Frequently Asked Questions
What is Familial Chylomicronemia Syndrome (FCS)?
FCS is a rare genetic disorder characterized by Lipoprotein Lipase Deficiency (LPLD), which prevents the body from breaking down fats, such as triglycerides (TGs), causing a build-up of TGs and dietary fats in the body. Eating even a little fat can cause a person with FCS to become ill. When the body has sustained high TG levels and is unable to digest fats, FCS patients are at risk for pancreatitis. Chronic symptoms include back pain and abdominal pain. Other symptoms include neurological problems and blood that appears fatty or “milky” when drawn. Left undiagnosed, FCS can have serious, life-threatening complications.
What is the BALANCE study?
BALANCE is a clinical study of an investigational antisense drug for treating Familial Chylomicronemia Syndrome (FCS). The study will help determine the safety and effectiveness of the potential new treatment. The drug is an antisense therapy for Familial Chylomicronemia Syndrome (FCS), and is designed to target and reduce the activity of a protein in the body. Targeting this protein may help break down dietary fat that can lower triglycerides in the blood and potentially help improve a patient’s quality of life for people living with FCS.
What is the investigational drug used in the BALANCE study?
This Phase 3 study will evaluate the efficacy of AKCEA‑APOCIII‑LRx on the percent change in fasting triglycerides (TGs). The drug is based on antisense technology, which is a therapy for reducing disease-causing proteins before they even begin. The study therapy (called AKCEA‑APOCIII‑LRx) is designed to target and reduce the activity of a protein in the body. Targeting this protein may help break down dietary fat and a clinically relevant reduction in triglycerides. The study drug therapy (investigational drug or placebo) will be administered as a subcutaneous injection (an injection under your skin).
What is an antisense therapeutic?
An antisense therapeutic is an RNA-targeted therapeutic that targets the root cause of the disease and aims to reduce disease-causing proteins before they are even created.
How long is the BALANCE study?
Individual participation in the BALANCE study will take place over 16 months. This time consists of a 12-month long study therapy period, and approximately a 3-month follow-up period.
What happens during the screening period?
During the screening period, you will be asked about your health and medical history. You’ll also have complimentary physical examinations and laboratory tests, if needed, to make sure you are healthy enough and ready to participate. If you choose to join the study, you will start a low-fat diet (20 grams of fat per day or less). You will remain on this diet for at least two weeks before proceeding to the next step in the study. You will also stay on this diet for the duration of the study. Study staff will provide counseling on how to maintain a low-fat diet throughout the study.
Because this is a research study, if, after you’ve completed some or all of the examinations required during the screening period, you may or may not be eligible to participate in the study. We will discuss with you the reasons why.
Will everyone who participates in the study receive the study drug?
If you are eligible and agree to participate in this study, you will either receive the study drug or placebo during the treatment period. A placebo is a substance that looks like and is taken the same way as the real drug, but has no active ingredients.
In clinical trials, a placebo is given to some participants so that the two groups—for BALANCE, it’s those taking the investigational drug vs. those taking the placebo—can be compared to show whether the drug being studied is safe and is effective. A placebo can also help researchers determine whether certain changes during the study happen by chance or actually in response to the investigational drug.
At the end of this study you may have the option to participate in an open label extension study where every participant will receive the investigational drug.
What precautions are being taken to help protect the health of at-risk study volunteers?
During the BALANCE study, your health is our number one priority. All medical guidelines are to be followed in study clinics including the use of gloves, properly preparing needles, and disposing of all materials. Masks, safety goggles, or face shields may also be used for added protection.
Will I be reimbursed for transportation, meals and parking?
All study participants may be reimbursed for miles traveled, parking, and a meal allowance for the days associated with the study visits.