Miglustat in Cystic Fibrosis

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00742092
• Date of registration: 26/08/2008
• Prospective Registration: No
• Primary sponsor: Actelion
• Public title: Miglustat in Cystic Fibrosis
• Scientific title: Single Center, Double-blind, Randomized, Placebo-controlled, Two-period/Two-treatment Crossover Study Investigating the Effect of Miglustat on the Nasal Potential Difference in Patients With Cystic Fibrosis Homozygous for the F508del Mutation
• Date of first enrolment: August 2008
• Target sample size: 11
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00742092
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
• Phase: Phase 2

Countries of recruitment

Belgium

Contacts

• Name: Patrick Lebecque, MD, PhD
• Affiliation: Catholic University of Louvain

Key inclusion & exclusion 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. Women of childbearing potential must use 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. A woman is considered
to have child-bearing potential unless she meets at least one of the following
criteria:

- 6 weeks post-surgical bilateral salpingo-oophorectomy or hysterectomy

- Premature ovarian failure confirmed by a specialist gynecologist

- Age > 50 years and not treated with any kind of HRT for at least 2 years prior
to screening, and with amenorrhea for at least 24 consecutive months prior to
screening and a serum FSH level of > 40 IU/L at screening.

- Age > 55 years and treated with HRT prior to screening with an appropriate
medical documentation of spontaneous amenorrhea for at least 24 months. For
female patients in the pediatric age range, a reliable method of contraception
must be considered, if appropriate.

- Male patients accepting for the duration of the study and for 3 months
thereafter to use a condom and not to procreate a child (not in case of
azoospermia)

- Cystic fibrosis patients homozygous for the F508del 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 of childbearing potential who will not undergo a pregnancy test prior
to enrollment into the study

- History of significant lactose intolerance

- History of neuropathy

- 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 investigationaldrug or therapy (e.g.,
gene therapy) within 1 month prior to randomization

- Breast-feeding, pregnant women or women who plan to become pregnant during the course
of the study.



Age minimum: 12 Years
Age maximum: N/A
Gender: Both

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: placebo

Drug: miglustat

Primary Outcome(s)

The sum of responses in nasal potential difference (NPD) after perfusion with isoproterenol and chloride-free buffer (TCS: Total Chloride Secretion), in the presence of amiloride. [Time Frame: Change from baseline (pre-dose on Day 1) to end-of-treatment (Day 8)]

Secondary Outcome(s)

Change in basline nasal potential difference (NPD) response [Time Frame: From baseline (pre-dose on Day 1) to end-of-treatment]

Secondary ID(s)

AC-056A202

Source(s) of Monetary Support

Please refer to primary and secondary sponsors