other Primary Eligibility Criteria
Potential participants must have 2 CFTR mutations that are not responsive to CFTR modulators. Additionally they must not have taken CFTR modulators within 28 days before the study. Potential participants who have participated in other studies of nucleic acid therapies (e.g. gen therapies, RNA-base therapies) must have not used those therapies within 12 months 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:
Study Sites
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Enrolling Soon
Alabama
University of Alabama at Birmingham, Birmingham, AL 35294
Contact
Heather Hathorne
Phone: +1 (205) 638-9568
Email: hyhathorne@uabmc.edu
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Enrolling Soon
California
Stanford University Medical Center, Palo Alto, CA 94304
Contact
Jacquelyn Spano
Phone: +1 (650) 721-1132
Email: jmzirbes@stanford.edu
-
Enrolling Soon
Colorado
National Jewish Health, Denver, CO 80206
Contact
Alix Wilson
Phone: +1 (303) 270-2517
Email: wilsona@njhealth.org
-
Enrolling Soon
Florida
University of Florida, Gainesville, FL 32610
Contact
Alexa Smith
Phone: +1
Email: alexa.howell@ufl.edu
-
Enrolling Soon
Illinois
Northwestern University, Chicago, IL 60611
Contact
Rachel Nelson
Phone: +1 (312) 695-0415
Email: rachel.nelson@northwestern.edu
-
Enrolling Soon
Iowa
University of Iowa, Iowa City, IA 52242
Contact
Mary Teresi
Phone: +1 (319) 384-7546
Email: mary-teresi@uiowa.edu
-
Enrolling Soon
Kansas
University of Kansas Medical Center, Kansas City, KS 66160
Contact
Lawrence Scott
Phone: +1 (913) 588-4020
Email: lscott2@kumc.edu
-
Enrolling
Massachusetts
Boston Children's Hospital, Brigham & Women's Hospital, Boston, MA 02115
Contact
Robert Fowler
Phone: +1 (617) 355-1834
Email: Robert.fowler@childrens.harvard.edu
-
Enrolling
Minnesota
The Minnesota Cystic Fibrosis Center, Minneapolis, MN 55455
Contact
Participant Contact University of Minnesota
Phone: +1
Email: cftrials@umn.edu
-
Enrolling Soon
Missouri
Washington University School of Medicine, St. Louis, MO 63110
Contact
Irma Bauer
Phone: +1 (314) 747-2940
Email: irmabauer@wustl.edu
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Enrolling Soon
New York
Columbia University Cystic Fibrosis Program, New York, NY 10032
Contact
Emily DiMango
Phone: +1 (212) 305-0290
Email: ead3@cumc.columbia.edu
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Enrolling Soon
Ohio
University of Cincinnati Medical Center, Cincinnati, OH 45267
Contact
Nicole Hummel
Phone: +1 (513) 558-7036
Email: hummelne@ucmail.uc.edu
-
Enrolling Soon
Ohio
Nationwide Children's Hospital, Columbus, OH 43205
Contact
Diana Gilmore
Phone: +1 (614) 722-4752
Email: diana.gilmore@nationwidechildrens.org
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Enrolling Soon
Pennsylvania
University of Pittsburgh Medical Center, Pittsburgh, PA 15224
Contact
Elizabeth Hartigan
Phone: +1 (412) 692-7060
Email: elizabeth.hartigan@chp.edu
-
Enrolling Soon
South Carolina
Medical University of South Carolina, Charleston, SC 29425
Contact
Ashley Warden
Phone: +1 (843) 792-4349
Email: jonesash@musc.edu
-
Enrolling Soon
Tennessee
Vanderbilt University Medical Center, Nashville, TN 37232
Contact
Brijesh Patel
Phone: +1 (615) 343-4865
Email: brijesh.patel@vumc.org
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Enrolling Soon
Texas
Baylor College of Medicine, Houston, TX 77030
Contact
Tracy Mosely
Phone: +1 (832) 822-3309
Email: tlmosely@texaschildrens.org
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Enrolling Soon
Utah
Intermountain Cystic Fibrosis Center, University of Utah, Salt Lake City, UT 84132
Contact
Kristyn Packer
Phone: +1 (801) 585-0401
Email: kristyn.packer@hsc.utah.edu
-
Enrolling Soon
Wisconsin
University of Wisconsin, Madison, WI 53792
Contact
Melanie Nelson
Phone: +1 (608) 265-4617
Email: mnelson@pediatrics.wisc.edu
Email a research coordinator to express interest in participating in this study.
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 a more comprehensive list of eligibility criteria and details on this study, visit ClinicalTrials.gov.
Other Primary Eligibility Criteria
Potential participants must have 2 CFTR mutations that are not responsive to CFTR modulators. Additionally they must not have taken CFTR modulators within 28 days before the study. Potential participants who have participated in other studies of nucleic acid therapies (e.g. gen therapies, RNA-base therapies) must have not used those therapies within 12 months prior to participating in this study.
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