Trial · NCT03851705

A Study of Inclisiran in Participants With Homozygous Familial Hypercholesterolemia (HoFH)

NCT03851705, Phase 3, Completed, Novartis Pharmaceuticals; results first posted 2022-11-10

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. A Study of Inclisiran in Participants With Homozygous Familial Hypercholesterolemia (HoFH). Trials, record NCT03851705. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03851705

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Trial

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

Title: A Study of Inclisiran in Participants With Homozygous Familial Hypercholesterolemia (HoFH)
NCT number: NCT03851705
Status: Completed
Phases: Phase 3
Enrollment: 56 participants, actual
Lead sponsor: Novartis Pharmaceuticals
Conditions: Homozygous Familial Hypercholesterolemia
Interventions: Inclisiran Sodium for injection, Placebo, Placebos
First posted: 2019-02-22
Last updated: 2023-01-30
Other registries' IDs: eudract:2018-000893-31
Results first posted: 2022-11-10

Source ClinicalTrials.gov

Registered outcomes52

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

List	Outcome	Time frame	Description
Primary	Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Day 150	Baseline, Day 150	Percentage Change in LDL-C levels from Baseline to Day 150
Secondary	Absolute Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Day 150	Baseline, Day 150	Absolute Change in LDL-C levels (mg/dL) from baseline to Day 150
Secondary	Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Subsequent Visits up to Day 180	Baseline, Days 90, 150, 180	Percentage Change in LDL-C levels from baseline to subsequent visits on Days 90, 150, and 180
Secondary	Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Subsequent Visits up to Day 720	Baseline, Days 270, 330, 450, 510, 630, 690, and 720	Percentage Change in LDL-C levels from baseline to subsequent visits up to Day 720
Secondary	Absolute Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Subsequent Visits up to Day 180	Baseline, Days 90, 150, 180	Absolute change in LDL-C levels (mg/dL) from baseline to subsequent visits on Days 90, 150 and 180 based on the
Secondary	Absolute Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Subsequent Visits up to Day 720	Baseline, Days 270, 330, 450, 510, 630, 690, and 720	Absolute change in LDL-C levels from Baseline to Subsequent Visits up to Day 720
Secondary	Percent Change in Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) From Baseline to Subsequent Visits up to Day 180	Baseline, Days 90, 150, 180	Percentage Change in PCSK9 from baseline to subsequent visits up to Day 180
Secondary	Percent Change in Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) From Baseline to Subsequent Visits up to Day 720	Baseline, Days 270, 330, 450, 510, 630, 690, and 720	Percentage Change in PCSK9 from baseline to subsequent visits up to Day 720
Secondary	Absolute Change in Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) From Baseline to Subsequent Visits up to Day 180	Baseline, Days 90, 150, 180	Absolute Change in PCSK9 from baseline to subsequent visits up to Day 180
Secondary	Absolute Change in Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) From Baseline to Subsequent Visits up to Day 720	Baseline, Days 270, 330, 450, 510, 630, 690, and 720	Absolute Change in PCSK9 from baseline to subsequent visits up to Day 720

1 to 10 of 52Source ClinicalTrials.gov

See all 52 registered outcomes

Outcomes52

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

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Day 150	Baseline, Day 150	ITT Population: All participants randomized into the study comprised the intent-to-treat (ITT) population.	Percentage change	LEAST_SQUARES_MEAN
SECONDARY	Absolute Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Day 150	Baseline, Day 150	ITT Population: All participants randomized into the study comprised the intent-to-treat (ITT) population.	mg/dL	LEAST_SQUARES_MEAN
SECONDARY	Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Subsequent Visits up to Day 180	Baseline, Days 90, 150, 180	ITT Population: All participants randomized into the study comprised the intent-to-treat (ITT) population.	Percentage change	LEAST_SQUARES_MEAN
SECONDARY	Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Subsequent Visits up to Day 720	Baseline, Days 270, 330, 450, 510, 630, 690, and 720	ITT Population (Part 2): The overall number of participants analyzed represents the intent-to-treat (ITT) population. The number analyzed per row represents participants with data at each time point.	Percentage change	MEAN
SECONDARY	Absolute Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Subsequent Visits up to Day 180	Baseline, Days 90, 150, 180	ITT Population: All participants randomized into the study comprised the intent-to-treat (ITT) population.	mg/dL	LEAST_SQUARES_MEAN
SECONDARY	Absolute Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Subsequent Visits up to Day 720	Baseline, Days 270, 330, 450, 510, 630, 690, and 720	ITT Population (Part 2): The overall number of participants analyzed represents the intent-to-treat (ITT) population. The number analyzed per row represents participants with data at each time point.	mg/dL	MEAN
SECONDARY	Percent Change in Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) From Baseline to Subsequent Visits up to Day 180	Baseline, Days 90, 150, 180	ITT Population: All participants randomized into the study comprised the intent-to-treat (ITT) population.	Percentage change	LEAST_SQUARES_MEAN
SECONDARY	Percent Change in Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) From Baseline to Subsequent Visits up to Day 720	Baseline, Days 270, 330, 450, 510, 630, 690, and 720	ITT Population (Part 2): The overall number of participants analyzed represents the intent-to-treat (ITT) population. The number analyzed per row represents participants with data at each time point.	Percentage Change	MEAN
SECONDARY	Absolute Change in Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) From Baseline to Subsequent Visits up to Day 180	Baseline, Days 90, 150, 180	ITT Population: All participants randomized into the study comprised the intent-to-treat (ITT) population.	ug/L	LEAST_SQUARES_MEAN
SECONDARY	Absolute Change in Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) From Baseline to Subsequent Visits up to Day 720	Baseline, Days 270, 330, 450, 510, 630, 690, and 720	ITT Population (Part 2): The overall number of participants analyzed represents the intent-to-treat (ITT) population. The number analyzed per row represents participants with data at each time point.	ug/L	MEAN

1 to 10 of 52Source ClinicalTrials.gov

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Results by group484

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

Outcome	Group	Category	Value	Spread	Units
Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Day 150	Part 1 - Placebo		2.39	95% Confidence Interval -19.98 to 24.75	Percentage change
Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Day 150	Part 1 - Inclisiran		0.70	95% Confidence Interval -14.03 to 15.44	Percentage change
Absolute Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Day 150	Part 1 - Placebo		-8.82	95% Confidence Interval -75.19 to 57.55	mg/dL
Absolute Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Day 150	Part 1 - Inclisiran		-2.35	95% Confidence Interval -36.34 to 31.63	mg/dL
Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Subsequent Visits up to Day 180	Part 1 - Placebo	Day 90	3.3	95% Confidence Interval -13.0 to 19.6	Percentage change
Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Subsequent Visits up to Day 180	Part 1 - Inclisiran	Day 90	-8.7	95% Confidence Interval -20.3 to 2.8	Percentage change
Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Subsequent Visits up to Day 180	Part 1 - Placebo	Day 150	-2.9	95% Confidence Interval -22.6 to 16.9	Percentage change
Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Subsequent Visits up to Day 180	Part 1 - Inclisiran	Day 150	1.8	95% Confidence Interval -12.5 to 16.1	Percentage change
Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Subsequent Visits up to Day 180	Part 1 - Placebo	Day 180	-1.5	95% Confidence Interval -17.0 to 14.1	Percentage change
Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Subsequent Visits up to Day 180	Part 1 - Inclisiran	Day 180	-7.2	95% Confidence Interval -19.0 to 4.6	Percentage change

1 to 10 of 484Source ClinicalTrials.gov

See all 484 results by group

Participant flow24

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

Period	Milestone	Group	Participants
Part 1: Double-Blind Period	STARTED	Part 1 - Inclisiran	37
Part 1: Double-Blind Period	STARTED	Part 1 - Placebo	19
Part 1: Double-Blind Period	STARTED	Part 2 - Placebo-Inclisiran	0
Part 1: Double-Blind Period	STARTED	Part 2 - Inclisiran-Inclisiran	0
Part 1: Double-Blind Period	COMPLETED	Part 1 - Inclisiran	34
Part 1: Double-Blind Period	COMPLETED	Part 1 - Placebo	19
Part 1: Double-Blind Period	COMPLETED	Part 2 - Placebo-Inclisiran	0
Part 1: Double-Blind Period	COMPLETED	Part 2 - Inclisiran-Inclisiran	0
Part 1: Double-Blind Period	NOT COMPLETED	Part 1 - Inclisiran	3
Part 1: Double-Blind Period	NOT COMPLETED	Part 1 - Placebo	0

1 to 10 of 24Source ClinicalTrials.gov

See all 24 participant flow

Adverse events396

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	Anaemia	Blood and lymphatic system disorders	Part 1: Inclisiran	1	37
other	Anaemia	Blood and lymphatic system disorders	Part 1: Placebo	1	19
other	Anaemia	Blood and lymphatic system disorders	Part 1:All Patients	2	56
other	Anaemia	Blood and lymphatic system disorders	Part 2: Inclisiran	1	53
other	Angina pectoris	Cardiac disorders	Part 1: Inclisiran	1	37
other	Angina pectoris	Cardiac disorders	Part 1: Placebo	0	19
other	Angina pectoris	Cardiac disorders	Part 1:All Patients	1	56
other	Angina pectoris	Cardiac disorders	Part 2: Inclisiran	2	53
other	Aortic valve incompetence	Cardiac disorders	Part 1: Inclisiran	0	37
other	Aortic valve incompetence	Cardiac disorders	Part 1: Placebo	0	19

1 to 10 of 396Source ClinicalTrials.gov

See all 396 adverse events

Adverse event totals12

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Part 1: Inclisiran	0	37
Total, all-cause mortality	Part 1: Placebo	0	19
Total, all-cause mortality	Part 1:All Patients	0	56
Total, all-cause mortality	Part 2: Inclisiran	3	53
Total, other adverse events	Part 1: Inclisiran	12	37
Total, other adverse events	Part 1: Placebo	6	19
Total, other adverse events	Part 1:All Patients	18	56
Total, other adverse events	Part 2: Inclisiran	29	53
Total, serious adverse events	Part 1: Inclisiran	2	37
Total, serious adverse events	Part 1: Placebo	1	19

1 to 10 of 12Source ClinicalTrials.gov

See all 12 adverse event totals

Papers2

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
33990512	DERIVED	PubMed
37850379	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 comparisons1

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

Comparison
Part 1 - Inclisiran vs Part 1 - Placebo: Proportion of Subjects With ≥30% LDL-C Reduction of From Baseline at Day 150

Source 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/NCT03851705

GET https://conductscience.org/api/trials/v1/NCT03851705/registered?page=2

API reference For AI assistants