Miglustat in Cystic Fibrosis

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Actelion
ClinicalTrials.gov Identifier:
NCT00742092
First received: August 26, 2008
Last updated: March 5, 2014
Last verified: March 2014
  Purpose

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
miglustat
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
placebo
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

  Eligibility

Ages Eligible for Study:   12 Years and older
Genders Eligible for Study:   Both
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. 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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00742092

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

No publications provided

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
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 August 21, 2014