Miglustat / OGT 918 in the Treatment of Cystic Fibrosis

Sponsor

Actelion (Industry)

Overall Status

Terminated

CT.gov ID

NCT00537602

Collaborator

(none)

Enrollment

Location

Arms

Duration (Months)

1.5

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Cystic Fibrosis	Drug: miglustat Drug: placebo	Phase 2

Study Design

Study Type:

Interventional

Actual Enrollment :

6 participants

Allocation:

Randomized

Intervention Model:

Crossover Assignment

Masking:

Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Primary Purpose:

Treatment

Official Title:

Single Center, Double-blind, Randomized, Placebo-controlled, 2-period/2-treatment Crossover Study Investigating the Effect of Miglustat on the Nasal Potential Difference in Patients With Cystic Fibrosis Homozygous for the ΔF508 Mutation

Study Start Date :

Nov 1, 2007

Actual Primary Completion Date :

Feb 1, 2008

Actual Study Completion Date :

Mar 1, 2008

Arms and Interventions

Arm	Intervention/Treatment
Experimental: A Oral miglustat capsules 200 mg t.i.d. for 1 week and a single 200 mg dose on day 8	Drug: miglustat Other Names: Zavesca OGT 918 1.5- (Butylimino) -1.5- Dideoxy-D-Glucitol
Placebo Comparator: B Oral placebo capsules matching in appearance miglustat capsules given t.i.d. for 1 week and a single dose on day 8	Drug: placebo

Arm

Intervention/Treatment

Experimental: A

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

Drug: miglustat

Other Names:

Zavesca

OGT 918

1.5- (Butylimino) -1.5- Dideoxy-D-Glucitol

Placebo Comparator: B

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

Drug: placebo

Outcome Measures

Primary Outcome Measures

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 Outcome Measures

Change in baseline NPD response [Baseline to end-of-treatment]

Eligibility Criteria

Criteria

Ages Eligible for Study:

12 Years and Older

Sexes Eligible for Study:

All

Accepts 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: 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