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

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Oslo University Hospital HF

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Cardiovascular Diseases [C14]

Decision date (initial)

2026-05-22

Transition trial

No

Low-intervention

Yes

Rare-disease indication

No

Vulnerable population

No

Funding sources

The Norwegian Regional Health Authority South-East

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Others

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).

Secondary objectives 1

1. To describe the diagnostic performance of a candidate biomarker (pattern of atorvastatin metabolites in blood) in context of distinguishing participants that are classified with statin-dependent muscle symptoms versus nocebo/misattributed symptoms.

Conditions and MedDRA coding

Familial hypercholesterolaemia

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 8

1. Participant must be ≥18 years of age at the time of signing the informed consent.
2. Participant having genetically verified diagnosis of heterozygous familial hypercholesterolemia or clinical diagnosis of familial hypercholesterolemia with Dutch Lipid Clinic Network Score ≥9.
3. 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.
4. 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).
5. 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).
6. Participant expected to be compliant with the requirements and restrictions listed in the informed consent form and in the protocol.
7. Participant having access to a smartphone, tablet or personal computer throughout the study.
8. Participant capable of giving signed informed consent.

Exclusion criteria 11

1. Participant hospitalized for an unplanned atherosclerotic cardiovascular event the past 6 months prior to study.
2. Participation in another interventional clinical study.
3. Participant with history of rhabdomyolysis.
4. Participant with history of myopathy (creatine kinase ≥10 times upper limit of the normal range)
5. Participant with history of liver affection (alanine aminotransferase and/or aspartate aminotransferase ≥3 times upper limit of the normal range) associated with statin treatment.
6. Participant with history of severe renal insufficiency last 12 months (estimated glomerular filtration rate < 30 mL/min/1.73m2).
7. 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.
8. 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).
9. 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.
10. Participant with short life expectancy (<24 months) due to other medical conditions.
11. Participant with elevated CK ≥5 times upper limit of the normal range at baseline.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. The individual mean difference in muscular symptom intensity over the last two weeks (week 5-6) between treatment periods with atorvastatin and placebo (at least 10 units and 25% difference on a 0-100 units rating scale).

Secondary endpoints 1

1. Area under the Receiver Operating Characteristic (ROC) curve, and diagnostic sensitivity and specificity.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Oslo University Hospital HF

Sponsor organisation

Oslo University Hospital HF

Address

Taarnbygget, Kirkeveien 166 Kirkeveien 166

City

Oslo

Postcode

0450

Country

Norway

Scientific contact point

Organisation

Oslo University Hospital HF

Contact name

Nils Tore Vethe

Public contact point

Organisation

Oslo University Hospital HF

Contact name

Nils Tore Vethe

Locations

1 EU/EEA country · 2 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 13 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications