Does a Nasal Instillation of Miglustat Normalize the Nasal Potential Difference in Cystic Fibrosis Patients ?

August 9, 2011 updated by: Cliniques universitaires Saint-Luc- Université Catholique de Louvain

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.

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Anticipated)

10

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Belgium
      • Brussels, Belgium, 1200
        • Cliniques Universitaires St Luc (Université Catholique de Louvain) 10 avenue Hippocrate

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

14 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Crossover Assignment
  • Masking: Triple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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 from baseline ( visit 1) and placebo to miglustat instillation

Secondary Outcome Measures

Outcome Measure
Time Frame
change in basal voltage value and in amiloride response ( reflecting sodium transport)
Time Frame: change from baseline (visit1) and placebo to miglustat instillation
change from baseline (visit1) and placebo to miglustat instillation

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Cliniques universitaires Saint-Luc- Université Catholique de Louvain

Investigators

  • 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)

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

July 1, 2009

Primary Completion (Actual)

May 1, 2011

Study Completion (Actual)

June 1, 2011

Study Registration Dates

First Submitted

July 23, 2009

First Submitted That Met QC Criteria

July 23, 2009

First Posted (Estimate)

July 24, 2009

Study Record Updates

Last Update Posted (Estimate)

August 10, 2011

Last Update Submitted That Met QC Criteria

August 9, 2011

Last Verified

August 1, 2011

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • MIG-99

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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