Does a Nasal Instillation of Miglustat Normalize the Nasal Potential Difference in Cystic Fibrosis Patients ?
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Purpose
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.
| Condition | Intervention | Phase |
|---|---|---|
|
Cystic Fibrosis |
Drug: Miglustat Drug: Placebo |
Phase 2 |
| Study Type: | Interventional |
| Study Design: | Allocation: Randomized Endpoint Classification: Efficacy Study Intervention Model: Crossover Assignment Masking: Double Blind (Subject, Caregiver, Investigator) Primary Purpose: Treatment |
| 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. |
- change in response to Chloride-free solution and isoproterenol ( reflecting chloride transport) [ Time Frame: change from baseline ( visit 1) and placebo to miglustat instillation ] [ Designated as safety issue: No ]
- change in basal voltage value and in amiloride response ( reflecting sodium transport) [ Time Frame: change from baseline (visit1) and placebo to miglustat instillation ] [ Designated as safety issue: No ]
| Estimated Enrollment: | 10 |
| Study Start Date: | July 2009 |
| Study Completion Date: | June 2011 |
| Primary Completion Date: | May 2011 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
No Intervention: Baseline
Visit 1
|
|
|
Active Comparator: Miglustat
Nasal instillation of Miglustat (visit 2 or 3)
|
Drug: Miglustat
Nasal instillation of miglustat
|
|
Placebo Comparator: Placebo
Nasal instillation of placebo (visit 3 or 2)
|
Drug: Placebo
Nasal instillation of placebo matching in appearance with the Miglustat instillation
|
Detailed Description:
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.
Eligibility| Ages Eligible for Study: | 14 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Cystic fibrosis patients homozygous for the F508del mutation as confirmed by genetic test
- 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:
- Acute respiratory tract infection or pulmonary exacerbation requiring antibiotic intervention within 2 weeks of visit 1
- 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
Contacts and Locations| Belgium | |
| 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) |
More Information
Publications:
| Responsible Party: | Patrick Lebecque, MD, PhD, Cliniques universitaires Saint Luc (Université Catholique de Louvain) |
| ClinicalTrials.gov Identifier: | NCT00945347 History of Changes |
| Other Study ID Numbers: | MIG-99 |
| Study First Received: | July 23, 2009 |
| Last Updated: | August 9, 2011 |
| Health Authority: | Belgium: Federal Agency for Medicinal Products and Health Products |
Keywords provided by Cliniques universitaires Saint-Luc- Université Catholique de Louvain:
|
Cystic fibrosis miglustat nasal instillation nasal potential difference |
Additional relevant MeSH terms:
|
Cystic Fibrosis Fibrosis Pancreatic Diseases Digestive System Diseases Lung Diseases Respiratory Tract Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases Pathologic Processes |
Miglustat Enzyme Inhibitors Molecular Mechanisms of Pharmacological Action Pharmacologic Actions Anti-HIV Agents Anti-Retroviral Agents Antiviral Agents Anti-Infective Agents Therapeutic Uses |
ClinicalTrials.gov processed this record on May 16, 2013