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Restore CFTR Protein Closed to Enrollment

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. 

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 more information about the results of this study and where it was conducted, 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

  • Closed to Enrollment

    Alabama

    University of Alabama at Birmingham, Birmingham, AL 35233

  • Closed to Enrollment

    Colorado

    National Jewish Health, Denver, CO 80206

  • Closed to Enrollment

    Florida

    Central Florida Pulmonary Group, Orlando, FL 32803

  • Closed to Enrollment

    Florida

    University of Florida, Gainesville, FL 32610

  • Closed to Enrollment

    Illinois

    Northwestern University, Chicago, IL 60611

  • Closed to Enrollment

    Indiana

    Indiana University Medical Center, Indianapolis, IN 46202

  • Closed to Enrollment

    Maine

    Maine Medical Center, Portland, ME 04102

  • Closed to Enrollment

    Maryland

    Johns Hopkins University, Baltimore, MD 21205

  • Closed to Enrollment

    Michigan

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

  • Closed to Enrollment

    Montana

    Billings Clinic, Billings, MT 59107

  • Closed to Enrollment

    Ohio

    University of Cincinnati Medical Center, Cincinnati, OH 45267

  • Closed to Enrollment

    Ohio

    Nationwide Children's Hospital, Columbus, OH 43205

  • Closed to Enrollment

    Ohio

    Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center, Cleveland, OH 44106

  • Closed to Enrollment

    Oregon

    Oregon Health & Science University, Portland, OR 97239

  • Closed to Enrollment

    Pennsylvania

    University of Pennsylvania, Philadelphia, PA 19104

  • Closed to Enrollment

    Pennsylvania

    University of Pittsburgh Medical Center, Pittsburgh, PA 15224

  • Closed to Enrollment

    Tennessee

    University of Tennessee Medical Center, Knoxville, TN 37920

  • Closed to Enrollment

    Utah

    Adult Cystic Fibrosis Center at the University of Utah, Salt Lake City, UT 84112

  • Closed to Enrollment

    Virginia

    Virginia Commonwealth University, Richmond, VA 23298

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 more information about the results of this study and where it was conducted, 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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