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Restore CFTR Function Completed with Results

Phase 1 exploratory study on the effect of the drug QR-010 on nasal cells (ProQR PQ-010-002)

This study evaluated the effectiveness of the drug eluforsen (when sprayed in the nose) to improve nasal cell chloride transport. This study was for people with CF who have at least one copy of the F508del CFTR mutation.

 Researchers evaluated the effectiveness of eluforsen on nasal cells by measuring nasal potential difference (NPD). Nasal potential difference is a measure of how well sodium and chloride move in and out of the nasal cells. This study was open-label, meaning that all study participants received eluforsen. Participants received 10mg of eluforsen (5mg/250 µL saline per nostril) three times a week for four weeks, for a total of 12 doses. NPD assessments involved recording the voltage from the left and right nostrils while the eluforsen solution was administered to the nose.

Eligibility

See other primary eligibility criteria for more information.

  • Age:

    18 Years and Older

  • Mutation(s):

    Two Copies F508del or One Copy F508del

  • FEV1% Predicted:

    40% or greater

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

Other Primary Eligibility Criteria

Must have CFTR gene mutations homozygous or compound heterozygous for the F508del mutation

Study Results

  • What We Learned:

    This study found that eluforsen increased total chloride transport in nasal cells of participants with two copies of F508del, but not in participants with only one copy of F508del. Eluforsen was well-tolerated in the study.

  • Primary Findings:

    Effectiveness:

    This study was conducted between October 2015 and July 2016. In this study, 14 participants completed the trial.

     

    The primary endpoint of the study was change in total chloride transport in nasal cells as measured by nasal potential difference (NPD).  In participants with two copies of F508del, treatment with eluforsen resulted in an increase in total chloride transport from the beginning to the end of the study (mean change -4.1 mV [90% CI -7.9; -0.4], p=0.04). In participants with only one copy of F508del, treatment with eluforsen did not result in an increase in total chloride transport.

     

    In addition to its effect on chloride transport, eluforsen also had a positive effect on sodium transport in participants with two copies of F508del (mean change +9.4 mV [90% Ci 1.1; 17.7], p=0.04).

    Safety:

    Eluforsen, when topically administered to the nose, was well-tolerated in the study.

  • Citation:

    J Cyst Fibros ;DOI 10.1016/j.jcf.2018.10.015 [Epub ahead of print];18(4):536-542

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

    No

  • Placebo Controlled: ?more info

    Yes

  • Length of Participation:

    7 weeks

  • Number of Study Visits:

    8

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 and Older

  • Mutation(s):

    Two Copies F508del or One Copy F508del

  • FEV1% Predicted:

    40% or greater

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

Other Primary Eligibility Criteria

Must have CFTR gene mutations homozygous or compound heterozygous for the F508del mutation

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