Trial Outcomes & Findings for Safety and Tolerability of Repatha® (Evolocumab) in Indian Participants With Homozygous Familial Hypercholesterolemia (NCT NCT03403374)

Last Updated: 2024-05-29

Results Overview

Includes both serious and non-serious adverse events (AEs). AE: any untoward medical occurrence in a participant. SAE: an AE that meets 1 on the following serious criteria: fatal; life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity; congenital anomaly/birth defect; other medically important serious event. TEAE: any AE starting on or after the first dose of study drug and up to and including 30 days after the end of study drug or the end of study date, whichever is earlier.

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

30 participants

Primary outcome timeframe

12 weeks

Results posted on

2024-05-29

Participant Flow

Participants were enrolled at 10 study centers in India. The first participant was enrolled on 04 August 2018 and the last participant was enrolled on 29 August 2019.

Participant milestones

Participant milestones
Measure	Evolocumab Evolocumab 420 mg subcutaneous (SC) once monthly (QM) or every 2 weeks (Q2W; for participants on apheresis).
Overall Study STARTED	30
Overall Study COMPLETED	29
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Evolocumab Evolocumab 420 mg subcutaneous (SC) once monthly (QM) or every 2 weeks (Q2W; for participants on apheresis).
Overall Study Lost to Follow-up	1

Baseline Characteristics

Safety and Tolerability of Repatha® (Evolocumab) in Indian Participants With Homozygous Familial Hypercholesterolemia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Evolocumab n=30 Participants Evolocumab 420 mg subcutaneous (SC) once monthly (QM) or every 2 weeks (Q2W; for participants on apheresis).
Age, Continuous	23.2 years STANDARD_DEVIATION 13.1 • n=5 Participants
Age, Customized Adolescents (12 - 17 years old)	13 Participants n=5 Participants
Age, Customized Adults (18 - 64 years old)	16 Participants n=5 Participants
Age, Customized Adults (65 - 84 years old)	1 Participants n=5 Participants
Sex: Female, Male Female	13 Participants n=5 Participants
Sex: Female, Male Male	17 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	30 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race/Ethnicity, Customized Asian	30 Participants n=5 Participants
Low-Density Lipoprotein Cholesterol (LDL-C)	473.5 mg/dL STANDARD_DEVIATION 135.2 • n=5 Participants
Apolipoprotein B (ApoB)	275.3 mg/dL STANDARD_DEVIATION 69.1 • n=5 Participants
Lipoprotein(a) (Lp[a])	201.3 nmol/L STANDARD_DEVIATION 177.6 • n=5 Participants

PRIMARY outcome

Timeframe: 12 weeks

Outcome measures

Outcome measures
Measure	Evolocumab n=30 Participants Evolocumab 420 mg subcutaneous (SC) once monthly (QM) or every 2 weeks (Q2W; for participants on apheresis).
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)	10 Participants

SECONDARY outcome

Timeframe: baseline, week 12

Population: Participants with a baseline and week 12 assessment

Outcome measures

Outcome measures
Measure	Evolocumab n=29 Participants Evolocumab 420 mg subcutaneous (SC) once monthly (QM) or every 2 weeks (Q2W; for participants on apheresis).
Percent Change From Baseline to Week 12 in Low-Density Lipoprotein Cholesterol (LDL-C)	-6.4 percent change Standard Deviation 22.7

SECONDARY outcome

Timeframe: baseline, week 12

Population: Participants with a baseline and week 12 assessment

Outcome measures

Outcome measures
Measure	Evolocumab n=29 Participants Evolocumab 420 mg subcutaneous (SC) once monthly (QM) or every 2 weeks (Q2W; for participants on apheresis).
Percent Change From Baseline to Week 12 in Apolipoprotein B (ApoB)	-6.0 percent change Standard Deviation 19.8

SECONDARY outcome

Timeframe: baseline, week 12

Population: Participants with a baseline and week 12 assessment

Outcome measures

Outcome measures
Measure	Evolocumab n=29 Participants Evolocumab 420 mg subcutaneous (SC) once monthly (QM) or every 2 weeks (Q2W; for participants on apheresis).
Percent Change From Baseline to Week 12 in Lipoprotein(a) (Lp[a])	-0.2 percent change Standard Deviation 26.2

Adverse Events

Evolocumab

Serious events: 2 serious events

Other events: 8 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Evolocumab n=30 participants at risk Evolocumab 420 mg subcutaneous (SC) once monthly (QM) or every 2 weeks (Q2W; for participants on apheresis).
Cardiac disorders Coronary artery disease	3.3% 1/30 • All-cause mortality: from enrollment (up to 4 weeks prior to treatment) through end of study (week 12). Serious and other adverse events: from the first dose of study treatment through end of treatment (at week 8) plus 4 weeks (week 12). All-cause mortality is reported for all participants enrolled in the study. Serious adverse events and other adverse events are reported for all participants who received at least one dose of study drug.
Nervous system disorders Syncope	3.3% 1/30 • All-cause mortality: from enrollment (up to 4 weeks prior to treatment) through end of study (week 12). Serious and other adverse events: from the first dose of study treatment through end of treatment (at week 8) plus 4 weeks (week 12). All-cause mortality is reported for all participants enrolled in the study. Serious adverse events and other adverse events are reported for all participants who received at least one dose of study drug.

Other adverse events

Other adverse events
Measure	Evolocumab n=30 participants at risk Evolocumab 420 mg subcutaneous (SC) once monthly (QM) or every 2 weeks (Q2W; for participants on apheresis).
Cardiac disorders Angina pectoris	3.3% 1/30 • All-cause mortality: from enrollment (up to 4 weeks prior to treatment) through end of study (week 12). Serious and other adverse events: from the first dose of study treatment through end of treatment (at week 8) plus 4 weeks (week 12). All-cause mortality is reported for all participants enrolled in the study. Serious adverse events and other adverse events are reported for all participants who received at least one dose of study drug.
General disorders Injection site pain	3.3% 1/30 • All-cause mortality: from enrollment (up to 4 weeks prior to treatment) through end of study (week 12). Serious and other adverse events: from the first dose of study treatment through end of treatment (at week 8) plus 4 weeks (week 12). All-cause mortality is reported for all participants enrolled in the study. Serious adverse events and other adverse events are reported for all participants who received at least one dose of study drug.
General disorders Injection site swelling	3.3% 1/30 • All-cause mortality: from enrollment (up to 4 weeks prior to treatment) through end of study (week 12). Serious and other adverse events: from the first dose of study treatment through end of treatment (at week 8) plus 4 weeks (week 12). All-cause mortality is reported for all participants enrolled in the study. Serious adverse events and other adverse events are reported for all participants who received at least one dose of study drug.
General disorders Peripheral swelling	3.3% 1/30 • All-cause mortality: from enrollment (up to 4 weeks prior to treatment) through end of study (week 12). Serious and other adverse events: from the first dose of study treatment through end of treatment (at week 8) plus 4 weeks (week 12). All-cause mortality is reported for all participants enrolled in the study. Serious adverse events and other adverse events are reported for all participants who received at least one dose of study drug.
Infections and infestations Pyoderma	3.3% 1/30 • All-cause mortality: from enrollment (up to 4 weeks prior to treatment) through end of study (week 12). Serious and other adverse events: from the first dose of study treatment through end of treatment (at week 8) plus 4 weeks (week 12). All-cause mortality is reported for all participants enrolled in the study. Serious adverse events and other adverse events are reported for all participants who received at least one dose of study drug.
Infections and infestations Varicella	3.3% 1/30 • All-cause mortality: from enrollment (up to 4 weeks prior to treatment) through end of study (week 12). Serious and other adverse events: from the first dose of study treatment through end of treatment (at week 8) plus 4 weeks (week 12). All-cause mortality is reported for all participants enrolled in the study. Serious adverse events and other adverse events are reported for all participants who received at least one dose of study drug.
Musculoskeletal and connective tissue disorders Arthralgia	3.3% 1/30 • All-cause mortality: from enrollment (up to 4 weeks prior to treatment) through end of study (week 12). Serious and other adverse events: from the first dose of study treatment through end of treatment (at week 8) plus 4 weeks (week 12). All-cause mortality is reported for all participants enrolled in the study. Serious adverse events and other adverse events are reported for all participants who received at least one dose of study drug.
Musculoskeletal and connective tissue disorders Arthritis	3.3% 1/30 • All-cause mortality: from enrollment (up to 4 weeks prior to treatment) through end of study (week 12). Serious and other adverse events: from the first dose of study treatment through end of treatment (at week 8) plus 4 weeks (week 12). All-cause mortality is reported for all participants enrolled in the study. Serious adverse events and other adverse events are reported for all participants who received at least one dose of study drug.
Respiratory, thoracic and mediastinal disorders Cough	3.3% 1/30 • All-cause mortality: from enrollment (up to 4 weeks prior to treatment) through end of study (week 12). Serious and other adverse events: from the first dose of study treatment through end of treatment (at week 8) plus 4 weeks (week 12). All-cause mortality is reported for all participants enrolled in the study. Serious adverse events and other adverse events are reported for all participants who received at least one dose of study drug.
Skin and subcutaneous tissue disorders Diabetic wound	3.3% 1/30 • All-cause mortality: from enrollment (up to 4 weeks prior to treatment) through end of study (week 12). Serious and other adverse events: from the first dose of study treatment through end of treatment (at week 8) plus 4 weeks (week 12). All-cause mortality is reported for all participants enrolled in the study. Serious adverse events and other adverse events are reported for all participants who received at least one dose of study drug.
Skin and subcutaneous tissue disorders Rash	3.3% 1/30 • All-cause mortality: from enrollment (up to 4 weeks prior to treatment) through end of study (week 12). Serious and other adverse events: from the first dose of study treatment through end of treatment (at week 8) plus 4 weeks (week 12). All-cause mortality is reported for all participants enrolled in the study. Serious adverse events and other adverse events are reported for all participants who received at least one dose of study drug.
Skin and subcutaneous tissue disorders Skin fissures	3.3% 1/30 • All-cause mortality: from enrollment (up to 4 weeks prior to treatment) through end of study (week 12). Serious and other adverse events: from the first dose of study treatment through end of treatment (at week 8) plus 4 weeks (week 12). All-cause mortality is reported for all participants enrolled in the study. Serious adverse events and other adverse events are reported for all participants who received at least one dose of study drug.

Additional Information

Study Director

Amgen Inc.

Phone: 866-572-6436

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The Clinical Trial Agreement generally does not restrict an investigator's discussion of trial results after completion. The Agreement permits Amgen a limited period of time to review material discussing trial results (typically up to 45 days and possible extension). Amgen may remove confidential information, but authors have final control and approval of publication content. For multicenter studies, the investigator agrees not to publish any results before the first multi-center publication.
Publication restrictions are in place

Restriction type: OTHER