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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New antibody could clear toxic proteins from organs in rare disease
Disease control OngoingThis study tests an experimental drug called CAEL-101 in 125 people with a rare disease called AL amyloidosis, where abnormal proteins build up in organs like the heart and kidneys. The drug is designed to remove those protein deposits. The goal is to see if it helps people live …
Phase: PHASE3 • Sponsor: Alexion Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jul 04, 2026 00:00 UTC
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New hope for rare bleeding disorder: coagadex trial targets AL amyloidosis
Disease control TerminatedThis phase 3 study tests Coagadex, a human clotting factor X, in 15 adults with acquired factor X deficiency caused by AL amyloidosis. The goal is to see if it safely stops active bleeding and helps manage bleeding during surgery. Participants receive the drug on demand for bleed…
Phase: PHASE3 • Sponsor: Kedrion S.p.A. • Aim: Disease control
Last updated Jul 01, 2026 00:00 UTC
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New drug aims to clear amyloid clumps and save hearts
Disease control OngoingThis phase 3 trial tests whether CAEL-101, an antibody that removes abnormal protein deposits from organs, can help people with AL amyloidosis live longer and avoid heart-related hospital stays. About 281 participants who have not yet received treatment for their plasma cell diso…
Phase: PHASE3 • Sponsor: Alexion Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 14:00 UTC
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New drug cocktail targets Hard-to-Treat amyloidosis
Disease control OngoingThis early-stage trial tests a combination of three drugs—venetoclax, ixazomib, and dexamethasone—in 24 patients with light chain amyloidosis that has returned or not responded to prior treatment. The study focuses on finding the safest dose and checking side effects. All partici…
Phase: PHASE1 • Sponsor: National Cancer Institute (NCI) • Aim: Disease control
Last updated Jun 27, 2026 13:06 UTC
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New drug shows promise for rare heart condition in chinese patients
Disease control OngoingThis study tests a drug called eplontersen in Chinese adults with a heart condition caused by abnormal protein buildup. The goal is to see if the drug lowers that protein and is safe over time. Participants will be randomly assigned to receive the drug or a placebo for 24 weeks, …
Phase: PHASE3 • Sponsor: AstraZeneca • Aim: Disease control
Last updated Jun 27, 2026 13:04 UTC
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Can a drug stop amyloid damage after a heart transplant?
Disease control OngoingThis study tests whether the drug tafamidis can safely stop the progression of ATTR amyloidosis in people who have already received a heart transplant. About 25 stable patients will take tafamidis daily for 12 months. Researchers will measure changes in blood protein levels, nerv…
Phase: PHASE4 • Sponsor: University of Texas Southwestern Medical Center • Aim: Disease control
Last updated Jun 27, 2026 13:02 UTC
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New drug combo aims to tame rare heart disease
Disease control OngoingThis phase 2 trial tests a drug combination including daratumumab for people with AL amyloidosis, a rare disease where abnormal proteins damage organs, especially the heart. The study has two groups: one gets the full combo right away, the other starts some drugs later. Researche…
Phase: PHASE2 • Sponsor: Janssen Research & Development, LLC • Aim: Disease control
Last updated Jun 27, 2026 13:02 UTC
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New hope for amyloidosis patients: Long-Term safety study of AT-02 underway
Disease control OngoingThis study is for people with systemic amyloidosis, a disease where abnormal proteins build up in organs. It tests the long-term safety of an experimental drug called AT-02. About 120 adults will receive the drug and be monitored for side effects and how the drug behaves in the b…
Phase: PHASE2 • Sponsor: Attralus, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:29 UTC
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Could flickering lights and tones help fight Alzheimer's? major trial underway
Disease control OngoingThis study tests a device that delivers specific light and sound patterns to the brain, aiming to slow the progression of mild-to-moderate Alzheimer's disease. About 670 participants will use the device at home for an hour daily over a year, with half receiving a sham (inactive) …
Phase: NA • Sponsor: Cognito Therapeutics, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:29 UTC
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New antibody therapy targets Hard-to-Treat AL amyloidosis
Disease control TerminatedThis trial aimed to test an experimental drug called CM-336 for people with AL amyloidosis that has returned, not responded to treatment, or only partially responded after initial therapy. The drug works by helping the immune system attack and destroy harmful cells. However, the …
Phase: NA • Sponsor: Institute of Hematology & Blood Diseases Hospital, China • Aim: Disease control
Last updated Jun 27, 2026 12:24 UTC
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New drug eplontersen tested for Long-Term safety in heart disease patients
Disease control ENROLLING_BY_INVITATIONThis study is checking the long-term safety of a drug called eplontersen in people with ATTR-CM, a heart condition caused by abnormal protein buildup. About 1400 adults who completed earlier studies will receive eplontersen injections. Researchers will monitor side effects, kidne…
Phase: PHASE3 • Sponsor: Ionis Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:08 UTC
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10-Year study tracks heart drug vyndamax in Real-World patients
Disease control OngoingThis study is monitoring 110 adults in Korea who are taking Vyndamax (tafamidis) for a rare heart condition called ATTR-CM. The goal is to track side effects and see how well the drug works over 10 years in everyday medical practice. Researchers will check heart function and symp…
Sponsor: Pfizer • Aim: Disease control
Last updated Jun 27, 2026 12:02 UTC
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New hope for AL amyloidosis patients who failed first-line therapy
Disease control OngoingThis study tests whether adding daratumumab back to pomalidomide and dexamethasone can improve complete remission rates in 15 adults with relapsed or refractory AL amyloidosis who have already received daratumumab. Participants receive the three-drug combination to control the di…
Phase: PHASE2 • Sponsor: Weill Medical College of Cornell University • Aim: Disease control
Last updated Jun 27, 2026 09:08 UTC
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New antibody aims to strip away Heart-Clogging protein in fatal heart disease
Disease control OngoingThis Phase 3 trial tests an experimental drug called ALXN2220 in about 1,180 adults with transthyretin amyloid cardiomyopathy (ATTR-CM), a condition where abnormal protein builds up in the heart, causing heart failure. The drug is designed to remove those protein deposits. Partic…
Phase: PHASE3 • Sponsor: Alexion Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 09:07 UTC
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Can deep sleep or nerve stimulation flush toxic proteins from the brain?
Disease control ENROLLING_BY_INVITATIONThis Phase 2 trial tests whether a sleep-deepening drug (lower-sodium oxybate) or a nerve-stimulating device (non-invasive vagus nerve stimulation), or both, can help clear amyloid-beta protein from the brain in people with cerebral amyloid angiopathy (CAA). Sixty participants wi…
Phase: PHASE2 • Sponsor: Leiden University Medical Center • Aim: Disease control
Last updated Jun 27, 2026 09:07 UTC
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Vutrisiran shows promise for Long-Term control of rare heart condition
Disease control ENROLLING_BY_INVITATIONThis study is for people with a rare heart condition called ATTR amyloidosis with cardiomyopathy, where abnormal protein builds up in the heart. It tests the long-term safety and effectiveness of a drug called vutrisiran, given as an injection every three months. About 700 adults…
Phase: PHASE3 • Sponsor: Alnylam Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 09:07 UTC
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Experimental heart drug pulled before first patient enrolled
Disease control TerminatedThis study aimed to test a monthly injection called ION-682884 in people with TTR amyloid cardiomyopathy, a condition where abnormal protein deposits damage the heart. Only patients who had already completed a 2-year study of a similar drug (inotersen) were eligible. The goal was…
Phase: PHASE2 • Sponsor: Brigham and Women's Hospital • Aim: Disease control
Last updated Jun 27, 2026 09:07 UTC
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New antibody combo aims to tame rare blood disease
Disease control OngoingThis early-phase trial tests whether adding the drug isatuximab to standard chemotherapy can safely treat people with high-risk AL amyloidosis, a rare disease where abnormal proteins damage organs. Eleven participants will receive the combination to see if it reduces toxicity and…
Phase: PHASE1 • Sponsor: Emory University • Aim: Disease control
Last updated Jun 27, 2026 09:06 UTC
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New strategy aims to tame common virus after stem cell transplants
Disease control OngoingThis study tests a modified plan to prevent and treat cytomegalovirus (CMV) infection in people with blood cancers who have had a donor stem cell transplant. CMV is a common virus that can cause serious problems after transplant. The study involves 153 participants and looks at w…
Sponsor: City of Hope Medical Center • Aim: Disease control
Last updated Jun 27, 2026 09:00 UTC
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Could sound waves help Alzheimer's drugs work better?
Disease control OngoingThis study tests whether using focused ultrasound to temporarily open the blood-brain barrier can safely help standard antibody therapy reach the brain in people with mild Alzheimer's or mild cognitive impairment. About 15 participants will receive the combined treatment. The goa…
Phase: EARLY_PHASE1 • Sponsor: Ali Rezai • Aim: Disease control
Last updated Jun 27, 2026 08:12 UTC
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Hope for rare heart disease: new drug aims to slow deadly protein clumps
Disease control OngoingThis study tests a drug called vutrisiran in 655 adults with a rare heart condition caused by abnormal protein buildup (ATTR amyloidosis with cardiomyopathy). The drug is given as a shot every 3 months and aims to reduce deaths and heart-related hospital stays. The goal is to see…
Phase: PHASE3 • Sponsor: Alnylam Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 08:10 UTC
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Second dose of experimental drug may help heart amyloidosis patients
Disease control OngoingThis study tests whether a second round of treatment with ALXN2220 is safe and can improve heart function in people with transthyretin amyloid cardiomyopathy, a condition where abnormal proteins build up in the heart. About 35 adults who previously received the drug will get it a…
Phase: PHASE2 • Sponsor: Neurimmune AG • Aim: Disease control
Last updated Jun 27, 2026 08:05 UTC
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New antibody treatment aims to keep myeloma in check after transplant
Disease control OngoingThis phase 2 trial tests whether daratumumab, an antibody that targets cancer cells, can improve outcomes for people with multiple myeloma after they receive a stem cell transplant. The study involves 31 participants and is currently active but not recruiting. The main goal is to…
Phase: PHASE2 • Sponsor: City of Hope Medical Center • Aim: Disease control
Last updated Jun 27, 2026 08:02 UTC
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New drug shows promise for Long-Term control of rare nerve disease
Disease control OngoingThis study looks at the long-term safety and effects of a drug called eplontersen in people with a rare inherited nerve disease called hATTR-PN. About 151 participants who completed earlier studies will receive regular injections of eplontersen. The goal is to see if the drug rem…
Phase: PHASE3 • Sponsor: Ionis Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 08:01 UTC
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Could a maintenance drug keep AL amyloidosis at bay?
Disease control OngoingThis phase 2 trial is testing whether the oral chemotherapy drug ixazomib can help control AL amyloidosis after initial treatment. Seventeen adults with the disease will take ixazomib along with low-dose dexamethasone to see if it delays organ progression or the need for more the…
Phase: PHASE2 • Sponsor: Memorial Sloan Kettering Cancer Center • Aim: Disease control
Last updated Jun 27, 2026 08:01 UTC
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Heart scan study sees if new drug clears sticky proteins
Disease control OngoingThis 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 Jun 27, 2026 08:00 UTC
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New hope for rare heart disease: experimental drug tested for long-term safety
Disease control OngoingThis study tests the long-term safety of an experimental drug called NNC6019-0001 in people with transthyretin amyloid cardiomyopathy, a rare heart disease that can lead to heart failure. All 80 participants have already completed an earlier study and will receive the drug every …
Phase: PHASE2 • Sponsor: Novo Nordisk A/S • Aim: Disease control
Last updated Jun 27, 2026 07:59 UTC
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Heart drug acoramidis tested for Long-Term safety in rare amyloidosis
Disease control OngoingThis study is checking the long-term safety of a drug called acoramidis in people with a rare heart condition called transthyretin amyloid cardiomyopathy. About 389 participants who finished a previous 30-month trial will take acoramidis twice daily for up to 5 years. Researchers…
Phase: PHASE3 • Sponsor: Eidos Therapeutics, a BridgeBio company • Aim: Disease control
Last updated Jun 27, 2026 07:54 UTC
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New chemo formula aims to boost transplant success in blood cancers
Disease control OngoingThis study tests a new, more stable version of the chemotherapy drug melphalan in people with multiple myeloma or AL amyloidosis who are getting a stem cell transplant. The goal is to find the best dose that reaches a target level in the body, while possibly causing fewer side ef…
Phase: PHASE1 • Sponsor: Memorial Sloan Kettering Cancer Center • Aim: Disease control
Last updated Jun 27, 2026 07:54 UTC
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Simple blood test could catch hidden heart disease in seniors
Diagnosis ENROLLING_BY_INVITATIONThis study is testing a way to find cardiac amyloidosis—a rare but serious heart condition—earlier in people aged 65 and older. Researchers in Turkey will screen 800 patients at family medicine clinics using simple blood tests and medical history. The goal is to see if this appro…
Sponsor: Kotyora Family Medicine Health Management and Education Association • Aim: Diagnosis
Last updated Jun 27, 2026 09:08 UTC
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Algorithm aims to spot rare heart disease in medical records
Diagnosis TerminatedThis study aimed to test a computer algorithm that uses insurance claims data to identify people with ATTR amyloidosis, a rare disease that affects the heart and nerves. Researchers planned to check how well the algorithm worked by reviewing medical records. However, the study wa…
Phase: EARLY_PHASE1 • Sponsor: Yale University • Aim: Diagnosis
Last updated Jun 26, 2026 18:38 UTC
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New nerve patch could ease pain after biopsy
Symptom relief OngoingThis study tests a biodegradable tube that is placed over a cut nerve during a leg biopsy. The goal is to see if it is safe and reduces common side effects like pain, redness, and swelling. Twenty adults with various nerve conditions are taking part, and half will get the tube wh…
Phase: NA • Sponsor: Mayo Clinic • Aim: Symptom relief
Last updated Jun 27, 2026 12:28 UTC
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New Drain-Clearing device could make Post-Surgery recovery smoother
Symptom relief ENROLLING_BY_INVITATIONThis Mayo Clinic study tests a new device designed to clear fluid and debris from surgical drain tubes more effectively than the current method of using fingers. The trial will enroll 500 adults who have abdominal surgery with Jackson-Pratt drains. Nurses will rate how easy the d…
Phase: NA • Sponsor: Mayo Clinic • Aim: Symptom relief
Last updated Jun 27, 2026 09:08 UTC
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New catheter cap could make dialysis more comfortable
Symptom relief OngoingThis study tests a new device called the Catheter Caps Case (C3) for dialysis patients. The goal is to see if it is more comfortable and easier to use than the usual gauze and tape. Ten adults with a dialysis catheter will try the device for 15 minutes, and researchers will measu…
Phase: NA • Sponsor: Mayo Clinic • Aim: Symptom relief
Last updated Jun 27, 2026 08:13 UTC
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New drug aims to reduce pain and fatigue after stem cell transplant in older patients
Symptom relief OngoingThis study tests whether a drug called siltuximab can reduce symptoms like weakness, fatigue, nausea, and pain after a stem cell transplant in patients aged 60-75 with multiple myeloma or AL amyloidosis. The drug blocks a protein linked to inflammation, which may help patients re…
Phase: PHASE2 • Sponsor: Memorial Sloan Kettering Cancer Center • Aim: Symptom relief
Last updated Jun 27, 2026 07:56 UTC
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Could ditching Beta-Blockers boost heart Patients' exercise?
Symptom relief ENROLLING_BY_INVITATIONThis 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: PHASE4 • Sponsor: Weill Medical College of Cornell University • Aim: Symptom relief
Last updated Jun 27, 2026 07:55 UTC
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German health data reveals how rare heart condition affects patients
Knowledge-focused OngoingThis study uses anonymous health insurance records from Germany to learn more about a rare heart condition called ATTR-CM, where abnormal protein builds up in the heart. Researchers will look at survival rates, hospital visits, and how common the disease is. No new treatments are…
Sponsor: Pfizer • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:36 UTC
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Blood marker may predict nerve damage onset in rare genetic disease
Knowledge-focused OngoingThis observational study is looking at a protein called neurofilament light chain (NfL) in the blood of people who carry a gene variant for hereditary ATTR amyloidosis, both those without symptoms and those with nerve damage. Researchers want to see if NfL levels can help detect …
Sponsor: Alnylam Pharmaceuticals • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:36 UTC
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Dialysis vein mystery: researchers hunt for clues to prevent blockages
Knowledge-focused OngoingThis study follows 96 people with end-stage kidney disease who need a brachiocephalic fistula for dialysis. Researchers collect data from exams, blood tests, and imaging to track changes in the vein over time. The goal is to learn what causes cephalic arch stenosis, a common narr…
Sponsor: University of Chicago • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:32 UTC
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Can new sensors improve dialysis monitoring in the ICU?
Knowledge-focused ENROLLING_BY_INVITATIONThis study looks at whether advanced monitoring devices can accurately measure blood thickness (hematocrit) and oxygen levels during continuous dialysis in critically ill patients with acute kidney injury or end-stage kidney failure. About 30 adults in the intensive care unit wil…
Sponsor: University of Pennsylvania • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:29 UTC
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Pedaling through dialysis: could High-Intensity cycling shield hearts?
Knowledge-focused OngoingThis study looks at whether high-intensity cycling during hemodialysis can protect the heart from damage that dialysis sometimes causes. Fifteen adults on dialysis will exercise at different intensities during their sessions. Researchers will use echocardiograms to measure heart …
Phase: NA • Sponsor: London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:24 UTC
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New registry tracks how ATTR-CM patients are treated in daily practice
Knowledge-focused OngoingThis study is a disease registry that collects information from routine medical care of about 350 adults diagnosed with transthyretin amyloid cardiomyopathy (ATTR-CM) in Taiwan, Hong Kong, and Malaysia. Researchers will look at patient characteristics, treatments received, and ho…
Sponsor: Pfizer • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:06 UTC
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Bronx database sheds light on underdiagnosed heart condition in women and minorities
Knowledge-focused OngoingThis study creates a database of 476 patients in the Bronx who were tested for transthyretin amyloid cardiomyopathy (ATTR-CM), a serious and often missed heart condition. Researchers collected health records, imaging results, and outcomes like hospitalizations and deaths. The goa…
Sponsor: Montefiore Medical Center • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:09 UTC
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Heart check: do Nerve-Targeting drugs also shield the heart in rare amyloidosis?
Knowledge-focused OngoingThis study follows 20 adults with hereditary transthyretin amyloidosis (ATTRv) who have both nerve and heart involvement. They are treated with patisiran or vutrisiran for two years. Researchers use MRI scans and questionnaires to see how the drugs affect heart structure and func…
Sponsor: Rennes University Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:08 UTC
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New SPECT method could reveal hidden heart issues in amyloidosis patients
Knowledge-focused OngoingThis study looks at 50 people with TTR amyloid cardiomyopathy, a condition where abnormal proteins build up in the heart. Researchers want to see if a special SPECT scan can measure blood flow in the heart's tiny vessels, which may be impaired even without blocked arteries. Parti…
Phase: NA • Sponsor: Centre Hospitalier Régional d'Orléans • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:05 UTC
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New program aims to help older dialysis patients manage falls, memory, and daily tasks
Knowledge-focused ENROLLING_BY_INVITATIONThis pilot study tests a program that screens older dialysis patients for common age-related problems like falls, memory trouble, and medication issues. A geriatrics team then gives personalized recommendations to the dialysis clinic. The goal is to see if this approach is practi…
Phase: NA • Sponsor: Duke University • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:05 UTC
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Heart scans reveal hidden protein buildup in rare disease
Knowledge-focused OngoingThis 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 Jun 27, 2026 08:00 UTC
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Monthly MRI reveals hidden brain bleeding patterns in elderly
Knowledge-focused OngoingThis study uses monthly MRI scans to closely watch brain changes in 75 people with cerebral amyloid angiopathy (CAA), a condition common in older adults that can cause bleeding and strokes. Researchers aim to understand how small bleeds and silent strokes develop over time. Parti…
Sponsor: Martin Dichgans • Aim: Knowledge-focused
Last updated Jun 26, 2026 16:35 UTC