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Restore CFTR Function Completed with Results

Phase 1b safety study of QR-010 in adults with CF (ProQR PQ-010-001)

This study evaluated the safety and tolerability of the inhaled drug eluforsen in people with CF who have two copies of the F508del CFTR mutation.

 This study consisted of two groups: a single ascending dose (SAD) group and a multiple ascending dose (MAD) group. In the SAD group, participants were randomly assigned to receive a one-time dose of inhaled eluforsen at one of four doses (6.25, 12.5, 25, or 50mg) or placebo. Participants in the MAD group were randomly assigned to receive one of four doses (6.25, 12.5, 25, or 50mg) of inhaled eluforsen or placebo three times a week for 4 weeks. 

Eligibility

See other primary eligibility criteria for more information.

  • Age:

    18 Years to 60 Years

  • Mutation(s):

    Two Copies F508del

  • FEV1% Predicted:

    70% or greater

For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.

Other Primary Eligibility Criteria

Must be homozygous for the F508del mutation

Study Results

  • What We Learned:

    This study showed that eluforsen was well-tolerated when tested at multiple dose levels. However, drug levels of eluforsen were very low even at the highest doses tested, and eluforsen did not have a significant impact on lung function or sweat chloride. Participants in the MAD group reported an improvement in respiratory symptoms in the CFQ-R RSS, a respiratory symptom questionnaire.

  • Primary Findings:

    Effectiveness:

    This study was conducted between August 2015 and July 2017. In the SAD group, 36 participants completed the study. In the MAD group, 31 of 34 participants completed the study. The three participants who did not complete the study stopped participating due to a drop in lung function of >15%. This was defined in the study protocol as a criterion for stopping participation.

     

    Overall, eluforsen was well-tolerated in both the SAD and MAD groups. No toxicities related to the dose level were seen in either group.

     

    Eluforsen drug levels in the body were found to be low even at the highest dose of 50mg in both the SAD and MAD groups. Additionally, eluforsen did not result in a significant impact on lung function or sweat chloride compared with placebo. Participants in the MAD group reported an improvement in respiratory symptoms in the CFQ-R RSS, a respiratory symptom questionnaire.

    Safety:

  • Citation:

    J Cyst Fibros ;DOI 10.1016/j.jcf.2019.05.014 [Epub ahead of print];S1569-1993(19):30766-30770

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:

    8 weeks

  • Number of Study Visits:

    10

Additional Information

  • Phase: ?more info

    Phase One

  • Study Sponsor: ?more info

    ProQr

  • Study Drugs:

    Eluforsen (QR-010)

Eligibility

See other primary eligibility criteria for more information.

  • Age:

    18 Years to 60 Years

  • Mutation(s):

    Two Copies F508del

  • FEV1% Predicted:

    70% or greater

For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.

Other Primary Eligibility Criteria

Must be homozygous for the F508del mutation

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