During the latest FCS patient-caregiver virtual meeting (link) hosted by the FCS Foundation, Ionis introduced the BALANCE study to the meeting attendance. Today, Ionis officially announced the initiation of the global Phase 3 BALANCE study via a press release (link).
According to the announcement, the BALANCE study is for AKCEA-APOCIII-LRX in patients with familial chylomicronemia syndrome. It will be “a global, multi-center, randomized, double-blind, placebo-controlled study enrolling up to 60 patients (age 18 and over) with FCS.” The study only recruits FCS patients and requires about 74 weeks to complete.
The BALANCE study has a page on clinicaltrials.gov which listed the details about the drug, the inclusion and exclusion criteria, and outcome measures. Please visit the page for more information. People who are interested in participating in the study can reach out to Ionis via phone or email listed below (adapted from clinicaltrials.gov):
|Contact: Ionis Pharmaceuticals
The HEART ACT:
The FCS Foundation, joined with the HAYSTACK Project and other leaders from the Rare Disease Community, created a bill called the HEART Act (Helping Experts Accelerate Rare Treatments), to ask Congress to make some important changes in the process we use to review and approve drugs to treat rare diseases in the U.S. Please go HERE or HERE and send messages the Congress and ask them to vote for the bill. Thanks!