- RecruitingPHASE3
MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)
This study looks at a new treatment called NTLA-2001 for a heart condition called transthyretin amyloidosis with cardiomyopathy (ATTR-CM). It compares a single dose of NTLA-2001 with a dummy drug (placebo) to see if it's safe and effective for people with this condition.
For: Transthyretin Amyloidosis (ATTR) With CardiomyopathyUnited States · Argentina · Australia - Ongoing, recruitingHuman Pharmacology (Phase I)- First administration to humans
Phase 1 Two-Part (Open-label, Single Ascending Dose (Part 1) and Open-label, Single Dose Expansion (Part 2)) Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN).
This study is testing a new medicine called NTLA-2001 for people with a rare condition called hereditary transthyretin amyloidosis (ATTRv-PN) that affects nerves. We want to see if the medicine is safe, how the body handles it, and if it has any effects on the condition.
For: Hereditary Transthyretin Amyloidosis with Polyneuropathy (ATTRv-PN)France · Sweden