Study Results

• What We Learned:

  This study enrolled three groups of participants, Group 1: healthy participants that do not have CF, Group 2: individuals with CF who have a gene mutation that results in partial CFTR function, and Group 3: individuals with CF who have mutations that result in no CFTR function. It found that Group 2 and 3 had similar lung function (FEV1) and that there was a significant difference in sweat chloride concentrations between each group.  Additional analyses of the biospecimens collected in the study to identify potential markers of disease severity are ongoing.

• Primary Findings:

  Effectiveness:

  This study was conducted between March 2015 and April 2018. The study enrolled 287 participants: 55 healthy participants, 40 participants with CF with partial CFTR function, and 164 participants with CF who have no CFTR function.

   

  Evaluation of lung function found that FEV1 in Group 2 was 86.1% and in Group 3 was 81.6%.  Sweat chloride concentrations were significantly different across the three groups; Group 1: – 22.0 mmol/L, Group 2: – 81.0 mmol/L, and Group 3: – 101.4 mmol/L.

   

  Additional analyses are ongoing. Inflammatory biomarker results will be added once available.

   

  These results have been provided from a NACFC abstract and have not been peer reviewed.

  Safety:

• Citation:

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