View clinical trials related to Amyloidosis.
Filter by:AL amyloidosis begins in the bone marrow where abnormal proteins misfold and create free light chains that cannot be broken down. These free light chains bind together to form amyloid fibrils that build up in the extracellular space of organs, affecting the kidneys, heart, liver, spleen, nervous system and digestive tract. The primary purpose of this study is to determine the recommended dose of CAEL-101 to facilitate progression of further clinical trials and evaluate safety and tolerability of CAEL-101 in combination with the standard of care (SoC) cyclophosphamide-bortezomib-dexamethasone (CyBorD) chemotherapy and daratumumab .
This phase II clinical trial aimed at influencing the improvement of major organ functions, especially the objective response rate, in Amyloid light-chain amyloidosis involving myocardium.
Patients above the age of 60 will be recruited at their carpal tunnel release surgery. Biopsies will be taken from the wrist and examined for the presence of amyloid protein. A amyloid-positive biopsy will refer the patients for cardiac examination including blod- and urine samples examined for transthyretin. If the result of the cardiac examination is suspected cardiac amyloidosis, the patient will be referred for a diagnostic TC99-DPD scintigraphy. If the scintigraphy is positive, the patient will be referred for right heart catherization (RHC) and an exercise test. Myocardial biopsies will be taken at the RHC and examined with electron microscopy and high resolution respirometry.
This study will test the hypothesis that in patients with previous daratumumab exposure, combination therapy of daratumumab, pomalidomide, and dexamethasone (DPd) will yield higher complete remission (CR) rates in relapsed/refractory amyloidosis than historical pomalidomide/dexamethasone treatment.
This is a worldwide safety surveillance study of pregnancy outcomes in women with hATTR-PN who may have been exposed or were not exposed to TEGSEDI prior to or during the pregnancy and of pediatric outcomes up to 1 year of age.
The DZHK TranslatiOnal Registry for CardiomyopatHies (DZHK TORCH) represents a unique resource of clinical data and high quality biological samples to enable innovative clinical and molecular studies on cardiomyopathies (CMP). As a multi-center German cardiomyopathy registry, TORCH has been prospectively admitting patients since December 2014. 2,300 patients were recruited as planned. Taken together, patient data showed that the prevalence of these diseases is much higher in men than in women, atrial fibrillation is common in all forms of CMPs as well as rare forms of disease indicate a higher risk and higher morbidity. This DZHK TORCH register is now to be expanded with a second phase (DZHK TORCH-Plus). The second phase DZHK TORCH-Plus consists of 4 main modules: 1. "Clinical phenotyping, follow-up & biosampling" 2. "Genomics", 3. "Inflammation" and 4. "Biomarker". The central aims are 1) to significantly increase the number of probands (n = 4340) in order to better address the different types of CMPs, especially patients with rare CMP forms such as LVNC and ARVC or with probably molecularly explainable cardiomyopathies (familial DCM), 2) to prolong the longitudinal with a further follow-up to achieve sufficient events and thereby derive clinical recommendations for risk assessment, 3) to increase the number of probands with state-of-the-art phenotyping, 4) to pinpoint the effect of myocardial inflammation, fibrosis, gender and to determine or predict genotypes based for outcome, 5) to validate novel biomarkers developed in other DZHK studies, and 6) to foster active cooperation with international CMP registries and partners from industry.
Carpal tunnel syndrome has been suggested to be an early sign of amyloidosi. The investigators will evaluate tissue samples obtained from patients undergoing carpal tunnel release surgery for amyloid.
The overall goal of the proposed research is to assess the feasibility of a randomized study evaluating the non-inferiority of an electronic Health (e-Health) delivery alternative (e.g. private web portal) as compared to return of actionable genetic research results with a genetic counselor.
The aging of the population is a reality in our society, with a strong increase in the number of elderly patients hospitalized for heart failure in our institutions. Heart failure in these patients is more present than to younger patients, with preserved ejection fraction form (HFpEF). Aging is responsible for the onset of senile amyloid cardiomyopathy. This pathology is still imperfectly understood and its link with the increase in the frequency of HFpEF is important. In addition, specific treatments have just shown their effectiveness. It is therefore urgent to better identify the prognostic predictive parameters of this cardiomyopathy. The pathophysiological involvement of the coronary microcirculation responsible for a true microvascular coronary disease (CMVD) has been described as predictive factor in all cardiomyopathies. However the implementation of preventive strategies and / or therapeutic of the coronary microcirculation dysfunction are limited because we lack of diagnostic tests available and applicable to large cohorts of patients. Our team INSERM U1039 Radiopharmaceutiques Biocliniques in collaboration with the laboratory GIPSA-lab (Grenoble Images Speech Signal Automatique), laboratory specialized in the signal analysis, has developed a new method of analysis allowing to measure the coronary microcirculation dysfunction usable in SPECT thanks to the measurement of a myocardial perfusion heterogeneity index (IHPM) (patented technique). The 3C registry (NCT03479580) is a registry studying the prevalence and cardiovascular prognosis of macro and microcirculatory coronary artery disease using the latest coronary evaluation techniques in patient with cardiomyopathy. This registry deployed on interventional cardiology centers on the Alpine Arc is therefore also addressed to patients with senile cardiomyopathy. The data collected will provide a better understanding of the factors influencing the prognosis of senile cardiomyopathy and the prognostic contribution of the measurement of the IHPM will be evaluated.
This study mainly evaluated the efficacy and safety of autologous stem cell transplantation for the treatment of AL amyloidosis, the role of induction and maintenance therapy in autologous stem cell transplantation, and the long-term efficacy and prognosis risk factors of autologous stem cell transplantation for the treatment of AL amyloidosis.