Pelacarsen for Aortic Stenosis

This trial tests a new treatment called pelacarsen (TQJ230) to determine if it can slow the progression of calcific aortic valve stenosis, a condition where the heart valve narrows and hardens. Participants will receive either a pelacarsen injection or a placebo once a month. The trial aims to assess pelacarsen's safety and effectiveness compared to a placebo. Suitable candidates for this trial include those with mild or moderate aortic valve stenosis, high levels of a specific blood component called Lp(a), and who are already managing their cardiovascular risk factors. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

The trial protocol does not specify if you need to stop taking your current medications. However, you must be optimally treated for existing cardiovascular risk factors, which might mean continuing certain medications.

Research has shown that pelacarsen has been tested for its ability to lower lipoprotein(a) levels. These studies found that pelacarsen can safely reduce these levels by 35–80%. Participants did not experience major safety issues, suggesting that the treatment is generally well-tolerated.

Unlike standard treatments for aortic stenosis, which often include surgical valve replacement or balloon valvuloplasty, Pelacarsen (TQJ230) offers a unique approach by targeting lipoprotein(a), a specific type of cholesterol that may contribute to the worsening of aortic stenosis. Researchers are excited about Pelacarsen because it is administered via a simple monthly subcutaneous injection, which could be more convenient and less invasive compared to surgical options. By potentially reducing lipoprotein(a) levels, Pelacarsen aims to address an underlying cause of the disease, rather than just alleviating symptoms or repairing existing damage.

Research shows that Pelacarsen, which participants in this trial may receive, can significantly lower levels of lipoprotein(a) (Lp(a)), linked to heart valve issues. Previous studies demonstrated that it safely reduced Lp(a) by 35–80%. Lowering Lp(a) is believed to slow the progression of calcific aortic valve stenosis, a condition where the heart valve becomes narrow and stiff. Pelacarsen is administered as a monthly injection under the skin. These early findings suggest it could effectively treat aortic stenosis by targeting and reducing Lp(a) levels.¹²³⁵⁶