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Miglustat in Cystic Fibrosis

Sponsor
Actelion (Industry)
Overall Status
Completed
CT.gov ID
NCT00742092
Collaborator
(none)
11
Enrollment
1
Location
2
Arms
4
Duration (Months)
2.7
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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 or Disease Intervention/Treatment Phase
Phase 2

Study Design

Study Type:
Interventional
Actual Enrollment :
11 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, 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
Study Start Date :
Aug 1, 2008
Actual Primary Completion Date :
Dec 1, 2008
Actual Study Completion Date :
Dec 1, 2008

Arms and Interventions

Arm Intervention/Treatment
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

Outcome Measures

Primary Outcome Measures

  1. 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. [Change from baseline (pre-dose on Day 1) to end-of-treatment (Day 8)]

Secondary Outcome Measures

  1. Change in basline nasal potential difference (NPD) response [From baseline (pre-dose on Day 1) to end-of-treatment]

Eligibility Criteria

Criteria

Ages Eligible for Study:
12 Years and Older
Sexes Eligible for Study:
All
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.

Contacts and Locations

Locations

Site City State Country Postal Code
1 Universite Catholique de Louvain Brussels Belgium B-1200

Sponsors and Collaborators

  • Actelion

Investigators

  • Principal Investigator: Patrick Lebecque, MD, PhD, Catholic University of Louvain

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Actelion
ClinicalTrials.gov Identifier:
NCT00742092
Other Study ID Numbers:
  • AC-056A202
First Posted:
Aug 27, 2008
Last Update Posted:
Mar 7, 2014
Last Verified:
Mar 1, 2014
Additional relevant MeSH terms:
Cystic Fibrosis
Fibrosis
Miglustat

Study Results

No Results Posted as of Mar 7, 2014