Amyloidosis

• Act Early: A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled Study of Acoramidis for Transthyretin Amyloidosis Prevention in the Young. Andrew Kates, MD. Eidos Therapeutics.
  CURRENTLY ENROLLING
  view on clinicaltrials.gov
• AG10 OLE:  An open-label extension and safety monitoring study of acoramidis in participants with symptomatic transthyretin amyloid cardiomyopathy who completed the phase 3 ATTRIBUTE-CM trial. Joshua Mitchell, MD. Eidos Therapeutics.
  view on clinicaltrials.gov
• An Open-Label Extension Study to Assess the Safety and Efficacy of Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy (ATTR Amyloidosis with Cardiomyopathy). Joshua Mitchell, MD. Alnylam Pharmaceuticals.
  view on clinicaltrials.gov
• ATTRIBUTE-CM:  Phase 3 study on the efficacy and safety of AG10 in subjects with symptomatic transthyretin amyloid cardiomyopathy. Joshua Mitchell, MD. Eidos Therapeutics.
  view on clinicaltrials.gov
• CARDIOTTRANSFORM:Phase 3 study evaluating the efficacy and safety of ION-682884 in patients with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). Joshua Mitchell, MD. Ionis pharmaceuticals, Inc.
  view on clinicaltrials.gov
• DepleTTR-CM: A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of Amyloid Depleter ALXN2220 in Adult Participants with Transthyretin Amyloid Cardiomyopathy (ATTR-CM). Joshua Mitchell, MD. Alexion Pharmaceuticals.
  view on clinicaltrials.gov
• MAESTTRO: A Non-interventional, Prospective, Multi-country Study Collecting Real-world Data on the Characteristics, Treatment Patterns, and Outcomes of Patients with Amyloid Transthyretin (ATTR) Amyloidosis. Joshua Mitchell, MD. AstraZeneca AB.
  CURRENTLY ENROLLING
  view on clinicaltrials.gov
• MAGNITUDE: A Phase 3, Multinational, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of NTLA-2001 in Participants with Transthyretin Amyloidosis with Cardiomyopathy.
  Pending enrollment, expected starting August 2025
  view on clinicaltrials.gov
• PATISIRAN EXPANDED ACCESS:  Expanded access protocol to provide patisiran to patients with transthyretin-mediated amyloidosis with cardiomyopathy. Joshua Mitchell, MD. Alnylam Pharmaceuticals, Inc.
  view on clinicaltrials.gov
• REVEAL- A Phase 3, Open-Label, Multicenter Study of I-124 evuzamitide as an Imaging Agent for the Diagnosis of Cardiac Amyloidosis using Positron Emission Tomography Computed Tomography (PET/CT).
  CURRENTLY ENROLLING
  view on clinicaltrials.gov
• Triton: TRITON-CM: A Phase 3 Global, Randomized, Double-Blind,Placebo-Controlled Study to Evaluate the Efficacy and Safety of Nucresiran in Patients with Transthyretin-Mediated Amyloidosis with Cardiomyopathy (ATTR amyloidosis with cardiomyopathy). Joshua Mitchell, MD. Alnylam Pharmaceuticals.
  Pending enrollment August 2025.