miglustat in cystic fibrosis

Conditions: Cystic Fibrosis

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


Eligibility 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.