CompletedPhase 3

An Open-label Extension Study to Assess the Long-term Safety and Efficacy of ISIS 301012 (Mipomersen) in Patients With Familial Hypercholesterolemia or Severe-Hypercholesterolemia

United States, Brazil, Canada144 participantsStarted 2008-04

Plain-language summary

To evaluate the safety and efficacy of extended dosing with mipomersen (ISIS 301012) in participants with familial hypercholesterolemia or severe hypercholesterolemia on lipid-lowering therapy who had completed either the 301012-CS5 (NCT00607373), 301012-CS7 (NCT00706849), 301012-CS17 (NCT00477594) or MIPO3500108 (NCT00794664) clinical drug trials.

Who can participate

Age range

12 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Satisfactory completion of dosing in their initial study (Protocol 301012-CS5 \[NCT00607373\], 301012-CS7 \[NCT00706849\], 301012-CS17 \[NCT00477594\], or MIPO3500108 \[NCT00794664\]) Exclusion Criteria: * Had any new condition or worsening of existing condition which in the opinion of the Investigator would make the participant unsuitable for enrollment, or could interfere with the participant participating in or completing the study

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Percent Change From Baseline in Low Density Lipoprotein Cholesterol (LDL-C)

Timeframe: Baseline up to Week 234; 24 weeks post treatment (up to 4.5 years)

Percent Change From Baseline in Apolipoprotein B (Apo B)

Timeframe: Baseline up to Week 234; 24 weeks post treatment (up to 4.5 years)

Percent Change From Baseline in Total Cholesterol

Timeframe: Baseline up to Week 234; 24 weeks post treatment (up to 4.5 years)

Percent Change From Baseline in Non High Density Lipoprotein Cholesterol (Non-HDL-C)

Timeframe: Baseline up to Week 234; 24 weeks post treatment (up to 4.5 years)

Trial details

NCT IDNCT00694109

SponsorKastle Therapeutics, LLC

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2014-09

Results submitted2015-09-11

View on ClinicalTrials.gov More Lipid Metabolism, Inborn Errors trials

Plain-language summary

Who can participate

Age range

12 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Percent Change From Baseline in Low Density Lipoprotein Cholesterol (LDL-C)

Timeframe: Baseline up to Week 234; 24 weeks post treatment (up to 4.5 years)

Percent Change From Baseline in Apolipoprotein B (Apo B)

Timeframe: Baseline up to Week 234; 24 weeks post treatment (up to 4.5 years)

Percent Change From Baseline in Total Cholesterol

Timeframe: Baseline up to Week 234; 24 weeks post treatment (up to 4.5 years)

Percent Change From Baseline in Non High Density Lipoprotein Cholesterol (Non-HDL-C)

Timeframe: Baseline up to Week 234; 24 weeks post treatment (up to 4.5 years)