An Study to Evaluate Rosuvastatin in Children and Adolesc... | NCT01078675

NCT01078675

Status Module

Sponsor/Collaborators Module

Oversight Module

Description Module

Conditions Module

Design Module

Interventional Study Design

Biospecimen

No data available

No data is available for this block.

Arms/Interventions Module

Label	Type	Description	Intervention Names
1	Experimental		Drug: rosuvastatin calcium

Name	Type	Description	Arm Group Labels	Other Names
rosuvastatin calcium	Drug	5 mg, oral, once daily, 24 months	1	Crestor

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Percent Change From Baseline in LDL-C	Negative values represent a decrease and positive values represent an increase. In total, 198 patients were treated. One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.	At Month 3, Month 12 and Month 24
Sexual Maturation by Tanner Staging at Baseline	Tanner stages (I-V) was used to characterize physical development in children and adolescent. The stages was based on external primary and secondary sex characteristics, such as the size of the breasts, genitalia, and development of pubic hair. Tanner stage is considered going up when the organs grow bigger.	At Baseline
Single Dose PK - Cmax	Serial plasma samples were taken at baseline (Week 0) at: 0.5 hours pre-dose and at 0.5, 1, 2, 3, 4, 5, 6, 9, 12 hours and on Day 1 at 24 hours after the single 10 mg dosing	Serial blood samples over 24 hours.
Percent Change From Baseline in Height	One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.	At Month 12 and Month 24
Sexual Maturation by Tanner Staging at Month 12	Tanner stages (I-V) was used to characterize physical development in children and adolescent. The stages was based on external primary and secondary sex characteristics, such as the size of the breasts, genitalia, and development of pubic hair. Tanner stage is considered going up when the organs grow bigger.	At Baseline
Sexual Maturation by Tanner Staging at Month 24

Secondary Outcomes

Measure	Description	Time Frame
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	One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.	At Month 3, Month 12 and Month 24
Change From Baseline in Max and Mean Carotid Intima and Media Wall Thickness (cIMT)

Eligibility Module

Contacts/Locations Module

Name	Affiliation	Role
John J.P. Kastelein, MD, PhD	Chairman, Dept. of Vascular Medicine, Academic Medical Center, Meibergdreef 9	Principal Investigator

Facility	Status	City	State	ZIP	Country	Contacts
Research Site		Cincinnati	Ohio		United States
Research Site

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
28592434	Derived	Braamskamp MJAM, Langslet G, McCrindle BW, Cassiman D, Francis GA, Gagne C, Gaudet D, Morrison KM, Wiegman A, Turner T, Miller E, Kusters DM, Raichlen JS, Martin PD, Stein EA, Kastelein JJP, Hutten BA. Effect of Rosuvastatin on Carotid Intima-Media Thickness in Children With Heterozygous Familial Hypercholesterolemia: The CHARON Study (Hypercholesterolemia in Children and Adolescents Taking Rosuvastatin Open Label). Circulation. 2017 Jul 25;136(4):359-366. doi: 10.1161/CIRCULATIONAHA.116.025158. Epub 2017 Jun 7.
24192652

Label	URL
D3561C00002 Study Report Synopsis	View source

IPD Sharing Statement Module

No data available

No data is available for this block.

Results Section

Participant Flow Module

ID	Title	Description
FG000	Rosuvastatin	Rosuvastatin 5 mg, 10 mg or 20 mg
FG001	Healthy Siblings	Controls to HeFH patients in the cIMT evaluations

Baseline Characteristics Module

Outcome Measures Module

Adverse Events Module

More Info Module

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Submission Tracking

No data available

No data is available for this block.

Condition Browse Module

Intervention Browse Module

Masking Info

Rosuvastatin; All treated patients. Healthy siblings; All patient who started this study

Baseline Measures

Title

Description

Population Description

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Denominator Units Selected

Denominators

Classes

Age, Continuous

Mean

Standard Deviation

Years

Title

Denominators

Categories

Years

Title	Measurements
	BG00011.6± 3.33 BG00111.5± 3.53 BG00211.55± 3.43

Sex: Female, Male

Count of Participants

Participants

Title

Denominators

Categories

Title	Measurements
Female	BG000110 BG00132 BG002

Race/Ethnicity, Customized

Number

Participants

Title

Denominators

Categories

Caucasian

Title	Measurements
	BG000178 BG00153 BG002

Type

Title

Description

Population Description

Reporting Status

Anticipated Posting Date

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Time Frame

Units Analyzed

Denominator Units Selected

Arm/Group Information

Denominators

Classes

Analyses

Primary

Percent Change From Baseline in LDL-C

Negative values represent a decrease and positive values represent an increase. In total, 198 patients were treated. One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.

Intent-to-treat analysis set and Per-protocol analysis set

Posted

Mean

Standard Deviation

Percentage change

At Month 3, Month 12 and Month 24

ID	Title	Description
OG000	Rosuvastatin	Rosuvastatin 5 mg, 10 mg or 20 mg

Units	Counts
Participants	OG000197

Title

Denominators

Categories

LDL-C %Change from Baseline at month 3

Title	Measurements
	OG000-37.86± 14.392

LDL-C %Change from Baseline at month 12

Title	Measurements
	OG000-43.67± 14.896

LDL-C %Change from Baseline at month 24

Group IDs	Group Description	Statistical Method	Statistical Comment	P-Value	P-Value Comment	Parameter Type	Parameter Value	Dispersion Type	Dispersion Value	Confidence Interval Sides	Confidence Interval %	CI Lower Limit	CI Upper Limit	CI Lower Limit Comment	CI Upper Limit Comment	Estimate Comment	Tested Non-Inferiority	Non-Inferiority Type	Non-Inferiority Comment	Other Analysis Description
OG000		ANCOVA	P-value is two-sided and based on ANCOVA using age group as the fixed factor, and study centre and the baseline value as covariates.	<0.001	P-value<0.001 at Month 24. No adjustment for multiple comparisons is made for individual age group.	Mean Difference (Final Values)	-42.88	Standard Deviation	18.222	2-Sided	-42.88	-45.44	-40.32				No	Superiority or Other

Primary

Sexual Maturation by Tanner Staging at Baseline

Tanner stages (I-V) was used to characterize physical development in children and adolescent. The stages was based on external primary and secondary sex characteristics, such as the size of the breasts, genitalia, and development of pubic hair. Tanner stage is considered going up when the organs grow bigger.

Safety analysis set

Posted

Number

Participants

At Baseline

ID	Title	Description
OG000	Rosuvastatin	Rosuvastatin 5 mg, 10 mg or 20 mg

Units	Counts
Participants	OG000197

Primary

Single Dose PK - Cmax

Serial plasma samples were taken at baseline (Week 0) at: 0.5 hours pre-dose and at 0.5, 1, 2, 3, 4, 5, 6, 9, 12 hours and on Day 1 at 24 hours after the single 10 mg dosing

Single dose PK analysis set

Posted

Mean

Standard Deviation

ng/mL

Serial blood samples over 24 hours.

ID	Title	Description
OG000	Rosuvastatin	Rosuvastatin 10 mg

Units	Counts
Participants	OG00012

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

One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.

Intent-to-treat analysis set (LOCF)

Posted

Mean

Standard Deviation

Percent change

At Month 3, Month 12 and Month 24

ID	Title	Description
OG000	Rosuvastatin	Rosuvastatin 5 mg, 10 mg or 20 mg

Units	Counts
Participants	OG000197

Secondary

Change From Baseline in Max and Mean Carotid Intima and Media Wall Thickness (cIMT)

One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.

Intent-to-treat analysis set (LOCF) and healthy siblings

Posted

Mean

Standard Deviation

At Month 12 and Month 24

ID	Title	Description
OG000	Rosuvastatin	Rosuvastatin 5 mg, 10 mg or 20 mg
OG001	Healthy Siblings	Controls to HeFH patients in the cIMT evaluations

Units	Counts
Participants	OG000

Secondary

Adverse Events

Number of participants with Various Categories of AE's. One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.

Safety analysis set

Posted

Number

Participant

2-year study period

ID	Title	Description
OG000	Rosuvastatin	Rosuvastatin 5 mg, 10 mg or 20 mg

Units	Counts
Participants	OG000197

Secondary

Total Duration of Exposure

Total duration of exposure was calculated as [last dose date of rosuva - first dose date of rosuva + 1 day]. One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.

Safety analysis set

Posted

Mean

Standard Deviation

Days

2-year study period

ID	Title	Description
OG000	Rosuvastatin	Rosuvastatin 5 mg, 10 mg or 20 mg

Units	Counts
Participants	OG000197

Primary

Percent Change From Baseline in Height

One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.

Safety Population

Posted

Mean

Standard Deviation

Percent change

At Month 12 and Month 24

ID	Title	Description
OG000	Rosuvastatin	Rosuvastatin 5 mg, 10 mg or 20 mg

Units	Counts
Participants	OG000197

Primary

Sexual Maturation by Tanner Staging at Month 12

Safety analysis set

Posted

Number

Participants

At Baseline

ID	Title	Description
OG000	Rosuvastatin	Rosuvastatin 5 mg, 10 mg or 20 mg

Units	Counts
Participants	OG000197

Primary

Sexual Maturation by Tanner Staging at Month 24

Safety analysis set

Posted

Number

Participants

At Baseline

ID	Title	Description
OG000	Rosuvastatin	Rosuvastatin 5 mg, 10 mg or 20 mg

Units	Counts
Participants	OG000197

Primary

Single Dose PK - Tmax

Serial plasma samples were taken at baseline (Week 0) at: 0.5 hours pre-dose and at 0.5, 1, 2, 3, 4, 5, 6, 9, 12 hours and on Day 1 at 24 hours after the single 10 mg dosing

Single dose PK analysis set

Posted

Mean

Standard Deviation

Serial blood samples over 24 hours

ID	Title	Description
OG000	Rosuvastatin	Rosuvastatin 10 mg

Units	Counts
Participants	OG00012

Primary

Single Dose PK - AUC(0-24)

Serial plasma samples were taken at baseline (Week 0) at: 0.5 hours pre-dose and at 0.5, 1, 2, 3, 4, 5, 6, 9, 12 hours and on Day 1 at 24 hours after the single 10 mg dosing

Single dose PK analysis set

Posted

Mean

Standard Deviation

ng*hr/mL

Serial blood samples over 24 hours

ID	Title	Description
OG000	Rosuvastatin	Rosuvastatin 10 mg

Units	Counts
Participants	OG00012

Secondary

Overal Treatment Adherence

Overall adherence rate was calculated as the weighted mean of adherence rates of all consecutive visits after baseline, in which the adherence rate between 2 consecutive visits was a percentage of the number of rosuvastatin taken divided by duration of exposure. One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.

Safety analysis set

Posted

Mean

Standard Deviation

Percent of doses

2-year study period

ID	Title	Description
OG000	Rosuvastatin	Rosuvastatin 5 mg, 10 mg or 20 mg

Units	Counts
Participants	OG000

Arm/Groups

ID	Title	Description	Deaths (Affected)	Deaths (At Risk)	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	Rosuvastatin	Rosuvastatin 5 mg, 10 mg or 20 mg			9	197	172	197

Serious Adverse Events

Term	Organ System	Assessment Type	Statistical Information
Appendicitis	Infections and infestations	Systematic Assessment	EG0001 events1 affected197 at risk
Urinary retention postoperative	Injury, poisoning and procedural complications	Systematic Assessment	EG0001 events1 affected197 at risk
Forearm fracture	Injury, poisoning and procedural complications	Systematic Assessment	EG0001 events1 affected197 at risk
Streptococcal toxic shock syndrome	Infections and infestations	Systematic Assessment	EG0001 events1 affected197 at risk
Femur fracture	Injury, poisoning and procedural complications	Systematic Assessment	EG0001 events1 affected197 at risk
Viral pericarditis	Infections and infestations	Systematic Assessment	EG0001 events1 affected197 at risk
Multiple fractures	Injury, poisoning and procedural complications	Systematic Assessment	EG0001 events1 affected197 at risk
Leg fracture	Injury, poisoning and procedural complications	Systematic Assessment	EG0001 events1 affected197 at risk
Epilepsy	Nervous system disorders	Systematic Assessment	EG0002 events1 affected197 at risk
Pectus carinatum	Congenital, familial and genetic disorders	Systematic Assessment	EG0001 events1 affected197 at risk

Other Adverse Events

Term	Organ System	Assessment Type	Statistical Information
Lymphadenopathy	Blood and lymphatic system disorders	Systematic Assessment	EG0002 events2 affected197 at risk
Pectus carinatum	Congenital, familial and genetic disorders	Systematic Assessment	EG0002 events1 affected197 at risk
Auricular swelling	Ear and labyrinth disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Deafness unilateral	Ear and labyrinth disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Ear discomfort	Ear and labyrinth disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Ear pain	Ear and labyrinth disorders	Systematic Assessment	EG0002 events2 affected197 at risk
Middle ear effusion	Ear and labyrinth disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Motion sickness	Ear and labyrinth disorders	Systematic Assessment	EG0002 events2 affected197 at risk
Early menarche	Endocrine disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Hypothyroidism	Endocrine disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Conjunctivitis	Eye disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Myopia	Eye disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Optic nerve disorder	Eye disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Abdominal discomfort	Gastrointestinal disorders	Systematic Assessment	EG0003 events3 affected197 at risk
Abdominal distension	Gastrointestinal disorders	Systematic Assessment	EG0002 events2 affected197 at risk
Abdominal pain	Gastrointestinal disorders	Systematic Assessment	EG00018 events13 affected197 at risk
Abdominal pain upper	Gastrointestinal disorders	Systematic Assessment	EG00021 events15 affected197 at risk
Constipation	Gastrointestinal disorders	Systematic Assessment	EG0004 events4 affected197 at risk
Dental discomfort	Gastrointestinal disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Diarrhoea	Gastrointestinal disorders	Systematic Assessment	EG0009 events6 affected197 at risk
Dyspepsia	Gastrointestinal disorders	Systematic Assessment	EG0004 events3 affected197 at risk
Flatulence	Gastrointestinal disorders	Systematic Assessment	EG0002 events2 affected197 at risk
Gastritis	Gastrointestinal disorders	Systematic Assessment	EG0006 events4 affected197 at risk
Intestinal obstruction	Gastrointestinal disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Irritable bowel syndrome	Gastrointestinal disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Mouth ulceration	Gastrointestinal disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Nausea	Gastrointestinal disorders	Systematic Assessment	EG00024 events18 affected197 at risk
Rectal spasm	Gastrointestinal disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Toothache	Gastrointestinal disorders	Systematic Assessment	EG0004 events4 affected197 at risk
Vomiting	Gastrointestinal disorders	Systematic Assessment	EG00024 events19 affected197 at risk
Asthenia	General disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Fatigue	General disorders	Systematic Assessment	EG0008 events7 affected197 at risk
Influenza like illness	General disorders	Systematic Assessment	EG00019 events16 affected197 at risk
Malaise	General disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Pyrexia	General disorders	Systematic Assessment	EG00010 events10 affected197 at risk
Hepatic pain	Hepatobiliary disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Allergy to animal	Immune system disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Hypersensitivity	Immune system disorders	Systematic Assessment	EG0004 events4 affected197 at risk
Seasonal allergy	Immune system disorders	Systematic Assessment	EG0004 events4 affected197 at risk
Amoebic dysentery	Infections and infestations	Systematic Assessment	EG0001 events1 affected197 at risk
Appendicitis	Infections and infestations	Systematic Assessment	EG0001 events1 affected197 at risk
Bronchitis	Infections and infestations	Systematic Assessment	EG0003 events3 affected197 at risk
Conjunctivitis infective	Infections and infestations	Systematic Assessment	EG0001 events1 affected197 at risk
Cystitis	Infections and infestations	Systematic Assessment	EG0005 events3 affected197 at risk
Ear infection	Infections and infestations	Systematic Assessment	EG0005 events4 affected197 at risk
Eye infection	Infections and infestations	Systematic Assessment	EG0001 events1 affected197 at risk
Furuncle	Infections and infestations	Systematic Assessment	EG0001 events1 affected197 at risk
Gastritis viral	Infections and infestations	Systematic Assessment	EG0001 events1 affected197 at risk
Gastroenteritis	Infections and infestations	Systematic Assessment	EG00015 events12 affected197 at risk
Gastroenteritis viral	Infections and infestations	Systematic Assessment	EG00024 events18 affected197 at risk
Gingival infection	Infections and infestations	Systematic Assessment	EG0001 events1 affected197 at risk
Groin infection	Infections and infestations	Systematic Assessment	EG0001 events1 affected197 at risk
Herpes zoster	Infections and infestations	Systematic Assessment	EG0001 events1 affected197 at risk
Impetigo	Infections and infestations	Systematic Assessment	EG0003 events3 affected197 at risk
Infectious mononucleosis	Infections and infestations	Systematic Assessment	EG0001 events1 affected197 at risk
Influenza	Infections and infestations	Systematic Assessment	EG00028 events20 affected197 at risk
Laryngitis	Infections and infestations	Systematic Assessment	EG0001 events1 affected197 at risk
Lower respiratory tract infection	Infections and infestations	Systematic Assessment	EG0001 events1 affected197 at risk
Mononucleosis syndrome	Infections and infestations	Systematic Assessment	EG0001 events1 affected197 at risk
Nail infection	Infections and infestations	Systematic Assessment	EG0001 events1 affected197 at risk
Nasopharyngitis	Infections and infestations	Systematic Assessment	EG000150 events88 affected197 at risk
Otitis externa	Infections and infestations	Systematic Assessment	EG0002 events2 affected197 at risk
Otitis media	Infections and infestations	Systematic Assessment	EG0003 events3 affected197 at risk
Pertussis	Infections and infestations	Systematic Assessment	EG0001 events1 affected197 at risk
Pharyngitis	Infections and infestations	Systematic Assessment	EG00010 events8 affected197 at risk
Pharyngitis streptococcal	Infections and infestations	Systematic Assessment	EG0001 events1 affected197 at risk
Pilonidal cyst	Infections and infestations	Systematic Assessment	EG0001 events1 affected197 at risk
Pneumonia	Infections and infestations	Systematic Assessment	EG0002 events2 affected197 at risk
Pyelonephritis	Infections and infestations	Systematic Assessment	EG0001 events1 affected197 at risk
Respiratory tract infection	Infections and infestations	Systematic Assessment	EG0003 events3 affected197 at risk
Rhinitis	Infections and infestations	Systematic Assessment	EG0001 events1 affected197 at risk
Sinusitis	Infections and infestations	Systematic Assessment	EG0004 events2 affected197 at risk
Tonsillitis	Infections and infestations	Systematic Assessment	EG0006 events4 affected197 at risk
Tooth infection	Infections and infestations	Systematic Assessment	EG0002 events2 affected197 at risk
Toxic shock syndrome streptococcal	Infections and infestations	Systematic Assessment	EG0001 events1 affected197 at risk
Upper respiratory tract infection	Infections and infestations	Systematic Assessment	EG00012 events11 affected197 at risk
Urinary tract infection	Infections and infestations	Systematic Assessment	EG0007 events7 affected197 at risk
Viral infection	Infections and infestations	Systematic Assessment	EG0002 events2 affected197 at risk
Viral pericarditis	Infections and infestations	Systematic Assessment	EG0001 events1 affected197 at risk
Viral upper respiratory tract infection	Infections and infestations	Systematic Assessment	EG0002 events2 affected197 at risk
Arthropod bite	Injury, poisoning and procedural complications	Systematic Assessment	EG0001 events1 affected197 at risk
Concussion	Injury, poisoning and procedural complications	Systematic Assessment	EG0003 events3 affected197 at risk
Contusion	Injury, poisoning and procedural complications	Systematic Assessment	EG0002 events2 affected197 at risk
Excoriation	Injury, poisoning and procedural complications	Systematic Assessment	EG0002 events1 affected197 at risk
Face injury	Injury, poisoning and procedural complications	Systematic Assessment	EG0001 events1 affected197 at risk
Fall	Injury, poisoning and procedural complications	Systematic Assessment	EG0004 events3 affected197 at risk
Femur fracture	Injury, poisoning and procedural complications	Systematic Assessment	EG0001 events1 affected197 at risk
Foot fracture	Injury, poisoning and procedural complications	Systematic Assessment	EG0001 events1 affected197 at risk
Forearm fracture	Injury, poisoning and procedural complications	Systematic Assessment	EG0001 events1 affected197 at risk
Hand fracture	Injury, poisoning and procedural complications	Systematic Assessment	EG0001 events1 affected197 at risk
Joint injury	Injury, poisoning and procedural complications	Systematic Assessment	EG0001 events1 affected197 at risk
Laceration	Injury, poisoning and procedural complications	Systematic Assessment	EG0002 events2 affected197 at risk
Ligament rupture	Injury, poisoning and procedural complications	Systematic Assessment	EG0001 events1 affected197 at risk
Ligament sprain	Injury, poisoning and procedural complications	Systematic Assessment	EG0005 events5 affected197 at risk
Limb injury	Injury, poisoning and procedural complications	Systematic Assessment	EG0001 events1 affected197 at risk
Lower limb fracture	Injury, poisoning and procedural complications	Systematic Assessment	EG0001 events1 affected197 at risk
Multiple fractures	Injury, poisoning and procedural complications	Systematic Assessment	EG0002 events1 affected197 at risk
Muscle strain	Injury, poisoning and procedural complications	Systematic Assessment	EG0001 events1 affected197 at risk
Periorbital haematoma	Injury, poisoning and procedural complications	Systematic Assessment	EG0001 events1 affected197 at risk
Post procedural complication	Injury, poisoning and procedural complications	Systematic Assessment	EG0001 events1 affected197 at risk
Post procedural swelling	Injury, poisoning and procedural complications	Systematic Assessment	EG0001 events1 affected197 at risk
Post-traumatic pain	Injury, poisoning and procedural complications	Systematic Assessment	EG0001 events1 affected197 at risk
Procedural pain	Injury, poisoning and procedural complications	Systematic Assessment	EG0003 events3 affected197 at risk
Road traffic accident	Injury, poisoning and procedural complications	Systematic Assessment	EG0001 events1 affected197 at risk
Scratch	Injury, poisoning and procedural complications	Systematic Assessment	EG0001 events1 affected197 at risk
Tendon injury	Injury, poisoning and procedural complications	Systematic Assessment	EG0002 events2 affected197 at risk
Tendon rupture	Injury, poisoning and procedural complications	Systematic Assessment	EG0002 events2 affected197 at risk
Traumatic haematoma	Injury, poisoning and procedural complications	Systematic Assessment	EG0001 events1 affected197 at risk
Urinary retention postoperative	Injury, poisoning and procedural complications	Systematic Assessment	EG0001 events1 affected197 at risk
Wrist fracture	Injury, poisoning and procedural complications	Systematic Assessment	EG0003 events3 affected197 at risk
Blood bilirubin increased	Investigations	Systematic Assessment	EG0001 events1 affected197 at risk
Blood creatine phosphokinase increased	Investigations	Systematic Assessment	EG0005 events4 affected197 at risk
Cardiac murmur	Investigations	Systematic Assessment	EG0003 events3 affected197 at risk
Haemoglobin decreased	Investigations	Systematic Assessment	EG0001 events1 affected197 at risk
Hepatic enzyme increased	Investigations	Systematic Assessment	EG0002 events1 affected197 at risk
Lymphocyte count abnormal	Investigations	Systematic Assessment	EG0001 events1 affected197 at risk
Renal bruit	Investigations	Systematic Assessment	EG0001 events1 affected197 at risk
Serum ferritin decreased	Investigations	Systematic Assessment	EG0003 events3 affected197 at risk
Weight decreased	Investigations	Systematic Assessment	EG0002 events2 affected197 at risk
Hypoglycaemia	Metabolism and nutrition disorders	Systematic Assessment	EG0002 events1 affected197 at risk
Arthralgia	Musculoskeletal and connective tissue disorders	Systematic Assessment	EG00015 events12 affected197 at risk
Back pain	Musculoskeletal and connective tissue disorders	Systematic Assessment	EG0006 events6 affected197 at risk
Bone disorder	Musculoskeletal and connective tissue disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Exostosis	Musculoskeletal and connective tissue disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Flank pain	Musculoskeletal and connective tissue disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Growing pains	Musculoskeletal and connective tissue disorders	Systematic Assessment	EG0004 events2 affected197 at risk
Joint stiffness	Musculoskeletal and connective tissue disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Joint swelling	Musculoskeletal and connective tissue disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Muscle spasms	Musculoskeletal and connective tissue disorders	Systematic Assessment	EG0003 events3 affected197 at risk
Muscular weakness	Musculoskeletal and connective tissue disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Musculoskeletal pain	Musculoskeletal and connective tissue disorders	Systematic Assessment	EG0004 events4 affected197 at risk
Musculoskeletal stiffness	Musculoskeletal and connective tissue disorders	Systematic Assessment	EG0005 events4 affected197 at risk
Myalgia	Musculoskeletal and connective tissue disorders	Systematic Assessment	EG00012 events11 affected197 at risk
Neck pain	Musculoskeletal and connective tissue disorders	Systematic Assessment	EG0002 events2 affected197 at risk
Osteochondrosis	Musculoskeletal and connective tissue disorders	Systematic Assessment	EG0002 events2 affected197 at risk
Pain in extremity	Musculoskeletal and connective tissue disorders	Systematic Assessment	EG0007 events7 affected197 at risk
Pain in jaw	Musculoskeletal and connective tissue disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Scoliosis	Musculoskeletal and connective tissue disorders	Systematic Assessment	EG0003 events3 affected197 at risk
Tendon disorder	Musculoskeletal and connective tissue disorders	Systematic Assessment	EG0002 events2 affected197 at risk
Tendon pain	Musculoskeletal and connective tissue disorders	Systematic Assessment	EG0004 events4 affected197 at risk
Tendonitis	Musculoskeletal and connective tissue disorders	Systematic Assessment	EG0002 events2 affected197 at risk
Skin papilloma	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	Systematic Assessment	EG0001 events1 affected197 at risk
Dizziness	Nervous system disorders	Systematic Assessment	EG0008 events6 affected197 at risk
Epilepsy	Nervous system disorders	Systematic Assessment	EG0004 events1 affected197 at risk
Headache	Nervous system disorders	Systematic Assessment	EG00076 events46 affected197 at risk
Migraine	Nervous system disorders	Systematic Assessment	EG00011 events3 affected197 at risk
Paraesthesia	Nervous system disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Post-traumatic headache	Nervous system disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Presyncope	Nervous system disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Psychomotor hyperactivity	Nervous system disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Syncope	Nervous system disorders	Systematic Assessment	EG00011 events6 affected197 at risk
Attention deficit/hyperactivity disorder	Psychiatric disorders	Systematic Assessment	EG0002 events2 affected197 at risk
Autism spectrum disorder	Psychiatric disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Depressed mood	Psychiatric disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Depression	Psychiatric disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Insomnia	Psychiatric disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Intentional self-injury	Psychiatric disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Sleep disorder	Psychiatric disorders	Systematic Assessment	EG0002 events2 affected197 at risk
Haematuria	Renal and urinary disorders	Systematic Assessment	EG0002 events2 affected197 at risk
Pollakiuria	Renal and urinary disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Polyuria	Renal and urinary disorders	Systematic Assessment	EG0002 events1 affected197 at risk
Pyuria	Renal and urinary disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Amenorrhoea	Reproductive system and breast disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Dysmenorrhoea	Reproductive system and breast disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Menstrual disorder	Reproductive system and breast disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Menstruation irregular	Reproductive system and breast disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Polycystic ovaries	Reproductive system and breast disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Scrotal pain	Reproductive system and breast disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Asthma	Respiratory, thoracic and mediastinal disorders	Systematic Assessment	EG0002 events2 affected197 at risk
Cough	Respiratory, thoracic and mediastinal disorders	Systematic Assessment	EG00011 events11 affected197 at risk
Epistaxis	Respiratory, thoracic and mediastinal disorders	Systematic Assessment	EG0009 events6 affected197 at risk
Nasal congestion	Respiratory, thoracic and mediastinal disorders	Systematic Assessment	EG0003 events2 affected197 at risk
Nasal obstruction	Respiratory, thoracic and mediastinal disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Oropharyngeal pain	Respiratory, thoracic and mediastinal disorders	Systematic Assessment	EG00013 events13 affected197 at risk
Rhinorrhoea	Respiratory, thoracic and mediastinal disorders	Systematic Assessment	EG0005 events5 affected197 at risk
Sinus congestion	Respiratory, thoracic and mediastinal disorders	Systematic Assessment	EG0004 events3 affected197 at risk
Sinus disorder	Respiratory, thoracic and mediastinal disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Acne	Skin and subcutaneous tissue disorders	Systematic Assessment	EG0008 events6 affected197 at risk
Alopecia	Skin and subcutaneous tissue disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Eczema	Skin and subcutaneous tissue disorders	Systematic Assessment	EG0004 events4 affected197 at risk
Nail disorder	Skin and subcutaneous tissue disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Pruritus	Skin and subcutaneous tissue disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Rash	Skin and subcutaneous tissue disorders	Systematic Assessment	EG0003 events3 affected197 at risk
Rash vesicular	Skin and subcutaneous tissue disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Skin lesion	Skin and subcutaneous tissue disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Xanthoma	Skin and subcutaneous tissue disorders	Systematic Assessment	EG0001 events1 affected197 at risk
Haematoma	Vascular disorders	Systematic Assessment	EG0001 events1 affected197 at risk

ID	Term
D006938	Hyperlipoproteinemia Type II

Ancestor Terms

ID	Term
D008052	Lipid Metabolism, Inborn Errors
D008661	Metabolism, Inborn Errors
D030342	Genetic Diseases, Inborn
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities
D006951	Hyperlipoproteinemias
D006949	Hyperlipidemias
D050171	Dyslipidemias
D052439	Lipid Metabolism Disorders
D008659	Metabolic Diseases
D009750	Nutritional and Metabolic Diseases

ID	Term
D000068718	Rosuvastatin Calcium

Ancestor Terms

ID	Term
D013449	Sulfonamides
D000577	Amides
D009930	Organic Chemicals
D005464	Fluorobenzenes
D006845	Hydrocarbons, Fluorinated
D006846	Hydrocarbons, Halogenated
D006838	Hydrocarbons
D013450	Sulfones
D013457	Sulfur Compounds
D011743	Pyrimidines
D006573	Heterocyclic Compounds, 1-Ring
D006571	Heterocyclic Compounds

Title

Denominators

Categories

Tanner Stage I at Baseline

Title	Measurements
	OG00081± NA

Tanner Stage II at Baseline

Title	Measurements
	OG00032± NA

Tanner Stage III at Baseline

Title	Measurements
	OG00018± NA

Tanner Stage IV at Baseline

Title	Measurements
	OG00044± NA

Tanner Stage V at Baseline

Title	Measurements
	OG00021± NA

Not Recorded at Baseline

Title	Measurements
	OG0001

Title

Denominators

Categories

HDL-C %Change from Baseline at Month 3

Title	Measurements
	OG0005.67± 17.445

HDL-C %Change from Baseline at Month 12

Title	Measurements
	OG0006.35± 16.725

HDL-C %Change from Baseline at Month 24

Title	Measurements
	OG00011.73± 19.996

TC %Change from Baseline at Month 3

Title	Measurements
	OG000-29.60± 11.433

TC %Change from Baseline at Month 12

Title	Measurements
	OG000-33.91± 12.050

TC %Change from Baseline at Month 24

Title	Measurements
	OG000-32.03± 14.530

Triglycerides %Change from Baseline at Month 3

Title	Measurements
	OG000-7.95± 34.482

Triglycerides %Change from Baseline at Month 12

Title	Measurements
	OG000-7.85± 37.530

Triglycerides %Change from Baseline at Month 24

Title	Measurements
	OG000-0.12± 37.682

non-HDL C %Change from Baseline at Month 3

Title	Measurements
	OG000-36.35± 13.368

non-HDL C %Change from Baseline at Month 12

Title	Measurements
	OG000-41.66± 14.235

non-HDL C %Change from Baseline at Month 24

Title	Measurements
	OG000-40.40± 17.555

LDL-C/HDL-C %Change from Baseline at Month 3

Title	Measurements
	OG000-39.66± 17.381

LDL-C/HDL-C %Change from Baseline at Month 12

Title	Measurements
	OG000-45.63± 17.082

LDL-C/HDL-C %Change from Baseline at Month 24

Title	Measurements
	OG000-46.95± 20.126

TC/HDL-C %Change from Baseline at Month 3

Title	Measurements
	OG000-31.77± 14.874

TC/HDL-C %Change from Baseline at Month 12

Title	Measurements
	OG000-36.54± 14.474

TC/HDL-C %Change from Baseline at Month 24

Title	Measurements
	OG000-37.39± 17.079

Trig/HDL-C %Change from Baseline at Month 3

Title	Measurements
	OG000-9.05± 41.765

Trig/HDL-C %Change from Baseline at Month 12

Title	Measurements
	OG000-10.50± 40.633

Trig/HDL-C %Change from Baseline at Month 24

Title	Measurements
	OG000-7.12± 40.585

non-HDL-C/HDL-C %Change from Baseline at Month 3

Title	Measurements
	OG000-37.98± 17.369

non-HDL-C/HDL-C %Change from Baseline at Month 12

Title	Measurements
	OG000-43.71± 16.731

non-HDL-C/HDL-C %Change from Baseline at Month 24

Title	Measurements
	OG000-44.74± 19.896

ApoA-I %Change from Baseline at Month 3

Title	Measurements
	OG0004.77± 14.680

ApoA-I %Change from Baseline at Month 12

Title	Measurements
	OG0001.41± 13.747

ApoA-I %Change from Baseline at Month 24

Title	Measurements
	OG0002.34± 15.027

ApoB %Change from Baseline at Month 3

Title	Measurements
	OG000-29.29± 12.456

ApoB %Change from Baseline at Month 12

Title	Measurements
	OG000-35.65± 12.424

ApoB %Change from Baseline at Month 24

Title	Measurements
	OG000-35.72± 15.710

ApoB/ApoA-I %Change from Baseline at Month 3

Title	Measurements
	OG000-31.30± 15.524

ApoB/ApoA-I %Change from Baseline at Month 12

Title	Measurements
	OG000-35.66± 13.920

ApoB/ApoA-I %Change from Baseline at Month 24

Title	Measurements
	OG000-35.94± 18.743

hsCRP %Change from Baseline at Month 3

Title	Measurements
	OG000512.57± 4724.249

hsCRP %Change from Baseline at Month 12

Title	Measurements
	OG00042.96± 226.954

hsCRP %Change from Baseline at Month 24

Title	Measurements
	OG00098.36± 565.444

Title

Denominators

Categories

Title	Measurements
	OG000703.5± 97.25

Title

Denominators

Categories

Tanner Stage I at Month 12

Title	Measurements
	OG00061± NA

Tanner Stage II at Month 12

Title	Measurements
	OG00031± NA

Tanner Stage III at Month 12

Title	Measurements
	OG00021± NA

Tanner Stage IV at Month 12

Title	Measurements
	OG00032± NA

Tanner Stage V at Month 12

Title	Measurements
	OG00042± NA

Not Recorded at Month 12

Title	Measurements
	OG00010

Title

Denominators

Categories

Tanner Stage I at Month 24

Title	Measurements
	OG00043± NA

Tanner Stage II at Month 24

Title	Measurements
	OG00033± NA

Tanner Stage III at Month 24

Title	Measurements
	OG00023± NA

Tanner Stage IV at Month 24

Title	Measurements
	OG00032± NA

Tanner Stage V at Month 24

Title	Measurements
	OG00064± NA

Not Recorded at Month 24

Title	Measurements
	OG0002

ID	Title	Description
BG000	Rosuvastatin	Rosuvastatin 5 mg, 10 mg or 20 mg
BG001	Healthy Siblings	Controls to HeFH patients in the cIMT evaluations
BG002	Total	Total of all reporting groups