Plozasiran Treatment for Adults and Adolescents with Familial Chylomicronemia Syndrome
This study aims to provide Plozasiran treatment to adults and adolescents diagnosed with Familial Chylomicronemia Syndrome, a rare genetic disorder.
Data Collection
Hyperlipidemias+8
+ Hyperlipoproteinemias
+ Lipid Metabolism, Inborn Errors
Recruiting
Available upon a request by a licensed MDSummary
This study focuses on using a drug called plozasiran to treat adults and adolescents diagnosed with Familial Chylomicronemia Syndrome (FCS), a condition that causes very high triglyceride levels in the blood. The study is designed for patients aged 15 years and older who have fasting triglycerides levels of 880 mg/dL or higher, and whose condition is not well-managed with standard lipid-lowering therapy. The main goal of this study is to evaluate if plozasiran can help improve the management of FCS. During the study, participants will receive 25 mg of plozasiran via a subcutaneous injection, which is a type of injection given under the skin. The first injection will be given on Day 1, and then every three months after that, making a total of 5 injections. The study will last for 15 months. The effects of plozasiran on FCS symptoms and overall health will be closely monitored throughout this period.
Protocol
This section provides details of the study plan, including how the study is designed and what the study is measuring.Eligibility
Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.Any sex
Biological sex of participants that are eligible to enroll.Over 15 Years
Range of ages for which participants are eligible to join.Conditions
Pathology
Criteria
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) * History of stroke, transient ischemic attack, or peripheral artery disease within 24 weeks of first dose (adults with FCS only) Note: Additional inclusion/exclusion criteria may apply per protocol
Study Centers
These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.This study has 1 location
Arrowhead Pharmaceuticals, Inc.
Pasadena, United StatesOpen Arrowhead Pharmaceuticals, Inc. in Google Maps