Miglustat in Cystic Fibrosis

Actelion Pharmaceuticals

Status and phase

Completed

Phase 2

Conditions

Cystic Fibrosis

Treatments

Drug: placebo

Drug: miglustat

Study type

Interventional

Funder types

Industry

Identifiers

NCT00742092

AC-056A202

Details and patient eligibility

About

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

Enrollment

11 patients

Sex

All

Ages

12+ years old

Volunteers

No Healthy Volunteers

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.