Estudio unicéntrico, a doble ciego, aleatorizado y controlado con placebo, cruzado de 2 brazos, para investigar el efecto de miglustat sobre la diferencia de potencial nasal en pacientes con fibrosis quística homocigotos para la mutación ?F508

Phase 1

• Conditions: fibrosis quística; MedDRA version: 8.1Level: LLTClassification code 10011762Term: Cystic fibrosis

• Registration Number: EUCTR2006-002049-35-ES
• Lead Sponsor: Actelion Pharmaceuticals Ltd

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2007-05-28
• Study Completion: 2008-02-22
• Last Update Posted: 19 April 2022

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 3

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

·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 into 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 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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified