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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 VX-522-001 MAD (VX21-522-001))
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 VX-522. Study participants will receive multiple doses of the study drug over 4 weeks and will have follow up study visits for 28 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.
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 to 90%
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 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:
8 months -
Number of Study Visits:
12
Additional Information
-
Phase: ?more info
Phase Two -
Study Sponsor: ?more info
Vertex -
Study Drugs:
Study Sites
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Enrolling
Alabama
University of Alabama at Birmingham, Birmingham, AL 35233
Contact
Heather Hathorne
Phone: +1 (205) 638-9568
Email: hyhathorne@uabmc.edu
-
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
California
Long Beach Memorial Medical Center, Long Beach, CA 90806
Contact
Angelica Rodriguez
Phone: +1
Email: ARodriguez9@memorialcare.org
-
Enrolling
Colorado
National Jewish Health, Denver, CO 80206
Contact
Alix Wilson
Phone: +1 (303) 270-2517
Email: wilsona@njhealth.org
-
Enrolling
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
Kansas
University of Kansas Medical Center, Kansas City, KS 66160
Contact
Lawrence Scott
Phone: +1 (913) 588-4020
Email: lscott2@kumc.edu
-
Enrolling
Maryland
Johns Hopkins University, Baltimore, MD 21205
Contact
Jeanne Pinto
Phone: +1 (410) 955-9782
Email: jpinto4@jh.edu
-
Enrolling
Massachusetts
Massachusetts General Hospital, Boston, MA 02114
Contact
Margot Hardcastle
Phone: +1 (617) 726-3719
Email: mhardcastle@mgh.harvard.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
-
Enrolling
New York
Columbia University Cystic Fibrosis Program, New York, NY 10032
Contact
Emily DiMango
Phone: +1 (212) 305-0290
Email: ead3@cumc.columbia.edu
-
Enrolling Soon
New York
Lenox Hill Cystic Fibrosis Center, New York, NY 10028
Contact
Teresa Demarco
Phone: +1 (212) 434-4790
Email: Tdemarco3@northwell.edu
-
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
Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center, Cleveland, OH 44106
Contact
Primary RC & Participant Contact General Contact
Phone: +1
Email: RainbowCFResearch@UHhospitals.org
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Enrolling Soon
Ohio
Nationwide Children's Hospital, Columbus, OH 43205
Contact
Diana Gilmore
Phone: +1 (614) 722-4752
Email: diana.gilmore@nationwidechildrens.org
-
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
South Carolina
Medical University of South Carolina, Charleston, SC 29425
Contact
Ashley Warden
Phone: +1 (843) 792-4349
Email: jonesash@musc.edu
-
Enrolling
Tennessee
Vanderbilt University Medical Center, Nashville, TN 37232
Contact
Brijesh Patel
Phone: +1 (615) 343-4865
Email: brijesh.patel@vumc.org
-
Enrolling Soon
Texas
Baylor College of Medicine, Houston, TX 77030
Contact
Tracy Mosely
Phone: +1 (832) 822-3309
Email: tlmosely@texaschildrens.org
-
Enrolling
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 to 90%
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 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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