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J Cyst Fibros. 2012 May;11(3):231-6. doi: 10.1016/j.jcf.2011.12.004. Epub 2012 Jan 27.

A randomized placebo-controlled trial of miglustat in cystic fibrosis based on nasal potential difference.

Author information

1
Pediatric Pulmonology & Cystic Fibrosis Unit, Cliniques St Luc, Université catholique de Louvain, 10 Avenue Hippocrate, 1200 Brussels, Belgium. anissa.leonard@uclouvain.be

Abstract

BACKGROUND:

Preclinical data suggest that miglustat could restore the function of the cystic fibrosis transmembrane conductance regulator gene in cystic fibrosis cells.

METHODS:

Single-center, randomized, double-blind, placebo-controlled, crossover Phase II study in 11 patients (mean±SD age, 26.3±7.7 years) homozygous for the F508del mutation received oral miglustat 200 mgt.i.d. or placebo for two 8-day cycles separated by a 14-day washout period. The primary endpoint was the change in total chloride secretion (TCS) assessed by nasal potential difference.

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.

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.

PMID:
22281182
DOI:
10.1016/j.jcf.2011.12.004
[Indexed for MEDLINE]
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