Cystic Fibrosis
Conditions
Keywords
cystic fibrosis, miglustat, Zavesca, Actelion, nasal potential difference, transmembrane conductance regulator (CFTR)
Brief summary
Cystic fibrosis is a genetic disease caused by mutation of the cystic fibrosis transmembrane conductance regulator (CFTR). The purpose of the study is to investigate the effects of miglustat on CFTR function in cystic fibrosis patients.
Interventions
DRUGmiglustat
DRUGplacebo
Sponsors
Actelion
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
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: only women who are surgically sterile, who are in the menopause (no menstruation for at least one year) or those of childbearing potential who are using 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. For children, a reliable method of contraception must be considered, if appropriate. * Accepting for the duration of the study and for 3 months thereafter to use a condom and not to procreate a child (males only) * Cystic fibrosis patients homozygous for the ΔF508 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 who will not undergo a pregnancy test prior to enrollment in the study * History of significant lactose intolerance * History of neuropathy * History of cataracts or known increased risk of cataract formation * Presence of clinically significant diarrhea (\>3 liquid stolls per days for \>7 days) without definable cause within 1 month prior to screening * Any known factor of disease that might interfere with treatment compliance, study conduct or interruption of the results such as drug or alcohol dependence or psychiatric disease * FEVI \<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 investigational drug or therapy (e.g., gene therapy) within 1 month prior to randomization
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change in nasal potential difference (NPD) in response to isoproterenol in chloride-free buffer in the presence of amiloride | Baseline (pre-dose on day 1) to end-of-treatment (day 8) |
Secondary
| Measure | Time frame |
|---|---|
| Change in baseline NPD response | Baseline to end-of-treatment |
Countries
Spain
Outcome results
None listed