View clinical trials related to Amyloidosis.
Filter by:Transthyretin cardiac amyloidosis (ATTR-CA) is a relentlessly progressive disease that can progress to end stage heart failure, at which point recently approved transthyretin production silencing or structure stabilizing therapies provide no clinical benefit. For well-selected individuals, heart transplantation is an excellent therapeutic option to improve survival. Historically, concomitant liver transplantation has been used to halt the progression of non-cardiac transthyretin amyloidosis (ATTR) manifestations, especially for individuals with TTR genotypes associated with significant neuropathy. However, despite this, patients continue to experience progressive non-cardiac manifestations, particularly gastrointestinal and neuropathic, which can have a substantial influence on post-heart transplantation morbidity. Concomitant liver transplantation is also associated with substantial morbidity and its future therapeutic role is questionable with recently established therapies for ATTR. Therefore, there is a clear unmet need to determine the utility and safety of ATTR targeted therapies for patients with recent heart transplantation for end-stage ATTR-CA. The central hypothesis of this proposal is that in patients who have received a heart transplantation for end-stage ATTR-CA, tafamidis therapy will be efficacious and well-tolerated. We aim to determine the safety and efficacy of tafamidis in stable patients who have undergone heart or combined heart/liver transplantation for ATTR (wild-type or variant) cardiac amyloidosis. The proposed study will be a single-arm intervention clinical trial with tafamidis. Because of the efficacy of tafamidis for both variant ATTR-CA and wild-type ATTR-CA, there is no clinical equipoise for an inactive-comparator placebo arm. The primary endpoint of this study will be serial change in plasma transthyretin (TTR) levels from baseline to 12 months at 3-month intervals. The secondary endpoints of this study will include serial changes in neuropathy assessments, modified body mass indices, incident transplant-specific adverse events, and pharmacokinetics of tafamidis. Observations from this study will establish the role of tafamidis use for the management of ATTR in patients after transplantation for end-stage ATTR-CA.
This phase I/II trial tests the safety, side effects, and best dose of venetoclax, daratumumab, and dexamethasone for the treatment of systemic light-chain amyloidosis in patients with a deoxyribonucleic acid (DNA) abnormality called a translocation involving chromosomes 11 and 14, or "t(11;14)". Venetoclax works by attaching to a protein called Bcl-2, in order to kill cancer cells. Daratumumab works by binding to a target on the surface of cancer cells called Cluster of differentiation 38 (CD38). When daratumumab binds to CD38, it enables the immune system to find the cancer cell and kill it. Dexamethasone is a type of drug called a corticosteroid. A corticosteroid is a drug made of artificial steroid hormones, that are used to treat symptoms such as inflammation (swelling and irritation to a part of the body). The combination of these medications may more effectively treat patients with systemic light-chain amyloidosis and t(11;14).
The purpose of this study is to assess the safety, tolerability, immunogenicity and pharmacodynamic effects of ACI-24.060 in subjects with prodromal Alzheimer's disease and in non-demented adults with Down syndrome.
The purpose of this study is assess safety, safest dose, and effectiveness of venetoclax in combination with dexamethasone in participants with t(11;14) positive relapsed (comes back) or refractory (did not get better) light chain amyloidosis.
The study aims, by generating a large registry of patients with ATTR amyloidosis, including data at diagnosis and during follow up, to describe the natural history of ATTR amyloidosis in a real-world setting and to define and validate prognostic models, response criteria applicable at any point of the disease. The registry will also be used for data sharing and to allow the possibility of a close collaboration amongst the amyloidosis experts of the ARTC and all the physicians around the Country involved in the diagnosis and management of systemic amyloidosis. Thanks to the online registry, the diagnostic facility of the ARTC will be made available to requesting physicians.
Systemic transthyretin amyloidosis is an aging-related disorder. It is usually associated with cardiac disease but also extends to other organs. Recent studies found that idiopathic carpal tunnel syndrome patients may have amyloid deposition in tenosynovial tissue. The main aims of this project are the characterization of the association between idiopathic carpal tunnel syndrome and transthyretin amyloidosis and its evaluation as a predictive factor of cardiac amyloidosis
Aortic stenosis (AS) is the most frequent valvulopathy in the general population in France and more generally in developed countries, due to populations aging. Its standard treatment is historically surgical aortic valve replacement (SAVR). In the 2000s, the management of this valvulopathy was revolutionized by the development of the technique of per-cutaneous aortic valve replacement (TAVI). TAVI opens the possibility of curative treatment to patients at high operational risk not operable by conventional surgery, and for whom outcome was affected with high mortality under medical treatment alone. Amyloidosis, a pathology with multiple etiologies, is a rare condition and its cardiac form (AC) even more (8 to 17 / 100,000 people / year). However, its prevalence is increasing. Some autopsies series have found prevalence up to 50% of cardiac amyloidosis with transthyretin (AC-TTR) after 60 years. In addition, recent data suggested that AC-TTR prevalence is higher in the population of patients with heart disease: 13% in heart failure with preserved ejection fraction and up to 16% in patients with AS. The outcome of patients with AC-TTR remains unknown after TAVI. Thus, the diagnosis of AC-TTR in patients undergoing TAVI represents an important issue. Indeed, a treatment stabilizing the process of accumulation of transthyretin deposits, effective on the survival of these patients, is now available. In addition, a non-invasive screening strategy for AC-TTR, alternative to biopsy, is now validated.
The primary objective of this study is to define the intracardiac flow imaging biomarkers in cardiac amyloidosis.
The primary aim is to establish a prospective cohort of patients with plasma cell disorders (PCDs). All of the hospitalized PCD patients who are willing to sign the informed consent form (ICF) will be included in this study. Clinical characteristics, treatment options and responses will be collected. Peripheral blood, bone marrow aspirate and urine samples before and after the treatment will banked for future research. Our team will focus on the clinical and pathological features of PCDs, the correlation between the minimal residual disease (MRD) status and prognosis, and the role of Tumor Microenvironment (TME) in the pathogenesis and progress of PCDs.
The purpose of this protocol is to generate a registry of Chinese patients with AL amyloidosis treated with subcutaneous/intravenous daratumumab alone or subcutaneous/intravenous daratumumab combined with chemotherapy.