Trial · NCT00706849

Efficacy and Safety Study of ISIS 301012 (Mipomersen) as Add-on in Familial Hypercholesterolemic Patients With Coronary Artery Disease

NCT00706849, Phase 3, Completed, Kastle Therapeutics, LLC; results first posted 2013-03-21

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Efficacy and Safety Study of ISIS 301012 (Mipomersen) as Add-on in Familial Hypercholesterolemic Patients With Coronary Artery Disease. Trials, record NCT00706849. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00706849

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Trial

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

Title: Efficacy and Safety Study of ISIS 301012 (Mipomersen) as Add-on in Familial Hypercholesterolemic Patients With Coronary Artery Disease
NCT number: NCT00706849
Status: Completed
Phases: Phase 3
Enrollment: 124 participants, actual
Lead sponsor: Kastle Therapeutics, LLC
Conditions: Heterozygous Familial Hypercholesterolemia, Coronary Artery Disease
Interventions: mipomersen sodium, placebo
First posted: 2008-06-30
Last updated: 2016-09-09
Results first posted: 2013-03-21

Source ClinicalTrials.gov

Registered outcomes20

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

List	Outcome	Time frame	Description
Primary	Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) at the Primary Efficacy Time Point	Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).	LDL cholesterol was measured in mg/dL. Samples were taken following an overnight fast. For patients with triglycerides \<400 mg/dL, LDL-C was obtained using Friedewald's calculation; and for patients with triglycerides ≥400 mg/dL, LDL-C was directly measured by the central laboratory using ultracentrifugation. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment.
Primary	LDL Cholesterol at Baseline and at the Primary Efficacy Time Point	Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).	The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment.
Secondary	Percent Change From Baseline in Apolipoprotein B at the Primary Efficacy Time Point	Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).	Apolipoprotein B was measured in mg/dL. Samples were taken following an overnight fast. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment.
Secondary	Apolipoprotein B at Baseline and at the Primary Efficacy Time Point	Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).	The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment.
Secondary	Percent Change From Baseline in Total Cholesterol at the Primary Efficacy Time Point	Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).	Total cholesterol was measured in mg/dL. Samples were taken following an overnight fast. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment.
Secondary	Total Cholesterol at Baseline and at the Primary Efficacy Time Point	Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).	The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment.
Secondary	Percent Change From Baseline in Non-High-Density Lipoprotein Cholesterol at the Primary Efficacy Time Point	Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).	Non-high-density lipoprotein cholesterol was measured in mg/dL. Samples were taken following an overnight fast. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment.
Secondary	Non-High-Density Lipoprotein Cholesterol at Baseline and at the Primary Efficacy Time Point	Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).	The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment.
Other	Percent Change From Baseline in Triglycerides at the Primary Efficacy Time Point	Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).	Triglycerides were measured in mg/dL. Samples were taken following an overnight fast. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment.
Other	Triglycerides at Baseline and at the Primary Efficacy Time Point	Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).	The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment.

1 to 10 of 20Source ClinicalTrials.gov

See all 20 registered outcomes

Outcomes20

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

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) at the Primary Efficacy Time Point	Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).	The Full Analysis Set, which consisted of all randomized patients who received at least 1 injection of study drug (mipomersen or placebo) and with a valid baseline and at least 1 post-baseline LDL-C measurement.	percentage of Baseline	MEAN
PRIMARY	LDL Cholesterol at Baseline and at the Primary Efficacy Time Point	Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).	Full Analysis Set	mg/dL	MEAN
SECONDARY	Percent Change From Baseline in Apolipoprotein B at the Primary Efficacy Time Point	Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).	Full Analysis Set	percentage of baseline	MEAN
SECONDARY	Apolipoprotein B at Baseline and at the Primary Efficacy Time Point	Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).	Full Analysis Set	mg/dL	MEAN
SECONDARY	Percent Change From Baseline in Total Cholesterol at the Primary Efficacy Time Point	Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).	Full Analysis Set	percentage of baseline	MEAN
SECONDARY	Total Cholesterol at Baseline and at the Primary Efficacy Time Point	Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).	Full Analysis Set	mg/dL	MEAN
SECONDARY	Percent Change From Baseline in Non-High-Density Lipoprotein Cholesterol at the Primary Efficacy Time Point	Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).	Full Analysis Set	percentage of baseline	MEAN
SECONDARY	Non-High-Density Lipoprotein Cholesterol at Baseline and at the Primary Efficacy Time Point	Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).	Full Analysis Set	mg/dL	MEAN
OTHER_PRE_SPECIFIED	Percent Change From Baseline in Triglycerides at the Primary Efficacy Time Point	Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).	Full Analysis Set	percentage of baseline	MEDIAN
OTHER_PRE_SPECIFIED	Triglycerides at Baseline and at the Primary Efficacy Time Point	Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).	Full Analysis Set	mg/dL	MEDIAN

1 to 10 of 20Source ClinicalTrials.gov

See all 20 outcomes

Results by group60

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

Outcome	Group	Category	Value	Spread	Units
Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) at the Primary Efficacy Time Point	Placebo		5.17	Standard Deviation 18.02	percentage of Baseline
Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) at the Primary Efficacy Time Point	Mipomersen		-28.02	Standard Deviation 26.99	percentage of Baseline
LDL Cholesterol at Baseline and at the Primary Efficacy Time Point	Placebo	Baseline	142.9	Standard Deviation 51.6	mg/dL
LDL Cholesterol at Baseline and at the Primary Efficacy Time Point	Mipomersen	Baseline	152.9	Standard Deviation 48.7	mg/dL
LDL Cholesterol at Baseline and at the Primary Efficacy Time Point	Placebo	Primary efficacy time point	146.4	Standard Deviation 43.4	mg/dL
LDL Cholesterol at Baseline and at the Primary Efficacy Time Point	Mipomersen	Primary efficacy time point	103.9	Standard Deviation 33.0	mg/dL
Percent Change From Baseline in Apolipoprotein B at the Primary Efficacy Time Point	Placebo		7.02	Standard Deviation 16.52	percentage of baseline
Percent Change From Baseline in Apolipoprotein B at the Primary Efficacy Time Point	Mipomersen		-26.31	Standard Deviation 22.16	percentage of baseline
Apolipoprotein B at Baseline and at the Primary Efficacy Time Point	Placebo	Baseline	126.8	Standard Deviation 33.2	mg/dL
Apolipoprotein B at Baseline and at the Primary Efficacy Time Point	Mipomersen	Baseline	132.8	Standard Deviation 33.9	mg/dL

1 to 10 of 60Source ClinicalTrials.gov

See all 60 results by group

Participant flow14

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

Period	Milestone	Group	Participants
Treatment Period	STARTED	Placebo	41
Treatment Period	STARTED	Mipomersen	83
Treatment Period	Full Analysis Set	Placebo	41
Treatment Period	Full Analysis Set	Mipomersen	82
Treatment Period	COMPLETED	Placebo	41
Treatment Period	COMPLETED	Mipomersen	73
Treatment Period	NOT COMPLETED	Placebo	0
Treatment Period	NOT COMPLETED	Mipomersen	10
Follow-up Period	STARTED	Placebo	3
Follow-up Period	STARTED	Mipomersen	28

1 to 10 of 14Source ClinicalTrials.gov

See all 14 participant flow

Adverse events564

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	Placebo	0	41
other	Anaemia	Blood and lymphatic system disorders	Mipomersen	1	83
other	Lymphadenopathy	Blood and lymphatic system disorders	Placebo	1	41
other	Lymphadenopathy	Blood and lymphatic system disorders	Mipomersen	1	83
other	Angina pectoris	Cardiac disorders	Placebo	0	41
other	Angina pectoris	Cardiac disorders	Mipomersen	2	83
other	Coronary artery disease	Cardiac disorders	Placebo	0	41
other	Coronary artery disease	Cardiac disorders	Mipomersen	1	83
other	Myocardial ischaemia	Cardiac disorders	Placebo	1	41
other	Myocardial ischaemia	Cardiac disorders	Mipomersen	1	83

1 to 10 of 564Source ClinicalTrials.gov

See all 564 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Placebo
Total, all-cause mortality	Mipomersen
Total, other adverse events	Placebo	38	41
Total, other adverse events	Mipomersen	83	83
Total, serious adverse events	Placebo	2	41
Total, serious adverse events	Mipomersen	6	83

Source ClinicalTrials.gov

Papers3

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
23060426	RESULT	PubMed
25614280	DERIVED	PubMed
27578134	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
Mipomersen vs Placebo: serious adverse events

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/NCT00706849

GET https://conductscience.org/api/trials/v1/NCT00706849/results?page=2

API reference For AI assistants