View clinical trials related to Amyloidosis.
Filter by:Open-label Phase 1b Dose Escalation/Dose Expansion study exploring the safety and efficacy of NXC-201 in patients with relapsed or refractory light chain amyloidosis (AL).
This phase III trial compares the effect of adding a stem cell transplant with melphalan after completing chemotherapy with daratumumab, cyclophosphamide, bortezomib and dexamethasone (Dara-VCD) versus chemotherapy with Dara-VCD alone for treating patients with newly diagnosed amyloid light chain (AL) amyloidosis. Melphalan is a chemotherapy given prior to a stem cell transplant. Giving chemotherapy before a peripheral blood stem cell transplant helps kill cancer cells in the body and helps make room in the patient's bone marrow for new blood-forming cells (stem cells) to grow. The stem cells are then returned to the patients to replace the blood forming cells that were destroyed by the chemotherapy. Daratumumab is in a class of medications called monoclonal antibodies. It binds to a protein called CD38, which is found on some types of immune cells and cancer cells, including myeloma cells. Daratumumab may block CD38 and help the immune system kill cancer cells. Chemotherapy drugs, such as cyclophosphamide and bortezomib, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Dexamethasone is in a class of medications called corticosteroids. It is used to lower the body's immune response to help stop the growth of cancer cells. Giving a stem cell transplant with melphalan after Dara-VCD may kill more cancer cells in patients with newly diagnosed AL amyloidosis.
ATTR-cardiac amyloidosis (CA) is present in 4% to 16% of elderly patients with severe calcific aortic stenosis (AS). The reasons for this association are not fully known. It is hypothesized that an amyloidotic infiltration of the aortic valve acts as a trigger for the development of endothelial damage and subsequent calcification. Elderly patients undergoing TAVI will be evaluated for the presence of ATTR-CA in Jordan.
The study design is a prospective registry including asymptomatic and symptomatic patients who carry a pathogenic TTR mutation. The study will enroll patients who meet the inclusion criteria and none of the exclusion criteria until 1000 patients are enrolled, at which point in time the study investigators will evaluate whether further patient accrual is meaningful.
To describe the demographics, clinical characteristics, treatment patterns and clinical outcomes of chronic inflammatory demyelinating polyneuropathy (CIDP), Guillain-Barre Syndrome (GBS), and heredofamilial amyloidosis (hATTR) adult patients at a single U.K. centre.
ATTR amyloidosis is a serious condition with significant morbidity and mortality. In Germany, there are numerous unreported cases of untreated patients, and diagnosing and initiating treatment often requires multiple specialized tests. To address this, a study is being conducted to determine if virtual reality (VR)-based patient education can improve diagnosis rates, treatment initiation, and medication adherence compared to standard education methods.
The B-AR project is based on a retrospective analysis of amyloidosis typing and the identification of their amylogenic protein.This work should be able to unite many clinicians, including pulmonologists and pathologists around a rare disease that still suffers from insufficient data.
The purpose of this clinical trial is to evaluate whether specific probiotic can reduce gastrointestinal symptoms and improves therapeutic response, on a background of Bortezomib+dexamethasone or Bortezomib+dexamethasone combined with daratumumab therapy, for naive AL amyloidosis patients.
Amyloid deposition in the heart is called cardiac amyloidosis (CA); 95% is immunoglobulin light chain amyloidosis (AL) and transthyretin amyloidosis (ATTR). Hereditary (ATTRm) or wild-type (ATTRwt) depends on whether the ATTRm gene is mutated or not. The most common mutation in Taiwan is A97S, 80% have left ventricular hypertrophy. The good prognosis depends on early diagnosis and correct treatment strategy. Bone-avid tracers such as 99mTc-PYP/DPD/HMDP could detect CA. The mechanism is not clear yet, which may be related to the microcalcification. AL amyloidosis is mostly between visual score grade 0-2, and ATTR-CM is usually gradeā„2 on PYP scan, or heart to contralateral (H/CL) ratio, and it might replace invasive myocardial biopsy. However, there are no large-scale clinical studies, lack of standardization data, and limited information in comparison between clinical and imaging parameters. This project will enroll patients with suspected or diagnosed with CA according to CA diagnostic algorithm. Clinical data and image parameters are collected and compared. The project aims to set up prediction models based on the multi-parameters of PYP scan using artificial intelligence technology, including imaging registration and alignment technology, and standardization. We further use the key cardiovascular data elements and imaging-derived database using model training network to extract image features to develop the diagnostic and prognostic prediction models, which are expected to validate the clinical significance and improve patient-centric performance and efficient clinical decision making.
Transthyretin (TTR) is a plasma protein mainly synthesized in the liver, recognized as a transporter of thyroxine and retinol-binding protein. Unstable changes in two types of TTR (wild type or variant) become misfolded, aggregate, and ultimately forms amyloid fibrils. Amyloid Transthyretin Cardiac amyloidosis (ATTR-CA) is an infiltrative cardiomyopathy caused by extracellular deposition of insoluble transthyretin (TTR) amyloid fibrils in the heart muscle. Cardiac amyloidosis (CA) has been recognized as a common cause of heart failure with preserved ejection fraction (HFpEF) among elderly persons, with increasing incidence. There are different ways of diagnosing ATTR-CA. These include cardiac magnetic resonance imaging, nuclear scintigraphy, and tissue biopsy, the gold standard. Tissues biopsy extracted from the adipose, lip salivary gland (LSG), and heart muscle (endomyocardial biopsy or EMB). Tissue diagnosis is the prerequisite of provincial support of the disease-modifying agent. However, with the convenience, ease, less risk of bleeding, and high sensitivity, LSG may offer an alternative to the more invasive EMB to diagnose suspected CA. To test the hypothesis that LSGB can replace EMB in tissue diagnosis of ATTR-CM. This Pilot study is designed to evaluate two invasive diagnostic methodologies: LSGB and the EMB. A total of 20 patients who underwent EMB within the last six months with confirmed Amyloid Transthyretin -wild type (ATTRwT) will be invited to participate. In addition, patients who signed the informed consent form will be scheduled for LSGB within two weeks.