Active clinical trials for "Amyloidosis"

RATIONALE: Chemotherapy plus interferon alfa may be effective for primary systemic amyloidosis. PURPOSE: Phase II trial to study the effectiveness of dexamethasone plus interferon alfa in treating patients who have primary systemic amyloidosis.

Exercise training in patients with heart failure and preserved ejection fraction (HFpEF) has been associated with an improvement in cardiorespiratory fitness and quality of life.

Primary objective: To identify older adults with transthyretin cardiac amyloidosis (ATTR-CA) early in the course of the illness, at a time when disease modifying therapies are most effective. The specific aims of this epidemiologic investigation include: To identify subjects with previous lumbar spinal stenosis (LSS) Surgery who have evidence of transthyretin (TTR) amyloid deposits in spinal specimens and could be at risk for ATTR cardiac amyloidosis. To evaluate for ATTR-CA among those with localized TTR in the spinal tissue. The study will also explore the following: The prevalence of amyloid in lumbar spinal stenosis specimens by Congo Red staining. The prevalence of TTR deposits among subjects with amyloid as determined by mass spectrometry. Evaluation of a novel artificial intelligence technique for that can identify amyloid histologically with standard H&E staining. Difference in ATTR-CA prevalence between subjects with TTR and indeterminate amyloid deposits in subject's spine by myocardial uptake of technetium pyrophosphate scan (Tc99-PYP).

The project aims to investigate the validity, and reliability of outcome measures of muscle strength, functioning (gait, balance, and fine motor skills), physical activity, and patient-reported outcome measures of functioning (gait, balance, and fine motor skills), and daily living among patients with polyneuropathy. Further, the project aims to compare physical activity and patient-reported outcome measures of functioning (gait, balance, and fine motor skills), and daily living among patients with polyneuropathy with physical activity and patient-reported outcome measures of functioning (gait, balance, and fine motor skills) and daily living in healthy adults.

To evaluate the efficacy and safety of eplontersen after administration for 65 weeks to patients with hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN), as compared to the NEURO-TTR trial (NCT01737398). For more information, please visit http://www.neuro-ttransform.com/.

Patients with multiple myeloma (a type of blood cancer affecting the white blood cells) or amyloidosis (abnormal buildup of a protein called amyloid in the body) are often given treatment with the drugs lenalidomide or pomalidomide. Some patients may experience an allergic reaction to these drugs which would mean stopping the treatment. The purpose of this research study is to see how safe and useful desensitization is in allowing patients to receive further treatment with lenalidomide or pomalidomide.

The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of ALN-TTRSC02 in healthy subjects.

A method of detecting amyloid in the retina has been developed. A specially designed retinal camera will directly visualze and record retinal amyloid and via image processing will generate a number: the retinal amyloid index (RAI). The amount of retinal amyloid correlates with cerebral amyloid and has a predictive value in Alzheimer's disease. Telomere attrition accounts for cellular aging and is felt to have a pivotal role in Alzheimer's disease. The investigators plan to screen individuals to select those having retinal amyloid then evaluate an oral telomerase activator to determine if its use can alter the RAI over time compared to placebo.

The principal aim of this study is to investigate the cardiac uptake of 89Zr-GSK2398852 in subjects with transthyretin cardiomyopathy amyloidosis (ATTR-CM), and its biodistribution to other organs. Low doses of GSK2398852 will be co-administered at levels not high enough for therapeutic benefit. This study will be conducted in two parts: Part A and Part B. Subjects in Part A will participate in up to two dosing sessions and subjects in Part B will participate in one dosing session. Subjects will undergo up to 3 PET scans at varying intervals after 89Zr-GSK2398852 administration. The total duration of study will be approximately 3 to 4 months for subjects in Part A and approximately 2 months for subjects in Part B. Part B of the study will be triggered based on data obtained in Part A and other emerging data.

The primary objective of this study was to evaluate safety and tolerability of ACZ885 in this extension study. This extension study offered the opportunity for participants who completed Epoch 4 of the preceding CACZ885N2301 (NCT02059291) study to continue to be treated with ACZ885 until approval in Japan of the drug in Periodic Fever Syndromes or until development of ACZ885 in Periodic Fever Syndromes was suspended.