Trial · NCT03397121

Trial to Evaluate the Effect of Inclisiran Treatment on Low Density Lipoprotein Cholesterol (LDL-C) in Subjects With Heterozygous Familial Hypercholesterolemia (HeFH)

NCT03397121, Phase 3, Completed, The Medicines Company; results first posted 2020-10-28

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Trial to Evaluate the Effect of Inclisiran Treatment on Low Density Lipoprotein Cholesterol (LDL-C) in Subjects With Heterozygous Familial Hypercholesterolemia (HeFH). Trials, record NCT03397121. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03397121

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Trial to Evaluate the Effect of Inclisiran Treatment on Low Density Lipoprotein Cholesterol (LDL-C) in Subjects With Heterozygous Familial Hypercholesterolemia (HeFH)
NCT number: NCT03397121
Status: Completed
Phases: Phase 3
Enrollment: 482 participants, actual
Lead sponsor: The Medicines Company
Conditions: Heterozygous Familial Hypercholesterolemia, Elevated Cholesterol
Interventions: Inclisiran, Placebo
First posted: 2018-01-11
Last updated: 2020-10-28
Other registries' IDs: eudract:2017-002472-30
Results first posted: 2020-10-28

Source ClinicalTrials.gov

Registered outcomes8

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Percent Change in LDL-C From Baseline To Day 510	Baseline, Day 510
Primary	Time-adjusted Percent Change in LDL-C From Baseline After Day 90 and up to Day 540	Baseline, Day 90	Assessments performed at Baseline, Day 90, Day 540, time-adjusted percent change at Day 90 reported
Secondary	Absolute Change in LDL-C From Baseline to Day 510	Baseline, Day 510
Secondary	Time-adjusted Absolute Change in LDL-C From Baseline After Day 90 and up to Day 540	Baseline, Day 90	Assessments performed at Baseline, Day 90, Day 540, absolute change at Day 90 reported
Secondary	Percentage Change in Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) From Baseline to Day 510	Baseline, Day 510
Secondary	Percentage Change in Total Cholesterol From Baseline to Day 510	Baseline, Day 510
Secondary	Percent Change in Apolipoprotein B (Apo-B) From Baseline To Day 510	Baseline, Day 510
Secondary	Percent Change in Non-high-density Lipoprotein (HDL)-C From Baseline To Day 510	Baseline, Day 510

Source ClinicalTrials.gov

Outcomes8

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Percent Change in LDL-C From Baseline To Day 510	Baseline, Day 510	ITT (intent-to-treat) Population	percent change	LEAST_SQUARES_MEAN
PRIMARY	Time-adjusted Percent Change in LDL-C From Baseline After Day 90 and up to Day 540	Baseline, Day 90	ITT (intent-to-treat) Population	percent change	LEAST_SQUARES_MEAN
SECONDARY	Absolute Change in LDL-C From Baseline to Day 510	Baseline, Day 510	ITT (intent-to-treat) Population	mg/dL	LEAST_SQUARES_MEAN
SECONDARY	Time-adjusted Absolute Change in LDL-C From Baseline After Day 90 and up to Day 540	Baseline, Day 90	ITT (intent-to-treat) Population	mg/dL	LEAST_SQUARES_MEAN
SECONDARY	Percentage Change in Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) From Baseline to Day 510	Baseline, Day 510	ITT (intent-to-treat) Population	percent change	LEAST_SQUARES_MEAN
SECONDARY	Percentage Change in Total Cholesterol From Baseline to Day 510	Baseline, Day 510	ITT (intent-to-treat) Population	percent change	LEAST_SQUARES_MEAN
SECONDARY	Percent Change in Apolipoprotein B (Apo-B) From Baseline To Day 510	Baseline, Day 510	ITT (intent-to-treat) Population	percent change	LEAST_SQUARES_MEAN
SECONDARY	Percent Change in Non-high-density Lipoprotein (HDL)-C From Baseline To Day 510	Baseline, Day 510	ITT (intent-to-treat) Population	percent change	LEAST_SQUARES_MEAN

Source ClinicalTrials.gov

Results by group16

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
Percent Change in LDL-C From Baseline To Day 510	Inclisiran	-41.15	95% Confidence Interval -44.52 to -37.77	percent change
Percent Change in LDL-C From Baseline To Day 510	Placebo	8.37	95% Confidence Interval 3.96 to 12.77	percent change
Time-adjusted Percent Change in LDL-C From Baseline After Day 90 and up to Day 540	Inclisiran	-38.08	95% Confidence Interval -41.03 to -35.14	percent change
Time-adjusted Percent Change in LDL-C From Baseline After Day 90 and up to Day 540	Placebo	6.22	95% Confidence Interval 3.26 to 9.17	percent change
Absolute Change in LDL-C From Baseline to Day 510	Inclisiran	-58.95	95% Confidence Interval -64.75 to -53.15	mg/dL
Absolute Change in LDL-C From Baseline to Day 510	Placebo	9.94	95% Confidence Interval 4.10 to 15.78	mg/dL
Time-adjusted Absolute Change in LDL-C From Baseline After Day 90 and up to Day 540	Inclisiran	-56.58	95% Confidence Interval -60.98 to -52.17	mg/dL
Time-adjusted Absolute Change in LDL-C From Baseline After Day 90 and up to Day 540	Placebo	6.17	95% Confidence Interval 1.72 to 10.62	mg/dL
Percentage Change in Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) From Baseline to Day 510	Inclisiran	-60.68	95% Confidence Interval -64.40 to -56.96	percent change
Percentage Change in Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) From Baseline to Day 510	Placebo	17.66	95% Confidence Interval 13.91 to 21.42	percent change

1 to 10 of 16Source ClinicalTrials.gov

See all 16 results by group

Participant flow6

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Inclisiran	242
Overall Study	STARTED	Placebo	240
Overall Study	COMPLETED	Inclisiran	235
Overall Study	COMPLETED	Placebo	231
Overall Study	NOT COMPLETED	Inclisiran	7
Overall Study	NOT COMPLETED	Placebo	9

Source ClinicalTrials.gov

Adverse events112

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk
other	Injection site reaction	General disorders	Inclisiran	22	241
other	Injection site reaction	General disorders	Placebo	0	240
other	Influenza	Infections and infestations	Inclisiran	13	241
other	Influenza	Infections and infestations	Placebo	21	240
other	Nasopharyngitis	Infections and infestations	Inclisiran	28	241
other	Nasopharyngitis	Infections and infestations	Placebo	20	240
other	Upper respiratory tract infection	Infections and infestations	Inclisiran	16	241
other	Upper respiratory tract infection	Infections and infestations	Placebo	16	240
other	Back pain	Musculoskeletal and connective tissue disorders	Inclisiran	17	241
other	Back pain	Musculoskeletal and connective tissue disorders	Placebo	10	240

1 to 10 of 112Source ClinicalTrials.gov

See all 112 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Inclisiran	1	241
Total, all-cause mortality	Placebo	1	240
Total, other adverse events	Inclisiran	96	241
Total, other adverse events	Placebo	67	240
Total, serious adverse events	Inclisiran	18	241
Total, serious adverse events	Placebo	33	240

Source ClinicalTrials.gov

Papers4

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
32197277	DERIVED	PubMed
38063346	DERIVED	PubMed
38757725	DERIVED	PubMed
41604274	DERIVED	PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons2

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Inclisiran vs Placebo: deaths from any cause	Trial comparisons
Inclisiran vs Placebo: serious adverse events	Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT03397121

GET https://conductscience.org/api/trials/v1/NCT03397121/events?page=2

API reference For AI assistants