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Personalized Exercise Training to Improve Functional Capacity in Transthyretin Cardiac Amyloidosis
Transthyretin cardiac amyloidosis causes debilitating heart failure in older adults. The proposed research will develop a personalized exercise training progra…
A Phase 2 Clinical Trial of Teclistamab and Daratumumab in Previously Untreated AL Amyloidosis
The purpose of this study is to investigate whether teclistamab-daratumumab combination is effective and safe in AL amyloidosis. The study treatment is divide…
Artificial Intelligence Guided Echocardiographic Screening of Rare Diseases
Despite rapidly advancing developments in targeted therapeutics and genetic sequencing, persistent limits in the accuracy and throughput of clinical phenotypin…
TRITON-CM: A Phase 3 Global, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Transthyretin-Mediated Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)
The purpose of this study is to: * Evaluate the efficacy of nucresiran compared to placebo on reducing all-cause mortality and cardiovascular (CV) events * Ev…
Registry for Adults With Plasma Cell Disorders (PCD's)
The primary purpose of this protocol is to create a registry of patients with plasma cell disorders (PCDs), including for example the cancer multiple myeloma (…
Prevalence and Prediction of Transthyretin Amyloidosis in Ambulatory Patients With Heart Failure With Preserved Ejection Fraction
Recent studies have shown that transthyretin amyloidosis (ATTR) can sometimes cause a type of heart failure where the pumping function of the heart is normal, …
Characterizing Iodine-124 Evuzumitide (AT-01) in Systemic Amyloidosis
This is a single center prospective study evaluating 124I-evuzumitide in patients with systemic amyloidosis. The purpose of this study is to 1) Establish the d…
MAGNITUDE: A Phase 3, Multinational, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)
To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.
A Phase 2, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacodynamics of Intrathecally Administered ALN-APP in Patients With Cerebral Amyloid Angiopathy (CAA)
The purpose of the study is to evaluate the effect of ALN-APP on measures of CAA disease progression and to characterize the safety, tolerability, and pharmaco…
Determining the Association of TTR Stabilizing Therapy With Circulating TTR Amyloid Aggregates Over Time in Patients With ATTR-CM: Longitudinal Biomarker Study
The objective of this study is to determine the association of clinically prescribed, on-label, TTR stabilizing therapy (e.g. tafamidis or acoramidis) with lev…
An Open-label Phase I/II Trial of Venetoclax-Dexamethasone in Relapsed and/or Refractory t(11;14) Systemic Light-Chain Amyloidosis
The purpose of this study is assess safety, safest dose, and effectiveness of venetoclax in combination with dexamethasone in participants with t(11;14) positi…
Dose Schedule Investigation of B-Cell Maturation Antigen (BCMA) Bispecific Antibody, Elranatamab, for Treatment of Newly Diagnosed Light Chain Amyloidosis
This research study is for people who have newly diagnosed with AL (light chain) amyloidosis and have not yet received any treatment for this condition. The pu…