Phase 3
Completed N=11
A Study to Evaluate the Efficacy and Safety of Rosuvastatin in Children and Adolescents With Homozygous Familial Hypercholesterolemia
Homozygous Familial Hypercholesterolemia (HoFH)
Source: ClinicalTrials.gov NCT02226198 ↗
Enrolled (actual)
11
Serious AEs
0.0%
Results posted
Jul 2016
Primary outcomePrimary: LDL-Cholesterol (mg/dL) — 396.0; 481.4 mg/dL — p=0.005
◆ Published Evidence
Established
57citations · ~6 / year
Efficacy of Rosuvastatin in Children With Homozygous Familial Hypercholesterolemia and Association With Underlying Genetic Mutations.
Summary
The purpose of the study is to establish the efficacy, safety and tolerability of rosuvastatin in children and adolescents with homozygous familial hypercholesterolemia.
Linked Publications
-
Efficacy of Rosuvastatin in Children With Homozygous Familial Hypercholesterolemia and Association With Underlying Genetic Mutations.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY LDL-Cholesterol (mg/dL) |
396.0; 481.4 | 0.005 sig |
| PRIMARY LDL-Cholesterol (mmol/L) |
10.26; 12.47 | 0.005 sig |
| SECONDARY TC (mg/dL) |
447.6; 539.0 | 0.003 sig |
| SECONDARY TC (mmol/L) |
11.59; 13.96 | 0.003 sig |
| SECONDARY Non-HDL C (mg/dL) |
412.1; 505.3 | 0.003 sig |
| SECONDARY Non-HDL C (mmol/L) |
10.67; 13.09 | 0.003 sig |
| SECONDARY ApoB (mg/dL) |
234.9; 267.9 | 0.024 sig |
| SECONDARY ApoB (g/L) |
2.35; 2.68 | 0.024 sig |
| SECONDARY HDL-C (mg/dL) |
35.5; 33.7 | 0.314 |
| SECONDARY HDL-C (mmol/L) |
0.92; 0.87 | 0.314 |
| SECONDARY LDL-C, Not on Apheresis (mg/dL) |
479.8; 594.7 | 0.080 |
| SECONDARY LDL-C, Not on Apheresis (mmol/L) |
12.43; 15.40 | 0.080 |
| SECONDARY LDL-C From End of Placebo (mg/dL) |
472.9; 491.3; 399.4; 421.7; 345.8; 394.7 | — |
| SECONDARY LDL-C From End of Placebo (mmol/L) |
12.25; 12.73; 10.34; 10.92; 8.96; 10.22 | — |
| SECONDARY Trough Concentrations |
7.387; 4.482 | — |
| SECONDARY Adverse Events |
4; 5; 1; 4; 5; 1 | — |
| SECONDARY AE's Leading to Discontinuation |
0; 0; 0 | — |
| SECONDARY Abnormal Serum Levels |
0; 0; 0; 0; 0; 1 | — |
| SECONDARY Height |
144.0; 140.9; 144.9; 141.1; 145.7; 142.0 | — |
| SECONDARY Height Z-score |
-0.42; -0.75; -0.29; -0.71; -0.18; -0.70 | — |
| SECONDARY Weight |
37.39; 40.19; 38.14; 40.36; 38.63; 42.30 | — |
| SECONDARY Tanner Stage |
2.0; 1.7; 2.3; 2.0 | — |
| SECONDARY TG (mg/dL) |
79.8; 119.5 | 0.004 sig |
| SECONDARY TG (mmol/L) |
0.90; 1.35 | 0.004 sig |
| SECONDARY LDL C/HDL C |
12.208; 15.600 | 0.006 sig |
| SECONDARY TC/HDL C |
13.704; 17.416 | 0.005 sig |
| SECONDARY Non-HDL C/HDL C |
12.704; 16.416 | 0.005 sig |
| SECONDARY ApoB/ApoA |
2.408; 2.873 | 0.013 sig |
| SECONDARY Urinalysis Abnormalitites |
3; 2; 1; 2; 3; 2 | — |
| SECONDARY ECG Abnormalities |
0; 0; 0 | — |
| SECONDARY Physical Exam Abnormalitites |
1; 2; 1; 2; 1; 2 | — |
| SECONDARY Abnormal Vital Signs |
— | — |
Eligibility Criteria
Inclusion Criteria
- 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.
- Male and female children and adolescents (aged 6 to 500 mg/dL (12.9 mmol/L) and triglyceride (TG) 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.
- 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.
- Current active liver disease or hepatic dysfunction (except a confirmed diagnosis of Gilbert's disease) as defined as elevations of 1.5 times the upper limit of normal (ULN) for any age in any of the following liver function tests at Visit 1: Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST), or bilirubin.
- 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.
Data sourced from ClinicalTrials.gov (NCT02226198) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.