Clinical Research

Primary (AL) Amyloidosis
IRB #H-34318/Idelalisib:  A Phase 2 Trial of Idelalisib in Patients with Immunoglobulin M (IgM)-Associated Primary (AL) Amyloidosis
IRB #H-31082/Pomalidomide:  A Phase 1/2 Trial of Pomalidomide and Dexamethasone in Subjects with Previously Treated AL Amyloidosis
IRB #31928/CUMC AAAJ7800:  Phase 2 Study of the Combination of Bendamustine and Dexamethasone in Patients with Relapsed AL Amyloidosis
IRB #2012-1122/Millennium C16011:  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
IRB #H-32474/Neotope NEOD001:  Phase 1, Open Label, Dose Escalation. Study of IV Admin of Single Agent NEOD001 in patients with Amyloidosis
IRB #H-32790/AMyC 11-MM-02:  Phase 1 Dose Escalation study of Carfilzomib in patients with previously treated AL Amyloidosis


Secondary (AA) Amyloidosis
IRB #H-30035/CL-503012:  The Kiacta Study – An International Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of the Efficacy and Safety of KIACTA in Preventing Renal Function Decline in Patients With AA Amyloidosis


Familial (ATTR) Amyloidosis
ISIS 420915-CS2:  The ISIS Study  A Phase 2/3 Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of ISIS 420915 in Patients With Familial Amyloid Polyneuropathy
B3461028:  The Tafamidis Study – A Multicenter, International, Phase 3, Double-Blind, Placebo-Controlled, Randomized Study to Evaluate the Efficacy, Safety, and Tolerability of Daily Oral Dosing of Tafamidis Meglumine (PF-06291826) 20 mg or 80 mg in Comparison to Placebo in Subjects Diagnosed With Transthyretin Cardiomyopathy (TTR-CM) 
ALN-TTR02-004:  The APOLLO Study – A Phase 3 Multicenter, Multinational, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of ALN-TTR02 in Transthyretin (TTR) Mediated Polyneuropathy(Familial Amyloidotic Polyneuropathy-FAP)