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Miglustat / OGT 918 in the Treatment of Cystic Fibrosis

Primary Purpose

Cystic Fibrosis

Status
Terminated
Phase
Phase 2
Locations
Spain
Study Type
Interventional
Intervention
miglustat
placebo
Sponsored by
Actelion
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cystic Fibrosis focused on measuring cystic fibrosis, miglustat, Zavesca, Actelion, nasal potential difference, transmembrane conductance regulator (CFTR)

Eligibility Criteria

12 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept 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

Sites / Locations

  • Corporacio Parc Tauli / Parc Tauli Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

A

B

Arm Description

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

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

Outcomes

Primary Outcome Measures

Change in nasal potential difference (NPD) in response to isoproterenol in chloride-free buffer in the presence of amiloride

Secondary Outcome Measures

Change in baseline NPD response

Full Information

First Posted
September 28, 2007
Last Updated
February 11, 2010
Sponsor
Actelion
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1. Study Identification

Unique Protocol Identification Number
NCT00537602
Brief Title
Miglustat / OGT 918 in the Treatment of Cystic Fibrosis
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 Type
Interventional

2. Study Status

Record Verification Date
February 2010
Overall Recruitment Status
Terminated
Why Stopped
Methodology applied did not meet all criteria required per guidelines
Study Start Date
November 2007 (undefined)
Primary Completion Date
February 2008 (Actual)
Study Completion Date
March 2008 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor
Actelion

4. Oversight

Data Monitoring Committee
No

5. 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis
Keywords
cystic fibrosis, miglustat, Zavesca, Actelion, nasal potential difference, transmembrane conductance regulator (CFTR)

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Crossover Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
6 (Actual)

8. Arms, Groups, and Interventions

Arm Title
A
Arm Type
Experimental
Arm Description
Oral miglustat capsules 200 mg t.i.d. for 1 week and a single 200 mg dose on day 8
Arm Title
B
Arm Type
Placebo Comparator
Arm Description
Oral placebo capsules matching in appearance miglustat capsules given t.i.d. for 1 week and a single dose on day 8
Intervention Type
Drug
Intervention Name(s)
miglustat
Other Intervention Name(s)
Zavesca, OGT 918, 1.5- (Butylimino) -1.5- Dideoxy-D-Glucitol
Intervention Type
Drug
Intervention Name(s)
placebo
Primary Outcome Measure Information:
Title
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)
Secondary Outcome Measure Information:
Title
Change in baseline NPD response
Time Frame
Baseline to end-of-treatment

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Paul van Giersbergen, PhD
Organizational Affiliation
Actelion
Official's Role
Study Director
First Name & Middle Initial & Last Name & Degree
Christian Domingo-Ribas, MD
Organizational Affiliation
Corporacio Parc Tauli
Official's Role
Principal Investigator
Facility Information:
Facility Name
Corporacio Parc Tauli / Parc Tauli Hospital
City
Barcelona
Country
Spain

12. IPD Sharing Statement

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Miglustat / OGT 918 in the Treatment of Cystic Fibrosis

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