Study design (if review, criteria of inclusion for studies)
Single-center, randomized, double-blind, placebo-controlled, crossover Phase II study
Participants
11 patients (mean±SD age, 26.3±7.7years) homozygous for the F508del mutation
Interventions
oral miglustat 200mgt.i.d. or placebo for two 8-day cycles separated by a 14-day washout period.
Outcome measures
The primary endpoint was the change in total chloride secretion (TCS) assessed by nasal potential difference.
Main results
No statistically significant changes in TCS, sweat chloride values or FEV(1) were detected. Pharmacokinetic and safety were similar to those observed in patients with other diseases exposed to miglustat.
Authors' conclusions
There was no evidence of a treatment effect on any nasal potential difference variable. Further studies with miglustat need to adequately address criteria for assessment of nasal potential difference.