A Study to Evaluate the 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
Intervention description: Active drug 1 or 2 tablets 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
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 9.0
Sex: All
Minimum age: 6 Years
Maximum age: 18 Years
Healthy volunteers: No
Keywords: LDL-C; HoFH; Hyperlipidemia

Summary:

      The purpose of the study is to evaluate the safety 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 [IEC] according to local regulations and guidelines). Study D3561C00004
             participants who have had their 18th birthday (adults) will be required to provide
             written informed consent. Communication should take place 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 who were aged 6 to <18 years at the onset of
             Study D3561C00004 (even if they had their 18th birthday during that study) with:

               -  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 TG <400 mg/dL (4.5
                  mmol/L) and at least 1 of the following criteria:

                    -  Tendinous and/or cutaneous xanthoma prior to 10 years of age; or

                    -  Documentation of HeFH 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. Were taking study drug at the end of Study D3561C00004 and are 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 of Study
             D3561C00004.

          2. Fasting serum glucose of >9.99 mmol/L (180 mg/dL) or glycosylated hemoglobin >9%
             during Study D3561C00004 or patients with a history of diabetic ketoacidosis within
             the past year.

          3. Uncontrolled hypothyroidism defined as thyroid stimulating hormone >1.5 times the
             upper limit of normal (ULN) at any time during Study D3561C00004.

          4. Evidence of active liver disease or hepatic dysfunction (except a confirmed diagnosis
             of Gilbert's disease) as defined as non-transient elevations of ALT or AST elevations
             ≥3 times the ULN or non-transient total bilirubin ≥2 times the ULN during the Study
             D3561C00004.

          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.