Treatment Protocol of Plozasiran in Adults and Adolescents With FCS

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

AVAILABLE

Study Classification

EXPANDED_ACCESS

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

This is a treatment program for the use of plozasiran in adults (AROAPOC3-EAP-002) and adolescents (AROAPOC3-EAP-003) with familial chylomicronemia syndrome (FCS). The program will enroll eligible patients ≥15 years of age, with fasting triglycerides ≥880 mg/dL (≥10 mmol/L) that is not adequately controlled with standard lipid-lowering therapy, and with a diagnosis of FCS. Patients will receive 25 mg of plozasiran by subcutaneous (sc) injection on Day 1 and every three months for a total of 5 injections. The duration of the program is 15 months.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Familial Chylomicronemia

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Plozasiran

25 mg ARO-APOC3 by sc injection administered by healthcare professional

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

ARO-APOC3

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* Fasting triglycerides (TG) ≥ 880mg/dL that is not sufficiently controlled on standard lipid-lowering therapy
* Established diagnosis of FCS based on documented history of fasting TG levels in excess of 1000 mg/dL on repeated testing (for at least 3 prior occasions), and at least one of the following: a supportive genetic test, documented history of recurrent episodes of acute pancreatitis not caused by alcohol or cholelithiasis, documented history of recurrent hospitalizations for severe abdominal pain without other explainable cause, documented history of childhood pancreatitis, family history of hypertriglyceridemia-induced pancreatitis
* Willing to follow dietary counseling based on local standard of care, consistent with an intake of ≤ 20 g of fat per day
* If on medications for management of type 2 diabetes the dosing regimen must be stable.
* Participants of childbearing potential must agree to use a highly effective form of contraception in addition to a male condom during the program and for at least 90 days after the last dose of plozasiran

Exclusion Criteria

* Diabetes mellitus with any of the following at Day 1: newly diagnosed within the past 24 weeks, HbA1c ≥9.0% within the past 4 weeks, meaningful medical events relating to poor glycemic control, changes in basal insulin regimen of more than =/- 10 units within 12 weeks if insulin-dependent
* Clinical evidence of primary hypothyroidism, primary subclinical hypothyroidism, or secondary hypothyroidism
* History of bleeding diathesis or coagulopathy
* Current diagnosis of nephrotic syndrome
* Eligible to receive any commercially available FDA-approved therapeutic for treatment of FCS unless proven to be ineffective or judged inappropriate by the treating physician
* History of acute coronary syndrome events (adults with FCS only)
* New York Heart Association Class III or IV heart failure or last known ejection fraction of \<30% (adults with FCS only)

Minimum Eligible Age

15 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Arrowhead Pharmaceuticals, Inc.

Pasadena, California, United States

Site Status AVAILABLE

Countries

Review the countries where the study has at least one active or historical site.

United States

Central Contacts

Reach out to these primary contacts for questions about participation or study logistics.

Arrowhead Medical

Role: CONTACT

Phone: 626-304-3400

Email: [email protected]

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

AROAPOC3-EAP-003

Identifier Type: OTHER

Identifier Source: secondary_id

AROAPOC3-EAP-002

Identifier Type: -

Identifier Source: org_study_id