BRONCHIECTASISPhase 4NCT01299194
Atorvastatin in Bronchiectasis in Patients With Pseudomonas Aeruginosa
A Phase 4 study testing ATORVASTATIN in people with BRONCHIECTASIS, run by University of Edinburgh at 1 site in United Kingdom.
Trial summary
Statins are a class of drug used to prevent heart attacks and strokes by lowering blood cholesterol levels. They have also been found to have a beneficial "side effect" of lowering the level of inflammation in the body. This is thought to be one of the reasons they are effective in treating heart attacks and strokes. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- BRONCHIECTASIS
- What is being tested?
- ATORVASTATIN
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 32 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- Nov 2010to May 2017
- Phase
- Phase 4
- 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 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients aged 18-80 will be recruited.
- All will have an established radiological diagnosis of bronchiectasis (CT of the chest).
- Patients colonised with Pseudomonas Aeruginosa.
- Able to give informed consent.
Who cannot join
- Current smokers or ex-smokers of less than 1 year; >15 pack year history
- Cystic fibrosis
- Active allergic bronchopulmonary aspergillosis
- Active tuberculosis
- Poorly controlled asthma
- Pregnancy or breast feeding
Show all 11Show fewer
- Known allergy to statins
- Active malignancy
- Chronic liver disease
- Established cardiovascular or cerebrovascular disease
- Statin use in the last year
Trial locations and contacts
1 site in 1 country.
United Kingdom1 site
Royal Infirmary of Edinburgh
Edinburgh, Scotland, EH16 4SA
About this study
Official title: A Randomised Controlled Trial of Atorvastatin as an Anti-Inflammatory Agent in Non-Cystic Fibrosis Bronchiectasis in Patients With Pseudomonas Aeruginosa
Statins are a class of drug used to prevent heart attacks and strokes by lowering blood cholesterol levels. They have also been found to have a beneficial "side effect" of lowering the level of inflammation in the body. This is thought to be one of the reasons they are effective in treating heart attacks and strokes. Laboratory experiments have shown that statins reduce lung inflammation in response to bacteria and this is a promising development for the treatment of chest infections.
Bronchiectasis is a chronic disabling lung disease characterised by chronic sputum production and recurrent chest infections. 2/3 of patients are chronically colonised with bacteria (normally the lungs are sterile) and this leads inflammation in the lung and in the rest of the body.
Read more
There are no effective treatments for bronchiectasis other than antibiotics for chest infections. With increasing antibiotic use, there is increasing antibiotic resistance and new treatments for this disease are needed.
The investigators intend to study Atorvastatin in patients with bronchiectasis with colonization with pseudomonas aeruginosa. The investigators will give Atorvastatin to 16 patients with this disease while 16 patients will receive placebo. This will be a crossover study where patients will receive atorvastatin or placebo for 3 months, followed by a statin wash out period of 6 weeks. Thereafter the groups will cross over and the group receiving atorvastatin will now receive placebo and those receiving placebo will receive atorvastatin for 3 months. The investigators will measure inflammation in their lungs and in the rest of their body before and after treatment with atorvastatin. The investigators will also assess their quality of life and number of chest infections over a 7.5 month period.
This pilot study will determine if there is any role for statins are an anti-inflammatory agent in patients with bronchiectasis.
Read the detailed description
BACKGROUND AND RATIONAL FOR STUDY Bronchiectasis is a chronic debilitating respiratory condition. Patients suffer daily cough, excess sputum production and recurrent chest infections because of inflamed and permanently damaged airways. It is a common with a Scottish incidence of 1 in 1,000 to 1 in 10,000. Over 600 patients in Edinburgh are monitored in secondary care. They frequently utilise primary and secondary care resources through consultations, A\&E attendances and inpatient admissions. The economic burden is huge- hospital admissions alone for bronchiectasis cost NHS Lothian just over 1 million pounds alone last year.
LIMITATIONS OF TREATMENT There are few evidence based long term treatments currently available. Long term antibiotics are a feasible option, but with the increasing problems of antimicrobial resistance and side effects, in particular Clostridium difficile and methicillin resistant Staphylococcus aureus (MRSA), there is an international drive to reduce antibiotic usage. There is an urgent need for novel non antibiotic treatments.
Statins as a potential new non antibiotic treatment in bronchiectasis Excessive neutrophilic airways inflammation is the central feature of bronchiectasis. This paradoxically both promotes bacterial colonisation and perpetuates damage to the airways creating a vicious cycle of bacterial colonisation and inflammation.1-3
Statins have been shown to have powerful anti-inflammatory effects.4-6 In animal models, statins can reduce neutrophil recruitment to the inflamed lung and reduce protease activity.7 Statin treatment has been shown to reduce epithelial cell adherence and invasion by Streptococcus pneumoniae in-vitro suggesting a role for statins in preventing bacterial colonisation.8 In healthy controls exposed to lipopolysaccharide to induce acute lung inflammation, pre-treatment with simvastatin reduced neutrophil accumulation in the lung and inhibited production of myeloperoxidase, tumour necrosis factor-alpha, matrix metalloproteinases and C-reactive protein.9 There was also an increase in neutrophil apoptosis, suggesting that statins may aid the resolution of inflammation in the airway.10
STUDY HYPOTHESIS We hypothesise that long term statin treatment will improve patients' symptoms through its anti-inflammatory effect. The beneficial effects on patient symptoms (cough, sputum volume, bacterial load, airway function, exercise tolerance, exacerbation frequency and health related quality of life) will be consequent on reduced neutrophilic airways inflammation.
Planned study
- This is a randomised double blind placebo controlled cross over trial to assess the efficacy of atorvastatin therapy in patients with clinically significant bronchiectasis.
- No such study has previously been undertaken (PUBMED Search "statins" and "bronchiectasis" 18 March 2010- no relevant articles).
- This is a unique proof of principle study assessing a new non antibiotic treatment that could benefit all patients with clinically significant bronchiectasis, without the side effect profile of long term antibiotics.
- Following this proof of principle study, we aim to design a large multi-centred study assessing long term statins as a new treatment.
- Stockley RA et al. Elastolytic activity of sputum and its relation to purulence and to lung function in patients with bronchiectasis. Thorax 1984;39(6):408-413.
- Hill AT et al. Association between airway bacterial load and markers of airway inflammation in patients with stable chronic bronchitis. Am J Med 2000;109(4):288-95.
- Inflammation: a two-edged sword-the model of bronchiectasis. Cole PJ. Eur J Respir Dis Suppl. 1986;147:6-15.
- Ridker PM et al. C-reactive protein levels and outcomes after statin therapy. N Engl J Med 2005;352:20-8.
- Terblanche M et al. Statins and Sepsis: multiple modifications at multiple levels. Lancet Infect Dis. 2007; 7(5):358-368.
- Vaughan CJ, Murphy MB, Buckley BM. Statins do more than just lower cholesterol. Lancet 1996;348:1079-82.
- Fessler MB et al. A role for HMG coenzyme A reductase in pulmonary inflammation and host defense. Am J Respir Crit Care Med 2005;171:606-15.
- Rosch JW et al. Statins protect against fulminant pneumococcal infection and cytolysin toxicity in a mouse model of sickle cell disease. J Clin Invest 2010; 120(2);627-35.
- Shyamsundar M et al. Simvastatin decreases lipopolysacchraide-induced pulmonary inflammation in healthy volunteers. Am J Respir Crit Care Med. 2009 179:1107-1114.
- Watt AP et al. Neutrophil apoptosis, proinflammatory mediators and cell counts in bronchiectasis. Thorax 2004;59(3):231-6.
- Conditions
- BRONCHIECTASIS
- Being tested
- ATORVASTATIN
- Keywords
- BRONCHIECTASIS, PSEUDOMONAS AERUGINOSA, STATIN, OUTCOME
- Medical subject headings
- Bronchiectasis, Pseudomonas Infections
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ATORVASTATINDrug
80mg once daily for 3 months
| Study group | Role | What they receive |
|---|---|---|
| ATORVASTATIN | Active comparator | Atorvastatin 80mg once daily for 3 months, 1.5 month wash out, then Placebo for 3 months Receives: Drug: ATORVASTATIN |
| PLACEBO | Placebo comparator | Placebo 3 months, then washout for 1.5 months, then Atorvastatin 80mg once daily Receives: Drug: ATORVASTATIN |
What is being measured
The primary outcome is the main question the study is designed to answer.
The primary endpoint of this study is a reduction in cough at 3 months compared to baseline as measured by the Leicester Cough Questionnaire score.
Measured: 7.5 months
Secondary outcomes (7)
pulmonary physiology and assessment of exercise capacity
Measured: 7.5 months
24 hour sputum volume
Measured: 7.5 months
qualitative and quantitative bacteriology
Measured: 7.5 months
health related quality of life and health care utilisation
Measured: 7.5 months
exacerbation frequency
Measured: 7.5 months
safety of statin therapy
Measured: 7.5 months
Airways and systemic inflammation
Measured: 7.5 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Feb 2011 (estimated)
- Study startNov 2010
- Primary completionJun 2015
- Study completionMay 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for BRONCHIECTASISStudies of the same condition, open and under way.
- More trials run by University of EdinburghThe 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 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 32 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Edinburgh
- Sponsor type
- University, hospital or other
- Collaborators
- NHS Lothian
- Responsible party
- Sponsor
- Investigators
- Adam T Hill, MBChB MD, Principal investigator, NHS Lothian
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01299194
- Sponsor's protocol ID
- 2010-022042-24
- Status last verified
- May 2017
- First submitted
- 21 Oct 2010
- Last updated
- 3 May 2017
- Results posted
- No
- Data monitoring committee
- No
Publications
Bedi P, Chalmers JD, Graham C, Clarke A, Donaldson S, Doherty C, Govan JRW, Davidson DJ, Rossi AG, Hill AT. A Randomized Controlled Trial of Atorvastatin in Patients With Bronchiectasis Infected With Pseudomonas Aeruginosa: A Proof of Concept Study. Chest. 2017 Aug;152(2):368-378. doi: 10.1016/j.chest.2017.05.017. Epub 2017 May 26.
PubMed 28554732
Study at a glance
From the registry record, last updated by the sponsor on 3 May 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomised Controlled Trial of Atorvastatin as an Anti-Inflammatory Agent in Non-Cystic Fibrosis Bronchiectasis in Patients With Pseudomonas Aeruginosa
- ClinicalTrials.gov ID
- NCT01299194
- Acronym / sponsor ID
- 2010-022042-24
- Conditions
- BRONCHIECTASIS
- Keywords
- BRONCHIECTASIS, PSEUDOMONAS AERUGINOSA, STATIN, OUTCOME
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 32 participants (actual)
- Lead sponsor
- University of EdinburghUniversity, hospital or other
- Collaborators
- NHS Lothian
- Study start
- Nov 2010
- Primary completion
- Jun 2015 (actual)
- Study completion
- May 2017 (actual)
- First posted
- 18 Feb 2011 (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.
Statins are a class of drug used to prevent heart attacks and strokes by lowering blood cholesterol levels. They have also been found to have a beneficial "side effect" of lowering the level of inflammation in the body. This is thought to be one of the reasons they are effective in treating heart attacks and strokes. Laboratory experiments have shown that statins reduce lung inflammation in response to bacteria and this is a promising development for the treatment of chest infections.
Bronchiectasis is a chronic disabling lung disease characterised by chronic sputum production and recurrent chest infections. 2/3 of patients are chronically colonised with bacteria (normally the lungs are sterile) and this leads inflammation in the lung and in the rest of the body.
There are no effective treatments for bronchiectasis other than antibiotics for chest infections. With increasing antibiotic use, there is increasing antibiotic resistance and new treatments for this disease are needed.
The investigators intend to study Atorvastatin in patients with bronchiectasis with colonization with pseudomonas aeruginosa. The investigators will give Atorvastatin to 16 patients with this disease while 16 patients will receive placebo. This will be a crossover study where patients will receive atorvastatin or placebo for 3 months, followed by a statin wash out period of 6 weeks. Thereafter the groups will cross over and the group receiving atorvastatin will now receive placebo and those receiving placebo will receive atorvastatin for 3 months. The investigators will measure inflammation in their lungs and in the rest of their body before and after treatment with atorvastatin. The investigators will also assess their quality of life and number of chest infections over a 7.5 month period.
This pilot study will determine if there is any role for statins are an anti-inflammatory agent in patients with bronchiectasis.
Detailed description
BACKGROUND AND RATIONAL FOR STUDY Bronchiectasis is a chronic debilitating respiratory condition. Patients suffer daily cough, excess sputum production and recurrent chest infections because of inflamed and permanently damaged airways. It is a common with a Scottish incidence of 1 in 1,000 to 1 in 10,000. Over 600 patients in Edinburgh are monitored in secondary care. They frequently utilise primary and secondary care resources through consultations, A\&E attendances and inpatient admissions. The economic burden is huge- hospital admissions alone for bronchiectasis cost NHS Lothian just over 1 million pounds alone last year.
LIMITATIONS OF TREATMENT There are few evidence based long term treatments currently available. Long term antibiotics are a feasible option, but with the increasing problems of antimicrobial resistance and side effects, in particular Clostridium difficile and methicillin resistant Staphylococcus aureus (MRSA), there is an international drive to reduce antibiotic usage. There is an urgent need for novel non antibiotic treatments.
Statins as a potential new non antibiotic treatment in bronchiectasis Excessive neutrophilic airways inflammation is the central feature of bronchiectasis. This paradoxically both promotes bacterial colonisation and perpetuates damage to the airways creating a vicious cycle of bacterial colonisation and inflammation.1-3
Statins have been shown to have powerful anti-inflammatory effects.4-6 In animal models, statins can reduce neutrophil recruitment to the inflamed lung and reduce protease activity.7 Statin treatment has been shown to reduce epithelial cell adherence and invasion by Streptococcus pneumoniae in-vitro suggesting a role for statins in preventing bacterial colonisation.8 In healthy controls exposed to lipopolysaccharide to induce acute lung inflammation, pre-treatment with simvastatin reduced neutrophil accumulation in the lung and inhibited production of myeloperoxidase, tumour necrosis factor-alpha, matrix metalloproteinases and C-reactive protein.9 There was also an increase in neutrophil apoptosis, suggesting that statins may aid the resolution of inflammation in the airway.10
STUDY HYPOTHESIS We hypothesise that long term statin treatment will improve patients' symptoms through its anti-inflammatory effect. The beneficial effects on patient symptoms (cough, sputum volume, bacterial load, airway function, exercise tolerance, exacerbation frequency and health related quality of life) will be consequent on reduced neutrophilic airways inflammation.
Planned study
- This is a randomised double blind placebo controlled cross over trial to assess the efficacy of atorvastatin therapy in patients with clinically significant bronchiectasis.
- No such study has previously been undertaken (PUBMED Search "statins" and "bronchiectasis" 18 March 2010- no relevant articles).
- This is a unique proof of principle study assessing a new non antibiotic treatment that could benefit all patients with clinically significant bronchiectasis, without the side effect profile of long term antibiotics.
- Following this proof of principle study, we aim to design a large multi-centred study assessing long term statins as a new treatment.
- Stockley RA et al. Elastolytic activity of sputum and its relation to purulence and to lung function in patients with bronchiectasis. Thorax 1984;39(6):408-413.
- Hill AT et al. Association between airway bacterial load and markers of airway inflammation in patients with stable chronic bronchitis. Am J Med 2000;109(4):288-95.
- Inflammation: a two-edged sword-the model of bronchiectasis. Cole PJ. Eur J Respir Dis Suppl. 1986;147:6-15.
- Ridker PM et al. C-reactive protein levels and outcomes after statin therapy. N Engl J Med 2005;352:20-8.
- Terblanche M et al. Statins and Sepsis: multiple modifications at multiple levels. Lancet Infect Dis. 2007; 7(5):358-368.
- Vaughan CJ, Murphy MB, Buckley BM. Statins do more than just lower cholesterol. Lancet 1996;348:1079-82.
- Fessler MB et al. A role for HMG coenzyme A reductase in pulmonary inflammation and host defense. Am J Respir Crit Care Med 2005;171:606-15.
- Rosch JW et al. Statins protect against fulminant pneumococcal infection and cytolysin toxicity in a mouse model of sickle cell disease. J Clin Invest 2010; 120(2);627-35.
- Shyamsundar M et al. Simvastatin decreases lipopolysacchraide-induced pulmonary inflammation in healthy volunteers. Am J Respir Crit Care Med. 2009 179:1107-1114.
- Watt AP et al. Neutrophil apoptosis, proinflammatory mediators and cell counts in bronchiectasis. Thorax 2004;59(3):231-6.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| ATORVASTATIN | Active comparator | Drug: ATORVASTATINAtorvastatin 80mg once daily for 3 months, 1.5 month wash out, then Placebo for 3 months |
| PLACEBO | Placebo comparator | Drug: ATORVASTATINPlacebo 3 months, then washout for 1.5 months, then Atorvastatin 80mg once daily |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Patients aged 18-80 will be recruited.
- All will have an established radiological diagnosis of bronchiectasis (CT of the chest).
- Patients colonised with Pseudomonas Aeruginosa.
- Able to give informed consent.
Key exclusion criteria (11 listed)
- Current smokers or ex-smokers of less than 1 year; >15 pack year history
- Cystic fibrosis
- Active allergic bronchopulmonary aspergillosis
- Active tuberculosis
- Poorly controlled asthma
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 |
|---|---|---|
| ATORVASTATIN80mg once daily for 3 months | Drug | ATORVASTATIN, PLACEBO |
Primary outcome
The primary endpoint of this study is a reduction in cough at 3 months compared to baseline as measured by the Leicester Cough Questionnaire score.
Time frame: 7.5 months
7 secondary outcomes
pulmonary physiology and assessment of exercise capacity
Time frame: 7.5 months
24 hour sputum volume
Time frame: 7.5 months
qualitative and quantitative bacteriology
Time frame: 7.5 months
health related quality of life and health care utilisation
Time frame: 7.5 months
exacerbation frequency
Time frame: 7.5 months
safety of statin therapy
Time frame: 7.5 months
Airways and systemic inflammation
Time frame: 7.5 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 May 2017.
- Derived from this study: PubMed 28554732Bedi P, Chalmers JD, Graham C, Clarke A, Donaldson S, Doherty C, Govan JRW, Davidson DJ, Rossi AG, Hill AT. A Randomized Controlled Trial of Atorvastatin in Patients With Bronchiectasis Infected With Pseudomonas Aeruginosa: A Proof of Concept Study. Chest. 2017 Aug;152(2):368-378. doi: 10.1016/j.chest.2017.05.017. Epub 2017 May 26.
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Bronchiectasis
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.
Adam T Hill, MBChB MD principal investigator
NHS Lothian
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing ATORVASTATIN in people with BRONCHIECTASIS, run by University of Edinburgh at 1 site in United Kingdom.
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 4 inclusion and 11 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: finished, as of the sponsor's last update on 3 May 2017. 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 United Kingdom. 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
The primary endpoint of this study is a reduction in cough at 3 months compared to baseline as measured by the Leicester Cough Questionnaire score. (time frame: 7.5 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01299194, which also lists 1 linked publication.
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 3 May 2017.