Hypercholesterolemia - The current trial will study children with Homozygous Familial Hypercholesterolemia (HoFH) MedDRA version: 17.1 Level: LLT Classification code 10054380 Term: Familial hypercholesterolemia System Organ Class: 100000004850
Conditions
Interventions
Trade Name: Crestor Tablets (calcium rosuvastatin) Film-Coated tablets
Product Name: Crestor
Product Code: ZD4522
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: calcium rosuvastatin
CAS
Sponsors
AstraZeneca AB
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1.Prior to any study related procedures being performed, provision of written informed consent from a parent/both parents or guardian and statement of assent from the child or adolescent (if required by Institutional Review Board [IRB] or Independent Ethics Committee [EC] according to local regulations and guidelines). Communication between the Investigator, patient/guardian and child/adolescent to confirm understanding and required compliance with the requirements of the study; 2.Male and female children and adolescents (aged 6 to 500 mg/dL (12.9 mmol/L) and triglyceride (TG) =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1.History of statin inducted myopathy or serious hypersensitivity reaction to other HMG CoA reductase inhibitors (statins), including rosuvastatin, at Visit 1; 2.Fasting serum glucose of >9.99 mmol/L (180 mg/dL) or glycosylated hemoglobin >9% at Visit 1 or patients with a history of diabetic ketoacidosis within the past year; 3.Uncontrolled hypothyroidism defined as thyroid stimulating hormone (TSH) >1.5 times the upper limit of normal (ULN) at Visit 1 or patients whose thyroid replacement therapy was initiated or modified within the last 3 months prior to Visit 2; 4.Current active liver disease or hepatic dysfunction (except a confirmed diagnosis of Gilbert’s disease) as defined as elevations of 1.5 times the ULN for any age in any of the following liver function tests at Visit 1: ALT, AST, or bilirubin; 5.Definite or suspected personal history or family history of clinically significant adverse drug reactions (ADRs), or hypersensitivity to drugs with a similar chemical structure to rosuvastatin as well as other statins;
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the efficacy of rosuvastatin 20 mg on low density lipoprotein cholesterol (LDL C), compared to placebo, after 6 weeks of treatment in pediatric patients with Homozygous Familial Hypercholesterolemia (HoFH);Secondary Objective: To assess the efficacy of rosuvastatin 20 mg on LDL C, compared to placebo, after 6 weeks of treatment in pediatric patients with HoFH not treated with apheresis To assess the efficacy of rosuvastatin 20 mg on lipid parameters other than LDL C, compared to placebo, after 6 weeks of treatment in pediatric patients with HoFH To assess the longitudinal profile of LDL C during treatment with rosuvastatin 20 mg To characterize the trough plasma exposure of rosuvastatin in pediatric patients with HoFH;Primary end point(s): "Describe efficacy in terms of low density lipoprotein cholesterol (LDL C) following 6 weeks rosuvastatin 20 mg or placebo treatment;Timepoint(s) of evaluation of this end point: Samples taken Day 42 (week 6), Day 84 (week 12) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1 Describe efficacy in terms of low density lipoprotein cholesterol (LDL C) following 6 weeks rosuvastatin 20 mg or placebo treatment in patients not treated with Apheresis 2 Describe efficacy in terms of High density lipoprotein cholesterol (HDL C), total cholesterol (TC), triglyceride (TG), non-HDL C, LDL-C/HDL C, TC/HDL C, non-HDL-C/HDL C, apolipoprotein B (ApoB), ApoB/apolipoprotein A 1 (ApoA 1) and ApoA 1 following 6 weeks of treatment with rosuvastatin 20 mg or placebo 3 Describe the longitudinal profile of LDL C as a change in LDL C from end of placebo period to 6, 12, and 18 weeks of therapy with rosuvastatin 20 mg 4, Describe the pharmacokinetic profile in terms of trough concentrations Safety Adverse events, including: • The frequency and severity of adverse events • Rate of discontinuations due to adverse events • Abnormal serum and urine laboratory values, ECGs, physical examinations, and vital signs Assessments of growth, including height (linear growth [cm and standard deviation (SD) score]), weight, and secondary characteristics of sexual maturation by Tanner stage performed at the beginning of the lead-in phase (Visit 2) and at Visit 7;Timepoint(s) of evaluation of this end point: 1 and 2: Samples taken Day 42 (week 6), Day 84 (week 12) 3. Samples taken Day 42 (week 6), Day 84 (week 12), Day 126 (week 18), Day 168 (week 24) 4. Samples taken Day 126 (week 18), Day 168 (week 24) Safety From screeing up to week 24 | — |
Countries
Australia, Belgium, Czech Republic, Denmark, Germany, Hong Kong, Israel, Netherlands, Norway, South Africa, Sweden, Taiwan, United Kingdom, United States
Contacts
Public ContactInformation Centre
AstraZeneca
Outcome results
None listed