An Open-label Single Center, Single Patient Study of an Experimental Antisense Oligonucleotide (ASO) Treatment in AA Amyloidosis
The purpose of this study is to assess the safety and efficacy of a subcutaneous injection of nL-SAA1-01in a patient with AA Amyloidosis.
NCT06397001 — AA Amyloidosis
Status: Active, not recruiting
http://inclinicaltrials.com/aa-amyloidosis/NCT06397001/
Daratumumab and Dexamethasone Combined With Pomalidomide (DPD) or ASCT in the Treatment of Newly Diagnosed Systemic Light Chain Amyloidosis (AL Amyloidosis): a Prospective, Single Center Clinical Trial
This is a prospective, single-center study exploratory clinical trial, aim to exploring the efficacy and safety of daratumumab in patients with AL amyloidosis, patients were divided into three groups: one group received long-term treatment with daratumumab based regimen, and the other group received autologous stem cell transplantation after two standard treatment courses with daratumumab based regimen, and the third group consists of newly diagnosed stage IIIb AL amyloidosis patients who plan to receive DPD treatment. The purpose of this study is to observe the efficacy and safety of Daratumumab, in the treatment of newly diagnosed systemic AL amyloidosis.
NCT06376214 — Light Chain (AL) Amyloidosis
Status: Recruiting
http://inclinicaltrials.com/light-chain-al-amyloidosis/NCT06376214/
Screening for AL Amyloidosis in Smoldering Multiple Myeloma
In this multicenter study, we will recruit 400 patients 40 years of age or older at 15 centers with a diagnosis of smoldering multiple myeloma (SMM), a group of patients for whom standard of care is observation not treatment. The main goal of this study is to screen for the diagnosis of light-chain amyloidosis (AL) before the onset of symptomatic disease and to develop a training set for a likelihood algorithm.
NCT06365060 — Smoldering Multiple Myeloma
Status: Not yet recruiting
http://inclinicaltrials.com/smoldering-multiple-myeloma/NCT06365060/
OverTTuRe: An Observational Multi-Country Study Collecting Real-World Secondary Data on the Characteristics, Treatment Patterns and Outcomes of Patients With ATTR Amyloidosis
The overall aim of this observational study is to generate real-world evidence on the pre- and post-diagnosis disease journeys, including baseline characteristics, treatment patterns and selected clinical, economic, and humanistic outcomes (for example Health Related Quality of Life (HRQoL), Neuropathy impairment score, activities of daily living (ADL) assessments) in patients with ATTR amyloidosis, and to better understand how the disease is presented.
NCT06355934 — ATTR Amyloidosis
Status: Recruiting
http://inclinicaltrials.com/attr-amyloidosis/NCT06355934/
Physiopathological Investigation of Unclassified GENotypes of Autoinflammatory Diseases and AA Amyloidosis
Patients with autoinflammatory diseases (AID) have recurrent episodes of systemic inflammation accompanied by nonspecific elevation of blood inflammation markers typically absent between attacks. A complication of autoinflammatory diseases is AA amyloidosis, which can lead to renal failure and dialysis. Advances in genetic analysis have led to the identification of new autoinflammatory diseases and thus new pathophysiological pathways. However, genetic analyses are sometimes confronted with results that are difficult to interpret. These are the Variants of Unknown Significance, for which genetic analysis alone does not allow to determine if the genetic mutation is responsible for the symptoms. genetic analysis sometimes has limitations in the diagnosis of AID which can only be overcome by pathophysiological studies of the variants found.
NCT06354322 — Autoinflammatory Disease
Status: Not yet recruiting
http://inclinicaltrials.com/autoinflammatory-disease/NCT06354322/
Assessment of the Association of New Biomarkers (GDF15, ST2, Galectin-3, TIMP-1, MMP-9, NfL) and Plasma Prothrombotic Potential in the Course of Cardiac Transthyretin Amyloidosis
The development of cardiac amyloidosis is caused by the deposition of misfolded, insoluble proteins in the extracellular matrix of tissues. An important element of the clinical picture of the disease is the increased risk of thromboembolic complications, independent of the occurrence of atrial fibrillation, and the presence of intracardiac thrombi. The pathomechanism may be related to an increase in filling pressure or amyloid infiltration leading to myocardial damage and endothelial dysfunction, which may activate the prothrombotic inflammatory cascade, resulting in increased thrombogenic potential. Currently, there is limited published data on the potential role of new heart failure biomarkers in the assessment of ATTR cardiomyopathy, particularly in the assessment of asymptomatic carriers of pathogenic TTR variants. Moreover, there are few literature reports on the direct assessment of the coagulation system in this group of patients, and the pathomechanism of the increased thromboembolic risk is unexplored. Purpose of the study: To assess the diagnostic value of biomarkers related to heart failure (growth differentiation factor-15 (GDF15), soluble suppression of tumorigenicity-2 (ST2), galectin-3), amyloidosis ( TTR, tissue inhibitor of metalloproteinase-1 (TIMP-1), matrix metalloproteinase-9 (MMP-9, matrix metalloproteinase-9), neurofilament light chain (NfL)) and the generation potential thrombin as a marker of the prothrombotic state in the course of ATTR. Methods: This prospective, single-center study will include consecutive patients diagnosed with ATTR cardiomyopathy (GROUP 1, n=30), asymptomatic carriers of pathogenic TTR variants (GROUP 2, n=30), and a matched control group of healthy volunteers (GROUP 3 , n=20). Material for research was collected and secured from all study participants. After giving informed consent, all patients will be tested using the ELISA method from peripheral blood (enzyme-linked immunosorbent assay) GDF15, ST2, TTR, TIMP-1, MMP-9, galectin-3, NfL. The values of these biomarkers will be compared in subgroups and correlated with clinical data, laboratory test results, echocardiography including analysis of left ventricular global strain (GLS), and scintigraphy. Additionally, the prothrombotic potential of plasma will be tested in both groups of patients using the calibrated automatic thrombogram (CAT) method, in accordance with the protocol previously used in the laboratory Expected results: The project will provide information on the value of biomarkers in the assessment of ATTR cardiomyopathy, especially in the assessment of asymptomatic carriers of pathogenic TTR variants, which may translate into the creation of a diagnostic algorithm for early identification of the development of the disease. Moreover, it will allow us to determine whether patients with cardiac ATTR are characterized by a prothrombotic state, which has not yet been described in the literature and may have potential clinical implications.
NCT06345235 — Transthyretin Amyloidosis
Status: Recruiting
http://inclinicaltrials.com/transthyretin-amyloidosis/NCT06345235/
A Study of Bortezomib, Pomalidomide, Dexamethasone in Patients With Systemic AL Amyloidosis
This is an open-label, multicenter, Phase 2 study in subjects with newly diagnosed systemic light chain (AL) amyloidosis. Approximately 40 subjects will receive therapy with bortezomib, pomalidomide, and dexamethasone. The primary outcome is hematologic very good partial response and complete response rate at 6 months.
NCT06342466 — Systemic Amyloidosis
Status: Recruiting
http://inclinicaltrials.com/systemic-amyloidosis/NCT06342466/
Multicenter Observational Retrospective-prospective Study of Prevalence and Clinical Characteristics of Transthyretin Amyloidosis Cardiomyopathy in Russian Patients With Heart Failure With Preserved Ejection Fraction in Real Clinical Practice
A multicenter observational retrospective-prospective study of prevalence and clinical characteristics of transthyretin amyloidosis (ATTR) cardiomyopathy (CM) in Russian patients with heart failure with preserved ejection fraction (HFpEF) in real clinical practice. The retrospective phase will entail secondary data collection from electronic or paper medical records of patients who are participating/participated in the PRIORITY-CHF study and have HFpEF. Those patients who have a high suspicion of having ATTR-CM and provided informed consent will be invited to participate in the prospective phase. The prospective phase will consist of three visits, during which a routine comprehensive cardiologic evaluation in order to confirm or exclude ATTR-CM diagnosis will be performed. In patients with confirmed ATTR-CM the material for genetic testing will be collected in order to specify the type of ATTR-amyloidosis
NCT06338839 — Transthyretin Amyloidosis Cardiomyopathy, Heart Failure With Preserved Ejection Fraction
Status: Recruiting
http://inclinicaltrials.com/other/NCT06338839/
Artificial Intelligence to Assist the Echocardiographic Identification of Transthyretin Cardiac Amyloidosis
The goal of this study is to develop an algorithm using artificial intelligence (AI) to assist identification of potential ATTR-CM cases using routine transthoracic echocardiography. The main questions it aims to answer are: - is the algorithm able to diagnose ATTR-CM - is the algorithm able to diagnose different types of ATTR-CM (ATTRv, ATTRwt) This is a non interventional study. Participant' echocardiographies will be, after deidentification, used to train, valid and test the algorithm.
NCT06328075 — Amyloid Cardiomyopathy
Status: Recruiting
http://inclinicaltrials.com/amyloid-cardiomyopathy/NCT06328075/
A Phase 1/2 Study of Linvoseltamab in Patients With Relapsed or Refractory Systemic Light Chain Amyloidosis
This study is researching an experimental drug called linvoseltamab ("study drug"). This study is focused on patients who have AL amyloidosis that has returned or have failed other therapies and need to be treated again. The study consists of 2 phases (Phase 1 and Phase 2): - In Phase 1, linvoseltamab will be given to a small number of participants to study the side effects of the study drug and to determine the recommended doses of the study drug to be given to participants in Phase 2. - In Phase 2, linvoseltamab will be given to more participants to continue to assess the side effects of the study drug and to evaluate the ability of linvoseltamab to treat AL amyloidosis. The study is looking at several other research questions, including: - How many participants treated with linvoseltamab have improvement in the abnormal proteins that cause organ problems and for how long - How many participants treated with linvoseltamab have improvement in the heart or kidney and for how long - What the right dosing regimen is for linvoseltamab - What side effects may happen from taking linvoseltamab - How much linvoseltamab is in your blood at different times - Whether the body makes antibodies against linvoseltamab (which could make the drug less effective or could lead to side effects)
NCT06292780 — Relapsed/Refractory Systemic Light Chain Amyloidosis
Status: Not yet recruiting
http://inclinicaltrials.com/relapsed-refractory-systemic-light-chain-amyloidosis/NCT06292780/