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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)
323 clinical trials for this condition and its sub-types, 133 tagged with Amyloidosis itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →Sub-types of Amyloidosis
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AL amyloidosis 93 trials · 99 incl. sub-types
2 sub-types
- Primary systemic amyloidosis 22 trials
- Primary localized amyloidosis 0 trials
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Wild type ATTR amyloidosis 95 trials
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Hereditary amyloidosis 19 trials · 79 incl. sub-types
9 sub-types
- Familial amyloid neuropathy 52 trials · 54 incl. sub-types Sub-types →
- Cerebral amyloid angiopathy 16 trials · 17 incl. sub-types Sub-types →
- APP-related brain and vascular amyloidosis 0 trials · 5 incl. sub-types Sub-types →
- Familial primary localized cutaneous amyloidosis 1 trial Sub-types →
- Finnish type amyloidosis 0 trials
- ITM2B amyloidosis 0 trials Sub-types →
- Familial visceral amyloidosis 0 trials Sub-types →
- Pulmonary amyloidosis 0 trials
- Variant ABeta2M amyloidosis 0 trials
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ABeta2M amyloidosis 0 trials · 34 incl. sub-types
2 sub-types
- Wild type ABeta2M amyloidosis 34 trials
- Variant ABeta2M amyloidosis 0 trials
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Primary cutaneous amyloidosis 4 trials · 5 incl. sub-types
5 sub-types
- Lichen amyloidosis 2 trials
- Familial primary localized cutaneous amyloidosis 1 trial Sub-types →
- Macular amyloidosis 1 trial
- Amyloidosis cutis dyschromia 0 trials
- Nodular cutaneous amyloidosis 0 trials
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AA amyloidosis 2 trials
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Soft tissue amyloid neoplasm 1 trial
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AApoAIV amyloidosis 0 trials
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AH amyloidosis 0 trials
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ALECT2 amyloidosis 0 trials
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Amyloidosis bronchopulmonary 0 trials
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Immunoglobulin heavy-and-light chain 0 trials
Most studied deeper sub-types
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Heart scan study sees if new drug clears sticky proteins
Disease control CompletedThis study looks at whether the drug eplontersen can reduce abnormal protein buildup in the heart for people with a condition called ATTR-CM. About 150 participants from a larger trial will get heart scans after 140 weeks of treatment. The goal is to see if the drug helps clear t…
Sponsor: Ionis Pharmaceuticals, Inc. • Aim: Disease control
Last updated Sep 13, 2026 00:00 UTC
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Can a new injection slow a rare heart disease?
Disease control CompletedThis trial is testing an experimental drug called eplontersen in people with transthyretin-mediated amyloid cardiomyopathy (ATTR CM), a condition where abnormal protein builds up in the heart and leads to heart failure. The study aims to see if the drug, given as a regular inject…
Phase 3 • Sponsor: Ionis Pharmaceuticals, Inc. • Aim: Disease control
Last updated Sep 05, 2026 00:00 UTC
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New drug patisiran aims to help hearts stiffened by rare protein disease
Disease control CompletedThis phase 3 trial tested the drug patisiran in 360 people with ATTR amyloidosis with cardiomyopathy, a condition where abnormal protein builds up in the heart, making it stiff and weak. Participants received either patisiran or a placebo intravenously. The study measured how far…
Phase 3 • Sponsor: Alnylam Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 12:10 UTC
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Gene editing breakthrough? CRISPR therapy targets rare amyloidosis
Disease control CompletedThis early-stage trial tests NTLA-2001, a CRISPR-based gene editing therapy, in 72 adults with hereditary ATTR amyloidosis—a condition where abnormal protein builds up in nerves or the heart. The treatment aims to turn off the faulty gene to reduce protein levels. The study focus…
Phase 1 • Sponsor: Intellia Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 09:00 UTC
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Promising combo therapy targets rare protein disease in major trial
Disease control CompletedThis study tested whether adding daratumumab to standard chemotherapy (CyBorD) helps people newly diagnosed with AL amyloidosis, a rare disease where abnormal proteins build up in organs. About 416 adults took part. The goal was to see if the combination leads to better blood res…
Phase 3 • Sponsor: Janssen Research & Development, LLC • Aim: Disease control
Last updated Jun 27, 2026 08:00 UTC
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A hidden warning in your wrist? surgery may reveal heart risk
Diagnosis CompletedThis study tests whether tissue taken during routine carpal tunnel release surgery can help diagnose amyloidosis, a condition where abnormal proteins build up in organs. People with bilateral carpal tunnel syndrome and certain risk factors—like age, heart issues, or family histor…
Sponsor: Henry Ford Health System • Aim: Diagnosis
Last updated Aug 16, 2026 00:00 UTC
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New PET tracer could spot rare heart disease without a biopsy
Diagnosis CompletedThis completed Phase 3 study tested whether a radioactive tracer called [18F]Florbetaben, used in PET scans, can accurately diagnose cardiac AL amyloidosis—a condition where abnormal proteins build up in the heart. 244 adults with suspected cardiac amyloidosis received a single P…
Phase 3 • Sponsor: Lantheus Germany GmbH • Aim: Diagnosis
Last updated Aug 05, 2026 00:00 UTC
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New PET scan agent could spot rare heart disease faster
Diagnosis CompletedThis study tested a new imaging drug called I-124 evuzamitide to see if it can accurately diagnose cardiac amyloidosis, a condition where abnormal proteins build up in the heart. 204 adults with suspected cardiac amyloidosis received a single dose followed by a PET/CT scan. The g…
Phase 3 • Sponsor: Sharmila Dorbala • Aim: Diagnosis
Last updated Jun 27, 2026 12:26 UTC
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Heart MRI breakthrough: new scan could replace needle biopsy for rare heart disease
Diagnosis CompletedThis study tested whether a special type of heart MRI, called T1 mapping, can accurately diagnose cardiac amyloidosis—a condition where abnormal proteins build up in the heart muscle. Researchers studied 112 older adults with heart failure and thickened heart walls. They compared…
Sponsor: University of Leipzig • Aim: Diagnosis
Last updated Jun 27, 2026 12:06 UTC
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New PET/MR scan could spot nerve amyloid without a biopsy
Diagnosis CompletedThis study tested whether a special PET/MR scan using a radioactive tracer called F-18 florbetapir can detect amyloid protein buildup in the nerves of people with amyloidosis. Eight adults with confirmed nerve amyloidosis or other nerve diseases took part. The goal was to see if …
Phase 1/2 • Sponsor: Mayo Clinic • Aim: Diagnosis
Last updated Jun 27, 2026 11:01 UTC
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AI-Powered ECG could spot hidden heart disease faster
Diagnosis CompletedThis study tested whether an artificial intelligence (AI) tool could help doctors detect cardiac amyloidosis, a rare but serious heart condition, earlier using standard ECG tests. Over 20,000 patients were involved, with some doctors using the AI-enhanced ECG and others continuin…
Sponsor: Mayo Clinic • Aim: Diagnosis
Last updated Jun 27, 2026 09:06 UTC
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AI spots silent heart condition in seconds
Diagnosis CompletedThis study tested a computer model that uses a patient's age, sex, medical history, and heart tests (ECG and echocardiogram) to estimate their risk of having cardiac amyloidosis, a serious but often undiagnosed heart condition. Researchers invited 50 high-risk patients for furthe…
Sponsor: Pierre Elias • Aim: Diagnosis
Last updated Jun 27, 2026 08:01 UTC
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Heart amyloid spotted by new PET tracer in pilot study
Diagnosis CompletedThis pilot study tested whether a radioactive tracer called F-18 florbetapir (Amyvid) can detect amyloid protein buildup in the heart using a PET scan. Researchers enrolled 23 people with known cardiac amyloidosis and a control group without the condition. The goal was to see if …
Phase 4 • Sponsor: Brigham and Women's Hospital • Aim: Diagnosis
Last updated Jun 27, 2026 07:59 UTC
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New PET tracer could sharpen amyloidosis diagnosis
Diagnosis CompletedThis phase 2 trial tested a radioactive tracer called 124I-AT-01 to see how well it can measure amyloid buildup in organs like the heart, kidneys, liver, and spleen using PET/CT scans. 33 adults with systemic amyloidosis received the tracer twice, six weeks apart. The goal was to…
Phase 2 • Sponsor: Attralus, Inc. • Aim: Diagnosis
Last updated Jun 26, 2026 13:34 UTC
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Laser zaps skin amyloid deposits in rare itching disorder
Symptom relief CompletedResearchers at Tanta University are testing whether fractional CO2 laser can reduce skin amyloid deposits and itching in people with primary cutaneous amyloidosis. The trial enrolls 22 adults with confirmed disease who have not used other treatments for at least six months. Parti…
Sponsor: Tanta University • Aim: Symptom relief
Last updated Sep 13, 2026 00:00 UTC
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Could ditching Beta-Blockers boost heart Patients' exercise?
Symptom relief CompletedThis study looks at whether older adults with a heart condition called cardiac amyloidosis feel better and can exercise more when they stop taking beta-blockers. Twenty participants will each go through two periods: one on their beta-blocker and one off it. Researchers will measu…
Phase 4 • Sponsor: Weill Medical College of Cornell University • Aim: Symptom relief
Last updated Jul 31, 2026 00:00 UTC
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Could a diabetes drug ease heart failure in rare amyloid disease?
Symptom relief CompletedThis study tested the drug dapagliflozin (a diabetes medicine) in 50 adults with heart failure due to amyloid cardiomyopathy, a rare condition where abnormal proteins stiffen the heart. Researchers measured changes in quality of life, walking distance, and heart stress markers ov…
Sponsor: National Medical Research Center for Cardiology, Ministry of Health of Russian Federation • Aim: Symptom relief
Last updated Jun 27, 2026 12:23 UTC
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Brain training for daily life: new approach may help seniors with memory loss
Symptom relief CompletedThis study tested whether a program called Strategy Training can help older adults with mild cognitive impairment (MCI) manage everyday tasks like shopping, paying bills, and taking medication. The training, delivered by occupational therapists, teaches goal-setting, scheduling, …
Sponsor: University of Pittsburgh • Aim: Symptom relief
Last updated Jun 27, 2026 09:10 UTC
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Heart scans reveal hidden protein buildup in rare disease
Knowledge-focused CompletedThis study uses MRI scans to measure amyloid protein buildup in the hearts of up to 150 people with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). Participants are already enrolled in a larger treatment trial. The goal is to see how amyloid levels change over time, not …
Sponsor: Ionis Pharmaceuticals, Inc. • Aim: Knowledge-focused
Last updated Sep 16, 2026 00:00 UTC
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500-Patient registry aims to unlock secrets of rare disease amyloidosis
Knowledge-focused CompletedThis study is creating a registry of 500 people with amyloidosis at Hospital Italiano de Buenos Aires. Researchers will track risk factors, diagnosis, treatment, and survival over time to better understand the disease. No new treatments are being tested—this is purely an observat…
Sponsor: Hospital Italiano de Buenos Aires • Aim: Knowledge-focused
Last updated Jul 23, 2026 00:00 UTC
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Wrist MRI may spot hidden amyloid clumps without needles
Knowledge-focused CompletedThis study tested whether a special MRI scan of the wrist can detect amyloid deposits in people with systemic amyloidosis, a condition where abnormal proteins build up in organs. Researchers enrolled 128 adults, including patients with cardiac amyloidosis and healthy volunteers. …
Sponsor: GCS Ramsay Santé pour l'Enseignement et la Recherche • Aim: Knowledge-focused
Last updated Jul 18, 2026 00:00 UTC
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Sleep hack may slow Alzheimer's: early study tests Time-in-Bed restriction
Knowledge-focused CompletedThis study tests whether spending less time in bed for four weeks can deepen sleep and boost memory in adults aged 65-85 who have poor sleep and are at risk for Alzheimer's. Researchers will measure brain activity, memory, and blood markers. The goal is to see if a simple behavio…
Early phase 1 • Sponsor: University of Pittsburgh • Aim: Knowledge-focused
Last updated Jul 08, 2026 00:00 UTC
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Hidden nerve disease found in carpal tunnel patients?
Knowledge-focused CompletedThis completed study in Russia checked how many people with carpal tunnel syndrome also have a rare inherited nerve condition called hereditary transthyretin amyloidosis. Researchers reviewed medical records of 721 patients and will test some for the genetic mutation. The goal is…
Sponsor: AstraZeneca • Aim: Knowledge-focused
Last updated Jun 27, 2026 13:03 UTC
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Hidden heart condition found in many with stiff heart failure
Knowledge-focused CompletedThis study looked at how often cardiac amyloidosis occurs in older Tunisian patients with a type of heart failure called HFpEF. Researchers used heart scans and imaging to find cases and identify early warning signs. The goal is to make diagnosis easier and faster for this seriou…
Sponsor: University Tunis El Manar • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:35 UTC
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Heart scans may predict survival in rare amyloid disease
Knowledge-focused CompletedThis study looked at 30 people newly diagnosed with cardiac AL amyloidosis, a rare disease where abnormal proteins damage the heart. Researchers used advanced MRI, echocardiogram, and PET scans to see if these images could predict survival and show how well chemotherapy works. Th…
Sponsor: Fondazione IRCCS Policlinico San Matteo di Pavia • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:07 UTC
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Protein clues could spot rare disease years early
Knowledge-focused CompletedThis study followed 37 people with a genetic risk for hereditary ATTR amyloidosis over five years. Researchers measured levels of misfolded proteins in the blood to see if they could detect the earliest signs of the disease. The goal is to develop a way to catch the condition bef…
Sponsor: The Cleveland Clinic • Aim: Knowledge-focused
Last updated Jun 27, 2026 11:00 UTC
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Could your nose warn of a rare heart disease?
Knowledge-focused CompletedThis study checked how common smell and taste problems are in 51 people with cardiac amyloidosis, a rare disease where abnormal proteins build up in the heart. Researchers used a simple smell test called Sniffin' Sticks to measure these senses. The goal was to see if these issues…
Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:05 UTC
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Tiny heart monitor could unlock secrets of deadly rhythms in amyloidosis
Knowledge-focused CompletedThis completed study looked at 110 people with cardiac amyloidosis, a condition where protein deposits build up in the heart. Researchers implanted a small device under the skin to continuously monitor heart rhythms for up to several years. The goal was to learn more about when a…
Sponsor: Marianna Fontana • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:12 UTC
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Brain scans reveal Age's role in Alzheimer's plaque buildup
Knowledge-focused CompletedThis study used special brain scans (PET imaging) to measure amyloid plaques in 60 people with Alzheimer's disease or related conditions like posterior cortical atrophy and logopenic progressive aphasia. The goal was to understand how age influences the amount of amyloid in the b…
Sponsor: Institut National de la Santé Et de la Recherche Médicale, France • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:53 UTC