Miglustat in Cystic Fibrosis

This study has been completed.
Information provided by (Responsible Party):
ClinicalTrials.gov Identifier:
First received: August 26, 2008
Last updated: March 5, 2014
Last verified: March 2014
Single Center, Double-Blind, Randomized, Placebo-Controlled, Two-Period/Two-Treatment Crossover Study Investigating the Effect of Miglustat on the Nasal Potential Difference in Patients With Cystic Fibrosis 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: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Single Center, Double-blind, Randomized, Placebo-controlled, Two-period/Two-treatment Crossover Study Investigating the Effect of Miglustat on the Nasal Potential Difference in Patients With Cystic Fibrosis Homozygous for the F508del Mutation

Resource links provided by NLM:

Further study details as provided by Actelion:

Primary Outcome Measures:
  • The sum of responses in nasal potential difference (NPD) after perfusion with isoproterenol and chloride-free buffer (TCS: Total Chloride Secretion), in the presence of amiloride. [ Time Frame: Change from baseline (pre-dose on Day 1) to end-of-treatment (Day 8) ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Change in basline nasal potential difference (NPD) response [ Time Frame: From baseline (pre-dose on Day 1) to end-of-treatment ] [ Designated as safety issue: No ]

Enrollment: 11
Study Start Date: August 2008
Study Completion Date: December 2008
Primary Completion Date: December 2008 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1
Drug: miglustat
Oral miglustat capsules 200 mg t.i.d. (three times a day) for 7 days and a single 200 mg dose on Day 8
Placebo Comparator: 2
Drug: placebo
Oral placebo capsules matching in appearance miglustat capsules given t.i.d. (three times a day) for 7 days and a single dose on Day 8


Ages Eligible for Study:   12 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

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. Women of childbearing potential must use 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. A woman is considered to have child-bearing potential unless she meets at least one of the following criteria:
    • 6 weeks post-surgical bilateral salpingo-oophorectomy or hysterectomy
    • Premature ovarian failure confirmed by a specialist gynecologist
    • Age > 50 years and not treated with any kind of HRT for at least 2 years prior to screening, and with amenorrhea for at least 24 consecutive months prior to screening and a serum FSH level of > 40 IU/L at screening.
    • Age > 55 years and treated with HRT prior to screening with an appropriate medical documentation of spontaneous amenorrhea for at least 24 months. For female patients in the pediatric age range, a reliable method of contraception must be considered, if appropriate.

      • Male patients accepting for the duration of the study and for 3 months thereafter to use a condom and not to procreate a child (not in case of azoospermia)
      • Cystic fibrosis patients homozygous for the F508del 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 of childbearing potential who will not undergo a pregnancy test prior to enrollment into the study
  • History of significant lactose intolerance
  • History of neuropathy
  • Presence of clinically significant diarrhea (> 3 liquid stools per day for > 7 days) without definable cause within 1 month prior to screening
  • Any known factor or disease that might interfere with treatment compliance, study conduct or interpretation of the results such as drug or alcohol dependence or psychiatric disease
  • FEV1 < 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 investigationaldrug or therapy (e.g., gene therapy) within 1 month prior to randomization
  • Breast-feeding, pregnant women or women who plan to become pregnant during the course of the study.
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Please refer to this study by its ClinicalTrials.gov identifier: NCT00742092

Universite Catholique de Louvain
Brussels, Belgium, B-1200
Sponsors and Collaborators
Principal Investigator: Patrick Lebecque, MD, PhD Catholic University of Louvain
  More Information

Responsible Party: Actelion
ClinicalTrials.gov Identifier: NCT00742092     History of Changes
Other Study ID Numbers: AC-056A202 
Study First Received: August 26, 2008
Last Updated: March 5, 2014
Health Authority: Belgium: Institutional Review Board
Belgium: The Federal Public Service (FPS) Health, Food Chain Safety and Environment
Belgium: Federal Agency for Medicines and Health Products, FAMHP

Additional relevant MeSH terms:
Cystic Fibrosis
Digestive System Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Lung Diseases
Pancreatic Diseases
Pathologic Processes
Respiratory Tract Diseases
Anti-HIV Agents
Anti-Infective Agents
Anti-Retroviral Agents
Antiviral Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Therapeutic Uses

ClinicalTrials.gov processed this record on May 02, 2016