Phase 3
N=315
An Study to Evaluate Rosuvastatin in Children and Adolescents With Familial Hypercholesterolaemia
Familial Hypercholesterolaemia
Bottom Line
View on ClinicalTrials.gov: NCT01078675 ↗Enrolled (actual)
315
Serious AEs
4.6%
Results posted
Feb 2015
Primary outcome: Primary: Percent Change From Baseline in LDL-C — -37.86; -43.67; -42.88 Percentage change — p=<0.001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- rosuvastatin calcium (Drug)
- Age
- Pediatric · 6+ yrs
- Sex
- All
- Sponsor
- AstraZeneca
- Primary completion
- Feb 2013
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent Change From Baseline in LDL-C |
-37.86; -43.67; -42.88 | <0.001 sig |
| PRIMARY Sexual Maturation by Tanner Staging at Baseline |
81; 32; 18; 44; 21; 1 | — |
| PRIMARY Single Dose PK - Cmax |
3.5717 | — |
| PRIMARY Percent Change From Baseline in Height |
3.20; 5.91 | — |
| PRIMARY Sexual Maturation by Tanner Staging at Month 12 |
61; 31; 21; 32; 42; 10 | — |
| PRIMARY Sexual Maturation by Tanner Staging at Month 24 |
43; 33; 23; 32; 64; 2 | — |
| PRIMARY Single Dose PK - Tmax |
2.664 | — |
| PRIMARY Single Dose PK - AUC(0-24) |
27.675 | — |
| SECONDARY Percent Change From Baseline in HDL-C, TC, TG, Non-HDL-C, LDL-C/HDL-C, TC/HDL-C, Non HDL C/HDL-C, ApoB, ApoA-1, and ApoB/ApoA-1 |
5.67; 6.35; 11.73; -29.60; -33.91; -32.03 | — |
| SECONDARY Change From Baseline in Max and Mean Carotid Intima and Media Wall Thickness (cIMT) |
0.00626; 0.01707; 0.00189; 0.01202; 0.00282; 0.01564 | — |
| SECONDARY Adverse Events |
172; 0; 3; 9; 29; 0 | — |
| SECONDARY Total Duration of Exposure |
703.5 | — |
| SECONDARY Overal Treatment Adherence |
89.6 | — |
Summary
This study is being carried out to see if the study medication, rosuvastatin, is effective in treating familial hypercholesterolaemia in children and adolescents, and to determine the long term (over 2 years) safety, tolerability and efficacy of the study medication in these patients.
This study will also measure levels of drug in the blood and see how well it is tolerated. This is known as pharmacokinetic (PK) analysis.
At baseline only a small number of patients will participate in a single dose PK phase over 24 hours.
In order to see if this medication works, a control group of healthy siblings will help the researchers to compare certain results.
Eligibility Criteria
Inclusion Criteria
- children and adolescents (aged 6 to less than 18 years) with Familial Hypercholesterolaemia
- Patients aged between 6 and less than 10 years of age must not be taking a statin medicine
Exclusion Criteria
- History of muscle or sensitivity reactions to any statin medicines
- Current active liver disease or dysfunction (except a confirmed diagnosis of Gilbert's disease)
Data sourced from ClinicalTrials.gov (NCT01078675). 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.