Cystic FibrosisPhase 2NCT00537602
Miglustat / OGT 918 in the Treatment of Cystic Fibrosis
A Phase 2 study testing miglustat in people with Cystic Fibrosis, run by Actelion at 1 site in Spain.
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. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- miglustat
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 6 participants
- Where
- 1 siteSpain
- Study dates
- Nov 2007to Mar 2008
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
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- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
1 site in 1 country.
About this 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
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
- Being tested
- miglustat, placebo
- Keywords
- cystic fibrosis, miglustat, Zavesca, Actelion, nasal potential difference, transmembrane conductance regulator (CFTR)
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
miglustatDrug
Also called Zavesca, OGT 918, 1.5- (Butylimino) -1.5- Dideoxy-D-Glucitol
placeboDrug
| Study group | Role | What they receive |
|---|---|---|
| A | Experimental | Oral miglustat capsules 200 mg t.i.d. for 1 week and a single 200 mg dose on day 8 Receives: 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 Receives: Drug: placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in nasal potential difference (NPD) in response to isoproterenol in chloride-free buffer in the presence of amiloride
Measured: Baseline (pre-dose on day 1) to end-of-treatment (day 8)
Secondary outcomes (1)
Change in baseline NPD response
Measured: Baseline to end-of-treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Oct 2007 (estimated)
- Study startNov 2007
- Primary completionFeb 2008
- Study completionMar 2008
Why it stopped: Methodology applied did not meet all criteria required per guidelines
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Actelion
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Paul van Giersbergen, PhD, Study director, ActelionChristian Domingo-Ribas, MD, Principal investigator, Corporacio Parc Tauli
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00537602
- Sponsor's protocol ID
- AC-056-201
- Status last verified
- Feb 2010
- First submitted
- 28 Sep 2007
- Last updated
- 12 Feb 2010
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Feb 2010. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT00537602
- Acronym / sponsor ID
- AC-056-201
- Conditions
- Cystic Fibrosis
- Keywords
- cystic fibrosis, miglustat, Zavesca, Actelion, nasal potential difference, transmembrane conductance regulator (CFTR)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Methodology applied did not meet all criteria required per guidelines
- Enrolment
- 6 participants (actual)
- Lead sponsor
- ActelionIndustry (pharma, biotech, devices)
- Study start
- Nov 2007
- Primary completion
- Feb 2008 (actual)
- Study completion
- Mar 2008 (actual)
- First posted
- 1 Oct 2007 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| A | Experimental | Drug: miglustatOral miglustat capsules 200 mg t.i.d. for 1 week and a single 200 mg dose on day 8 |
| B | Placebo comparator | Drug: placeboOral placebo capsules matching in appearance miglustat capsules given t.i.d. for 1 week and a single dose on day 8 |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (12 listed)
- 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:
- 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)
Key exclusion criteria (14 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| miglustatAlso: Zavesca, OGT 918, 1.5- (Butylimino) -1.5- Dideoxy-D-Glucitol | Drug | A |
| placebo | Drug | B |
Primary outcome
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)
1 secondary outcome
Change in baseline NPD response
Time frame: Baseline to end-of-treatment
Results and safety
The study was stopped early (reason given: Methodology applied did not meet all criteria required per guidelines). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Feb 2010.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Paul van Giersbergen, PhD study director
Actelion
Christian Domingo-Ribas, MD principal investigator
Corporacio Parc Tauli
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing miglustat in people with Cystic Fibrosis, run by Actelion at 1 site in Spain.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 12 Feb 2010. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Spain. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Methodology applied did not meet all criteria required per guidelines). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Feb 2010.