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Miglustat / OGT 918 in the Treatment of Cystic Fibrosis

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ClinicalTrials.gov Identifier: NCT00537602
Recruitment Status : Terminated (Methodology applied did not meet all criteria required per guidelines)
First Posted : October 1, 2007
Last Update Posted : February 12, 2010
Sponsor:
Information provided by:
Actelion

Brief Summary:
Cystic fibrosis is a genetic disease caused by mutation of the cystic fibrosis transmembrane conductance regulator (CFTR). The purpose of the study is to investigate the effects of miglustat on CFTR function in cystic fibrosis patients.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: miglustat Drug: placebo Phase 2

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 6 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Single Center, Double-blind, Randomized, Placebo-controlled, 2-period/2-treatment Crossover Study Investigating the Effect of Miglustat on the Nasal Potential Difference in Patients With Cystic Fibrosis Homozygous for the ΔF508 Mutation
Study Start Date : November 2007
Actual Primary Completion Date : February 2008
Actual Study Completion Date : March 2008

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis
Drug Information available for: Miglustat

Arm Intervention/treatment
Experimental: A
Oral miglustat capsules 200 mg t.i.d. for 1 week and a single 200 mg dose on day 8
Drug: miglustat
Other Names:
  • Zavesca
  • OGT 918
  • 1.5- (Butylimino) -1.5- Dideoxy-D-Glucitol

Placebo Comparator: B
Oral placebo capsules matching in appearance miglustat capsules given t.i.d. for 1 week and a single dose on day 8
Drug: placebo



Primary Outcome Measures :
  1. Change in nasal potential difference (NPD) in response to isoproterenol in chloride-free buffer in the presence of amiloride [ Time Frame: Baseline (pre-dose on day 1) to end-of-treatment (day 8) ]

Secondary Outcome Measures :
  1. Change in baseline NPD response [ Time Frame: Baseline to end-of-treatment ]


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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Aged 12 years and older
  • Male or female
  • Non-pregnant women who are to remain non-pregnant for 3 months after the end of the study: only women who are surgically sterile, who are in the menopause (no menstruation for at least one year) or those of childbearing potential who are using a reliable method of contraception. Reliable methods of contraception for female patients include the following:

    • Barrier type devices (e.g., female condom, diaphragm and contraceptive sponge) used ONLY in combination with a spermicide
    • Intrauterine devices
    • Oral contraceptive agent
    • Depo-Provera™ (medroxyprogesterone acetate)
    • Levonorgestrel implants Abstention, the rhythm method or contraception by the partner alone are NOT reliable methods of contraception.

For children, a reliable method of contraception must be considered, if appropriate.

  • Accepting for the duration of the study and for 3 months thereafter to use a condom and not to procreate a child (males only)
  • Cystic fibrosis patients homozygous for the ΔF508 mutation as confirmed by genetic test
  • Signed informed consent prior to any study-mandated procedure

Exclusion Criteria:

  • Any condition prohibiting the correct measurement of the NPD such as upper respiratory tract infection
  • Acute upper respiratory tract or pulmonary exacerbation requiring antibiotic intervention within 2 weeks of screening
  • Severe renal impairment (creatinine clearance < 30 ml/min as per Cockroft and Gault)
  • Female patients who will not undergo a pregnancy test prior to enrollment in the study
  • History of significant lactose intolerance
  • History of neuropathy
  • History of cataracts or known increased risk of cataract formation
  • Presence of clinically significant diarrhea (>3 liquid stolls per days for >7 days) without definable cause within 1 month prior to screening
  • Any known factor of disease that might interfere with treatment compliance, study conduct or interruption of the results such as drug or alcohol dependence or psychiatric disease
  • FEVI <25% of predicted normal
  • Oxygen saturation at rest <88%
  • Active or passive smoking as measured using the Smokelyzer®
  • Hypersensitivity to miglustat or any excipients
  • Planned treatment or treatment with another investigational drug or therapy (e.g., gene therapy) within 1 month prior to randomization

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00537602


Locations
Spain
Corporacio Parc Tauli / Parc Tauli Hospital
Barcelona, Spain
Sponsors and Collaborators
Actelion
Investigators
Study Director: Paul van Giersbergen, PhD Actelion
Principal Investigator: Christian Domingo-Ribas, MD Corporacio Parc Tauli

Responsible Party: Paul van Giersbergen , PhD, Actelion
ClinicalTrials.gov Identifier: NCT00537602     History of Changes
Other Study ID Numbers: AC-056-201
First Posted: October 1, 2007    Key Record Dates
Last Update Posted: February 12, 2010
Last Verified: February 2010

Keywords provided by Actelion:
cystic fibrosis
miglustat
Zavesca
Actelion
nasal potential difference
transmembrane conductance regulator (CFTR)

Additional relevant MeSH terms:
Fibrosis
Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Miglustat
1-Deoxynojirimycin
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Anti-HIV Agents
Anti-Retroviral Agents
Antiviral Agents
Anti-Infective Agents
Glycoside Hydrolase Inhibitors
Hypoglycemic Agents
Physiological Effects of Drugs