Phase 3 N=9 Treatment

A Study to Evaluate the Safety of Rosuvastatin in Children and Adolescents With Homozygous Familial Hypercholesterolemia

Homozygous Familial Hypercholesterolemia (HoFH)

Bottom Line

View on ClinicalTrials.gov: NCT02434497 ↗

Enrolled (actual)

Serious AEs

0.0%

Results posted

Feb 2018

Primary outcome: Primary: The Number of Participants Who Experianced Adverse Events and Serious Adverse Events — 5 participants

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: Rosuvastatin 20mg (Drug)
Age: Pediatric, Adult · 6+ yrs
Sex: All
Sponsor: AstraZeneca
Primary completion: Nov 2016

Outcome Measures

Outcome	Result	p-value
PRIMARY The Number of Participants Who Experianced Adverse Events and Serious Adverse Events	5	—
PRIMARY Safety and Tolerability in Terms of Number of Participants Who Had Adverse Events, Discontinuations Due to Adverse Events	—	—
PRIMARY Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Basophils/Leukocytes (%) >Upper Limite of Normal (ULN)	0; 0; 0; 0; 0; 0	—
PRIMARY Safety and Tolerability in Terms of Growth, Height	142.5; 136.8; 143.5; 136.8; 144.0; 137.6	—
PRIMARY Safety and Tolerability in Terms of Abnormalitites in Sexual Maturation	—	—
PRIMARY Safety and Tolerability in Terms of Growth, Height SD-score (or Z-score)	-0.43; -1.05; -0.48; -1.05; -0.41; -1.19	—
PRIMARY Safety and Tolerability in Terms of Growth, Weight	36.25; 37.42; 37.20; 38.02; 37.93; 38.16	—
PRIMARY Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Alanine Aminotransferase (U/L) >ULN	0; 0; 0; 0; 0; 1	—
PRIMARY Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Albumin (g/dL) >ULN	0; 1; 0; 0; 0; 0	—
PRIMARY Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Aspartate Aminotransferase (U/L) >ULN	0; 0; 0; 0; 0; 0	—
PRIMARY Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Bicarbonate (Mol/L) <LLN	0; 0; 0; 1; 1; 1	—
PRIMARY Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Bicarbonate (Mol/L) >ULN	0; 0; 0; 0; 0; 0	—
PRIMARY Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Ery. Mean Corpuscular HGB Concentration (g/dL) <LLN	2; 3; 0; 2; 2; 2	—
PRIMARY Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Ery. Mean Corpuscular HGB (pg) <LLN	1; 3; 1; 2; 1; 2	—
PRIMARY Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Ery. Mean Corpuscular Volume (fL) <LLN	1; 2; 1; 2; 1; 2	—
PRIMARY Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Ery. Mean Corpuscular Volume (fL) >ULN	0; 0; 0; 0; 0; 0	—
PRIMARY Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Erythrocytes (10^12/L) <LLN	1; 0; 0; 0; 0; 1	—
PRIMARY Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Erythrocytes (10^12/L) >ULN	0; 1; 0; 0; 0; 1	—
PRIMARY Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Hematocrit (%) <LLN	2; 2; 2; 3; 1; 2	—
PRIMARY Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Hemoglobin (g/dL) <LLN	2; 3; 2; 3; 1; 3	—
PRIMARY Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Leukocytes >ULN	0; 0; 0; 0; 0; 0	—
PRIMARY Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Lymphocytes/Leukocytes (%) <LLN	0; 0; 0; 0; 0; 0	—
PRIMARY Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Lymphocytes/Leukocytes (%) >ULN	0; 1; 0; 1; 0; 1	—
PRIMARY Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Monocytes/Leukocytes (%) >ULN	0; 0; 0; 0; 0; 0	—
PRIMARY Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Platelets (10^9/L) >ULN	1; 0; 0; 1; 0; 0	—
PRIMARY Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Blood Urea Nitrogen (mg/dL) <LLN	1; 0; 1; 0; 1; 0	—
PRIMARY Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Chloride (mmol/L) >ULN	0; 1; 0; 1; 0; 0	—
PRIMARY Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Creatine Kinase (U/L) >ULN	0; 0; 1; 0; 0; 0	—
PRIMARY Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Glucose (mg/dL) >ULN	0; 0; 0; 0; 0; 0	—
PRIMARY Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Lactate Dehydrogenase (U/L) <LLN	0; 0; 0; 0; 0; 0	—
PRIMARY Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Phosphate (mg/dL) >ULN	0; 0; 0; 0; 0; 0	—
PRIMARY Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Protein (g/dL) >ULN	0; 0; 0; 0; 0; 1	—
PRIMARY Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Sodium (mmol/L) <LLN	2; 0; 1; 0; 0; 0	—
PRIMARY Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Urate (mg/dL) >ULN	1; 0; 1; 1; 1; 0	—
PRIMARY Safety and Tolerability in Terms of Abnormal Urine Laboratory Values, Urine Ketones	0; 0; 0; 0; 0; 0	—
PRIMARY Safety and Tolerability in Terms of Abnormal Urine Laboratory Values, Urine Occult Blood	1; 1; 2; 1; 1; 2	—
PRIMARY Safety and Tolerability in Terms of Abnormal Urine Laboratory Values, Urine Protein	2; 1; 1; 1; 2; 1	—
PRIMARY Safety and Tolerability in Terms of Abnormal ECG, Abnormalities	0; 0; 0; 0; 0; 0	—
PRIMARY Safety and Tolerability in Terms of Abnormal Physical Exams, Cardiovascular	0; 0; 0; 0; 0; 0	—
PRIMARY Safety and Tolerability in Terms of Abnormal Physical Exams, General Appearance	1; 2; 1; 2; 1; 2	—
PRIMARY Safety and Tolerability in Terms of Abnormal Physical Exams, Head and Neck	0; 1; 0; 1; 0; 1	—
PRIMARY Safety and Tolerability in Terms of Abnormal Physical Exams, Musculoskeletal/Extremities	0; 0; 0; 0; 0; 0	—
PRIMARY Safety and Tolerability in Terms of Abnormal Physical Exams, Skin	3; 2; 3; 2; 3; 2	—
PRIMARY Safety and Tolerability in Terms of Abnormal Vital Signs	0; 0; 0; 0; 0; 0	—
SECONDARY Percent Change in LDL-C From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis	-21.0; -18.6; -14.7; -12.1; -21.3; -20.5	—
SECONDARY Percent Change in HDL-C From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis	12.3; 19.0; 5.2; 4.4; 4.4; 9.4	—
SECONDARY Percent Change in Total Cholesterol (TC) From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis	-19.4; -16.8; -14.0; -11.8; -19.9; -18.3	—
SECONDARY Percent Change in Triglycerides (TG) From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis	-17.3; -17.4; -16.2; -22.0; -23.3; -21.7	—
SECONDARY Percent Change in Non-HDL-C From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis	-21.7; -19.2; -15.7; -13.3; -22.1; -21.0	—
SECONDARY Percent Change in LDL-C/HDL-C From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis	-39.4; -41.2; -36.1; -35.0; -48.6; -42.2	—
SECONDARY Percent Change in TC/HDL-C From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis	-30.6; -29.4; -20.5; -16.6; -24.1; -29.2	—
SECONDARY Percent Change in Non-HDL-C/HDL-C From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis	-32.9; -31.5; -22.3; -18.1; -26.3; -31.8	—
SECONDARY Percent Change in ApoB From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis	-20.5; -18.7; -15.2; -10.5; -17.2; -19.2	—
SECONDARY Percent Change in ApoA-1 From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis	8.0; 7.0; 5.3; 6.5; 1.0; 10.7	—
SECONDARY Percent Change in ApoB/ApoA-1 From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis	-23.3; -22.1; -18.9; -15.7; -17.0; -26.0	—
SECONDARY Pharmacokinetic Profile in Terms of Trough Concentrations in Pediatric HoFH Taking a Daily Dose of Rosuvastatin 40mg	9.00; 7.14	—

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

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.
Male and female children and adolescents who were aged 6 to 500 mg/dL (12.9 mmol/L) and TG 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.
Uncontrolled hypothyroidism defined as thyroid stimulating hormone >1.5 times the upper limit of normal (ULN) at any time during Study D3561C00004.
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.
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.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02434497). 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.