A 52-Week Open-Label Extension and Safety Study of Pitavastatin in High-Risk Hyperlipidaemia in Childhood (NK-104-4.02EU).

Recruitment & Eligibility

Status: Completed

Sex: Not specified

Target Recruitment: 40

Inclusion Criteria

1. Male or female *6 years of age and <17 years of age at Visit 1;;2. Have fasting LDL-C levels *160 mg/dL (4.1 mmol/L) or LDL-C *130 mg/dL (3.4 mmol/L) if any of the following additional risk factors are present:;* Male;;* A family history of premature cardiovascular disease defined as a myocardial infarction before age 50 in a second-degree relative or before age 60 in a first-degree relative with at least 1 relative (parent, grandparent, or sibling) affected;;* Presence of low HDL-C (<45 mg/dL) or high TG (>150 mg/dL);;* Presence of high lipoprotein(a) (>75 nmol/L);;* Presence of type 2 diabetes mellitus diagnosed by treating physician according to current guidances; or;* Presence of hypertension defined as systolic and diastolic blood pressures above the 95th percentile for age and size;;3. Have not taken any lipid-lowering medications in the 5 weeks prior to screening or in the 4 weeks prior to the lipid qualifying visit at Week -1;;4. Have been adherent to an appropriate diet for at least 8 weeks;;5. Females who are post-menarche must not be pregnant or breast feeding and, if sexually active, must be using a reliable form of contraception; and;6. Written informed consent and assent (if necessary) obtained as required per local regulations.

Exclusion Criteria

1. Unable or unwilling to take study drug;;2. Fasting TG >400 mg/dL (4.5 mmol/L);;3. Homozygous familial hypercholesterolaemia;;4. Other secondary causes of hyperlipidaemia (eg, hypothyroidism, human immunodeficiency virus;infection, systemic lupus erythematosus, organ transplantation, previous malignancy, nephrotic;syndrome, glycogen storage disease);;5. Previous history of statin intolerance, adverse effects with other statin use, or hypersensitivity to any;components of the study drug;;6. Need for non-statin lipid-lowering medications;;7. Apheresis therapy;;8. Use of any concomitant medication which may interfere with the objectives of the study;;9. Type 1 diabetes mellitus;;10. Poorly controlled type 2 diabetes mellitus defined as haemoglobin A1c >9.0% at screening;;11. Severe renal impairment defined as serum creatinine >2.0 mg/dL at screening;;12. Uncontrolled hypertension;;13. Untreated thyroid disease;;14. Severe hepatic impairment, active liver disease, or persistent elevation of alanine transaminase or;aspartate transaminase >3 × the upper limit of normal (ULN);;15. Active muscle disease or creatine kinase >3 × ULN (unless explained by exercise);;16. Screening laboratory values within the following age/gender appropriate reference ranges as assessed;by the central laboratory:;* Haemoglobin <10 g/dL for males or <9 g/dL for females or;* Alkaline phosphatase >2 × ULN for age;;17. Any other laboratory abnormality that could compromise patient safety because of study;participation;;18. Malignancy during the past 5 years;;19. Current smoker or history of drug or alcohol abuse;;20. Hospitalisation for any cause within 30 days prior to the administration of study drug;;21. History of major surgery in the 3 months prior to screening;;22. Any medical condition which, in the judgment of the Investigator, would jeopardize the evaluation of;safety and/or constitute a significant safety risk to the patient; or;23. Participation in another clinical study involving an investigational drug during the course of this;study or within 30 days prior to signing the informed consent/assent form for this study.

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>The safety endpoints of this study are the following:<br /><br>- Adverse events;<br /><br>- Clinical laboratory parameters (including assessment of renal function and<br /><br>adrenal, gonadal, and pituitary hormones);<br /><br>- Vital signs;<br /><br>- Electrocardiogram (ECG) parameters; and<br /><br>- Physical examinations (including Tanner staging).</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>The efficacy endpoints of this study are the following:<br /><br>- Percent change in LDL-C from baseline over 52 weeks of treatment;<br /><br>- Percentages of patients who achieve AHA minimal (130 mg/dL [3.4 mmol/L]) and<br /><br>ideal (110 mg/dL [2.8 mmol/L]) LDL-C targets over 52 weeks of treatment;<br /><br>- Percent changes in HDL-C, non-high-density lipoprotein cholesterol<br /><br>(non-HDL-C), TC, TG, apolipoprotein A1 (Apo A1), and Apo B from baseline over<br /><br>52 weeks of treatment; and<br /><br>- Changes in TC:HDL-C ratio, non-HDL-C:HDL-C ratio, and Apo B:Apo A1 ratio from<br /><br>baseline over 52 weeks of treatment</p><br>