. 2023 Nov 20;2023(11):CD010966. doi: 10.1002/14651858.CD010966.pub4

Horsley 2017.

*Study characteristics*
Methods	Phase 1b RCT Parallel design Multicentre: 14 centres in Australia, Czech Republic, Germany and the UK Duration: 28 days
Participants	27 participants aged 18 or over, homozygous for F508del and with FEV₁ % predicted at least 40% at baseline FDL169 400 mg (n = 6) Age, mean (range): 31.5 (18 to 56) years Sex: 3 males, 3 females FEV₁ % predicted, mean (SD): 82.2 (22.5)% Sweat chloride, mean (SD): 96.9 (9.5) mmol/L CFQ‐R score (respiratory domain), mean (SD): 89.7 (8.1) FDL169 600 mg (n = 6) Age, mean (range): 37.7 (18 to 62) years Sex: 3 males, 3 females FEV₁ % predicted, mean (SD): 59.3 (9.9)% Sweat chloride, mean (SD): 101.8 (10.1) mmol/L CFQ‐R score (respiratory domain), mean (SD): 74.0 (11.5) FDL169 800 mg (n = 8) Age, mean (range): 26.5 (21 to 37) years Sex: 4 males, 4 females FEV₁ % predicted, mean (SD): 85.3 (13.5)% Sweat chloride, mean (SD): 98.5 (9.8) mmol/L CFQ‐R score (respiratory domain), mean (SD): 77.0 (18.8) Placebo (n = 7) Age, mean (range): 30.4 (20 to 51) years Sex: 2 males, 5 females FEV₁ % predicted, mean (SD): 64.3 (21.5)% Sweat chloride, mean (SD): 104.4 (14.1) mmol/L CFQ‐R score (respiratory domain), mean (SD): 75.4 (11.6)
Interventions	Cohort 1 (n = 15): FDL169 400 mg 3x daily (n = 6) versus FDL169 600 mg 3x daily (n = 6) versus placebo (n = 3) Cohort 2 (n = 12): FDL169 800 mg 3x daily (n = 8) versus placebo (n = 4)
Outcomes	Safety and tolerability including adverse effects, laboratory tests, ECG and vital signs Pharmacokinetics of numerous doses of FDL169 Exploratory outcomes: changes in "CFTR activity" (sweat chloride levels), pulmonary function (FEV₁ % predicted, respiratory symptoms), CFQ‐R respiratory domain
Funding source	Flatley Discovery Lab was the sponsor for this Phase 1b study
Notes	We also obtained a poster from the author, which was presented at a conference. A study is planned for 2019 to evaluate the corrector FDL169 in combination with potentiator FDL176 in individuals with CF homozygous for F508del.
*Risk of bias*
Bias	Authors' judgement	Support for judgement
Random sequence generation (selection bias)	Unclear risk	States that participants were randomised, but does not state the method by which they were randomised.
Allocation concealment (selection bias)	Unclear risk	Does not state methods of allocation concealment.
Blinding of participants and personnel (performance bias) All outcomes	Unclear risk	Does not state who was and was not blinded during the study.
Blinding of outcome assessment (detection bias) All outcomes	Unclear risk	Does not state how outcome assessors were blinded during the study.
Incomplete outcome data (attrition bias) All outcomes	Low risk	All participants who were randomised are accounted for.
Selective reporting (reporting bias)	Low risk	All outcomes stated in the methods are reported in the results.
Other bias	Unclear risk	As the only information available was as part of a poster and a full detailed publication has not been published; it is difficult to say with any certainty whether there are other sources of bias in the process of this study.