EFFECT-FD: Effect of adding bezaFibrate to standard lipid lowering therapy on non-Fasting CholesTerol in patients with Familial
Dysbetalipoproteinemia

1. Presence of a genetically confirmed apolipoprotein E2 homozygote
genotype or an autosomal dominant FD genotype in combination with (at
least) one of the following clinical characteristics:

o (History of) presence of xanthoma;

o ApoB/TC ratio <0.15 or;

o Use of lipid-lowering medication.

2. Age >18 years (on the day of signing informed consent). Both males and
females will be included.

3. Women are postmenopausal and not receiving hormone therapy.

4. Any type of lipid lowering treatment, including lifestyle.

o Use of any type of fibrate (including but not limited to gemfibrozil,
bezafibrate, fenofibrate and ciprofibrate);

o Intolerance, known allergy or hypersensitivity to fibrate or any component of the medication;

o Unable to drink oral fatload;

o History of cholelithiasis or other biliary diseases;

o History of rhabdomyolysis;

o History of organ transplantation and/or immunosuppressive medication;

o Uncontrolled diabetes mellitus: HbA1c >69 mmol/mol;

o Untreated (sub)clinical hypothyroidism: defined as TSH ¡Ý5.0 mcIU/mL;

o Impaired renal function (estimated glomerular filtration rate (eGFR) <60
mL/min/1.73m2 based on MDRD equation at the screening visit), need of hemodialysis or other clinically significant renal disease;

o Active liver disease or impaired liver function (AST/ALT >1.5x ULN);

o Creatinine kinase (CK) >3 x ULN;

o Poorly controlled blood pressure with systolic blood pressure >180mmHg or diastolic blood pressure >100mmHg at the screening visit;

o Active malignancy (¡Ü 2 year prior to informed consent);

o Human Immunodeficiency Virus (HIV) or AIDS;

o Celiac disease or other disorder associated with significant intestinal
malabsorption;

o Alcohol abuse/excessive alcohol use, defined as >14 alcoholic consumptions per week.

o Use of anti-tuberculosis medication;

o Use of anti-epileptics;

o Use of oral anticoagulants;

o Use of monoamine oxidase (MAO) inhibitors (antidepressant);

o Use of cytochrome P450 3A4 (CYP3A4) inhibitors: oral antimycotics (fluconazol, itraconazol, ketoconazole, voriconazol, posaconazol), ciclosporin, grapefruit juice, mycines (azithromycin, clarithromycin, erythromycin), diltiazem, verapamil and amiodaron;

o Galactose-intolerance, Lapp-lactose deficiency or glucose-galactose
malabsorption.

o Current participation or participation in a study with an investigational compound or device within 30 days of signing informed consent.