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)
322 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
-
AL amyloidosis 92 trials · 98 incl. sub-types
2 sub-types
- Primary systemic amyloidosis 22 trials
- Primary localized amyloidosis 0 trials
-
Wild type ATTR amyloidosis 95 trials
-
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
-
ABeta2M amyloidosis 0 trials · 34 incl. sub-types
2 sub-types
- Wild type ABeta2M amyloidosis 34 trials
- Variant ABeta2M amyloidosis 0 trials
-
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
-
AA amyloidosis 2 trials
-
Soft tissue amyloid neoplasm 1 trial
-
AApoAIV amyloidosis 0 trials
-
AH amyloidosis 0 trials
-
ALECT2 amyloidosis 0 trials
-
Amyloidosis bronchopulmonary 0 trials
-
Immunoglobulin heavy-and-light chain 0 trials
Most studied deeper sub-types
-
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 2 • Sponsor: Janssen Research & Development, LLC • Aim: Disease control
Last updated Aug 30, 2026 00:00 UTC
-
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 3 • Sponsor: Alexion Pharmaceuticals, Inc. • Aim: Disease control
Last updated Aug 12, 2026 00:00 UTC
-
Can a targeted drug trio tame a rare amyloid disease?
Disease control OngoingThis phase 2 trial is testing whether combining daratumumab, venetoclax, and dexamethasone can produce a deep and rapid response in people newly diagnosed with light-chain (AL) amyloidosis who carry a specific genetic marker called t(11;14). The study enrolls 36 participants and …
Phase 2 • Sponsor: Peking Union Medical College Hospital • Aim: Disease control
Last updated Jul 30, 2026 00:00 UTC
-
Engineered immune cells take aim at a rare Protein-Clogging disease
Disease control OngoingThis study tests a new treatment called NXC-201 CAR-T for people with AL amyloidosis, a rare disease where abnormal proteins build up in organs. The therapy uses a patient's own immune cells, modified to target and destroy the cells producing these proteins. The trial is open to …
Phase 1/2 • Sponsor: Nexcella Inc. • Aim: Disease control
Last updated Jul 16, 2026 00:00 UTC
-
New drug eplontersen tested for Long-Term safety in heart disease patients
Disease control By invitation onlyThis 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 3 • Sponsor: Ionis Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:08 UTC
-
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 2 • Sponsor: Weill Medical College of Cornell University • Aim: Disease control
Last updated Jun 27, 2026 09:08 UTC
-
Vutrisiran shows promise for Long-Term control of rare heart condition
Disease control By invitation onlyThis 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 3 • Sponsor: Alnylam Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 09:07 UTC
-
Experimental heart drug pulled before first patient enrolled
Disease control CancelledThis 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 2 • Sponsor: Brigham and Women's Hospital • Aim: Disease control
Last updated Jun 27, 2026 09:07 UTC
-
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 3 • Sponsor: Alnylam Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 08:10 UTC
-
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 2 • Sponsor: Neurimmune AG • Aim: Disease control
Last updated Jun 27, 2026 08:05 UTC
-
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 1 • Sponsor: Memorial Sloan Kettering Cancer Center • Aim: Disease control
Last updated Jun 27, 2026 07:54 UTC
-
AI eye on the heart: could a computer catch a rare, Often-Missed disease?
Diagnosis By invitation onlyThis trial tests whether an artificial intelligence tool called EchoNet-LVH can help screen for cardiac amyloidosis, a rare but serious heart condition that is often misdiagnosed. The AI analyzes routine echocardiograms to flag suspicious patterns that might indicate the disease.…
Sponsor: Cedars-Sinai Medical Center • Aim: Diagnosis
Last updated Jul 24, 2026 00:00 UTC
-
Could a common hand condition reveal hidden heart disease? new imaging trial investigates
Diagnosis By invitation onlyThis study tests whether a radioactive tracer called 124I-evuzamitide can detect amyloid deposits in the heart and elsewhere in people aged 60 and older who have lumbar spinal stenosis or carpal tunnel syndrome. Participants will receive a single dose of the tracer and undergo a …
Phase 2 • Sponsor: University of Tennessee Graduate School of Medicine • Aim: Diagnosis
Last updated Jul 23, 2026 00:00 UTC
-
Simple blood test could catch hidden heart disease in seniors
Diagnosis By invitation onlyThis 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
-
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…
Sponsor: Mayo Clinic • Aim: Symptom relief
Last updated Jun 27, 2026 12:28 UTC
-
New study tracks Real-World safety of TEGSEDI for rare nerve disease
Knowledge-focused OngoingThis study follows 240 patients with hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN) to monitor the long-term safety of the drug TEGSEDI. Researchers will compare patients who have taken TEGSEDI with those who have not, focusing on side effects like low platel…
Sponsor: Akcea Therapeutics • Aim: Knowledge-focused
Last updated Jul 31, 2026 00:00 UTC
-
New PET scan could reveal hidden heart scarring in rare disease
Knowledge-focused OngoingThis study tested whether a special PET imaging probe called [68Ga]CBP8 can measure scarring (fibrosis) in the hearts of people with cardiac amyloidosis, a rare condition where abnormal proteins build up in the heart. Researchers compared 18 participants with cardiac amyloidosis …
Phase 3 • Sponsor: Brigham and Women's Hospital • Aim: Knowledge-focused
Last updated Jul 08, 2026 00:00 UTC