Clinical trial NCT00850460: Quality of Life in Patients With Statin-Associated Myopathy
Official title: Quality of Life and Metabolic Alterations in Patients With Statin-Associated Myopathy
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE4
Conditions: Statin Adverse Reaction
Lead sponsor: Rockefeller University
Sponsor class: OTHER
Collaborators: name: Cornell University
name: Adelphi University
First posted: 2009-02-25
Enrollment: 14
Sex: ALL
Minimum age (years): 40
Maximum age (years): 60
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL

Brief 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. 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:

1. improved Individualized Neuromuscular Quality of Life (INQoL) and Short Form-36 (SF-36) scores (primary end point)
2. alleviation of muscle symptoms,
3. increased utilization of fatty acids as a fuel source reflected by the metabolic test results
4. decreased intramyocellular lipid (IMCL)
5. 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:

1. improved INQoL and SF-36 scores (primary end point)
2. alleviation of muscle symptoms,
3. increased utilization of fatty acids as a fuel source reflected by the metabolic test results
4. decreased IMCL
5. improved insulin sensitivity.

Eligibility criteria: Inclusion Criteria:

* 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

Exclusion Criteria:

* 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)
* 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

Primary outcomes:
measure: Individualized Neuromuscular Quality of Life (INQoL) Mean Scores From Week 0 to Week 8
timeFrame: Week 0 to Week 8
measure: Individualized Short Form-36 (SF-36) Mean Scores (Physical Component) From Week 0 to Week 8
timeFrame: Week 0 to Week 8
Secondary outcomes:
Other outcomes:
Locations:
city: New York
country: United States
facility: Rockefeller University
state: New York
Study officials:
affiliation: Rockefeller University
name: Patricia D Maningat, MD
role: PRINCIPAL_INVESTIGATOR
References: