Clinical Research

Repository: All Types of Amyloidosis

H-22838: Data, Tissue, Blood, And Urine Repository For Amyloid Diseases 2000-455G
H-33317: Serum Biomarker Identification of Cardiac Amyloidosis in Elderly African Americans

 

AL Amyloidosis

NCT05250973: A Phase 2, Multicohort Study of Daratumumab-Based Therapies in Participants With Amyloid Light Chain (AL) Amyloidosis (AQUARIUS)
NCT04973137: A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled, Efficacy and Safety Study of Birtamimab Plus Standard of Care vs. Placebo Plus Standard of Care in Mayo Stage IV Subjects With Light Chain (AL) Amyloidosis (AFFIRM-AL)
NCT04512235: A Phase 3, Double-Blind, Multicenter Study to Evaluate the Efficacy and Safety of CAEL-101 and Plasma Cell Dyscrasia Treatment Versus Placebo and Plasma Cell Dyscrasia Treatment in Plasma Cell Dyscrasia Treatment Naïve Patients With Mayo Stage IIIa AL Amyloidosis (CARES-302)
NCT04504825: A Phase 3, Double-Blind, Multicenter Study to Evaluate the Efficacy and Safety of CAEL-101 and Plasma Cell Dyscrasia Treatment Versus Placebo and Plasma Cell Dyscrasia Treatment in Plasma Cell Dyscrasia Treatment Naïve Patients With Mayo Stage IIIb AL Amyloidosis (CARES-301)
NCT04270175: Daratumumab, Pomalidomide, and Dexamethasone (DPd) in Relapsed/Refractory Light Chain Amyloidosis Patients Previously Exposed to Daratumumab
NCT04847453: A Phase 1/1a Study of Venetoclax, MLN9708 (Ixazomib Citrate) and Dexamethasone for Relapsed/Refractory Light Chain Amyloidosis
NCT04316442: A Phase 1b/2a, Open-Label, Dose-Escalation Study of the Safety and Efficacy of an Anti-CD38 Antibody Drug Conjugate (STI-6129) in Patients With Relapsed or Refractory Systemic AL Amyloidosis

 

ATTRwt (Wild Type) Amyloidosis

H-28609: A Study of Serum Transthyretin Aggregates And Association With Disease Progression In Patients With Senile Systemic Amyloidosis

 

ATTRmt (Mutant TTR, Familial) Amyloidosis

ISIS 420915-CS3:  IONIS OPEN LABEL EXTENSION STUDY: An Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of ISIS 420915 in Patients with Familial Amyloid Polyneuropathy (FAP)
B3461045TAFAMIDIS OPEN LABEL EXTENSION STUDY: A Multicenter, International, Phase 3, Double-Blind, Randomized, Extension Study to Evaluate the Safety of Daily Oral Dosing of Tafamidis Meglumine (PF-06291826) 20 mg or 80 mg in Subjects Diagnosed With Transthyretin Cardiomyopathy (TTR-CM)
ALN-TTR02-006: PATISIRAN OPEN LABEL EXTENSION STUDY: A Multicenter, Open-Label, Extension Study to Evaluate the Long-term Safety and Efficacy of Patisiran in Patients With Familial Amyloidotic Polyneuropathy Who Have Completed a Prior Patisiran Clinical Study
ALN-TTR02-007: PATISIRAN EXPANDED ACCESS PROGRAM: Expanded Access Protocol of Patisiran for Patients With Hereditary Transthyretin-Mediated Amyloidosis (hATTR Amyloidosis) With Polyneuropathy