A Phase IIB Study of NEOD001 versus Placebo to Treat Light Chain Amyloidosis in Previously Treated Patients with Impaired Heart Function

Full Title

A Phase 2b, Randomized, Double-blind, Placebo-controlled Study of NEOD001 in Previously Treated Subjects With Light Chain (AL) Amyloidosis Who have Persistent Cardiac Dysfunction


Amyloidosis is a disorder in which abnormal, misfolded proteins build up in tissues and organs, preventing them from working properly. Clumps of the abnormal misfolded proteins are called amyloid deposits. In patients with light chain amyloidosis, abnormal plasma cells in the bone marrow are responsible for producing a type of protein called an “immunoglobulin light-chain protein” that misfolds. Light chain amyloidosis may be treated with chemotherapy, but the organs affected may not recover because the amyloid deposits remain.

NEOD001 is an investigational drug that targets the amyloid deposits in tissues and organs. It has been shown in laboratory studies to reduce amyloid build-up and may improve abnormal organ function. In this study, patients with previously treated amyloidosis affecting the heart will be randomly assigned to receive either NEOD001 or a placebo. NEOD001 is given intravenously (by vein).


To be eligible for this study, patients must meet several criteria, including but not limited to the following:

  • Patients must have light chain amyloidosis and impaired heart function.
  • Patients must have received at least one prior regimen of chemotherapy for amyloidosis.
  • This study is for patients age 18 and older.

For more information about this study and to inquire about eligibility, please contact Dr. Heather Landau at 212-639-8808.