Miglustat / OGT 918 in the Treatment of Cystic Fibrosis - Full Text View

Sponsor:	Actelion
Information provided by:	Actelion
ClinicalTrials.gov Identifier:	NCT00537602

Purpose

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	Intervention	Phase
Cystic Fibrosis	Drug: miglustat Drug: placebo	Phase II

Study Type:	Interventional
Study Design:	Treatment, Randomized, Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Placebo Control, Crossover Assignment, Safety/Efficacy Study
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

Resource links provided by NLM:

Genetics Home Reference related topics: cystic fibrosis Help Me Understand Genetics

MedlinePlus related topics: Cystic Fibrosis

Drug Information available for: SC 48334

U.S. FDA Resources

Further study details as provided by Actelion:

Primary Outcome Measures:

Change in nasal potential difference (NPD) in response to isoproterenol in chloride-free buffer in the presence of amiloride [ Time Frame: Baseline (pre-dose on day 1) to end-of-treatment (day 8) ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Change in baseline NPD response [ Time Frame: Baseline to end-of-treatment ] [ Designated as safety issue: No ]

Enrollment:	6
Study Start Date:	September 2007
Estimated Study Completion Date:	December 2008
Primary Completion Date:	August 2008 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
A: Experimental Oral miglustat capsules 200 mg t.i.d. for 1 week and a single 200 mg dose on day 8	Drug: miglustat
B: Placebo Comparator Oral placebo capsules matching in appearance miglustat capsules given t.i.d. for 1 week and a single dose on day 8	Drug: placebo

Eligibility

Ages Eligible for Study:	12 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00537602

Locations

Spain
Corporacio Parc Tauli / Parc Tauli Hospital
Barcelona, Spain

Sponsors and Collaborators

Actelion

Investigators

Study Director:	Paul van Giersbergen, PhD	Actelion
Principal Investigator:	Christian Domingo-Ribas, MD	Corporacio Parc Tauli

More Information

Additional Information:

Actelion trials website This link exits the ClinicalTrials.gov site

No publications provided

Responsible Party:	Actelion ( Paul van Giersbergen , PhD )
Study ID Numbers:	AC-056-201
Study First Received:	September 28, 2007
Last Updated:	September 25, 2008
ClinicalTrials.gov Identifier:	NCT00537602 History of Changes
Health Authority:	Spain: Ethics Committee; Spain: Spanish Agency of Medicines

Keywords provided by Actelion:

cystic fibrosis
miglustat
Zavesca

Actelion
nasal potential difference
transmembrane conductance regulator (CFTR)

Additional relevant MeSH terms:

Anti-Infective Agents
Anti-HIV Agents
Molecular Mechanisms of Pharmacological Action
Fibrosis
Enzyme Inhibitors
Antiviral Agents
Pharmacologic Actions
Miglustat
Digestive System Diseases

Pathologic Processes
Anti-Retroviral Agents
Cystic Fibrosis
Respiratory Tract Diseases
Genetic Diseases, Inborn
Therapeutic Uses
Lung Diseases
Pancreatic Diseases
Infant, Newborn, Diseases

ClinicalTrials.gov processed this record on February 08, 2010