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Restore CFTR Function Enrolling
RESTORE-CF: Phase 1/2 study of MRT5005 drug in adults with cystic fibrosis (Parts A, B and B Expansion) (Translate Bio MRT5005-101)
This study is taking place at multiple care centers across the U.S. It will look at the safety and tolerability of different doses of the nebulized drug MRT5005 in adults with CF.
This study is placebo-controlled, meaning that some participants will receive MRT5005, and others will receive a placebo. Researchers will test the safety and tolerability of MRT5005 by tracking adverse events. They will also assess how much of the drug gets into the body.
Participants in this study will have up to 21 study visits depending on what part of the study they are enrolled in. All participants will be followed for 12 months after their last dose of study drug.
Email a research coordinator to express interest in participating in this study.
Eligibility
See other primary eligibility criteria for more information.
-
Age:
18 Years and Older -
Mutation(s):
See Other Primary Eligibility Criteria -
FEV1% Predicted:
50 to 90%
For a more comprehensive list of eligibility criteria and details on this study, visit ClinicalTrials.gov.
Other Primary Eligibility Criteria
Patients who have two Class I or II CFTR gene mutations are eligible to participate in the study. Unclassified mutations will be considered. Patients who are receiving lumacaftor/ivacaftor combination drug (ORKAMBI) or are receiving tezacaftor/ivacaftor and ivacaftor combination drug (SYMDEKO) are eligible for the study; however, patients must have been on stable treatment with this medication for at least 28 days prior to the screening visit, and should remain on it for the duration of the study. Patients who have a Class III, IV, or V CFTR gene mutation in at least 1 allele or who are receiving treatment with ivacaftor monotherapy (KALYDECO) are NOT eligible for this study. Patients on triple combination elexacaftor/tezacaftor/ivacaftor therapy (TRIKAFTA) are NOT eligible for this study; however, study participants may begin triple combination therapy 2 months following the last dose of MRT5005.
Study Design
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Study Type: ?more info
Interventional -
Randomized Study: ?more info
Yes -
Placebo Controlled: ?more info
Yes -
Length of Participation:
1 years -
Number of Study Visits:
21
Additional Information
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Phase: ?more info
Phase One -
Study Sponsor: ?more info
Translate Bio -
Study Drugs:
Study Sites
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Enrolling
Alabama
University of Alabama at Birmingham, Birmingham, AL 35294
Contact
Heather Hathorne
Phone: +1 (205) 638-9568
Email: hhathorne@peds.uab.edu
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Enrolling
Colorado
National Jewish Health, Denver, CO 80206
Contact
Alix Wilson
Phone: +1 (303) 270-2333
Email: wilsona@njhealth.org
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Enrolling
Florida
University of Florida, Gainesville, FL 32610
Contact
Noni Graham
Phone: +1 (352) 294-5195
Email: Noni.graham@medicine.ufl.edu
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Enrolling
Illinois
Northwestern University, Chicago, IL 60611
Contact
Rachel Nelson
Phone: +1 (312) 695-0415
Email: rachel.nelson@northwestern.edu
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Enrolling
Indiana
Indiana University Medical Center, Indianapolis, IN 46202
Contact
Lisa Bendy
Phone: +1 (317) 278-7152
Email: lbendy@iupui.edu
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Enrolling
Maine
Maine Medical Center, Portland, ME 04102
Contact
Harmony Renna
Phone: +1 (207) 662-5917
Email: rennah@mmc.org
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Enrolling
Maryland
Johns Hopkins University, Baltimore, MD 21205
Contact
Britany Zeglin
Phone: +1 (443) 287-8983
Email: bzeglin1@jhmi.edu
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Enrolling Soon
Michigan
University of Michigan, Michigan Medicine, Ann Arbor, MI 48109
Contact
Dawn Kruse
Phone: +1 (734) 615-3266
Email: dmkruse@med.umich.edu
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Enrolling
Ohio
University of Cincinnati Medical Center, Cincinnati, OH 45267
Contact
Nicole Hummel
Phone: +1 (513) 558-7036
Email: Nicole.Hummel@UCHealth.com
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Enrolling
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
Ohio
Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center, Cleveland, OH 44106
Contact
Kathleen Hilliard
Phone: +1 (216) 844-7489
Email: kathleen.hilliard@uhhospitals.org
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Enrolling
Oregon
Oregon Health Sciences University, Portland, OR 97239
Contact
Brendan Klein
Phone: +1 (503) 418-8108
Email: kleinb@ohsu.edu
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Enrolling
Pennsylvania
University of Pennsylvania, Philadelphia, PA 19104
Contact
Sara Deitrick
Phone: +1 (215) 615-0276
Email: Sara.deitrick@uphs.upenn.edu
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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
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Enrolling
Tennessee
University of Tennessee Medical Center, Knoxville, TN 37920
Contact
Phone: +1
Email:
-
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
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Enrolling
Virginia
Virginia Commonwealth University, Richmond, VA 23298
Contact
Ryan Hayden
Phone: +1 (804) 628-3921
Email: ryan.hayden@vcuhealth.org
Email a research coordinator to express interest in participating in this study.
Eligibility
See other primary eligibility criteria for more information.
-
Age:
18 Years and Older -
Mutation(s):
See Other Primary Eligibility Criteria -
FEV1% Predicted:
50 to 90%
For a more comprehensive list of eligibility criteria and details on this study, visit ClinicalTrials.gov.
Other Primary Eligibility Criteria
Patients who have two Class I or II CFTR gene mutations are eligible to participate in the study. Unclassified mutations will be considered. Patients who are receiving lumacaftor/ivacaftor combination drug (ORKAMBI) or are receiving tezacaftor/ivacaftor and ivacaftor combination drug (SYMDEKO) are eligible for the study; however, patients must have been on stable treatment with this medication for at least 28 days prior to the screening visit, and should remain on it for the duration of the study. Patients who have a Class III, IV, or V CFTR gene mutation in at least 1 allele or who are receiving treatment with ivacaftor monotherapy (KALYDECO) are NOT eligible for this study. Patients on triple combination elexacaftor/tezacaftor/ivacaftor therapy (TRIKAFTA) are NOT eligible for this study; however, study participants may begin triple combination therapy 2 months following the last dose of MRT5005.
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