Miglustat / OGT 918 in the Treatment of Cystic Fibrosis

Cystic Fibrosis Clinical Trial

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT00537602
• Other study ID #: AC-056-201
• Status: Terminated
• Phase: Phase 2
• First received: September 28, 2007
• Last updated: February 11, 2010
• Start date: November 2007
• Est. completion date: March 2008

Study information

• Verified date: February 2010
• Source: Actelion
• Contact: n/a
• Is FDA regulated: No
• Health authority: Spain: Ethics CommitteeSpain: Spanish Agency of Medicines
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 6
• Est. completion date: March 2008
• Est. primary completion date: February 2008
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 12 Years and older

Eligibility

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 ?F508 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

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• miglustat

• placebo

Locations

Country	Name	City	State
Spain	Corporacio Parc Tauli / Parc Tauli Hospital	Barcelona

Sponsors (1)

Lead Sponsor	Collaborator
Actelion

Country where clinical trial is conducted

Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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)	No
Secondary	Change in baseline NPD response		Baseline to end-of-treatment	No