MedStar Health Begins Phase 3 MAGNITUDE Gene-Editing Study, Treats First U.S. Heart Patient

A groundbreaking study is underway at MedStar Washington Hospital Center, where the first U.S. heart patient has been enrolled to test a new gene-editing therapy for transthyretin amyloidosis with cardiomyopathy (ATTR-CM). This international clinical trial, named MAGNITUDE, aims to assess the safety and effectiveness of this innovative treatment for ATTR-CM, a lesser-known cause of heart failure.

The MAGNITUDE trial is a Phase 3, multinational, multicenter, double-blind, placebo-controlled study. It focuses on a one-time intravenous infusion of a gene-editing therapy in patients suffering from ATTR-CM. ATTR is a protein disorder where the transthyretin (TTR) protein accumulates in various parts of the body, notably the heart and nerves, leading to amyloidosis. When this condition affects the heart, it is referred to as ATTR-CM, a potentially fatal disease. The new treatment aims to halt the production of the abnormal TTR protein that triggers the disorder.

Participants in the study will be randomly assigned to receive either NTLA-2001, the investigational medication, or a placebo. NTLA-2001 uses the CRISPR/Cas9 gene-editing system to locate and deactivate the TTR gene in the liver, where most of the TTR protein is produced. This groundbreaking therapy is delivered as a single intravenous infusion.

Dr. Farooq Sheikh, the medical director of the Advanced Heart Failure Program at MedStar Washington Hospital Center and the principal investigator of the MAGNITUDE study, expressed excitement about the trial. "Our center is thrilled to enroll the first U.S. patient in this pivotal study, exploring a new treatment avenue for ATTR-CM," Sheikh stated. "The MedStar Health Infiltrative Cardiomyopathy/Advanced Heart Failure Program is dedicated to enhancing the lives of our patients locally and globally."

NTLA-2001 is the first CRISPR-based therapy to be administered intravenously for gene editing inside the human body. Early results from Phase 1 and 2 clinical trials indicated that NTLA-2001 consistently led to a significant and sustained reduction in TTR protein levels.

Globally, an estimated 50,000 individuals suffer from hereditary ATTR amyloidosis, while between 200,000 and 500,000 people live with wild-type ATTR amyloidosis, which predominantly affects the heart and leads to ATTR-CM. Currently, there is no known cure for these conditions.

The MAGNITUDE trial plans to enroll around 765 participants worldwide and is one of over 175 cardiovascular research studies conducted annually at MedStar Health.