Statin Adverse ReactionPhase 4NCT00850460
Quality of Life in Patients With Statin-Associated Myopathy
A Phase 4 study testing Statins in people with Statin Adverse Reaction, run by Rockefeller University at 1 site in United States.
Trial summary
The proposed study will focus on possible effects of statins on muscle strength and why they become tired more easily, quality of life, and measurements to understand why muscles are not able to fully utilize fats. Read more about this study
- Who is it for?
- Ages 40 to 60 years
- Conditions the study is for
- Statin Adverse Reaction
- What is being tested?
- Statins
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 14 participants
- Where
- 1 siteUnited States
- Study dates
- Feb 2009to Feb 2013
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 40 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- males and females 30-60 yrs old
- experiencing muscle pain, weakness, numbness or cramping that they perceive to interfere with activities of daily living (ADLs), but able to ambulate independently (in order to perform exercise tests)
- muscle symptoms started/ occurred within one year of starting statin treatment or within one year of changing statin brand or dose adjustment
- currently taking a statin (has been taking medications ≥ 80% of the time or at least 5 days/week)
- ≤ 15% probability of having a cardiovascular (CV) event in the next 10 years calculated using an online CV risk calculator (while on current statin) for the questionnaire portion; AND with a low or a moderate American College of Sports Medicine (ACSM) risk stratification for a cardiovascular event during a treadmill test for the full metabolic study
- must agree to have a letter sent to inform the health care provider who prescribed the statin of study participation except for subjects referred by Metropolitan Hospital physicians
Who cannot join
- concomitant treatment with other lipid-lowering agents
- impaired liver or kidney function ( alanine aminotransferase (ALT) or asparate aminotransferase (AST) ≥ 3x upper limit of normal, creatinine ≥ 3x or creatine phosphokinase (CPK) ≥ 5x upper limit of normal)
- untreated hypo or hyperthyroidism
- current treatment with other medications known to increase risk of myopathy (e.g. cyclosporine, azithromycin, erythromycin and other macrolide antibiotics, azole antifungals, fusidic acid, digoxin)
- documented history of muscle disorder or myopathy other than statin-associated myopathy
- anemia (Hb< 110 g/dL)
Show all 27Show fewer
- cancer within 5 years of enrollment except basal or squamous cell carcinoma (CA) of the skin
- diabetes
- HIV-1 infection
- Uncontrolled blood pressure ≥ 160/100
- known coronary artery disease or peripheral vascular disease
- chronic illnesses such as lupus, rheumatoid arthritis, psoriasis
- any condition, that at the investigators' discretion would impact/ bias the study data
- long term oral, nasal, or inhaler steroid use > 6 months
- on Hormone Therapy except for thyroid replacement
- alcohol consumption ≥ 40 g/day (3 glasses/day wine or beers or binge drinking ≥ 4 glasses/night)
- engaged in significant amounts of sport or strenuous leisure activity (> 60 min four times per week)
- surgery in the past 6 months except for minor excision/incision procedures,
- 12-L electrocardiogram demonstrating old/new myocardial infarction/ ischemia or other findings that, at the cardiologist's discretion, may put the subject at high risk
- cognitive impairment that prevents comprehension of questionnaires
- inability to read English (questionnaire language)
- Exclusions for the metabolic study:
- currently taking beta blockers
- body mass index > 28 kg/m2
- premenopausal females < 50 yrs (menopause defined as 12 consecutive months without menstruation (in order to avoid the confounding effect of the menstrual cycle phase on fuel selection)
- physical disability or previous injury that prevents safe exercise testing
- do not meet the magnetic resonance spectroscopy (MRS) prescreening criteria
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Quality of Life and Metabolic Alterations in Patients With Statin-Associated Myopathy
The proposed study will focus on possible effects of statins on muscle strength and why they become tired more easily, quality of life, and measurements to understand why muscles are not able to fully utilize fats. The investigators are specifically interested in statin users and the impact of muscle symptoms on daily activities and quality of life.
This study hypothesize that patients with likely statin-associated myopathy have a metabolic dysregulation in fuel utilization such that compared to patients continuing statins, those on placebo will show:
- improved Individualized Neuromuscular Quality of Life (INQoL) and Short Form-36 (SF-36) scores (primary end point)
- alleviation of muscle symptoms,
- increased utilization of fatty acids as a fuel source reflected by the metabolic test results
- decreased intramyocellular lipid (IMCL)
- improved insulin sensitivity.
Read the detailed description
The proposed study will focus on possible effects of statins on muscle strength and quality of life, and measurements to understand why muscles of statin users are not able to fully utilize fats. The investigators are specifically interested in statin users and the impact of muscle symptoms on daily activities and quality of life.
This study hypothesizes that patients with likely statin-associated myopathy have a metabolic dysregulation in fuel utilization such that compared to patients continuing statins, those on placebo will show:
- improved INQoL and SF-36 scores (primary end point)
- alleviation of muscle symptoms,
- increased utilization of fatty acids as a fuel source reflected by the metabolic test results
- decreased IMCL
- improved insulin sensitivity.
- Conditions
- Statin Adverse Reaction
- Being tested
- Statins, Placebo
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
StatinsDrug
Also called atorvastatin, simvastatin, pravastatin, rosuvastatin
Subjects will be randomized to continue their statin dosage or placebo for 8 weeks. They will stay on the same dosage as prescribed by their physician. Usual dosage for atorvastatin 10-80 mg/tab once daily by mouth; simvastatin 20-80 mg/tab once daily by mouth; pravastatin 10-80 mg/tab once daily by mouth; rosuvastatin 5-20 mg/tab once daily by mouth.
PlaceboDrug
Also called lactose pills
Placebo pills will consist of lactose and will be given one capsule once daily
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Lactose placebo pill Receives: Drug: Placebo |
| Statins | Active comparator | Statin medications Receives: Drug: Statins |
What is being measured
The primary outcome is the main question the study is designed to answer.
Individualized Neuromuscular Quality of Life (INQoL) Mean Scores From Week 0 to Week 8
Scores from the self-administered INQoL questionnaire will be compared at the start of the study (Week 0) and at the end (Week 8) between the statin-treated group and the placebo group. Scores range from 0-100, with 100 being a better outcome. Measures reported are the means of Week 0 and week 8, measures of dispersion is the range of the results (3 per group).
Measured: Week 0 to Week 8
Individualized Short Form-36 (SF-36) Mean Scores (Physical Component) From Week 0 to Week 8
Scores from the self-administered SF-36 (Physical component) questionnaire were measured at the start (Week 0) of the study and at the end (Week 8) among patients in the placebo- and statin-treated group. Mean scores range from 0 (minimum) - 100 (maximum) with higher mean scores reflecting better outcomes. Measures reported are the means of Week 0 and week 8, measures of dispersion is the range of scores.
Measured: Week 0 to Week 8
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Feb 2009 (estimated)
- Study startFeb 2009
- Primary completionFeb 2013
- Study completionFeb 2013
Why it stopped: Investigator left institution and no PI has been found to continue the study
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Statin Adverse ReactionStudies 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 4
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- Rockefeller University
- Sponsor type
- University, hospital or other
- Collaborators
- Cornell University, Adelphi University
- Responsible party
- Sponsor
- Investigators
- Patricia D Maningat, MD, Principal investigator, Rockefeller University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00850460
- Sponsor's protocol ID
- PAM-0655
- Status last verified
- Sep 2016
- First submitted
- 23 Feb 2009
- Last updated
- 7 Nov 2016
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Nov 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Quality of Life and Metabolic Alterations in Patients With Statin-Associated Myopathy
- ClinicalTrials.gov ID
- NCT00850460
- Acronym / sponsor ID
- PAM-0655
- Conditions
- Statin Adverse Reaction
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Investigator left institution and no PI has been found to continue the study
- Enrolment
- 14 participants (actual)
- Lead sponsor
- Rockefeller UniversityUniversity, hospital or other
- Collaborators
- Cornell University, Adelphi University
- Study start
- Feb 2009
- Primary completion
- Feb 2013 (actual)
- Study completion
- Feb 2013 (actual)
- First posted
- 25 Feb 2009 (estimated)
- Results
- Posted 7 Nov 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The proposed study will focus on possible effects of statins on muscle strength and why they become tired more easily, quality of life, and measurements to understand why muscles are not able to fully utilize fats. The investigators are specifically interested in statin users and the impact of muscle symptoms on daily activities and quality of life.
This study hypothesize that patients with likely statin-associated myopathy have a metabolic dysregulation in fuel utilization such that compared to patients continuing statins, those on placebo will show:
- improved Individualized Neuromuscular Quality of Life (INQoL) and Short Form-36 (SF-36) scores (primary end point)
- alleviation of muscle symptoms,
- increased utilization of fatty acids as a fuel source reflected by the metabolic test results
- decreased intramyocellular lipid (IMCL)
- improved insulin sensitivity.
Detailed description
The proposed study will focus on possible effects of statins on muscle strength and quality of life, and measurements to understand why muscles of statin users are not able to fully utilize fats. The investigators are specifically interested in statin users and the impact of muscle symptoms on daily activities and quality of life.
This study hypothesizes that patients with likely statin-associated myopathy have a metabolic dysregulation in fuel utilization such that compared to patients continuing statins, those on placebo will show:
- improved INQoL and SF-36 scores (primary end point)
- alleviation of muscle symptoms,
- increased utilization of fatty acids as a fuel source reflected by the metabolic test results
- decreased IMCL
- improved insulin sensitivity.
Study design
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: PlaceboLactose placebo pill |
| Statins | Active comparator | Drug: StatinsStatin medications |
Who may qualify
- Ages
- 40 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (6 listed)
- males and females 30-60 yrs old
- experiencing muscle pain, weakness, numbness or cramping that they perceive to interfere with activities of daily living (ADLs), but able to ambulate independently (in order to perform exercise tests)
- muscle symptoms started/ occurred within one year of starting statin treatment or within one year of changing statin brand or dose adjustment
- currently taking a statin (has been taking medications ≥ 80% of the time or at least 5 days/week)
- ≤ 15% probability of having a cardiovascular (CV) event in the next 10 years calculated using an online CV risk calculator (while on current statin) for the questionnaire portion; AND with a low or a moderate American College of Sports Medicine (ACSM) risk stratification for a cardiovascular event during a treadmill test for the full metabolic study
Key exclusion criteria (27 listed)
- concomitant treatment with other lipid-lowering agents
- impaired liver or kidney function ( alanine aminotransferase (ALT) or asparate aminotransferase (AST) ≥ 3x upper limit of normal, creatinine ≥ 3x or creatine phosphokinase (CPK) ≥ 5x upper limit of normal)
- untreated hypo or hyperthyroidism
- current treatment with other medications known to increase risk of myopathy (e.g. cyclosporine, azithromycin, erythromycin and other macrolide antibiotics, azole antifungals, fusidic acid, digoxin)
- documented history of muscle disorder or myopathy other than statin-associated myopathy
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 |
|---|---|---|
| StatinsAlso: atorvastatin, simvastatin, pravastatin, rosuvastatinSubjects will be randomized to continue their statin dosage or placebo for 8 weeks. They will stay on the same dosage as prescribed by their physician. Usual dosage for atorvastatin 10-80 mg/tab once daily by mouth; simvastatin 20-80 mg/tab once daily by mouth; pravastatin 10-80 mg/tab once daily by mouth; rosuvastatin 5-20 mg/tab once daily by mouth. | Drug | Statins |
| PlaceboAlso: lactose pillsPlacebo pills will consist of lactose and will be given one capsule once daily | Drug | Placebo |
Primary outcome
Individualized Neuromuscular Quality of Life (INQoL) Mean Scores From Week 0 to Week 8
Time frame: Week 0 to Week 8
Scores from the self-administered INQoL questionnaire will be compared at the start of the study (Week 0) and at the end (Week 8) between the statin-treated group and the placebo group. Scores range from 0-100, with 100 being a better outcome. Measures reported are the means of Week 0 and week 8, measures of dispersion is the range of the results (3 per group).
Individualized Short Form-36 (SF-36) Mean Scores (Physical Component) From Week 0 to Week 8
Time frame: Week 0 to Week 8
Scores from the self-administered SF-36 (Physical component) questionnaire were measured at the start (Week 0) of the study and at the end (Week 8) among patients in the placebo- and statin-treated group. Mean scores range from 0 (minimum) - 100 (maximum) with higher mean scores reflecting better outcomes. Measures reported are the means of Week 0 and week 8, measures of dispersion is the range of scores.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Nov 2016.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Patricia D Maningat, MD principal investigator
Rockefeller University
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 Statins in people with Statin Adverse Reaction, run by Rockefeller University at 1 site in United States.
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 6 inclusion and 27 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 7 Nov 2016. 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 States. 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?
Yes. Results are posted in the registry (first posted 7 Nov 2016). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Individualized Neuromuscular Quality of Life (INQoL) Mean Scores From Week 0 to Week 8 (time frame: Week 0 to Week 8). The study lists 2 primary outcomes in all. 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 7 Nov 2016.