On July 31, the Amyloidosis Research Consortium (ARC) held a webinar to discuss the many clinical trials currently underway for different types of amyloidosis, including transthyretin amyloid cardiomyopathy (ATTR-CM).
During the webinar, Kristen Hsu, executive director of research at ARC, provided updates on some of the clinical trials that are recruiting patients, collecting data or being planned for the future.
Clinical trials currently recruiting
At the moment, several studies are actively seeking participants, Hsu said.
Read more about ATTR-CM therapies
The ACT-EARLY study is open to individuals who carry a mutation known to cause ATTR but do not yet have symptoms. Participants will be given acoramidis twice daily to see whether treatment prevents or delays disease development.
TRITON-CM will study nucresiran, a therapy that silences the transthyretin gene. Patients with ATTR-CM both with and without a confirmed mutation known to cause the disease can enroll in the study. Participants cannot have received any transthyretin depleters previously, although transthyretin stabilizer use is allowed.
The MAGNITUDE study will aim to evaluate the safety and effectiveness of nex-z, a gene editing therapy administered through a one-time infusion, in patients with ATTR-CM. Enrollment is currently open to patients who have never received vutrisiran and have not taken patisiran, inotersen or eplontersen within 12 months of entering the study.
Clinical trials underway
Hsu also highlighted some key trials with upcoming milestones to be aware of.
Results for the CARDIO-TTRansform study are expected some time in 2026. This trial is evaluating eplontersen, a transthyretin gene silencer, in patients with ATTR-CM.
Coramitug, a drug that depletes amyloid buildups, is also being studied in patients with ATTR-CM. The results of a Phase 2 trial of coramitug should be released later this year.
Lastly, the DepleTTR-CM study is investigating ALXN2220, another transthyretin depleter therapy. This study recently completed recruitment, with a total of 1,158 patients enrolled. The final patient is expected to complete treatment roughly two years from now, with results to be analyzed shortly thereafter, Hsu said.
Other trials and future steps
In addition to the trials described in the webinar, there are a number of other studies being conducted to improve the diagnosis of ATTR. These include REVEAL and CArdiag, which are looking into tracers to help diagnose different types of cardiac amyloidosis, including ATTR-CM. These studies are open to individuals with a suspected diagnosis of cardiac amyloidosis.
Because many of the existing studies are primarily examining safety and efficacy, some companies are planning future studies that will more closely analyze the impacts of certain drugs on symptoms and quality of life.
“ARC developed an online tool called MAP [My Amyloidosis Pathfinder] to help patients with amyloidosis get connected to treatment centers and clinical trials specific to their type of amyloidosis and symptoms,” Hsu added. “It’s a great way to stay informed and ultimately help advance research towards better treatment and a cure for amyloidosis.”
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