Amyloidosis

2013-08 A Phase 3, Randomized, Controlled, Open-Label, Multicenter, Safety and Efficacy Study of Dexamethasone Plus MLN9708 or Physician’s Choice of Treatment Administered to Patients With Relapsed or Refractory Systemic Light Chain (AL) Amyloidosis.

Principle Investigator: Dr. Maurizio Zangari

Primary Objectives:

To determine whether dexamethasone plus MLN9708 improves hematologic response (PR +VGPR + CR) versus a physician’s choice of a chemotherapy regimen as selected from the list of offered treatment options in patients diagnosed with relapsed or refractory AL amyloidosis

To determine whether dexamethasone plus MLN9708 improves 2-year vital organ (that is, heart or kidney) deterioration and mortality rate versus a physician’s choice of a chemotherapy regimen as selected from the list of offered treatment options in patients diagnosed with relapsed or refractory AL amyloidosis. Cardiac deterioration is defined as the need for hospitalization for heart failure. Kidney deterioration is defined as progression to end-stage renal disease (ESRD) with the need for maintenance dialysis or renal transplantation.

Key secondary objectives:

To determine overall survival (OS)

To determine the complete hematologic response rate (CR)

Eligibility:

Adult patients with biopsy-proven systemic AL amyloidosis with relapsed or refractory disease.