A Research Study to Look at How a New Medicine Called NNC6019-0001 Works and How Safe it is for People Who Have Heart Disease Due to Transthyretin (TTR) Amyloidosis

Summary

This study is testing a potential new medicine, NNC6019-0001, for people who have a heart disease due to TTR amyloidosis.The study will look at if this medicine can reduce the symptoms of a heart disease due to TTR amyloidosis, such as heart failure. Participants will either get NNC6019-0001 (apotential new medicine) or placebo (a medicine which has no effect on the body). Which treatment participants get is decided by chance. The chance of getting NNC6019-0001 is two times higher than getting placebo. NNC6019-0001 is not yet approved in any country or region in the world. It is a new medicine that doctors cannot prescribe yet. Participants will get an infusion of the study medicine 13 times, once every 4 weeks. The study will last for about 64 weeks after the first dose of medicine. Participants cannot participate in this study if they have a heart disease other than a heart disease due to TTR amyloidosis.

Timeline

Start

2022-08-02

Primary completion

2025-02-17

Completion

2025-05-13

Publications

• Fontana M, Garcia-Pavia P, Grogan M, Shah SJ, Engelmann MDM, Hovingh GK, Kristen AV, Lim-Watson MZ, Malling B, Kar S, Revanna M, Sarswat N, Tsujita K, Alexander KM, Maurer MS. Coramitug, a Humanized Monoclonal Antibody for the Treatment of Transthyretin Amyloid Cardiomyopathy: A Phase 2, Randomized, Multicenter, Double-Blind, Placebo-Controlled Trial. Circulation. 2026 Jan 27;153(4):214-225. doi: 10.1161/CIRCULATIONAHA.125.077304. Epub 2025 Nov 10.
• Suhr OB, Grogan M, Silva AMD, Karam C, Garcia-Pavia P, Drachman B, Zago W, Tripuraneni R, Kinney GG. PRX004 in variant amyloid transthyretin (ATTRv) amyloidosis: results of a phase 1, open-label, dose-escalation study. Amyloid. 2025 Mar;32(1):14-21. doi: 10.1080/13506129.2024.2420809. Epub 2024 Oct 29.

Drugs

Indications