Miglustat / OGT 918 in the Treatment of Cystic Fibrosis

NCT ID: NCT00537602

Last Updated: 2010-02-12

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2
• Total Enrollment: 6 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2007-11-30
• Study Completion Date: 2008-03-31

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.

Conditions

Cystic Fibrosis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

A

Oral miglustat capsules 200 mg t.i.d. for 1 week and a single 200 mg dose on day 8

Group Type: EXPERIMENTAL

miglustat

Intervention Type: DRUG

B

Oral placebo capsules matching in appearance miglustat capsules given t.i.d. for 1 week and a single dose on day 8

Group Type: PLACEBO_COMPARATOR

placebo

Intervention Type: DRUG

Interventions

miglustat

Intervention Type: DRUG

placebo

Intervention Type: DRUG

Other Intervention Names

Zavesca; OGT 918; 1.5- (Butylimino) -1.5- Dideoxy-D-Glucitol

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: 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

• Minimum Eligible Age: 12 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Actelion

INDUSTRY

Sponsor Role: lead

Responsible Party

Actelion

Principal Investigators

Paul van Giersbergen, PhD

Role: STUDY_DIRECTOR

Actelion

Christian Domingo-Ribas, MD

Role: PRINCIPAL_INVESTIGATOR

Corporacio Parc Tauli

Locations

Corporacio Parc Tauli / Parc Tauli Hospital

Barcelona, , Spain

Countries

Spain

Other Identifiers

AC-056-201

Identifier Type: -

Identifier Source: org_study_id