Clinical Trial Finder

Back to the Clinical Trial Finder

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.

 

Learn More

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

  • 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

  • Phase: ?more info

    Phase One

  • Study Sponsor: ?more info

    Translate Bio

  • Study Drugs:

    MRT5005

Study Sites

  • Enrolling

    Alabama

    University of Alabama at Birmingham, Birmingham, AL 35294

    Contact

    Heather Hathorne

    Phone: +1 (205) 638-9568

    Email:

  • Enrolling

    Colorado

    National Jewish Health, Denver, CO 80206

    Contact

    Alix Wilson

    Phone: +1 (303) 270-2333

    Email:

  • Enrolling

    Florida

    University of Florida, Gainesville, FL 32610

    Contact

    Noni Graham

    Phone: +1 (352) 294-5195

    Email:

  • Enrolling

    Illinois

    Northwestern University, Chicago, IL 60611

    Contact

    Rachel Nelson

    Phone: +1 (312) 695-0415

    Email:

  • Enrolling

    Indiana

    Indiana University Medical Center, Indianapolis, IN 46202

    Contact

    Lisa Bendy

    Phone: +1 (317) 278-7152

    Email:

  • Enrolling

    Maine

    Maine Medical Center, Portland, ME 04102

    Contact

    Harmony Renna

    Phone: +1 (207) 662-5917

    Email:

  • Enrolling

    Maryland

    Johns Hopkins University, Baltimore, MD 21205

    Contact

    Britany Zeglin

    Phone: +1 (443) 287-8983

    Email:

  • Enrolling Soon

    Michigan

    University of Michigan, Michigan Medicine, Ann Arbor, MI 48109

    Contact

    Dawn Kruse

    Phone: +1 (734) 615-3266

    Email:

  • Enrolling

    Ohio

    University of Cincinnati Medical Center, Cincinnati, OH 45267

    Contact

    Nicole Hummel

    Phone: +1 (513) 558-7036

    Email:

  • Enrolling

    Ohio

    Nationwide Children's Hospital, Columbus, OH 43205

    Contact

    Diana Gilmore

    Phone: +1 (614) 722-4752

    Email:

  • 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:

  • Enrolling

    Oregon

    Oregon Health Sciences University, Portland, OR 97239

    Contact

    Brendan Klein

    Phone: +1 (503) 418-8108

    Email:

  • Enrolling

    Pennsylvania

    University of Pennsylvania, Philadelphia, PA 19104

    Contact

    Sara Deitrick

    Phone: +1 (215) 615-0276

    Email:

  • Enrolling Soon

    Pennsylvania

    University of Pittsburgh Medical Center, Pittsburgh, PA 15224

    Contact

    Elizabeth Hartigan

    Phone: +1 (412) 692-7060

    Email:

  • 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:

  • Enrolling

    Virginia

    Virginia Commonwealth University, Richmond, VA 23298

    Contact

    Ryan Hayden

    Phone: +1 (804) 628-3921

    Email:

Learn More

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. 

Related Topics

  • 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.

  • 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.

  • Trikafta™

    The following is a collection of information and news about Trikafta™, the triple-combination modulator therapy that was approved by the U.S. Food and Drug Administration on Oct. 21, 2019.

Sign up for clinical trial alerts

Get email updates about clinical trials that matter to you.

Check the Drug Development Pipeline

We’re attacking CF from every angle. Learn about the status of CF drugs in development.

Learn More