Cystic FibrosisPhase 1/2CFStatinNCT01092572
Statins To Treat Adult Cystic Fibrosis
A Phase 1/2 study testing Simvastatin in people with Cystic Fibrosis and Systemic Inflammation, run by University of British Columbia.
Trial summary
Cystic fibrosis (CF) is a lethal genetic condition that affects 30,000 children and adults in the United States. Although CF management has improved substantially over the past two decades, there is still no cure and most patients with CF die before reaching their 50th birthday, largely due to lung failure. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cystic Fibrosis, Systemic Inflammation
- What is being tested?
- Simvastatin
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- May 2010to May 2010
- Phase
- Phase 1/2
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients of provincial legal age of majority in British Columbia (≥19 years of age);
- Confirmed diagnosis of CF based on the following criteria:
- One or more clinical features consistent with the CF phenotype
- A genotype with identifiable classes I or II CFTR mutations
- Ability to provide informed consent.
- Clinically stable at enrollment as assessed by the treating physician.
- Ability to comply with medication use, study visits and study procedures, such as spirometry, and venipunctures.
Who cannot join
- Allergy or clinical reaction to simvastatin.
- The following abnormal lab values within the last six months or at screening:
- AST/ALT > 1.5 ULN, CK > 1.5 ULN, and eGFR < 40ml/min/1.73m2.
- Use of intravenous antibiotics or oral quinolones within 14 days of screening.
- With the exception of Azithromycin the use of oral antibiotics including prophylactic antibiotics (e.g., augmentin, tetracycline, cloxacillin, cephalosporins, trimethoprim/sulfamethoxazole) within 14 days of screening.
- Initiation of high dose ibuprofen, dornase alpha, hypertonic saline or aerosolized antibiotics within 30 days of screening.
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- On medications that are known to have potential serious interactions with simvastatin (as listed on page 10 of this protocol).
- Use of systemic corticosteroids within 30 days of screening.
- Investigational drug use within 30 days of screening.
- Other major organ dysfunction excluding pancreatic dysfunction.
- History of lung transplantation or currently on lung transplant list.
- Pregnant, breast feeding, or if post-menarche female, unwilling to practice birth control during participation in the study.
- Chronic users of niacin, azole antifungals (itraconazole, ketoconazole, voriconazole), telithromycin, fibric acid derivatives, HIV protease inhibitors, amiodarone, digoxin and/or cyclosporine (to decrease the risk of statin-related myotoxicity).
- Patients who are colonized or infected with Burkholderia cepacia complex are excluded.
About this study
Official title: The Effect of Simvastatin on Systemic Inflammation in Adult Cystic Fibrosis Subjects: A Pilot Study
Cystic fibrosis (CF) is a lethal genetic condition that affects 30,000 children and adults in the United States. Although CF management has improved substantially over the past two decades, there is still no cure and most patients with CF die before reaching their 50th birthday, largely due to lung failure. There is growing evidence that excess lung and blood inflammation that occurs in response to infections in the lungs cause CF patients to be sicker. Simvastatin is a drug that is used to lower cholesterol, but many researchers have found that this drug may also treat blood and lung inflammation. In this study, we will determine whether or not simvastatin can treat blood and lung inflammation in patients with CF and most importantly determine whether or not it can make these patients feel better and have better lung function.
Read the detailed description
Study Objectives
- To determine the effect of 12 weeks of 40 mg once daily simvastatin on general inflammatory molecules, IL-6 and CRP in the blood of CF patients.
- To determine the effect of simvastatin on LPS-related pathway molecules in the blood.
- To determine the effect of simvastatin on inflammatory pneumo-proteins in the blood.
- To determine exacerbation and safety data on statins in preparation for a large phase III trial of statins in CF.
Study Endpoints
The primary endpoint will be the quantitative changes in serum levels of CRP.
Secondary endpoints will include:
- blood biomarkers IL6, LPS related proteins, LPS, LBP, sCD14 and EndoCAb, and pneumoproteins, SPD and CCL18; and molecules such as TNF-α and IL-1beta;
- changes in FEV1 over 12 weeks ; and
- exacerbations over 12 weeks
- Conditions
- Cystic Fibrosis, Systemic Inflammation
- Being tested
- Simvastatin, placebo
- Keywords
- cystic fibrosis, adult cystic fibrosis
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SimvastatinDrug
simvastatin 40 mg per day orally for 12 weeks.
placeboDrug
placebo 1 tablet once daily for 12 weeks
| Study group | Role | What they receive |
|---|---|---|
| Simvastatin 40 mg/d | Experimental | simvastatin 40 mg per day taken orally Receives: Drug: Simvastatin |
| Sugar pill | Placebo comparator | Not described Receives: Drug: placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
C-reactive protein
The difference in the change in plasma C-reactive protein concentrations from baseline to 12 weeks of treatment between those randomized to simvastatin 40 mg/d and those randomized to placebo
Measured: 12 weeks
Secondary outcomes (3)
Changes in forced expiratory volume in one second (FEV1)
The differences in the above parameters over 12 weeks between those assigned to simvastatin 40 mg/d and those assigned to placebo.
Measured: 12 weeks
Changes in exacerbation rates
The differences in the above parameters over 12 weeks between those assigned to simvastatin 40 mg/d and those assigned to placebo.
Measured: 12 weeks
Changes in blood pro-inflammatory markers such as IL-6, TNF, IL-1beta, LPS, LBP, sCD14, EndoCAB, SP-D, CCL-18)
The differences in the above parameters over 12 weeks between those assigned to simvastatin 40 mg/d and those assigned to placebo.
Measured: 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Mar 2010 (estimated)
- Study startMay 2010
- Primary completionMay 2010
- Study completionMay 2010
Why it stopped: Lack of funding
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.
- More trials run by University of British ColumbiaThe sponsor's other registered studies.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of British Columbia
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Paul Man, MD, Principal investigator, University of British Columbia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01092572
- Sponsor's protocol ID
- CF-Man-Statin-1
- Acronym
- CFStatin
- Status last verified
- Jan 2014
- First submitted
- 23 Mar 2010
- Last updated
- 30 Jan 2014
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Jan 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Effect of Simvastatin on Systemic Inflammation in Adult Cystic Fibrosis Subjects: A Pilot Study
- ClinicalTrials.gov ID
- NCT01092572
- Acronym / sponsor ID
- CFStatin / CF-Man-Statin-1
- Conditions
- Cystic Fibrosis, Systemic Inflammation
- Keywords
- cystic fibrosis, adult cystic fibrosis
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Lack of funding
- Enrolment
- 0 participants (actual)
- Lead sponsor
- University of British ColumbiaUniversity, hospital or other
- Study start
- May 2010
- Primary completion
- May 2010 (actual)
- Study completion
- May 2010 (actual)
- First posted
- 25 Mar 2010 (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 (CF) is a lethal genetic condition that affects 30,000 children and adults in the United States. Although CF management has improved substantially over the past two decades, there is still no cure and most patients with CF die before reaching their 50th birthday, largely due to lung failure. There is growing evidence that excess lung and blood inflammation that occurs in response to infections in the lungs cause CF patients to be sicker. Simvastatin is a drug that is used to lower cholesterol, but many researchers have found that this drug may also treat blood and lung inflammation. In this study, we will determine whether or not simvastatin can treat blood and lung inflammation in patients with CF and most importantly determine whether or not it can make these patients feel better and have better lung function.
Detailed description
Study Objectives
- To determine the effect of 12 weeks of 40 mg once daily simvastatin on general inflammatory molecules, IL-6 and CRP in the blood of CF patients.
- To determine the effect of simvastatin on LPS-related pathway molecules in the blood.
- To determine the effect of simvastatin on inflammatory pneumo-proteins in the blood.
- To determine exacerbation and safety data on statins in preparation for a large phase III trial of statins in CF.
Study Endpoints
The primary endpoint will be the quantitative changes in serum levels of CRP.
Secondary endpoints will include:
- blood biomarkers IL6, LPS related proteins, LPS, LBP, sCD14 and EndoCAb, and pneumoproteins, SPD and CCL18; and molecules such as TNF-α and IL-1beta;
- changes in FEV1 over 12 weeks ; and
- exacerbations over 12 weeks
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Simvastatin 40 mg/d | Experimental | Drug: Simvastatinsimvastatin 40 mg per day taken orally |
| Sugar pill | Placebo comparator | Drug: placebo |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Patients of provincial legal age of majority in British Columbia (≥19 years of age);
- Confirmed diagnosis of CF based on the following criteria:
- Ability to provide informed consent.
- Clinically stable at enrollment as assessed by the treating physician.
- Ability to comply with medication use, study visits and study procedures, such as spirometry, and venipunctures.
Key exclusion criteria (14 listed)
- Allergy or clinical reaction to simvastatin.
- The following abnormal lab values within the last six months or at screening:
- AST/ALT > 1.5 ULN, CK > 1.5 ULN, and eGFR < 40ml/min/1.73m2.
- Use of intravenous antibiotics or oral quinolones within 14 days of screening.
- With the exception of Azithromycin the use of oral antibiotics including prophylactic antibiotics (e.g., augmentin, tetracycline, cloxacillin, cephalosporins, trimethoprim/sulfamethoxazole) within 14 days of screening.
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 |
|---|---|---|
| Simvastatinsimvastatin 40 mg per day orally for 12 weeks. | Drug | Simvastatin 40 mg/d |
| placeboplacebo 1 tablet once daily for 12 weeks | Drug | Sugar pill |
Primary outcome
C-reactive protein
Time frame: 12 weeks
The difference in the change in plasma C-reactive protein concentrations from baseline to 12 weeks of treatment between those randomized to simvastatin 40 mg/d and those randomized to placebo
3 secondary outcomes
Changes in forced expiratory volume in one second (FEV1)
Time frame: 12 weeks
Changes in exacerbation rates
Time frame: 12 weeks
Changes in blood pro-inflammatory markers such as IL-6, TNF, IL-1beta, LPS, LBP, sCD14, EndoCAB, SP-D, CCL-18)
Time frame: 12 weeks
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Jan 2014.
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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 Man, MD principal investigator
University of British Columbia
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Simvastatin in people with Cystic Fibrosis and Systemic Inflammation, run by University of British Columbia.
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 7 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: withdrawn, as of the sponsor's last update on 30 Jan 2014. Status can change between updates, so confirm with the study contact before making plans.
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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 withdrawn before enrolling participants, so there are no results.
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?
C-reactive protein (time frame: 12 weeks). 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 30 Jan 2014.