Status:
COMPLETED
Miglustat in Cystic Fibrosis
Lead Sponsor:
Actelion
Conditions:
Cystic Fibrosis
Eligibility:
All Genders
12+ years
Phase:
PHASE2
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 Fi...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
August 1 2008
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 1 2008
Estimated Enrollment :
11 Patients enrolled
Trial Details
Trial ID
NCT00742092
Start Date
August 1 2008
End Date
December 1 2008
Last Update
March 7 2014
Active Locations (1)
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1
Universite Catholique de Louvain
Brussels, Belgium, B-1200