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Mucociliary Clearance Completed with Results

BALANCE-CF 1: Study of BI 1265162, a mucus clearance drug, in teens and adults with CF (BI-1399-0003)

This study evaluated the safety and effectiveness of BI 1265162, a drug intended to improve the clearance of mucus from the lungs.

 Participants in the study were randomly assigned to receive one of four doses (20, 50, 100, and 200 µg) of BI 1265162 or placebo for 4 weeks.

Eligibility

  • Age:

    12 Years and Older

  • Mutation(s):

    No Mutation Requirement

  • FEV1% Predicted:

    40 to 90%

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

Study Results

  • What We Learned:

    This study was terminated early after a planned interim efficacy (futility) review of the data. The protocol specified that if the increase in ppFEV1 was <1.5% and the decrease (improvement) in LCI was <0.3 units the study would be terminated. At this interim data review, the highest dose of BI (200 µg) resulted in a 0.1% decrease of ppFEV1change an increase in LCI of 0.8 LCI units, neither of which met the pre-defined threshold. Overall, BI 1265162 was well-tolerated. One participant in the BI 200 µg group discontinued due to chest discomfort of mild intensity.

  • Primary Findings:

    Effectiveness:

    This study was conducted between October 2019 and April 2020. The study enrolled 52 adult participants (20 µg N=6, 50 µg N=5, 100 µg N=5, 200 µg N=18, placebo N=18). 

     

    The primary efficacy endpoint was the change in lung function (ppFEV1) from baseline after 4 weeks of treatment. This study was terminated early after a planned interim efficacy (futility) review of the data demonstrated that the change in lung function between BI 1265162 and placebo did not meet a minimum threshold pre-defined before the start of the study.

     

    The protocol specified that if the increase in ppFEV1 was <1.5% and the decrease (improvement) in LCI was <0.3 units the study would be terminated. At this interim data review, the highest dose of BI (200 µg) resulted in a 0.1% decrease of ppFEV1change an increase in LCI of 0.8 LCI units, neither of which met the pre-defined threshold.  Additionally, the data showed that the change in lung function between the 200 µg BI group and placebo was -0.8% ppFEV1 (95% CI -6.6 to 4.9) and 2.1 LCI units (95% CI -2.4 to 6.5).

     

    A 9.4-point improvement in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain was observed in the 200μg group versus placebo.

    Safety:

    Overall, BI 1265162 was well-tolerated up to doses of 200 µg. One participant in the BI 200 µg group discontinued due to chest discomfort of mild intensity.

  • Citation:

    Eur Respir J ;DOI 10.1183/13993003.00746-2021 [Epub ahead of print]

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:

    7 weeks

  • Number of Study Visits:

    5

Additional Information

  • Phase: ?more info

    Phase Two

  • Study Sponsor: ?more info

    Boehringer-Ingelheim

  • Study Drugs:

    BI 1265162

Eligibility

  • Age:

    12 Years and Older

  • Mutation(s):

    No Mutation Requirement

  • FEV1% Predicted:

    40 to 90%

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

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