View clinical trials related to Amyloidosis.
Filter by:To evaluate the efficacy and safety of Vel-Dex therapy (bortezomib and dexamethasone) followed by autologous hematopoietic stem cell transplantation as an initial treatment in patients with newly diagnosed AL.
The study is being done to see if the combination of bendamustine and dexamethasone will help people with amyloidosis that has returned after standard treatment, and to to estimate the partial hematologic response rate (PHR).
The primary purpose of this study is to assess the efficacy and safety of treatment with Kiacta in adult patients with AA Amyloidosis.
RATIONALE: Infection prophylaxis and management may help prevent cytomegalovirus (CMV) infection caused by a stem cell transplant. PURPOSE:This clinical trial studies infection prophylaxis and management in treating cytomegalovirus infection in patients with hematologic malignancies previously treated with donor stem cell transplant.
Primary outcome measure: - Hematologic response rate to the association of Lenalidomide, Cyclophosphamide and Dexamethasone. Secondary outcome measures: - Organ response rate. - Predictors of response (cardiac biomarkers, serum free light chains). - Toxicity - Safety (type, frequency, severity and relationship of adverse events to the study drug). - Duration of response. - Time to progression. - Overall survival
This study is being conducted to explore the potential benefits of a twelve-month doxycycline (at the best tolerated dose of 200 mg/day) and tauroursodeoxycholic acid (750 mg/day) treatment on disease progression in patients affected by transthyretin amyloidosis, including: 1) patients not eligible for liver transplantation; 2) patients eligible for liver transplantation, as a "bridge" therapy between the time of diagnosis and surgery, with the aim of stabilizing the disease; 3) patients showing disease progression after liver transplantation performed since at least 1 year. It is a phase II, therapeutic exploratory, two-part, 18-month, single centre, prospective study. Part I is a 12-month, open label treatment period in which doxycycline (200 mg/day, continuously) and tauroursodeoxycholic acid (750 mg/day continuously) are administered to 40 consenting subjects with transthyretin amyloidosis. Part II is a withdrawal period in which subjects will be monitored for disease progression. During part I, subjects will be evaluated at baseline (study Day 0), and then after 3, 6, 9 and 12 months of doxycycline plus tauroursodeoxycholic acid treatment or at premature treatment discontinuation; during part II, they will be assessed at months 15 and 18. Monthly phone contacts and blood tests will be performed to monitor potential adverse events.
Purpose of this study is to determine whether keeping SAA on normal or near normal level will delay progression of renal failure in patients with amyloidosis secondary to FMF.
Background: - Allergic inflammation is central to allergy-related diseases and disorders, such as asthma, food allergies, and atopic dermatitis. Atopic dermatitis, commonly called eczema is a chronic, noncontagious skin condition, usually starting in the first years of life, which causes itching and scaling of an individual s skin. Because atopic dermatitis is a common condition in children who have allergy-related diseases, including asthma, researchers are interested in studying both individuals with atopic dermatitis and their close relatives (parents and children) to better understand how allergy-related diseases develop and progress. In addition, some patients with inherited disorders with features including atopic dermatitis or other aspects of allergy such as food allergy, asthma, hay fever, hives, and others, will also be seen. Objectives: - To study the natural history of diseases of allergic inflammation, such as atopic dermatitis or genetic disorders associated with allergic inflammation. Eligibility: - Children and adolescents between 1 month and 21 years of age who have a documented history of moderate to severe atopic dermatitis. - Individuals between 1 month and 80 years of age who have a suspected genetic or inherited allergy disorder related to atopic dermatitis or allergic pathways. - Child and adult relatives of eligible participants will also be studied on this protocol. Design: - The study will require one initial visit to the National Institutes of Health Clinical Center (lasting 1-5 days), as well as any required follow-up visits for treatment and research studies. Participants will receive treatment for atopic dermatitis and other allergic diseases as part of the study for up to 1 year. - Participants will have some or all of the following tests as part of this study: - A detailed physical examination and medical history - Allergy skin prick testing to examine participants' responses to different allergens. - Blood samples for additional allergen testing, testing the immune system, and other research purposes - Skin punch biopsy to take a skin sample - Lung function tests to measure airflow from the lungs and inflammation - Food-related tests to diagnose potential food allergies - Leukapheresis to collect white blood cells only - Research samples, including stool specimens, saliva samples, buccal swabs (to collect cells from the inside of the cheek), and skin cell samples - Clinical digital photography to provide images of affected and healthy skin. - Participants will be asked to return for follow-up visits and tests for up to 1 year after the initial visit(s).
The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of a single dose of ALN-TTR01 in patients with transthyretin (TTR) mediated amyloidosis (ATTR).
We hope to determine the importance of different genes (including B receptors) in anthracycline-induced cardiomyopathy. This has important benefits to patients exposed to anthracyclines, as this could help determine whether certain individuals have increased susceptibility to cardiac injury.