A Double-Blind, Randomised, Placebo-Controlled, Parallel-Group, 12-Week Study of Pitavastatin in High-Risk Hyperlipidaemia in Childhood P/266/2011, P267/2011, P268/2011

Phase 3

Completed

• Conditions: hyperlipidemie; high cholesterol; Hyperlipidaemia

• Registration Number: NL-OMON37577
• Lead Sponsor: Kowa Research Europe

Brief Summary

Trial is onging in other countries

Detailed Description

Not available

Study Timeline

• Study Completion: 2013-03-08
• Last Update Posted: 29 April 2024

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 randomisation;;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);;09 December 2011 vii12;* 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 primary efficacy endpoint of this study is the percent change in LDL-C from<br /><br>baseline to Week 12 endpoint.</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>The secondary efficacy endpoints of this study are the following:<br /><br>- Percent change in LDL-C from baseline over 12 weeks of treatment (Week 4,<br /><br>Week 8, and Week 12);<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 12 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>12 weeks of treatment; and<br /><br>- Changes in TC:HDL-C ratio, non-HDL-C:HDL-C ratio, and Apo B:Apo A1 ratio<br /><br>from baseline over 12 weeks of treatment.</p><br>