A study to assess the efficacy and safety of ALXN2220 in participants with transthyretin amyloid cardiomyopathy (ATTR-CM)

Zusammenfassung der Studie

This study will investigate the investigational substance ALXN2220 (a so-called amyloid-reducing agent) in patients with transthyretin amyloidosis (ATTR, deposits of misfolded proteins) with cardiomyopathy (CM, disease of the heart muscle), referred to as ATTR-CM. The aim is to determine how effective and safe ALXN2220 is. Patients with ATTR-CM experience symptoms of heart muscle weakness (e.g., shortness of breath or difficulty exercising) and many die within a few years of the disease. Current treatment options for ATTR-CM include TTR stabilizers (medications that reduce deposits of misfolded proteins in the heart) and therapies to treat the symptoms of heart muscle weakness (e.g., diuretics, medications that promote urine production and its excretion). However, there is currently no treatment that can remove already deposited misfolded proteins that are present in the heart. Therefore, the only option for patients with advanced ATTR-CM may be a heart transplant. ALXN2220 works differently than the currently available treatment options. ALXN binds to the deposited misfolded proteins and activates the immune system, hoping that these will be removed from the heart and other organs, and that patients will experience an improvement in their heart muscle weakness and other symptoms. About 1000 women and men aged between 18 and 90 years will participate in this study worldwide, including about 10 from Switzerland.

(BASEC)

Untersuchte Intervention

All participants will be randomly assigned to a treatment in a 2:1 ratio.

The probability of receiving the investigational substance ALXN2220 is 2 out of 3 (approximately 66%). The probability of receiving the placebo (a placebo looks like the experimental drug but does not contain the active ingredient in the investigational substance) is 1 out of 3 (approximately 33%).

Before treatment with ALXN2220 or the placebo can begin, a screening will be conducted to verify that all inclusion criteria for participation in the study are met.

Participation in this study will last 2-4 years with visit appointments approximately every 4 weeks. Each visit appointment may vary in duration, from a short visit of about 4 hours to about 6 hours for longer visits.

During these visits, ALXN2220 (experimental group) or the placebo (control group) will be administered by infusion (controlled administration or the introduction of larger volumes of fluid into the body).

(BASEC)

Untersuchte Krankheit(en)

Adults with transthyretin amyloidosis with cardiomyopathy (ATTR-CM)

(BASEC)

Sponsor

AstraZeneca AG, Switzerland

(BASEC)

Name der bewilligenden Ethikkommission (bei multizentrischen Studien nur die Leitkommission)

Ethikkommission Zürich

(BASEC)

Datum der Bewilligung durch die Ethikkommission

19.04.2024

(BASEC)

Ergebnisse der Studie