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
  • 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.
  • 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.
    CURRENTLY ENROLLING
  • APOLLO-B:  Phase 3 study evaluating the efficacy and safety of Patisiran in patients with transthyretin amyloidosis with cardiomyopathy. Joshua Mitchell, MD. Alnylam Pharmaceuticals.
  • ATTRIBUTE-CM:  Phase 3 study on the efficacy and safety of AG10 in subjects with symptomatic transthyretin amyloid cardiomyopathy. Joshua Mitchell, MD. Eidos Therapeutics.
  • 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.
  • 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.
    CURRENTLY ENROLLING
  • 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
  • PATISIRAN EXPANDED ACCESS:  Expanded access protocol to provide patisiran to patients with transthyretin-mediated amyloidosis with cardiomyopathy. Joshua Mitchell, MD. Alnylam Pharmaceuticals, Inc.