Part A of the PROSPECT study identified biomarkers of CFTR function that could be used to monitor disease progression.
Three groups of participants were enrolled in the study: Group 1: healthy participants that do not have CF, Group 2: individuals with CF who have at least one gene mutation that results in partial CFTR function, and Group 3: individuals with CF who have mutations that result in no CFTR function. Researchers measured lung function and sweat chloride and collected sputum, blood, urine, and stool samples for biomarker measurements. Group 1 participated for two weeks and had two visits. Groups 2 and 3 participated for three months and had three visits.
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.
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.
Part A of the PROSPECT study enrolled two groups of people with CF: One group included people with CF that have CF mutations that result in parital CFTR function; the second group included people with CF that have mutations with no CFTR function (specifically those with two copies of F508del).