ong term investigation of Pitavastatin for high-cholesterol in childre
- Conditions
- 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
- EUCTR2011-004983-32-FR
- Lead Sponsor
- Kowa Research Europe, Ltd.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 120
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.
Are the trial subjects under 18? yes
Number of subjects for this age range: 120
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 assess the safety of pitavastatin 1 mg QD, 2 mg QD, and 4 mg QD in children or adolescent patients with high-risk hyperlipidaemia over a period of 52 weeks.;Secondary Objective: The secondary objective of this study is to assess the persistence of efficacy with pitavastatin over 52 weeks by measuring lipid parameters and attainment of American Heart Association (AHA) minimal (130 mg/dL [3.4 mmol/L]) and ideal (110 mg/dL [2.8 mmol/L]) targets for LDL-C.;Primary end point(s): • Adverse events;<br>• Clinical laboratory parameters (including assessment of renal function and<br>adrenal, gonadal, and pituitary hormones);<br>• Vital signs;<br>• Electrocardiogram (ECG) parameters; and<br>• Physical examinations (including Tanner staging).;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 52 weeks of treatment;<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 52 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 52 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 52 weeks of treatment.;Timepoint(s) of evaluation of this end point: every visit