Investigating Pitavastatin for High Cholesterol in Childre
- Conditions
- high-risk hyperlipidaemiaMedDRA version: 14.1Level: PTClassification code 10062060Term: HyperlipidaemiaSystem Organ Class: 10027433 - Metabolism and nutrition disordersMedDRA version: 14.1Level: PTClassification code 10020603Term: HypercholesterolaemiaSystem Organ Class: 10027433 - Metabolism and nutrition disordersMedDRA version: 14.1Level: LLTClassification code 10016205Term: Familial hyperlipidaemiaSystem Organ Class: 10010331 - Congenital, familial and genetic disordersMedDRA version: 14.1Level: LLTClassification code 10057099Term: Heterozygous familial hypercholesterolaemiaSystem Organ Class: 10010331 - Congenital, familial and genetic disordersMedDRA version: 14.1Level: LLTClassification code 10049593Term: Familial hypercholesterolaemiaSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
- Registration Number
- EUCTR2011-004964-32-NO
- Lead Sponsor
- Kowa Research Europe, Ltd.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 96
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.
Are the trial subjects under 18? yes
Number of subjects for this age range: 96
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
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 clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: The primary objective of this study is to compare the efficacy of pitavastatin 1 mg QD, 2 mg QD, and 4 mg QD to placebo in terms of the percentage reduction in LDL-C in children or adolescent patients with high-risk hyperlipidaemia at steady state (Week 12).;Secondary Objective: The secondary objectives of this study are the following:<br>• To compare the efficacy of pitavastatin 1 mg QD, 2 mg QD, and 4 mg QD to placebo in terms of the change or percent change in secondary lipid parameters in children and adolescent patients with high-risk hyperlipidaemia over 12 weeks;<br>• To measure PK parameters at each dose level; and<br>• To compare the safety and tolerability of pitavastatin 1 mg QD, 2 mg QD, and 4 mg QD to placebo in children and adolescent patients with high-risk hyperlipidaemia over 12 weeks.;Primary end point(s): The primary efficacy endpoint of this study is the percent change in LDL-C from baseline to Week 12 endpoint.;Timepoint(s) of evaluation of this end point: see above
- Secondary Outcome Measures
Name Time Method Secondary end point(s): • Percent change in LDL-C from baseline over 12 weeks of treatment (Week 4, Week 8, and Week 12);<br>• Percentages of patients who achieve AHA minimal (130 mg/dL [3.4 mmol/L]) and ideal (110 mg/dL [2.8 mmol/L]) LDL-C targets over 12 weeks of treatment;<br>• Percent changes in HDL-C, non-high-density lipoprotein cholesterol (non-HDL-C), TC, TG, apolipoprotein A1 (Apo A1), and Apo B from baseline over 12 weeks of treatment; and<br>• Changes in TC:HDL-C ratio, non-HDL-C:HDL-C ratio, and Apo B:Apo A1 ratio from baseline over 12 weeks of treatment.;Timepoint(s) of evaluation of this end point: see above