Heterozygous Familial HypercholesterolemiaPhase 3RADICHOL IINCT00706849

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

A Phase 3 study testing mipomersen sodium in people with Heterozygous Familial Hypercholesterolemia and Coronary Artery Disease, run by Kastle Therapeutics, LLC at 48 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

The purpose of this study is to determine whether mipomersen safely and effectively lowers low-density lipoprotein cholesterol (LDL-C) in patients with Heterozygous Familial Hypercholesterolemia (HeFH) and coronary artery disease (CAD) who are already on a stable dose of other lipid-lowering agents (including… Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Heterozygous Familial Hypercholesterolemia, Coronary Artery Disease
What is being tested?: mipomersen sodium
Type of treatment: Injection or infusion
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 124 participants
Where: 48 sitesin 2 countries
Study dates: Jul 2008to May 2010
Phase: Phase 3
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Diagnosis of Heterozygous Familial Hypercholesterolemia (HeFH)
Diagnosis of Coronary Artery Disease (CAD)
Stable lipid-lowering therapy for 12 weeks
On maximally tolerated statin therapy with at least 1 statin at a dose greater than zero, per Investigator judgment
Stable low-fat diet for 8 weeks
Stable weight for 6 weeks

Who cannot join

Significant health problems in recent past including heart attack, stroke, coronary syndrome, unstable angina, heart failure, significant arrhythmia, orthostatic hypotension, uncontrolled hypertension, blood disorders, liver disease, cancer, or digestive problems
Receiving apheresis treatment or last apheresis treatment within 8 weeks

Trial locations and contacts

48 sites in 2 countries.

Country

City, postcode or hospital

48 sites in 2 countries

United States37 sites

Study site
Ann Arbor, Michigan, 48109-5853
Study site
Biddeford, Maine, 04005
Study site
Boston, Massachusetts, 02114
Study site
Bridgeport, Connecticut, 06606
Study site
Charlotte, North Carolina, 28204
Study site
Charlotte, North Carolina, 28211
Study site
Chicago, Illinois, 60610
Study site
Cincinnati, Ohio, 45212
Study site
Concord, New Hampshire, 03301
ResEvo, LLC
Cuyahoga Falls, Ohio, 44223
Study site
Dallas, Texas, 75220
Study site
Dallas, Texas, 75226
Study site
Durham, North Carolina, 27710
Study site
Franklin, Ohio, 45005
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Grapevine, Texas, 76051
Study site
Houston, Texas, 77093
Study site
Kansas City, Kansas, 66160
Study site
La Jolla, California, 92093
Study site
Las Vegas, Nevada, 89144
Study site
Los Angeles, California, 90048
Study site
Melbourne, Florida, 32901
Study site
Mission Viejo, California, 92691
Study site
Naperville, Illinois, 60540
Study site
Nashville, Tennessee, 37232
Study site
New York, New York, 10032
The Rogosin Institute Comprehensive Lipid Control Center
New York, New York, 10065
Study site
Newport Beach, California, 92260
Study site
Oklahoma City, Oklahoma, 73120
Study site
Pensacola, Florida, 32514
Study site
Portland, Oregon, 97239
Study site
Salt Lake City, Utah, 84108
Study site
San Antonio, Texas, 78229
Study site
Santa Ana, California, 92705
Study site
Scarborough, Maine, 04074
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Seattle, Washington, 98104
Study site
St Louis, Missouri, 63110
Study site
Thousand Oaks, California, 91360

Other trials in United States

Canada11 sites

Study site
Calgary, Alberta, T2E 7C5
Study site
Chicoutimi, Quebec, G7H 5H6
Study site
London, Ontario, N6A 5K8
Study site
Montreal, Quebec, H1T 1C8
Study site
Montreal, Quebec, H2W 1R7
Study site
Saint Foy, Quebec, G1V 4M6
Study site
Sherbrooke, Quebec, J1H 5N4
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Toronto, Ontario, M5C 2T2
Study site
Toronto, Ontario, M5G 2C4
Study site
Vancouver, British Columbia, V6Z 1Y6
Study site
Winnipeg, Manitoba, R3A 1R9

Other trials in Canada

About this study

Official title: A Randomized, Double-Blind, Placebo-Controlled Study to Assess Efficacy and Safety of ISIS 301012 as Add-on Therapy in Heterozygous Familial Hypercholesterolemia Subjects With Coronary Artery Disease

Read the detailed description

Familial hypercholesterolemia (FH) is an autosomal dominant genetic disorder of lipoprotein metabolism characterized by markedly elevated LDL-C, premature onset of atherosclerosis and development of xanthomata. Patients with heterozygous familial hypercholesterolemia typically have total plasma cholesterol between 350 to 550 mg/dL and disease onset in their third and fourth decade.

Mipomersen (ISIS 301012) is an antisense drug that reduces a protein in the liver cells called apolipoprotein B (apo-B). Apo-B plays a role in producing low density lipoprotein cholesterol (the "bad" cholesterol) and moving it from the liver to one's bloodstream. High LDL-C is an independent risk factor for the development of coronary heart disease (CHD) or other diseases of blood vessels. It has been shown that lowering LDL-C reduces the risk of heart attacks and other major adverse cardiovascular events.

This study consisted of a 26-week treatment period and a 24-week post-treatment follow-up period (with the exception of patients who enrolled in the open-label extension study [Study 301012-CS6; NCT00694109]). The treatment period spanned the time during which the study treatment was administered until the later of the primary efficacy time point (PET) or 14 days beyond the last day of study drug administration. The post-treatment follow-up period began the day after completion of the treatment period and ended on the day of the patient's last contact date within the study.

Following treatment and Week 28 evaluations, eligible patients who tolerated study drug could elect to enroll in an open-label extension study (301012-CS6). Patients who were not eligible for or who elected not to enroll in the open-label extension study and patients who discontinued during the 26-week treatment period were followed in this study for an additional 24 weeks from administration of the last dose of study drug.

Conditions: Heterozygous Familial Hypercholesterolemia, Coronary Artery Disease
Being tested: mipomersen sodium, placebo
Keywords: familial hypercholesterolemia
Medical subject headings: Coronary Artery Disease, Hyperlipoproteinemia Type II

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

mipomersen sodiumDrug
Also called ISIS 301012, Kynamro™
200 mg /mL
placeboDrug
1 mL matching placebo

Study groups in NCT00706849
Study group	Role	What they receive
Mipomersen	Experimental	Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks. Receives: Drug: mipomersen sodium
Placebo	Placebo comparator	Participants received a placebo subcutaneous injection once a week for 26 weeks. Receives: Drug: placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) at the Primary Efficacy Time Point
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.
Measured: 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 at Baseline and at the Primary Efficacy Time Point
The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment.
Measured: 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).

Secondary outcomes (6)

Percent Change From Baseline in Apolipoprotein B at the Primary Efficacy Time Point
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.
Measured: 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 at Baseline and at the Primary Efficacy Time Point
The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment.
Measured: 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).
Percent Change From Baseline in Total Cholesterol at the Primary Efficacy Time Point
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.
Measured: 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 at Baseline and at the Primary Efficacy Time Point
The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment.
Measured: 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).
Percent Change From Baseline in Non-High-Density Lipoprotein Cholesterol at the Primary Efficacy Time Point
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.
Measured: 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 at Baseline and at the Primary Efficacy Time Point
The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment.
Measured: 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).

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Jul 2008May 2010

First posted30 Jun 2008 (estimated)
Study startJul 2008
Primary completionDec 2009
Study completionMay 2010

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (3)Papers the sponsor has linked to this study.
Other trials for Heterozygous Familial HypercholesterolemiaStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 3
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 124 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00706849
Sponsor's protocol ID: 301012-CS7
Acronym: RADICHOL II
Status last verified: Aug 2016
First submitted: 26 Jun 2008
Last updated: 9 Sep 2016
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

Publications

Stein EA, Dufour R, Gagne C, Gaudet D, East C, Donovan JM, Chin W, Tribble DL, McGowan M. Apolipoprotein B synthesis inhibition with mipomersen in heterozygous familial hypercholesterolemia: results of a randomized, double-blind, placebo-controlled trial to assess efficacy and safety as add-on therapy in patients with coronary artery disease. Circulation. 2012 Nov 6;126(19):2283-92. doi: 10.1161/CIRCULATIONAHA.112.104125. Epub 2012 Oct 11.
PubMed 23060426
Duell PB, Santos RD, Kirwan BA, Witztum JL, Tsimikas S, Kastelein JJP. Long-term mipomersen treatment is associated with a reduction in cardiovascular events in patients with familial hypercholesterolemia. J Clin Lipidol. 2016 Jul-Aug;10(4):1011-1021. doi: 10.1016/j.jacl.2016.04.013. Epub 2016 May 9.
PubMed 27578134
Santos RD, Raal FJ, Catapano AL, Witztum JL, Steinhagen-Thiessen E, Tsimikas S. Mipomersen, an antisense oligonucleotide to apolipoprotein B-100, reduces lipoprotein(a) in various populations with hypercholesterolemia: results of 4 phase III trials. Arterioscler Thromb Vasc Biol. 2015 Mar;35(3):689-99. doi: 10.1161/ATVBAHA.114.304549. Epub 2015 Jan 22.
PubMed 25614280

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Active, not recruitingObservational study
Assessment of the Prevalence of Steatotic Liver Disease Associated With Metabolic Dysfunction in Patients With Heterozygous Familial Hypercholesterolemia
Heterozygous Familial Hypercholesterolemia
Not yet recruitingPhase 1/2
EDIT-401 in Adults Who Require Additional Lowering of Low-Density Lipoprotein Cholesterol (LDL-C)
Heterozygous Familial Hypercholesterolemia (HeFH)
Recruiting
The ORIGIN-FH Study
Heterozygous Familial Hypercholesterolemia (HeFH), Homozygous Familial Hypercholesterolemia (HoFH)
RecruitingPhase 2/3
A Study of Enlicitide Decanoate (MK-0616, an Oral PCSK9 Inhibitor) in Children and Adolescents With Heterozygous Familial Hypercholesterolemia (MK-0616-029)
Heterozygous Familial Hypercholesterolemia (HeFH)
Not yet recruitingPhase 2
A Study of VERVE-102 (LY4636581) in Adults With or at High Risk for Cardiovascular Disease
Atherosclerosis, Cardiovascular Disease

All Heterozygous Familial Hypercholesterolemia trials

Study at a glance

From the registry record, last updated by the sponsor on 9 Sep 2016. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Randomized, Double-Blind, Placebo-Controlled Study to Assess Efficacy and Safety of ISIS 301012 as Add-on Therapy in Heterozygous Familial Hypercholesterolemia Subjects With Coronary Artery Disease
ClinicalTrials.gov ID: NCT00706849
Acronym / sponsor ID: RADICHOL II / 301012-CS7
Conditions: Heterozygous Familial Hypercholesterolemia, Coronary Artery Disease
Keywords: familial hypercholesterolemia
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 124 participants (actual)
Lead sponsor: Kastle Therapeutics, LLCIndustry (pharma, biotech, devices)
Collaborators: Ionis Pharmaceuticals, Inc.
Study start: Jul 2008
Primary completion: Dec 2009 (actual)
Study completion: May 2010 (actual)
First posted: 30 Jun 2008 (estimated)
Results: Posted 21 Mar 2013

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 2

Arm	Type	Receives
Mipomersen	Experimental	Drug: mipomersen sodiumParticipants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Placebo	Placebo comparator	Drug: placeboParticipants received a placebo subcutaneous injection once a week for 26 weeks.

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (6 listed)

Diagnosis of Heterozygous Familial Hypercholesterolemia (HeFH)
Diagnosis of Coronary Artery Disease (CAD)
Stable lipid-lowering therapy for 12 weeks
On maximally tolerated statin therapy with at least 1 statin at a dose greater than zero, per Investigator judgment
Stable low-fat diet for 8 weeks

Key exclusion criteria (2 listed)

Significant health problems in recent past including heart attack, stroke, coronary syndrome, unstable angina, heart failure, significant arrhythmia, orthostatic hypotension, uncontrolled hypertension, blood disorders, liver disease, cancer, or digestive problems
Receiving apheresis treatment or last apheresis treatment within 8 weeks

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
mipomersen sodiumAlso: ISIS 301012, Kynamro™200 mg /mL	Drug	Mipomersen
placebo1 mL matching placebo	Drug	Placebo

Primary outcome

Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) at the Primary Efficacy Time Point
Time frame: 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.
LDL Cholesterol at Baseline and at the Primary Efficacy Time Point
Time frame: 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.

6 secondary outcomes

Percent Change From Baseline in Apolipoprotein B at the Primary Efficacy Time Point
Time frame: 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 at Baseline and at the Primary Efficacy Time Point
Time frame: 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).
Percent Change From Baseline in Total Cholesterol at the Primary Efficacy Time Point
Time frame: 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 at Baseline and at the Primary Efficacy Time Point
Time frame: 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).
Percent Change From Baseline in Non-High-Density Lipoprotein Cholesterol at the Primary Efficacy Time Point
Time frame: 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 at Baseline and at the Primary Efficacy Time Point
Time frame: 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).

Results and safety

Results posted to the registry on 21 Mar 2013, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Placebo	41	41
Mipomersen	83	73

Primary outcome: Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) at the Primary Efficacy Time Point

Time frame: 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).. Mean (Standard Deviation). Unit: percentage of Baseline

Measure	Placebo n = 41	Mipomersen n = 82
Value	5.17 (18.02)	-28.02 (26.99)

Placebo vs Mipomersen: p <0.001, t-test, 2 sided

Population analysed: 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.

Primary outcome: LDL Cholesterol at Baseline and at the Primary Efficacy Time Point

Measure	Placebo n = 41	Mipomersen n = 82
Baseline	142.9 (51.6)	152.9 (48.7)
Primary efficacy time point	146.4 (43.4)	103.9 (33.0)

Population analysed: Full Analysis Set

6 secondary outcomes are reported in the full results.

Adverse events

Time frame: Up to Week 28 (2 weeks after last dose). Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Placebo	2 of 41 (4.9%)	38 of 41 (92.7%)	Not reported
Mipomersen	6 of 83 (7.2%)	83 of 83 (100%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 9 Sep 2016.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Results publication: PubMed 23060426Stein EA, Dufour R, Gagne C, Gaudet D, East C, Donovan JM, Chin W, Tribble DL, McGowan M. Apolipoprotein B synthesis inhibition with mipomersen in heterozygous familial hypercholesterolemia: results of a randomized, double-blind, placebo-controlled trial to assess efficacy and safety as add-on therapy in patients with coronary artery disease. Circulation. 2012 Nov 6;126(19):2283-92. doi: 10.1161/CIRCULATIONAHA.112.104125. Epub 2012 Oct 11.
Derived from this study: PubMed 27578134Duell PB, Santos RD, Kirwan BA, Witztum JL, Tsimikas S, Kastelein JJP. Long-term mipomersen treatment is associated with a reduction in cardiovascular events in patients with familial hypercholesterolemia. J Clin Lipidol. 2016 Jul-Aug;10(4):1011-1021. doi: 10.1016/j.jacl.2016.04.013. Epub 2016 May 9.
Derived from this study: PubMed 25614280Santos RD, Raal FJ, Catapano AL, Witztum JL, Steinhagen-Thiessen E, Tsimikas S. Mipomersen, an antisense oligonucleotide to apolipoprotein B-100, reduces lipoprotein(a) in various populations with hypercholesterolemia: results of 4 phase III trials. Arterioscler Thromb Vasc Biol. 2015 Mar;35(3):689-99. doi: 10.1161/ATVBAHA.114.304549. Epub 2015 Jan 22.

Related trials for Heterozygous Familial Hypercholesterolemia

Active, not recruitingObservational study
Assessment of the Prevalence of Steatotic Liver Disease Associated With Metabolic Dysfunction in Patients With Heterozygous Familial Hypercholesterolemia
Heterozygous Familial Hypercholesterolemia
Not yet recruitingPhase 1/2
EDIT-401 in Adults Who Require Additional Lowering of Low-Density Lipoprotein Cholesterol (LDL-C)
Heterozygous Familial Hypercholesterolemia (HeFH)
Recruiting
The ORIGIN-FH Study
Heterozygous Familial Hypercholesterolemia (HeFH), Homozygous Familial Hypercholesterolemia (HoFH)
RecruitingPhase 2/3
A Study of Enlicitide Decanoate (MK-0616, an Oral PCSK9 Inhibitor) in Children and Adolescents With Heterozygous Familial Hypercholesterolemia (MK-0616-029)
Heterozygous Familial Hypercholesterolemia (HeFH)
Not yet recruitingPhase 2
A Study of VERVE-102 (LY4636581) in Adults With or at High Risk for Cardiovascular Disease
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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Medical Monitor study director

Genzyme, a Sanofi Company

48 sites in 2 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing mipomersen sodium in people with Heterozygous Familial Hypercholesterolemia and Coronary Artery Disease, run by Kastle Therapeutics, LLC at 48 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 9 Sep 2016. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 48 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 21 Mar 2013). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) at the Primary Efficacy Time Point (time frame: 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 study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00706849, which also lists 3 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Sep 2016.