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Restore CFTR Protein Completed with Results

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 evaluated the safety, tolerability, and biological activity of MRT5005 in adults with Cystic Fibrosis who have either two Class I or two Class II CFTR gene mutations.

 This was a placebo-controlled study designed to look at the safety, tolerability, and biological activity of MRT5005 in adults. In Part A, participants received a single dose of either 8mg, 16mg, 20mg, or 24mg of the study drug. In Part B, participants received five weekly doses of either 8mg, 12mg, 16mg, or 20mg of the study drug. Part C was not executed. In Part D, participants received 5 daily 4mg doses of the study drug. Safety and tolerability were assessed by tracking adverse events.  

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 Results

  • What We Learned:

    While MRT5005 was mostly well tolerated, several participants developed fevers after dosing. Additionally, no improvement in lung function was observed. 

  • Primary Findings:

    Effectiveness:

    This small study of multiple different doses of MRT5005 did not show any improvement in lung function.

     

    Safety:

    This study was conducted between May 2018 and December 2021. 

    In the single dose part of the study, one participant receiving a 20mg dose experienced a severe pulmonary exacerbation 23 days after dosing and possibly related to treatment. Two participants experienced hypersensitivity reactions: one had received a single 20mg dose and the other participant had received three daily 4mg doses). 
    Fevers and increased body temperature were the most common study drug related reaction and were treated with standard medications like acetaminophen or ibuprofen. Two participants discontinued the study due to fever or hypersensitivity. One after receiving the first 16mg dose in Part B and one after receiving the third of five planned doses in Part D.

  • Citation:

    J Cyst Fibros 2023;22(4):656-664

For current information about the overall development status of this drug, please check the Drug Development Pipeline.

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

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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