A Study to Evaluate the Efficacy and Safety of Rosuvastatin in Children and Adolescents With Homozygous Familial Hypercholesterolemia
Condition: Homozygous Familial Hypercholesterolemia (HoFH)
MeSH terms: Hyperlipoproteinemia Type II; Homozygous Familial Hypercholesterolemia; Hypercholesterolemia
Intervention: Rosuvastatin 20mg; Placebo
Intervention description: Active drug will be taken taken orally, QD, either in the morning or in the evening; Will be taken taken orally, QD, either in the morning or in the evening
Intervention MeSH terms: Rosuvastatin Calcium
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Crossover Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Enrollment: 20.0
Sex: All
Minimum age: 6 Years
Maximum age: 17 Years
Healthy volunteers: No
Keywords: LDL-C; HoFH; Hyperlipidemia

Summary:

      The purpose of the study is to establish the efficacy, safety and tolerability of
      rosuvastatin in children and adolescents with homozygous familial hypercholesterolemia.
    

Eligibility 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 <18 years) with at least 1 of the
             following criteria:

             Documentation of genetic testing confirming 2 mutated alleles of the LDL receptor gene
             locus; and/or

             Documented untreated LDL C >500 mg/dL (12.9 mmol/L) and triglyceride (TG) <300 mg/dL
             (3.4 mmol/L) and at least 1 of the following criteria:

               1. Tendinous and/or cutaneous xanthoma prior to 10 years of age; or

               2. Documentation of HoFH in both parents by:

                    -  genetic and/or

                    -  clinical criteria

          3. Negative pregnancy test (b human chorionic gonadotropin analysis) prior to baseline in
             females of child bearing potential:

               -  Female patients of child bearing potential must adhere to a pregnancy prevention
                  method (abstinence, chemical, or mechanical) during the study and 3 months
                  following the last dose.

               -  Male patients should refrain from fathering a child (including sperm donation)
                  during the study and up to 3 months following the last dose; and

          4. Willing to follow all study procedures including adherence to dietary guidelines,
             study visits, fasting blood draws, and compliance with study treatment regimens.

        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 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.

          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.