Quality of Life in Patients With Statin-Associated Myopathy

NCT ID: NCT00850460

Last Updated: 2016-11-07

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE4
• Total Enrollment: 14 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2009-02-28
• Study Completion Date: 2013-02-28

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.

Conditions

Statin Adverse Reaction

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Blinding Strategy: TRIPLE

Study Groups

Placebo

Lactose placebo pill

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Placebo pills will consist of lactose and will be given one capsule once daily

Statins

Statin medications

Group Type: ACTIVE_COMPARATOR

Statins

Intervention Type: DRUG

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.

Interventions

Statins

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.

Intervention Type: DRUG

Placebo

Placebo pills will consist of lactose and will be given one capsule once daily

Intervention Type: DRUG

Other Intervention Names

atorvastatin, simvastatin, pravastatin, rosuvastatin; lactose pills

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

• Minimum Eligible Age: 40 Years
• Maximum Eligible Age: 60 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Cornell University

OTHER

Sponsor Role: collaborator

Adelphi University

OTHER

Sponsor Role: collaborator

Rockefeller University

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Patricia D Maningat, MD

Role: PRINCIPAL_INVESTIGATOR

Rockefeller University

Locations

Rockefeller University

New York, New York, United States

Countries

United States

Other Identifiers

PAM-0655

Identifier Type: -

Identifier Source: org_study_id