A listing of amyloidosis medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.
The purpose of this study is to find out if we can detect a disease of the heart called cardiac transthyretin (TTR) amyloidosis early. People develop cardiac TTR amyloidosis due to an accumulation of a transthyretin protein in the heart. This can happen as part of an aging process (wild …
A Phase 3 Global, Open-Label, Randomized Study to Evaluate the Efficacy and Safety of ION-682884 in Patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy. For those with : a. Stage 1 or Stage 2 according to the Familial Amyloid Polyneuropathy (FAP) or Coutinho Stage b. Documented genetic mutation in the TTR gene …
This patisiran expanded access protocol (EAP) is an open-label, multicenter, single-arm study designed to provide pre-approval access to investigational patisiran for patients with ATTR amyloidosis with cardiomyopathy who, at baseline, have an inadequate response to or cannot tolerate standard of care. An intravenous infusion of study drug (patisiran 0.3 mg/kg) …
The purpose of this study is to evaluate the efficacy and safety of vutrisiran (ALN-TTRSC02) in patients with hereditary transthyretin amyloidosis (hATTR amyloidosis). Participants will receive vutrisiran or the reference comparator patisiran during the Treatment Period.
This is a prospective global multicenter long-term observational study designed to document the clinical outcomes of patients with hereditary transthyretin-mediated amyloidosis or wild-type transthyretin-mediated amyloidosis and the safety of patisiran (Onpattro) when used in patients with ATTRv amyloidosis. This is data collection study only and no study medication no visits …
AL (or light chain) amyloidosis begins in the bone marrow where abnormal proteins misfold and create free light chains that cannot be broken down. These free light chains bind together to form amyloid fibrils that build up in the extracellular space of organs, affecting the kidneys, heart, liver, spleen, nervous …
TEGSEDI (also known as inotersen) is a medicine that has been approved in the United States, Europe and Canada to treat polyneuropathy (nerve damage) caused by hereditary transthyretin amyloidosis (hATTR-PN) in adults. hATTR-PN is a rare genetic disease that worsens over time if left untreated. This research study will examine …
We plan to enroll up to 25 (target of 20 fully evaluable) adult subjects in this study who have an established diagnosis of advanced cardiac amyloidosis (AL). Subjects will be included in 2 subject cohorts of approximately 10 subjects each. In one cohort positron emission tomography/computed tomography (PET/CT) imaging will …
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