This is a global, multicenter, Phase 2b, randomized, double-blind, placebo-controlled, two-arm, parallel-group efficacy and safety study of NEOD001 as a single agent administered intravenously in adults with AL amyloidosis who had a hematologic response to previous treatment for their amyloidosis (e.g., chemotherapy, autologous stem cell transplant [ASCT]) and have persistent cardiac dysfunction.
- Must have confirmed diagnosis of systemic AL amyloidosis
- Must have had at least one prior systemic chemotherapeutic regimen for AL amyloidosis
- Must have cardiac involvement
- Must not have received Plasma cell directed chemotherapy within 6 months
- Must not have received autologous stem cell transplant (ASCT) within 12 months
- Must not have non-AL amyloidosis
Subjects will be asked to provide blood samples in addition to standard of care labs at screening, every first day of a cycle, and at end of treatment. These samples will range in volume from about 3 tablespoons in volume to 1.5 tablespoons. Subjects will be asked to collect urine for 24 hours for a urine analysis. In addition, participants will be asked to fill out quality of life questionnaires and to do a six minute walk test. Treatment infusions will occur over 60 minutes, and it is estimated that subjects will be involved in the study for about 14 months.