What side effects are associated with this type of intervention?

Common treatments for Chylomicronemia Syndrome that inhibit APOC3 to reduce triglyceride levels, such as Plozasiran, are still under investigation. However, similar treatments often include side effects like injection site reactions, flu-like symptoms, and potential liver enzyme elevations. Other general side effects for lipid-lowering therapies can include gastrointestinal issues, muscle pain, and increased risk of infections. It is important to monitor these side effects closely and discuss them with a healthcare provider.

What prior FDA approvals exist?

The FDA has approved several treatments for Chylomicronemia Syndrome, focusing on reducing triglyceride levels. One notable drug is volanesorsen, an antisense oligonucleotide that inhibits the production of apolipoprotein C-III (APOC3), similar to the mechanism of Plozasiran. Volanesorsen has been approved for familial chylomicronemia syndrome (FCS) to help manage severe hypertriglyceridemia. Other treatments include dietary modifications and fibrates, although these are less specific in targeting APOC3.

What other types of research exist?

Promising alternatives to Plozasiran for Chylomicronemia Syndrome patients include mipomersen, an antisense therapy targeting apoB messenger RNA, and gene editing technologies like CRISPR-Cas9, which are being explored for lipid disorders. Additionally, novel lipid-lowering drugs in clinical trials may offer new options.

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Antisense Oligonucleotide

ARO-APOC3 for Chylomicronemia Syndrome (PALISADE Trial)

Phase 3

Waitlist Available

Research Sponsored by Arrowhead Pharmaceuticals

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Fasting TG ≥ 10 mmol/L (≥ 880 mg/dL) at screening refractory to standard lipid lowering therapy

Diagnosis of FCS

Must not have

On treatment with human immunodeficiency virus (HIV) antiretroviral therapy

Active pancreatitis within 12 weeks before Day 1

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

Pivotal Trial

Summary

This trial is testing a new drug called plozasiran in adults with a rare genetic disorder that causes very high blood fat levels. The drug aims to lower these fat levels by reducing a specific protein in the blood.

Who is the study for?

Adults with familial chylomicronemia syndrome (FCS) who are willing to follow dietary advice and have very high fasting triglyceride levels. They must use effective contraception, not be pregnant or breastfeeding, and cannot have had major surgery or uncontrolled conditions like hypertension recently.

What is being tested?

The trial is testing ARO-APOC3 against a placebo in adults with FCS. Participants will receive either the drug or placebo through subcutaneous injections over a period, followed by an open-label extension where all get ARO-APOC3.

What are the potential side effects?

Potential side effects of ARO-APOC3 may include reactions at the injection site, changes in liver function tests, possible allergic responses, and other systemic symptoms that will be closely monitored throughout the study.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My fasting triglycerides are very high despite taking standard treatments.

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I have been diagnosed with Familial Chylomicronemia Syndrome (FCS).

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I am not pregnant or breastfeeding.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am currently on HIV medication.

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I have not had pancreatitis in the last 3 months.

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I have heart failure that affects my daily activities.

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My blood pressure is not controlled by medication.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: ARO-APOC3 (plozasiran)Experimental Treatment1 Intervention

4 doses of plozasiran by subcutaneous (sc) injection (randomized period) 8 doses of plozasiran by sc injection (open-label period)

Group II: PlaceboPlacebo Group1 Intervention

calculated volume to match active treatment by sc injection (randomized period)

0 / 7Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Chylomicronemia Syndrome treatments, such as APOC3 inhibitors like Plozasiran, work by reducing the production of apolipoprotein C-III, a protein that inhibits the breakdown of triglycerides. By lowering APOC3 levels, these treatments enhance the clearance of triglycerides from the bloodstream. This mechanism is crucial for patients as it directly addresses the impaired triglyceride metabolism, reducing the risk of severe complications like pancreatitis.

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