Trial Assessing Long Term USe of PCSK9 Inhibition in Subj... | NCT01624142

ID	Type	Description	Link
2011-005400-15	EudraCT Number

Label	Type	Description	Intervention Names
Evolocumab	Experimental	Participants received 420 mg evolocumab every month (participants not on lipid apheresis) or every 2 weeks (participants on lipid apheresis) for up to 5 years. Participants could switch dosing regimens at week 12 or 24 based on LDL-C and serum unbound proprotein convertase subtilisin/kexin type 9 (PCSK9) levels.	Biological: Evolocumab

Name	Type	Description	Arm Group Labels	Other Names
Evolocumab	Biological	Evolocumab was administered by subcutaneous injection either once a month (QM) or once every two weeks (Q2W).	Evolocumab

Measure	Description	Time Frame
Number of Participants With Adverse Events	The severity of each adverse event (AE) was graded according to the National Cancer Institute Common Terminology Criteria for AEs (NCI-CTCAE) grading scale, where grade 1 = mild AE, grade 2 = moderate AE, grade 3 = severe AE, grade 4 = life-threatening AE and grade 5 = death due to AE.	From first dose of study drug in Study 20110271 up to 30 days after the last dose or until the end of study date, whichever was earlier; median duration of treatment was 48.7 months.

Measure	Description	Time Frame
Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C)		Baseline and weeks 4, 6, 8, 12, 16, 20, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, 204, and 216
Percent Change From Baseline in Non-high-density Lipoprotein Cholesterol (Non-HDL-C)

Name	Affiliation	Role
MD	Amgen	Study Director

Facility	Status	City	State	ZIP	Country	Contacts
Research Site		Los Angeles	California	90048	United States
Research Site

PubMed Identifier	Type	Citation	Retractions
28215937	Background	Raal FJ, Hovingh GK, Blom D, Santos RD, Harada-Shiba M, Bruckert E, Couture P, Soran H, Watts GF, Kurtz C, Honarpour N, Tang L, Kasichayanula S, Wasserman SM, Stein EA. Long-term treatment with evolocumab added to conventional drug therapy, with or without apheresis, in patients with homozygous familial hypercholesterolaemia: an interim subset analysis of the open-label TAUSSIG study. Lancet Diabetes Endocrinol. 2017 Apr;5(4):280-290. doi: 10.1016/S2213-8587(17)30044-X. Epub 2017 Feb 16.
29353350

Label	URL
AmgenTrials clinical trials website	View source

ID	Title	Description
FG000	HoFH	Participants with homozygous familial hypercholesterolemia (HoFH) received 420 mg evolocumab every month (participants not on lipid apheresis) or every 2 weeks (participants on lipid apheresis) for up to 5 years. Participants could switch dosing regimens at week 12 or 24 based on LDL-C and serum unbound proprotein convertase subtilisin/kexin type 9 (PCSK9) levels.

Type	Includes Protocol	Includes SAP	Includes ICF	Document Label	Document Date	Document Uploaded Date	Document File Name
Prot	Yes	No	No	Study Protocol	Dec 2, 2015	Dec 14, 2018

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

Number of Participants With Adverse Events

The severity of each adverse event (AE) was graded according to the National Cancer Institute Common Terminology Criteria for AEs (NCI-CTCAE) grading scale, where grade 1 = mild AE, grade 2 = moderate AE, grade 3 = severe AE, grade 4 = life-threatening AE and grade 5 = death due to AE.

All participants who received at least one dose of evolocumab in Study 20110271

Posted

Count of Participants

Participants

From first dose of study drug in Study 20110271 up to 30 days after the last dose or until the end of study date, whichever was earlier; median duration of treatment was 48.7 months.

ID	Title	Description
OG000	HoFH	Participants with homozygous familial hypercholesterolemia (HoFH) received 420 mg evolocumab every month (participants not on lipid apheresis) or every 2 weeks (participants on lipid apheresis) for up to 5 years. Participants could switch dosing regimens at week 12 or 24 based on LDL-C and serum unbound proprotein convertase subtilisin/kexin type 9 (PCSK9) levels.
OG001	Severe FH	Participants with severe (non-HoFH) familial hypercholesterolemia (FH) received 420 mg evolocumab every month (participants not on lipid apheresis) or every 2 weeks (participants on lipid apheresis) for up to 5 years. Participants could switch dosing regimens at week 12 or 24 based on LDL-C and serum unbound PCSK9 levels.

Units	Counts
Participants	OG000106 OG001194

Title

Denominators

Categories

All adverse events (AEs)

Title	Measurements
	OG00094 OG001174

Adverse events ≥ grade 2

Title	Measurements
	OG000

Secondary

Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C)

Participants who received at least 1 dose of evolocumab in Study 20110271 and with available data at each time point.

Posted

Median

Inter-Quartile Range

percent change

Baseline and weeks 4, 6, 8, 12, 16, 20, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, 204, and 216

ID	Title	Description
OG000	HoFH	Participants with homozygous familial hypercholesterolemia (HoFH) received 420 mg evolocumab every month (participants not on lipid apheresis) or every 2 weeks (participants on lipid apheresis) for up to 5 years. Participants could switch dosing regimens at week 12 or 24 based on LDL-C and serum unbound proprotein convertase subtilisin/kexin type 9 (PCSK9) levels.
OG001	Severe FH	Participants with severe (non-HoFH) familial hypercholesterolemia (FH) received 420 mg evolocumab every month (participants not on lipid apheresis) or every 2 weeks (participants on lipid apheresis) for up to 5 years. Participants could switch dosing regimens at week 12 or 24 based on LDL-C and serum unbound PCSK9 levels.

Units	Counts
Participants

Secondary

Percent Change From Baseline in Non-high-density Lipoprotein Cholesterol (Non-HDL-C)

Participants who received at least 1 dose of evolocumab in Study 20110271 and with available data at each time point.

Posted

Median

Inter-Quartile Range

percent change

Baseline and weeks 4, 6, 8, 12, 16, 20, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, 204, and 216

ID	Title	Description
OG000	HoFH	Participants with homozygous familial hypercholesterolemia (HoFH) received 420 mg evolocumab every month (participants not on lipid apheresis) or every 2 weeks (participants on lipid apheresis) for up to 5 years. Participants could switch dosing regimens at week 12 or 24 based on LDL-C and serum unbound proprotein convertase subtilisin/kexin type 9 (PCSK9) levels.
OG001	Severe FH	Participants with severe (non-HoFH) familial hypercholesterolemia (FH) received 420 mg evolocumab every month (participants not on lipid apheresis) or every 2 weeks (participants on lipid apheresis) for up to 5 years. Participants could switch dosing regimens at week 12 or 24 based on LDL-C and serum unbound PCSK9 levels.

Units	Counts
Participants

Secondary

Percent Change From Baseline in Lipoprotein (a)

Participants who received at least 1 dose of evolocumab in Study 20110271 and with available data at each time point.

Posted

Median

Inter-Quartile Range

percent change

Baseline and weeks 4, 6, 8, 12, 16, 20, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, 204, and 216

ID	Title	Description
OG000	HoFH	Participants with homozygous familial hypercholesterolemia (HoFH) received 420 mg evolocumab every month (participants not on lipid apheresis) or every 2 weeks (participants on lipid apheresis) for up to 5 years. Participants could switch dosing regimens at week 12 or 24 based on LDL-C and serum unbound proprotein convertase subtilisin/kexin type 9 (PCSK9) levels.
OG001	Severe FH	Participants with severe (non-HoFH) familial hypercholesterolemia (FH) received 420 mg evolocumab every month (participants not on lipid apheresis) or every 2 weeks (participants on lipid apheresis) for up to 5 years. Participants could switch dosing regimens at week 12 or 24 based on LDL-C and serum unbound PCSK9 levels.

Units	Counts
Participants

Secondary

Percent Change From Baseline in Apolipoprotein B

Participants who received at least 1 dose of evolocumab in Study 20110271 and with available data at each time point.

Posted

Median

Inter-Quartile Range

percent change

Baseline and weeks 4, 6, 8, 12, 16, 20, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, 204, and 216

ID	Title	Description
OG000	HoFH	Participants with homozygous familial hypercholesterolemia (HoFH) received 420 mg evolocumab every month (participants not on lipid apheresis) or every 2 weeks (participants on lipid apheresis) for up to 5 years. Participants could switch dosing regimens at week 12 or 24 based on LDL-C and serum unbound proprotein convertase subtilisin/kexin type 9 (PCSK9) levels.
OG001	Severe FH	Participants with severe (non-HoFH) familial hypercholesterolemia (FH) received 420 mg evolocumab every month (participants not on lipid apheresis) or every 2 weeks (participants on lipid apheresis) for up to 5 years. Participants could switch dosing regimens at week 12 or 24 based on LDL-C and serum unbound PCSK9 levels.

Units	Counts
Participants

Secondary

Percent Change From Baseline in Total Cholesterol/HDL-C Ratio

Participants who received at least 1 dose of evolocumab in Study 20110271 and with available data at each time point.

Posted

Median

Inter-Quartile Range

percent change

Baseline and weeks 4, 6, 8, 12, 16, 20, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, 204, and 216

ID	Title	Description
OG000	HoFH	Participants with homozygous familial hypercholesterolemia (HoFH) received 420 mg evolocumab every month (participants not on lipid apheresis) or every 2 weeks (participants on lipid apheresis) for up to 5 years. Participants could switch dosing regimens at week 12 or 24 based on LDL-C and serum unbound proprotein convertase subtilisin/kexin type 9 (PCSK9) levels.
OG001	Severe FH	Participants with severe (non-HoFH) familial hypercholesterolemia (FH) received 420 mg evolocumab every month (participants not on lipid apheresis) or every 2 weeks (participants on lipid apheresis) for up to 5 years. Participants could switch dosing regimens at week 12 or 24 based on LDL-C and serum unbound PCSK9 levels.

Units	Counts
Participants

Secondary

Percent Change From Baseline in Apolipoprotein B/Apolipoprotein A1 Ratio

Participants who received at least 1 dose of evolocumab in Study 20110271 and with available data at each time point.

Posted

Median

Inter-Quartile Range

percent change

Baseline and weeks 4, 6, 8, 12, 16, 20, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, 204, and 216

ID	Title	Description
OG000	HoFH	Participants with homozygous familial hypercholesterolemia (HoFH) received 420 mg evolocumab every month (participants not on lipid apheresis) or every 2 weeks (participants on lipid apheresis) for up to 5 years. Participants could switch dosing regimens at week 12 or 24 based on LDL-C and serum unbound proprotein convertase subtilisin/kexin type 9 (PCSK9) levels.
OG001	Severe FH	Participants with severe (non-HoFH) familial hypercholesterolemia (FH) received 420 mg evolocumab every month (participants not on lipid apheresis) or every 2 weeks (participants on lipid apheresis) for up to 5 years. Participants could switch dosing regimens at week 12 or 24 based on LDL-C and serum unbound PCSK9 levels.

Units	Counts
Participants

Secondary

Percentage of Participants With a 15% or Greater Reduction in LDL-C

Participants who received at least 1 dose of evolocumab in Study 20110271 and with available data at each time point.

Posted

Number

percentage of participants

Baseline and weeks 4, 6, 8, 12, 16, 20, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, 204, and 216

ID	Title	Description
OG000	HoFH	Participants with homozygous familial hypercholesterolemia (HoFH) received 420 mg evolocumab every month (participants not on lipid apheresis) or every 2 weeks (participants on lipid apheresis) for up to 5 years. Participants could switch dosing regimens at week 12 or 24 based on LDL-C and serum unbound proprotein convertase subtilisin/kexin type 9 (PCSK9) levels.
OG001	Severe FH	Participants with severe (non-HoFH) familial hypercholesterolemia (FH) received 420 mg evolocumab every month (participants not on lipid apheresis) or every 2 weeks (participants on lipid apheresis) for up to 5 years. Participants could switch dosing regimens at week 12 or 24 based on LDL-C and serum unbound PCSK9 levels.

Units	Counts
Participants

Serious Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Anaemia	Blood and lymphatic system disorders	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Acute coronary syndrome	Cardiac disorders	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Acute myocardial infarction	Cardiac disorders	MedDRA 21.0	Systematic Assessment	EG0002 affected106 at risk EG0012 affected194 at risk EG0024 affected300 at risk
Angina pectoris	Cardiac disorders	MedDRA 21.0	Systematic Assessment	EG0004 affected106 at risk EG0017 affected194 at risk EG00211 affected300 at risk
Angina unstable	Cardiac disorders	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0013 affected194 at risk EG0023 affected300 at risk
Aortic valve disease	Cardiac disorders	MedDRA 21.0	Systematic Assessment	EG0001 affected106 at risk EG0010 affected194 at risk EG0021 affected300 at risk
Aortic valve stenosis	Cardiac disorders	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Cardiac failure	Cardiac disorders	MedDRA 21.0	Systematic Assessment	EG0001 affected106 at risk EG0010 affected194 at risk EG0021 affected300 at risk
Cardiac failure congestive	Cardiac disorders	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Cardiogenic shock	Cardiac disorders	MedDRA 21.0	Systematic Assessment	EG0001 affected106 at risk EG0010 affected194 at risk EG0021 affected300 at risk
Coronary artery disease	Cardiac disorders	MedDRA 21.0	Systematic Assessment	EG0003 affected106 at risk EG0011 affected194 at risk EG0024 affected300 at risk
Coronary artery occlusion	Cardiac disorders	MedDRA 21.0	Systematic Assessment	EG0001 affected106 at risk EG0010 affected194 at risk EG0021 affected300 at risk
Coronary artery stenosis	Cardiac disorders	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Left ventricular dysfunction	Cardiac disorders	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Myocardial infarction	Cardiac disorders	MedDRA 21.0	Systematic Assessment	EG0001 affected106 at risk EG0010 affected194 at risk EG0021 affected300 at risk
Myocardial ischaemia	Cardiac disorders	MedDRA 21.0	Systematic Assessment	EG0001 affected106 at risk EG0014 affected194 at risk EG0025 affected300 at risk
Pericardial effusion	Cardiac disorders	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Supravalvular aortic stenosis	Cardiac disorders	MedDRA 21.0	Systematic Assessment	EG0001 affected106 at risk EG0010 affected194 at risk EG0021 affected300 at risk
Cerebrovascular arteriovenous malformation	Congenital, familial and genetic disorders	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Vertigo	Ear and labyrinth disorders	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Cataract	Eye disorders	MedDRA 21.0	Systematic Assessment	EG0001 affected106 at risk EG0011 affected194 at risk EG0022 affected300 at risk
Glaucoma	Eye disorders	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Macular degeneration	Eye disorders	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Macular hole	Eye disorders	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Colitis	Gastrointestinal disorders	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Diarrhoea	Gastrointestinal disorders	MedDRA 21.0	Systematic Assessment	EG0001 affected106 at risk EG0010 affected194 at risk EG0021 affected300 at risk
Gingival cyst	Gastrointestinal disorders	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Haemorrhoids	Gastrointestinal disorders	MedDRA 21.0	Systematic Assessment	EG0001 affected106 at risk EG0010 affected194 at risk EG0021 affected300 at risk
Asthenia	General disorders	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Chest discomfort	General disorders	MedDRA 21.0	Systematic Assessment	EG0001 affected106 at risk EG0010 affected194 at risk EG0021 affected300 at risk
Chest pain	General disorders	MedDRA 21.0	Systematic Assessment	EG0001 affected106 at risk EG0015 affected194 at risk EG0026 affected300 at risk
Complication associated with device	General disorders	MedDRA 21.0	Systematic Assessment	EG0001 affected106 at risk EG0010 affected194 at risk EG0021 affected300 at risk
Non-cardiac chest pain	General disorders	MedDRA 21.0	Systematic Assessment	EG0001 affected106 at risk EG0013 affected194 at risk EG0024 affected300 at risk
Cholecystitis acute	Hepatobiliary disorders	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Cholelithiasis	Hepatobiliary disorders	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0012 affected194 at risk EG0022 affected300 at risk
Hepatic cyst	Hepatobiliary disorders	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Non-alcoholic steatohepatitis	Hepatobiliary disorders	MedDRA 21.0	Systematic Assessment	EG0001 affected106 at risk EG0010 affected194 at risk EG0021 affected300 at risk
Appendicitis	Infections and infestations	MedDRA 21.0	Systematic Assessment	EG0001 affected106 at risk EG0010 affected194 at risk EG0021 affected300 at risk
Cellulitis	Infections and infestations	MedDRA 21.0	Systematic Assessment	EG0001 affected106 at risk EG0011 affected194 at risk EG0022 affected300 at risk
Diverticulitis	Infections and infestations	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0012 affected194 at risk EG0022 affected300 at risk
Osteomyelitis chronic	Infections and infestations	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Pneumonia	Infections and infestations	MedDRA 21.0	Systematic Assessment	EG0001 affected106 at risk EG0012 affected194 at risk EG0023 affected300 at risk
Postoperative wound infection	Infections and infestations	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Sepsis	Infections and infestations	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Septic shock	Infections and infestations	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Urosepsis	Infections and infestations	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Accidental overdose	Injury, poisoning and procedural complications	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Arteriovenous fistula site haemorrhage	Injury, poisoning and procedural complications	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Arteriovenous fistula thrombosis	Injury, poisoning and procedural complications	MedDRA 21.0	Systematic Assessment	EG0001 affected106 at risk EG0010 affected194 at risk EG0021 affected300 at risk
Fall	Injury, poisoning and procedural complications	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Fibula fracture	Injury, poisoning and procedural complications	MedDRA 21.0	Systematic Assessment	EG0001 affected106 at risk EG0010 affected194 at risk EG0021 affected300 at risk
Fracture	Injury, poisoning and procedural complications	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Head injury	Injury, poisoning and procedural complications	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Lower limb fracture	Injury, poisoning and procedural complications	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Rib fracture	Injury, poisoning and procedural complications	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Spinal compression fracture	Injury, poisoning and procedural complications	MedDRA 21.0	Systematic Assessment	EG0001 affected106 at risk EG0010 affected194 at risk EG0021 affected300 at risk
Spinal fracture	Injury, poisoning and procedural complications	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Subarachnoid haemorrhage	Injury, poisoning and procedural complications	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Tendon rupture	Injury, poisoning and procedural complications	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0012 affected194 at risk EG0022 affected300 at risk
Wound	Injury, poisoning and procedural complications	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Alanine aminotransferase increased	Investigations	MedDRA 21.0	Systematic Assessment	EG0002 affected106 at risk EG0010 affected194 at risk EG0022 affected300 at risk
Aspartate aminotransferase increased	Investigations	MedDRA 21.0	Systematic Assessment	EG0001 affected106 at risk EG0010 affected194 at risk EG0021 affected300 at risk
Blood creatine phosphokinase increased	Investigations	MedDRA 21.0	Systematic Assessment	EG0001 affected106 at risk EG0010 affected194 at risk EG0021 affected300 at risk
Cardiac stress test abnormal	Investigations	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Liver function test abnormal	Investigations	MedDRA 21.0	Systematic Assessment	EG0001 affected106 at risk EG0010 affected194 at risk EG0021 affected300 at risk
Transaminases increased	Investigations	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Troponin increased	Investigations	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Arthralgia	Musculoskeletal and connective tissue disorders	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0012 affected194 at risk EG0022 affected300 at risk
Intervertebral disc protrusion	Musculoskeletal and connective tissue disorders	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Muscle atrophy	Musculoskeletal and connective tissue disorders	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Posture abnormal	Musculoskeletal and connective tissue disorders	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Adenocarcinoma pancreas	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Breast cancer in situ	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	MedDRA 21.0	Systematic Assessment	EG0001 affected106 at risk EG0010 affected194 at risk EG0021 affected300 at risk
Colon cancer metastatic	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Invasive lobular breast carcinoma	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Metastases to liver	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Ovarian germ cell teratoma	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	MedDRA 21.0	Systematic Assessment	EG0001 affected106 at risk EG0010 affected194 at risk EG0021 affected300 at risk
Prostate cancer	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Small cell lung cancer	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Carotid artery occlusion	Nervous system disorders	MedDRA 21.0	Systematic Assessment	EG0001 affected106 at risk EG0010 affected194 at risk EG0021 affected300 at risk
Carotid artery stenosis	Nervous system disorders	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Cerebral ischaemia	Nervous system disorders	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Cerebrovascular accident	Nervous system disorders	MedDRA 21.0	Systematic Assessment	EG0001 affected106 at risk EG0010 affected194 at risk EG0021 affected300 at risk
Dementia with Lewy bodies	Nervous system disorders	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Embolic stroke	Nervous system disorders	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Headache	Nervous system disorders	MedDRA 21.0	Systematic Assessment	EG0001 affected106 at risk EG0010 affected194 at risk EG0021 affected300 at risk
Hemiparesis	Nervous system disorders	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Ischaemic stroke	Nervous system disorders	MedDRA 21.0	Systematic Assessment	EG0001 affected106 at risk EG0010 affected194 at risk EG0021 affected300 at risk
Seizure	Nervous system disorders	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Syncope	Nervous system disorders	MedDRA 21.0	Systematic Assessment	EG0001 affected106 at risk EG0011 affected194 at risk EG0022 affected300 at risk
Transient ischaemic attack	Nervous system disorders	MedDRA 21.0	Systematic Assessment	EG0001 affected106 at risk EG0010 affected194 at risk EG0021 affected300 at risk
Abortion spontaneous	Pregnancy, puerperium and perinatal conditions	MedDRA 21.0	Systematic Assessment	EG0001 affected106 at risk EG0010 affected194 at risk EG0021 affected300 at risk
Delirium	Psychiatric disorders	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Acute kidney injury	Renal and urinary disorders	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Nephrolithiasis	Renal and urinary disorders	MedDRA 21.0	Systematic Assessment	EG0001 affected106 at risk EG0011 affected194 at risk EG0022 affected300 at risk
Renal infarct	Renal and urinary disorders	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Menorrhagia	Reproductive system and breast disorders	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Uterine prolapse	Reproductive system and breast disorders	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Acute respiratory failure	Respiratory, thoracic and mediastinal disorders	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Asthma	Respiratory, thoracic and mediastinal disorders	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Epistaxis	Respiratory, thoracic and mediastinal disorders	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Pleural effusion	Respiratory, thoracic and mediastinal disorders	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Pleuritic pain	Respiratory, thoracic and mediastinal disorders	MedDRA 21.0	Systematic Assessment	EG0001 affected106 at risk EG0010 affected194 at risk EG0021 affected300 at risk
Aortic stenosis	Vascular disorders	MedDRA 21.0	Systematic Assessment	EG0003 affected106 at risk EG0012 affected194 at risk EG0025 affected300 at risk
Peripheral artery occlusion	Vascular disorders	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk
Peripheral artery stenosis	Vascular disorders	MedDRA 21.0	Systematic Assessment	EG0000 affected106 at risk EG0011 affected194 at risk EG0021 affected300 at risk

Other Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Abdominal pain	Gastrointestinal disorders	MedDRA 21.0	Systematic Assessment	EG0007 affected106 at risk EG0017 affected194 at risk EG00214 affected300 at risk
Diarrhoea	Gastrointestinal disorders	MedDRA 21.0	Systematic Assessment	EG0007 affected106 at risk EG00122 affected194 at risk EG00229 affected300 at risk
Chest pain	General disorders	MedDRA 21.0	Systematic Assessment	EG0004 affected106 at risk EG00115 affected194 at risk EG00219 affected300 at risk
Fatigue	General disorders	MedDRA 21.0	Systematic Assessment	EG0008 affected106 at risk EG00119 affected194 at risk EG00227 affected300 at risk
Influenza like illness	General disorders	MedDRA 21.0	Systematic Assessment	EG0002 affected106 at risk EG00110 affected194 at risk EG00212 affected300 at risk
Injection site bruising	General disorders	MedDRA 21.0	Systematic Assessment	EG0004 affected106 at risk EG00110 affected194 at risk EG00214 affected300 at risk
Injection site pain	General disorders	MedDRA 21.0	Systematic Assessment	EG0006 affected106 at risk EG0015 affected194 at risk EG00211 affected300 at risk
Oedema peripheral	General disorders	MedDRA 21.0	Systematic Assessment	EG0003 affected106 at risk EG00111 affected194 at risk EG00214 affected300 at risk
Bronchitis	Infections and infestations	MedDRA 21.0	Systematic Assessment	EG0004 affected106 at risk EG00116 affected194 at risk EG00220 affected300 at risk
Gastroenteritis	Infections and infestations	MedDRA 21.0	Systematic Assessment	EG0006 affected106 at risk EG00112 affected194 at risk EG00218 affected300 at risk
Influenza	Infections and infestations	MedDRA 21.0	Systematic Assessment	EG00017 affected106 at risk EG00121 affected194 at risk EG00238 affected300 at risk
Nasopharyngitis	Infections and infestations	MedDRA 21.0	Systematic Assessment	EG00017 affected106 at risk EG00136 affected194 at risk EG00253 affected300 at risk
Sinusitis	Infections and infestations	MedDRA 21.0	Systematic Assessment	EG0007 affected106 at risk EG0017 affected194 at risk EG00214 affected300 at risk
Upper respiratory tract infection	Infections and infestations	MedDRA 21.0	Systematic Assessment	EG00018 affected106 at risk EG00117 affected194 at risk EG00235 affected300 at risk
Urinary tract infection	Infections and infestations	MedDRA 21.0	Systematic Assessment	EG0008 affected106 at risk EG00112 affected194 at risk EG00220 affected300 at risk
Alanine aminotransferase increased	Investigations	MedDRA 21.0	Systematic Assessment	EG0006 affected106 at risk EG0013 affected194 at risk EG0029 affected300 at risk
Blood creatine phosphokinase increased	Investigations	MedDRA 21.0	Systematic Assessment	EG0006 affected106 at risk EG0014 affected194 at risk EG00210 affected300 at risk
Arthralgia	Musculoskeletal and connective tissue disorders	MedDRA 21.0	Systematic Assessment	EG0006 affected106 at risk EG00123 affected194 at risk EG00229 affected300 at risk
Back pain	Musculoskeletal and connective tissue disorders	MedDRA 21.0	Systematic Assessment	EG0009 affected106 at risk EG00117 affected194 at risk EG00226 affected300 at risk
Muscle spasms	Musculoskeletal and connective tissue disorders	MedDRA 21.0	Systematic Assessment	EG0004 affected106 at risk EG00115 affected194 at risk EG00219 affected300 at risk
Myalgia	Musculoskeletal and connective tissue disorders	MedDRA 21.0	Systematic Assessment	EG0006 affected106 at risk EG00124 affected194 at risk EG00230 affected300 at risk
Pain in extremity	Musculoskeletal and connective tissue disorders	MedDRA 21.0	Systematic Assessment	EG0004 affected106 at risk EG00111 affected194 at risk EG00215 affected300 at risk
Dizziness	Nervous system disorders	MedDRA 21.0	Systematic Assessment	EG0004 affected106 at risk EG00112 affected194 at risk EG00216 affected300 at risk
Headache	Nervous system disorders	MedDRA 21.0	Systematic Assessment	EG00017 affected106 at risk EG00116 affected194 at risk EG00233 affected300 at risk
Anxiety	Psychiatric disorders	MedDRA 21.0	Systematic Assessment	EG00010 affected106 at risk EG0010 affected194 at risk EG00210 affected300 at risk
Cough	Respiratory, thoracic and mediastinal disorders	MedDRA 21.0	Systematic Assessment	EG0005 affected106 at risk EG00114 affected194 at risk EG00219 affected300 at risk
Hypertension	Vascular disorders	MedDRA 21.0	Systematic Assessment	EG0009 affected106 at risk EG00115 affected194 at risk EG00224 affected300 at risk

ID	Term
D006938	Hyperlipoproteinemia Type II
D006937	Hypercholesterolemia
D000090542	Homozygous Familial Hypercholesterolemia

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	Title	Description
BG000	HoFH	Participants with homozygous familial hypercholesterolemia (HoFH) received 420 mg evolocumab every month (participants not on lipid apheresis) or every 2 weeks (participants on lipid apheresis) for up to 5 years. Participants could switch dosing regimens at week 12 or 24 based on LDL-C and serum unbound proprotein convertase subtilisin/kexin type 9 (PCSK9) levels.
BG001	Severe FH	Participants with severe (non-HoFH) familial hypercholesterolemia (FH) received 420 mg evolocumab every month (participants not on lipid apheresis) or every 2 weeks (participants on lipid apheresis) for up to 5 years. Participants could switch dosing regimens at week 12 or 24 based on LDL-C and serum unbound PCSK9 levels.
BG002	Total	Total of all reporting groups

ID	Title	Description	Deaths (Affected)	Deaths (At Risk)	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	HoFH	Participants with homozygous familial hypercholesterolemia (HoFH) received 420 mg evolocumab every month (participants not on lipid apheresis) or every 2 weeks (participants on lipid apheresis) for up to 5 years. Participants could switch dosing regimens at week 12 or 24 based on LDL-C and serum unbound proprotein convertase subtilisin/kexin type 9 (PCSK9) levels.	3	106	29	106	80	106
EG001	Severe FH	Participants with severe (non-HoFH) familial hypercholesterolemia (FH) received 420 mg evolocumab every month (participants not on lipid apheresis) or every 2 weeks (participants on lipid apheresis) for up to 5 years. Participants could switch dosing regimens at week 12 or 24 based on LDL-C and serum unbound PCSK9 levels.	6	194	57	194	134	194
EG002	Total	Participants received 420 mg evolocumab every month (participants not on lipid apheresis) or every 2 weeks (participants on lipid apheresis) for up to 5 years. Participants could switch dosing regimens at week 12 or 24 based on LDL-C and serum unbound PCSK9 levels.	9	300	86	300	214	300