Does a Nasal Instillation of Miglustat Normalize the Nasal Potential Difference in Cystic Fibrosis Patients ?
|ClinicalTrials.gov Identifier: NCT00945347|
Recruitment Status : Completed
First Posted : July 24, 2009
Last Update Posted : August 10, 2011
|Condition or disease||Intervention/treatment||Phase|
|Cystic Fibrosis||Drug: Miglustat Drug: Placebo||Phase 2|
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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||10 participants|
|Intervention Model:||Crossover Assignment|
|Masking:||Triple (Participant, Care Provider, Investigator)|
|Official Title:||Does a Nasal Instillation of Miglustat Normalize the Nasal Potential Difference in Cystic Fibrosis Patients Homozygous for the F508del Mutation? A Randomized, Double Blind Placebo-controlled Study.|
|Study Start Date :||July 2009|
|Actual Primary Completion Date :||May 2011|
|Actual Study Completion Date :||June 2011|
No Intervention: Baseline
Active Comparator: Miglustat
Nasal instillation of Miglustat (visit 2 or 3)
Nasal instillation of miglustat
Placebo Comparator: Placebo
Nasal instillation of placebo (visit 3 or 2)
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 ]
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00945347
|Cliniques Universitaires St Luc (Université Catholique de Louvain) 10 avenue Hippocrate|
|Brussels, Belgium, 1200|
|Principal Investigator:||Patrick LEBECQUE, MD, PhD||Cliniques Universitaires St Luc (Université Catholique de Louvain )|
|Principal Investigator:||Teresinha LEAL, MD, PhD||Cliniques Universitaires St. Luc ( Université Catholique de Louvain)|