CompletedPhase 2

Phase 2 Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Chylomicronemia Syndrome (FCS)

Canada3 participantsStarted 2017-12-21

Plain-language summary

This is a single center, open-label study to evaluate the efficacy of AKCEA-ANGPTL3-LRx for reduction of triglyceride (TG) levels in participants with FCS.

Who can participate

Age range

18 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.

Key Inclusion Criteria: * Genetically confirmed chylomicronemia syndrome. * Fasting triglycerides greater than or equal to (\>=) 750 milligrams per deciliter (mg/dL) \[8.4 millimoles per liter (mmol/L)\] at Screening. Key Exclusion Criteria: * Diabetes mellitus if newly diagnosed or if glycated hemoglobin (HbA1c) \>= 9.0%. * Active pancreatitis within 2 weeks of screening. * Acute coronary syndrome within 6 months of screening. * Major surgery within 3 months of screening. * Treatment with Glybera therapy within 2 years of screening. * Previous treatment with AKCEA-ANGPTL3-LRx. * Have any other conditions in the opinion of the investigator which could interfere with the patient 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

Absolute Change From Baseline to Month 3 in Fasting Triglycerides (TG)

Timeframe: Baseline to Month 3

Percent Change From Baseline to Month 3 in Fasting Triglycerides (TG)

Timeframe: Baseline to Month 3

Trial details

NCT IDNCT03360747

SponsorAkcea Therapeutics

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2018-06-12

Results submitted2020-12-11

View on ClinicalTrials.gov More Familial Chylomicronemia Syndrome trials

Who can participate

Age range

18 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.