Miglustat / OGT 918 in the Treatment of Cystic Fibrosis

NCT00537602 | Terminated Phase 2

• 1 other identifier: AC-056-201
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

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

Keywords

cystic fibrosis; miglustat; Zavesca; Actelion; nasal potential difference; transmembrane conductance regulator (CFTR)

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• Change in baseline NPD response

  Baseline to end-of-treatment

Study Arms (2)

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

Interventions

miglustat DRUG

Also known as: Zavesca, OGT 918, 1.5- (Butylimino) -1.5- Dideoxy-D-Glucitol

A

placebo DRUG

B

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Actelion: lead

Study Sites (1)

Corporacio Parc Tauli / Parc Tauli Hospital

Barcelona, Spain

Location

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

miglustat

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Officials

• Paul van Giersbergen, PhD

  Actelion

  STUDY DIRECTOR

• Christian Domingo-Ribas, MD

  Corporacio Parc Tauli

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY

Study Record Dates

• First Submitted: September 28, 2007
• First Posted: October 1, 2007
• Study Start: November 1, 2007
• Primary Completion: February 1, 2008
• Study Completion: March 1, 2008
• Last Updated: February 12, 2010

Record last verified: 2010-02

Locations

ES (1)