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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) (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 and will evaluate how it affects the airway cells by measuring changes in CFTR activity after treatment.
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.
Study Design
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Study Type: ?more info
Interventional -
Randomized Study: ?more info
Yes -
Placebo Controlled: ?more info
Yes -
Length of Participation:
4 months -
Number of Study Visits:
18
Additional Information
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Phase: ?more info
Phase One -
Study Sponsor: ?more info
Translate Bio -
Study Drugs:
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.
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: hhathorne@peds.uab.edu
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Enrolling
Colorado
National Jewish Health, Denver, CO 80206
Contact
Connie St. Clair
Phone: +1 (303) 270-2517
Email: StClairC@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
Florida
Central Florida Pulmonary Group, Orlando, FL 32803
Contact
Kenneth Kesser
Phone: +1 (407) 841-1100
Email: bkesser@cfpulmonary.com
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Enrolling Soon
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 Soon
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
Montana
Billings Clinic, Billings, MT 59107
Contact
Jerimiah Lysinger
Phone: +1 (406) 238-5137
Email: JLysinger@billingsclinic.org
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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
Victoria Fleck
Phone: +1 (215) 662-3115
Email: Victoria.Fleck@uphs.upenn.edu
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Enrolling
Tennessee
University of Tennessee Medical Center, Knoxville, TN 37920
Contact
Phone: +1
Email:
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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
Clinical Studies 101
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Questions to Ask When Considering a Specific Trial
Clinical trials that test potential drugs and therapies in people with cystic fibrosis are a major part of CF research. They take place at Cystic Fibrosis Foundation-accredited care centers all over the United States and enroll people with CF of all ages.
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Patient Safety Is a Priority
Nothing is more important than patient safety in developing new treatments for cystic fibrosis. The U.S. government requires that all clinical trials are supervised for safety, and the Cystic Fibrosis Foundation adds additional measures to protect study volunteers.
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How Do Clinical Trials Work
Each clinical trial has a study sponsor and a protocol. Potential drugs must move through four well-defined phases of testing in patients.
