Does a Nasal Instillation of Miglustat Normalize the Nasal Potential Difference in Cystic Fibrosis Patients ?
NCT ID: NCT00945347
Last Updated: 2011-08-10
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE2
10 participants
INTERVENTIONAL
2009-07-31
2011-06-30
Brief Summary
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Detailed Description
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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.
Conditions
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Study Design
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RANDOMIZED
CROSSOVER
TREATMENT
TRIPLE
Study Groups
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Baseline
Visit 1
No interventions assigned to this group
Miglustat
Nasal instillation of Miglustat (visit 2 or 3)
Miglustat
Nasal instillation of miglustat
Placebo
Nasal instillation of placebo (visit 3 or 2)
Placebo
Nasal instillation of placebo matching in appearance with the Miglustat instillation
Interventions
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Miglustat
Nasal instillation of miglustat
Placebo
Nasal instillation of placebo matching in appearance with the Miglustat instillation
Eligibility Criteria
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Inclusion Criteria
* Aged 14 years and older
* Male or female (non-pregnant women who are to remain non-pregnant for 3 months after the end of the study)
* FEV1 \> 50% of predicted normal
Exclusion Criteria
* Any condition prohibiting the correct measurement of the NPD such as respiratory tract infection
* Active or passive smoking
* Allergic chronic rhinitis
* History of significant lactose intolerance
* History of neuropathy
* History of cataracts or known increased risk of cataract formation
* Hypersensitivity to miglustat or any excipients
* Planned treatment or treatment with another investigational drug or therapy within 1 month prior to randomisation
14 Years
ALL
No
Sponsors
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Cliniques universitaires Saint-Luc- Université Catholique de Louvain
OTHER
Responsible Party
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Cliniques universitaires Saint Luc (Université Catholique de Louvain)
Principal Investigators
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Patrick LEBECQUE, MD, PhD
Role: PRINCIPAL_INVESTIGATOR
Cliniques Universitaires St Luc (Université Catholique de Louvain )
Teresinha LEAL, MD, PhD
Role: PRINCIPAL_INVESTIGATOR
Cliniques Universitaires St. Luc ( Université Catholique de Louvain)
Locations
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Cliniques Universitaires St Luc (Université Catholique de Louvain) 10 avenue Hippocrate
Brussels, , Belgium
Countries
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References
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Lubamba B, Lebacq J, Lebecque P, Vanbever R, Leonard A, Wallemacq P, Leal T. Airway delivery of low-dose miglustat normalizes nasal potential difference in F508del cystic fibrosis mice. Am J Respir Crit Care Med. 2009 Jun 1;179(11):1022-8. doi: 10.1164/rccm.200901-0049OC. Epub 2009 Mar 19.
Norez C, Noel S, Wilke M, Bijvelds M, Jorna H, Melin P, DeJonge H, Becq F. Rescue of functional delF508-CFTR channels in cystic fibrosis epithelial cells by the alpha-glucosidase inhibitor miglustat. FEBS Lett. 2006 Apr 3;580(8):2081-6. doi: 10.1016/j.febslet.2006.03.010. Epub 2006 Mar 10.
Noel S, Wilke M, Bot AG, De Jonge HR, Becq F. Parallel improvement of sodium and chloride transport defects by miglustat (n-butyldeoxynojyrimicin) in cystic fibrosis epithelial cells. J Pharmacol Exp Ther. 2008 Jun;325(3):1016-23. doi: 10.1124/jpet.107.135582. Epub 2008 Feb 28.
Norez C, Antigny F, Noel S, Vandebrouck C, Becq F. A cystic fibrosis respiratory epithelial cell chronically treated by miglustat acquires a non-cystic fibrosis-like phenotype. Am J Respir Cell Mol Biol. 2009 Aug;41(2):217-25. doi: 10.1165/rcmb.2008-0285OC. Epub 2009 Jan 8.
Heneghan M, Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2023 Nov 20;11(11):CD010966. doi: 10.1002/14651858.CD010966.pub4.
Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3.
Other Identifiers
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MIG-99
Identifier Type: -
Identifier Source: org_study_id
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