View clinical trials related to Amyloidosis.
Filter by:This is a Phase II, single-arm, multicentre study of Daratumumab (16mg/kg IV route) in adult patients with Light-Chain (AL) Amyloidosis who are not in VGPR or better after previous treatment. A sample size of 40 patients who meet all eligibility criteria will be enrolled to receive study treatment. Patients will receive treatment until either disease progression or toxicity has occurred with a maximum planned of six 28-day cycles. Daratumumab will be administrated every week for the first 2 cycles then. every 2 weeks from cycle 3 through cycle 6. Patients will also receive best supportive care (BSC) to mitigate Daratumumab side-effects, and to address underlying Amyloidosis, including blood product transfusions, antimicrobials, and (as appropriate) growth factors including granulocyte colony-stimulating factors for neutropenia, erythropoietin for anaemia, and/or transfusions for thrombocytopenia
The aim of the study is to evaluate coronary flow reserve (CFR), index of microcirculatory resistance (IMR), and proportion of overt microvascular disease, defined as depressed CFR as well as elevated IMR in patients with cardiac amyloidosis. The second objective of this study is to compare results of non-invasive test including serum light chain amount, Doppler echocardiography with 2D strain, and cardiac perfusion MRI. The third object of this study is to evaluate the association between physiologic indices and pathologically measured percent area involvement of interstitium.
The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of ALN-TTRSC02 in healthy subjects.
The investigators will prospectively evaluate tissue samples obtained from patients undergoing carpal tunnel release surgery for amyloid in the soft tissue that is removed. Patients who have tissue that stains positive for amyloid will undergo cardiac testing to look for evidence of cardiac involvement.
The purpose of this study is to determine whether the efficacy of gemcitabine is comparable with the efficacy of the standard chemotherapy with vinorelbine for mobilization of autologous stem cells in myeloma patients
Periodic Fever, Aphthous stomatitis, Pharyngitis and cervical Adenitis (PFAPA) is one of the most common, least explored periodic fever syndrome in childhood. This study aims to investigate whether a single dose of an interleukin beta (IL-1) antagonist, Canakinumab will be able to abort PFAPA flares in patients who experience a flare in an average of 2 weeks or less. This will be a single arm open label pilot study. 10 patients will be recruited from 1 center (Pediatric rheumatology unit -Schneider children's medical center of Israel). Patients in ages 2-10 years old who are diagnosed with PFAPA according to clinical criteria at least 3 months prior to enrollment and who are under regular care for this disease (single dose of glucocorticoids during flare) and who suffer from more than 4 PFAPA flares for the last 2 months, will be screened for this study. In the second documented flare, patients will be enrolled to receive a single dose of subcutaneous (SC) Canakinumab 4 mg/kg. The primary outcome is defined as - 50% reduction in PFAPA flares for the next 2 consecutive months as reported by the patient (use of diary) and documented by the patient primary care physician and/ or the researcher in a monthly follow up visits. Secondary outcome measure are define as time to flare (days) and Parent/patient quality of life assessment measured by 100mm visual analog scale (VAS).
Evaluate safety and toxicity/adverse events associated with delivery of low dose whole brain irradiation in patients with early Alzheimer's dementia according to the National Institute of Neurological and Communicative Disorders and Stroke and the Alzheimer's Disease and Related Disorders Association (NINCDS-ADRDA) criteria.. As a secondary goal it will establish whether or not the intervention with low dose whole brain irradiation stabilizes or decreases cerebral amyloid deposits and whether these correlate with the recognized progression of Alzheimer's dementia. The investigators will also collect information from the FDG and Amyvid® PET Scans to determine if there is any correlation between neurocognitive/quality of life scores and changes in amyloid plaque size, number and location.
This protocol seeks to enroll smoldering multiple myeloma (SMM) and monoclonal gammopathy of undetermined significant (MGUS) patients with λ light chain (LC) involvement, a group of patients for whom standard of care is observation not treatment. Patients with SMM and MGUS have a precursor plasma cell disorder from which light chain amyloidosis (AL) can evolve. In this trial, enrolled subjects will have blood and if available bone marrow cells evaluated by molecular testing to determine their clonal λ LC variable region (VL) germline gene. Seventy percent of AL cases involve just 7 germline donors, 5 of which are λ germline donors. The hypothesis that will be tested with this protocol is that the presence of AL germline genes associated with AL in patients with a pre-existing diagnosis of λ SMM or λ MGUS indicates the presence of AL or risk of progression to AL.
In this study, the investigators seek to evaluate bone marrow and blood samples and treatment responses to see if Minimal Residual Disease (MRD) can be used as a predictive method of response to treatment in amyloidosis.
International, multicenter, observational, longitudinal study to identify biomarker/s for the development of a new MS-based biomarker for the early and sensitive diagnosis of Transthyretin-Related Familial Amyloidotic Polyneuropathy from blood and number of correctly identified patients with Transthyretin-Related Familial Amyloidotic Polyneuropathy