fibrosis quística MedDRA version: 8.1 Level: LLT Classification code 10011762 Term: Cystic fibrosis
Conditions
Interventions
Trade Name: Zavesca
Product Name: miglustat
Product Code: OGT 918
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: Miglustat
CAS Number: 72599-27-0
Current Sponsor code: OGT 918
Other descripti
Sponsors
Actelion Pharmaceuticals Ltd
Eligibility
Sex/Gender
All
Inclusion 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: 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: Ø barrier type devices (e.g., female condom, diaphragm and contraceptive sponge) used ONLY in combination with a spermicide Ø intrauterine devices Ø oral contraceptive agent Ø Depo-Provera TM (medroxyprogesterone acetate) Ø 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 ?F508 mutation as confirmed by genetic test · Signed informed consent prior to any study-mandated procedure. Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
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 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 investigational drug or therapy (e.g., gene therapy) within 1 month prior to randomization
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To demonstrate that miglustat restores the function of the cystic fibrosis transmembrane conductance regulator (CFTR) in patients with cystic fibrosis homozygous for the ?F508 mutation as reflected in nasal potential difference (NPD).;Secondary Objective: . To investigate the effect of miglustat on the concentration of sodium and chloride in sweat in this patient population. · To investigate the safety and tolerability of miglustat in this patient population.;Primary end point(s): Primary endpoint: Change from baseline (pre-dose on day 1) to end-of-treatment (day 8) in nasal potential difference (NPD) in response to isoproterenol in chloride-free buffer in the presence of amiloride Assumptions: · A difference of 3.2 mV to the isoproterenol challenge between placebo and miglustat treatment (as measured after the morning dose on day 8 of each period) represents about 20% of the response seen in non cystic fibrosis subjects, which is considered clinically significant. · No carry-over effect is expected with a washout of 2 weeks. · No period effect is expected · It is assumed that the NPD is normally distributed and that the standard deviation of the difference of 2 repeat measurements is 4.4 mV. Secondary endpoints: · Change from baseline to end-of-treatment in baseline NPD response · Change from baseline to end-of-treatment in amiloride-sensitive NP · Change from baseline to end-of-treatment in low-chloride sensitive NPD in the presence of amiloride · Change from baseline to end-of-treatment in ATP-sensitive NPD in the presence of chloride-free buffer, amiloride, and isoproterenol · Change from baseline to end-of-treatment in sweat sodium and chloride concentration Baseline and end-of-treatment are defined as predose on day 1 and 8, respectively, of both treatment periods | — |
Countries
Spain
Outcome results
None listed