A Phase 2 interventional study of Miglustat and Placebo in Cystic Fibrosis, sponsored by Cliniques universitaires Saint-Luc- Université Catholique de Louvain. Completed at 1 site in Belgium. Open to participants aged 14 Years and older. Per ClinicalTrials.gov, last updated 2011-08-10.
Sponsored by Cliniques universitaires Saint-Luc- Université Catholique de Louvain · Phase 2, Interventional, and Treatment
The purpose of this study is to investigate within a short delay the effect of nasal instillation of Miglustat on nasal potential difference in cystic fibrosis patients homozygous for the F508del mutation.
Miglustat is an inhibitor of α-glucosidases and other enzymes. Oral miglustat is currently marketed in Europa and US for the treatment of Gaucher type 1 patients for whom enzyme replacement treatment is not an option.
Gastro-intestinal side effects are common with this formulation. This medication has been shown to have a beneficial effect both on Cl- an Na+ transports in cystic fibrosis epithelial cells. In addition, a single airway delivery of low-dose Miglustat normalizes nasal potential difference (NPD) in F508del cystic fibrosis mice. NPD abnormalities specific of CF patients are considered to reflect the primary defect of CFTR protein so that any curative treatment is expected to correct them at least partially.
In the field of respiratory pharmacology, it is a general rule that the inhaled route is to be favoured whenever possible : it is usually more effective despite much lower doses and systemic absorption (which also implies lower costs and improved tolerance).
The aim of this study is to investigate the effect of a single local administration of Miglustat on NPD measurements in CF patients homozygous for the F508del mutation.
1,581 studies on the registry are indexed under Cystic Fibrosis; 190 are open to participants now.
This study's planned enrollment of 10 is below the median of 36 across 1,034 interventional studies indexed under Cystic Fibrosis.
Browse Cystic Fibrosis studies →Cliniques universitaires Saint-Luc- Université Catholique de Louvain is the lead sponsor of 342 studies on the registry; 61 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Visit 1
Nasal instillation of Miglustat (visit 2 or 3)
Drug: Miglustat
Nasal instillation of placebo (visit 3 or 2)
Drug: Placebo
Nasal instillation of miglustat
Nasal instillation of placebo matching in appearance with the Miglustat instillation
change in response to Chloride-free solution and isoproterenol ( reflecting chloride transport)
Time frame: change from baseline ( visit 1) and placebo to miglustat instillation
change in basal voltage value and in amiloride response ( reflecting sodium transport)
Time frame: change from baseline (visit1) and placebo to miglustat instillation
This study is completed, as verified in Aug 2011. You cannot join it, but the record below documents what was studied.
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Cliniques universitaires Saint-Luc- Université Catholique de Louvain