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Amyloidosis clinical trials

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NCT ID: NCT06469372 Recruiting - Cardiac Amyloidosis Clinical Trials

Cardiac Amyloidosis Discovery Trial

Start date: May 28, 2024
Phase: N/A
Study type: Interventional

This is a single center, diagnostic clinical trial in which the investigators aim to prospectively validate a deep learning model that identifies patients with features suggestive of cardiac amyloidosis, including transthyretin cardiac amyloidosis (ATTR-CA). Cardiac Amyloidosis is an age-related infiltrative cardiomyopathy that causes heart failure and death that is frequently unrecognized and underdiagnosed. The investigators have developed a deep learning model that identifies patients with features of ATTR-CA and other types of cardiac amyloidosis using echocardiographic, ECG, and clinical factors. By applying this model to the population served by NewYork-Presbyterian Hospital, the investigators will identify a list of patients at highest predicted risk for having undiagnosed cardiac amyloidosis. The investigators will then invite these patients for further testing to diagnose cardiac amyloidosis. The rate of cardiac amyloidosis diagnosis of patients in this study will be compared to rate of cardiac amyloidosis diagnosis in historic controls from the following two groups: (1) patients referred for clinical cardiac amyloidosis testing at NewYork-Prebysterian Hospital and (2) patients enrolled in the Screening for Cardiac Amyloidosis With Nuclear Imaging in Minority Populations (SCAN-MP) study.

NCT ID: NCT06455748 Recruiting - Clinical trials for Amyloid Light-chain Amyloidosis

Daratumumab/Daratumumab and Hyaluronidase-fihj in Combination With Pomalidomide and Dexamethasone for the Treatment of Patients With Newly Diagnosed AL Amyloidosis: a Prospective, Multicenter, Single-arm Study

Start date: March 1, 2024
Phase: N/A
Study type: Interventional

This is a prospective and single arm clinical study. The goal of this clinical trial is to observe and evaluate the efficacy and safety of Daratumumab/daratumumab and hyaluronidase-fihj in combination with pomalidomide and dexamethasone in the treatment of patients with newly diagnosed AL amyloidosis.

NCT ID: NCT06414746 Recruiting - Clinical trials for Carpal Tunnel Syndrome

Hereditary Transthyretin Amyloidosis Polyneuropathy in Patients With Carpal Tunnel Syndrome in Russia

LOCUS
Start date: December 29, 2023
Phase:
Study type: Observational

This is a multicenter observational study consisting of retrospective and prospective phases. The retrospective phase will entail secondary data collection from electronic or paper medical records of patients who underwent surgery for CTS to assess their probability of having ATTR PN.

NCT ID: NCT06393712 Recruiting - Clinical trials for Cerebral Amyloid Angiopathy

A Phase 2 Trial of ALN-APP in Patients With Cerebral Amyloid Angiopathy

cAPPricorn-1
Start date: May 2024
Phase: Phase 2
Study type: Interventional

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 pharmacodynamics (PD) of ALN-APP in adult patients with sporadic CAA (sCAA) and Dutch-type CAA (D-CAA). The study will be conducted over 2 periods: a 24-month double-blind treatment period and an optional 18-month open-label extension (OLE) period. The estimated duration of study participation, inclusive of screening, treatment, and additional safety follow-up, is up to 50 months.

NCT ID: NCT06393465 Recruiting - Clinical trials for Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)

Real-World Effectiveness of High-Dose Tafamidis on Neurologic Disease Progression in Mixed-Phenotype Transthyretin Amyloid Cardiomyopathy

Start date: May 19, 2024
Phase:
Study type: Observational

This study will examine the clinical effectiveness of Tafamidis in patients with Mixed Phenotype Transthyretin Amyloidosis using data that already exist in patients' medical records

NCT ID: NCT06383143 Recruiting - AL Amyloidosis Clinical Trials

Promoting Diagnosis and Management of AL in Italy (ProDigALIty)

ProDigALIty
Start date: May 1, 2023
Phase:
Study type: Observational [Patient Registry]

The investigators plan to establish a dedicated network of Italian Hematologic Departments interconnected with the Amyloidosis Research and Treatment Center in Pavia to: 1. Implement a biomarker-based screening strategy to promote early diagnosis of AL amyloidosis among at-risk patients, including patients with monoclonal gammopathy of undetermined significance, MGUS, and altered free light chain ratio (aFLCR), and patients with smoldering multiple myeloma (SMM) 2. Expedite and facilitate patients' referral and their enrollment in ongoing pre-clinical/clinical studies, also to reflect a broader spectrum of the real-world population of patients with AL amyloidosis in Italy; 3. Investigate the clinical utility of novel diagnostic technologies, including light chain sequencing and N-glycosylation analysis

NCT ID: NCT06376214 Recruiting - Clinical trials for Light Chain (AL) Amyloidosis

Daratumumab for Patients With Light Chain Amyloidosis

Start date: January 1, 2023
Phase: N/A
Study type: Interventional

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.

NCT ID: NCT06372301 Recruiting - Aortic Stenosis Clinical Trials

Dobutamine Stress Echocardiography in LF/LG Aortic Stenosis and Wild-type Transthyretin Amyloid Cardiomyopathy

DobAttrAS
Start date: April 2, 2024
Phase: N/A
Study type: Interventional

The goal of this prospective clinical study is improve the diagnosis of Low-flow low-gradient aortic stenosis (LF/LG AS), in patients with co-existing wild-type transthyretin cardiac amyloidosis (ATTRwt). The main question it aims to answer is whether the classic dobutamine-stress echocardiography can be used to determine AS severity in patients with ATTRwt and LF/LG AS This question will be tried to answer by comparing dobutamine stress echocardiography, with the invasively measured aortic valve area (which is considered as the gold standard). In addition we aim to assess the degree of myocardial fibrosis and amyloid infiltration, assessed by light microscopy and cardiac magnetic resonance (CMRI) and evaluation of myocyte mitochondrial function by high resolution respirometry and their relation to AS severity and hemodynamic response to dobutamine.

NCT ID: NCT06367842 Recruiting - Clinical trials for Carpal Tunnel Syndrome

Orthopaedic Specimen Pathology and Early Diagnosis of ATTR Cardiopathy (ATTR-ORTHO)

ATTR-ORTHO
Start date: February 27, 2024
Phase:
Study type: Observational

The goal of this observational study is to learn about the frequency of ATTR amyloid, cardiac involvement and associated features, in 150 patients aged 50 or more years, and operated for an idiopathic carpal tunnel syndrome, lumbar spine stenosis or total hip or knee arthroplasty for primary osteoarthritis. The main questions to be answered are: 1. What is the frequency of ATTR deposits in samples retrieved during surgery? 2. What is the frequency of cardiac involvement in ATTR positive patients? 3. What are the preoperative predictors of ATTR deposits? Participants will - have operative samples stained by Congo red in search of amyloid, which will be typed by immunochemistry in positive samples, - undergo a multimodal imaging search for cardiac involvement, if ATTR is identified, - undergo a preoperative complete clinical examination, including collection of medical history, ECG, biochemical tests, and imaging (ultrasound scans of rotator cuff and hip capsule in all participating patients, of the carpal tunnel in patients operated at this site, and MRI + standing profile radiography of the lumbar spine, in patients operated for lumbar stenosis) - ATTR positive patients will be proposed to be followed-up by a reference center, with the aim of an early diagnosis of cardiac involvement, allowing efficient mamagement. Researchers will assess the frequency of ATTR deposits at each operated site, the frequency or ATTR cardiopathy in ATTR + patients, and will compare demographic, clinical, biochemical, and imaging features in patients with and without ATTR deposits, to guide the indications of pathological examination during these frequent orthopedic surgeries

NCT ID: NCT06365593 Recruiting - Clinical trials for Hereditary Transthyretin Amyloidosis

hATTR Polyneuropathy in Russia

PRIMER
Start date: December 29, 2023
Phase:
Study type: Observational

A multicenter observational retrosPective Registry of patIents with transthyretin aMyloid polynEuropathy (hATTR-PN) and chRonic idiopathic axonal polyneuropathy (CIAP) in the population of the Russian Federation (PRIMER) There are no comprehensive epidemiological data on patients with hereditary ATTR-PN (hATTR-PN) and CIAP in the Russian Federation. Therefore, there is a need to conduct a large-scale observational study in the Russian population to obtain information on clinical, electrophysiological and demographic characteristics of patients with hATTR-PN and CIAP. Obtaining the study data will help to identify the patients with axonal polyneuropathy, who deserve TTR gene sequencing, and therefore to allow early treatment and potentially modify disease progression in patients.