Amyloidosis
MONDO:0019065A disorder characterized by the localized or diffuse accumulation of amyloid protein in various anatomic sites. It may be primary, due to clonal plasma cell proliferations; secondary, due to long standing infections, chronic inflammatory disorders, or malignancies; or familial. It may affect the nerves, skin, tongue, joints, heart, liver, spleen, kidneys and adrenal glands.
Also known as: amyloid, amyloid disease, amyloidoses, amyloidosis, amyloidosis (disease)
284 clinical trials for this condition and its sub-types.
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Broader categories
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Experimental NK cell therapy for blood cancers hits early hurdle
Disease control TerminatedThis early-stage study tested a new drug called SAR445514, which helps the body's natural killer (NK) cells attack cancer cells in people with multiple myeloma or light-chain amyloidosis that had returned or stopped responding to other treatments. The study was terminated early, …
Phase: PHASE1 • Sponsor: Sanofi • Aim: Disease control
Last updated Jun 27, 2026 12:37 UTC
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Experimental drug aims to stop rare heart disease in its tracks
Disease control TerminatedThis study tested a drug called inotersen in 31 people with a rare condition where a faulty protein builds up in the heart, causing damage. The drug is designed to lower levels of that protein. The trial was stopped early, so it's unclear if it works, but researchers hoped to see…
Phase: PHASE2 • Sponsor: Brigham and Women's Hospital • Aim: Disease control
Last updated Jun 27, 2026 09:07 UTC
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Experimental antibody trial for rare heart disease halted early
Disease control TerminatedThis phase 3 study tested an experimental drug called birtamimab in 208 people with advanced AL amyloidosis, a rare disease where abnormal proteins damage organs like the heart. Participants received either birtamimab or a placebo, plus standard chemotherapy. The goal was to see …
Phase: PHASE3 • Sponsor: Prothena Biosciences Ltd. • Aim: Disease control
Last updated Jun 27, 2026 08:04 UTC
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Promising drug combo for rare amyloidosis hits snag: trial ends early
Disease control TerminatedThis study tested a drug called ixazomib plus dexamethasone against other standard treatments for people with relapsed AL amyloidosis, a rare disease where abnormal proteins build up in organs. The trial aimed to see if the combination improved blood markers and slowed heart or k…
Phase: PHASE3 • Sponsor: Millennium Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 26, 2026 13:13 UTC
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Tafamidis tablet vs capsule: which works better?
Knowledge-focused TerminatedThis early-stage study aimed to compare how a tablet form of tafamidis is absorbed in the body compared to the existing capsule form. It involved 24 healthy adults who took a single dose of each form under fed conditions. The study was terminated early, so results may be limited.
Phase: PHASE1 • Sponsor: Pfizer • Aim: Knowledge-focused
Last updated Jun 28, 2026 00:00 UTC
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Hidden heart condition: study seeks to uncover missed diagnosis in heart failure patients
Knowledge-focused TerminatedThis study aims to find out how common transthyretin amyloidosis cardiomyopathy (ATTR-CM) is in Russian patients with a certain type of heart failure. Researchers will review medical records and then invite some patients for extra heart tests to confirm or rule out ATTR-CM. The g…
Sponsor: AstraZeneca • Aim: Knowledge-focused
Last updated Jun 26, 2026 17:47 UTC