A Phase III Study of Standard Chemotherapy with or without NEOD001 Patients with Newly Diagnosed Light Chain Amyloidosis

Full Title

A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled, 2-Arm, Efficacy and Safety Study of NEOD001 Plus Standard of Care vs. Placebo Plus Standard of Care in Subjects with Light Chain (AL) Amyloidosis

Purpose

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.

The purpose of this study is to see if adding an investigational drug called NEOD001 (which targets the amyloid deposits in tissues and organs) to standard chemotherapy is more effective than standard chemotherapy alone in patients with newly diagnosed light chain amyloidosis. Patients in this study will be randomly assigned to receive standard chemotherapy (the drugs cyclophosphamide, bortezomib, and dexamethasone) with either NEOD001 or a placebo (inactive drug).

NEOD001 is an antibody that is given intravenously (by vein). Cyclophosphamide is given intravenously, bortezomib is given intravenously or by subcutaneous (under the skin) injection, and dexamethasone is taken orally (by mouth).

Eligibility

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

  • Patients must be newly diagnosed with light chain amyloidosis and may not have received prior treatment.
  • Patients planning to have an autologous stem cell transplant may not participate.
  • 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.

Protocol

15-250

Phase

III

Investigator

Co-Investigators