A study to evaluate the efficacy and safety of Rosuvastatin in Children and Adolescents with Homozygous Familial Hypercholesterolemia - HYDRA

Study identifier:D3561C00004

ClinicalTrials.gov identifier:N/A

EudraCT identifier:N/A

CTIS identifier:N/A

Study Complete

Official Title

A Randomized, Double-blind, Placebo-controlled, Multi-center, Cross-over Study of Rosuvastatin in Children and Adolescents (aged 6 to <18 years) with Homozygous Familial Hypercholesterolemia (HoFH)

Medical condition

Homozygous Familial Hypercholesterolemia (HoFH)

Phase

Phase 3

Healthy volunteers

Study drug

Rosuvastatin 20mg, Placebo

Sex

All

Actual Enrollment

Study type

Interventional

Age

6 Years - 17 Years

Date

Study Start Date: 01 Nov 2014

Primary Completion Date: 01 Jul 2015

Study Completion Date: 01 Jul 2015

Study design

Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Double Blind
Primary Purpose: Treatment

Verification:

Verified 01 Jul 2015 by AstraZeneca

Collaborators

Inclusion and exclusion 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:
a. Tendinous and/or cutaneous xanthoma prior to 10 years of age; or
b. 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.

No locations available

Arms	Assigned Interventions
Active Comparator: Rosuvastatin 6-week treatment period, and after crossover finished a 12-week efficacy maintenance phase for all patients	Drug: Rosuvastatin 20mg Active drug will be taken taken orally, QD, either in the morning or in the evening
Placebo Comparator: Placebo 6 weeks treatment during crossover	Drug: Placebo Will be taken taken orally, QD, either in the morning or in the evening

Documents available

Trial Results Summaries
Available here

Contacts

Contact

AstraZeneca Clinical Study Information Center

1-877-240-9479

[email protected]