Reassessment of statin-associated muscle symptoms in adults with familial hypercholesterolemia

Trial details

Who runs it

Oslo University Hospital HF

Trial code

EU 2025-521589-83-00

Record updated

2026. május 22.

Original description

To determine the proportion of patients with statin-dependent muscle symptoms and nocebo/misattributed muscle symptoms among adults with familial hypercholesterolemia and self-perceived statin-associated muscle symptoms (SAMS).Read full original descriptionShow less

Reassessment of statin-associated musscle symptoms in adults with familial hypercholesterolemia: A phase IV randomized double-blinded n-of-1 crossover trial including periods with atorvastatin, placebo and no study treatment

To determine the proportion of patients with statin-dependent muscle symptoms and nocebo/misattributed muscle symptoms among adults with familial hypercholesterolemia and self-perceived statin-associated muscle symptoms (SAMS).

Inclusion: Participant must be ≥18 years of age at the time of signing the informed consent; Participant having genetically verified diagnosis of heterozygous familial hypercholesterolemia or clinical diagnosis of familial hypercholesterolemia with Dutch Lipid Clinic Network Score ≥9; Participant with a history of self-perceived statin-associated muscle symptoms (i.e. muscle symptoms subjectively associated with statin treatment) on at least two different types of statins; Participant having discontinued statin treatment due to associated muscle symptoms (must have been off statin treatment for at least 2 weeks prior to first assessment for study inclusion); If woman of childbearing potential (defined as all premenopausal female that are not permanently sterile); participant must take a pregnancy test with negative result and express willingness to highly effective contraceptive use or adequate sexual abstinence during the study intervention (until the end of intervention); Participant expected to be compliant with the requirements and restrictions listed in the informed consent form and in the protocol; Participant having access to a smartphone, tablet or personal computer throughout the study; Participant capable of giving signed informed consent.

Exclusion: Participant hospitalized for an unplanned atherosclerotic cardiovascular event the past 6 months prior to study; Participant currently using other drug(s) referred to as contraindicated during atorvastatin treatment, or not compatible with atorvastatin 80 mg, according to the Summary of Product Characteristics; Participant with short life expectancy (<24 months) due to other medical conditions; Participant with history of rhabdomyolysis; Participant with history of myopathy (creatine kinase ≥10 times upper limit of the normal range); Participant with history of liver affection (alanine aminotransferase and/or aspartate aminotransferase ≥3 times upper limit of the normal range) associated with statin treatment; Participant with history of severe renal insufficiency last 12 months (estimated glomerular filtration rate < 30 mL/min/1.73m2); Participant with any condition (e.g. psychiatric illness, dementia, substance abuse), that in the principal investigator’s opinion could put the subject at significant risk, confound the study results or interfere significantly with the subject participation in the study; Participant having any contraindication(s) for atorvastatin listed in the Summary of Product Characteristics (including known hypersensitivity to the ingredients, alanine aminotransferase ≥3 times upper limit of the normal range at baseline, pregnancy and breastfeeding); Participant with elevated CK ≥5 times upper limit of the normal range at baseline; Participation in another interventional clinical study.

EU CTIS 2025-521589-83-00