Primary systemic amyloidosis
MONDO:0017816Primary systemic amyloidosis (PSA) is a form of AL amyloidosis caused by the aggregation and deposition of insoluble amyloid fibrils derived from misfolded monoclonal immunoglobulin light chains usually produced by a plasma cell tumor and characterized by multiple organ involvement.
Also known as: systemic AL amyloidosis, systemic Immunoglobulin Light chain amyloidosis, systemic amyloidosis
30 clinical trials for this condition and its sub-types.
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New hope for rare amyloidosis: targeted drug combo enters trial
Disease control Recruiting nowThis phase 2 trial tests a drug called sonrotoclax, combined with dexamethasone and sometimes daratumumab, in 39 people with a specific genetic form of AL amyloidosis (t(11;14)). The goal is to see if the treatment can quickly reduce harmful protein levels and improve organ funct…
Phase: PHASE2 • Sponsor: Peking University People's Hospital • Aim: Disease control
Last updated Jul 30, 2026 00:00 UTC
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New antibody therapy shows promise for rare amyloidosis
Disease control Recruiting nowThis phase 2 trial is testing a drug called CM336 in 21 people newly diagnosed with AL amyloidosis, a rare disease where abnormal proteins build up in organs. CM336 is a bispecific antibody that helps the immune system target and destroy the cells making these proteins. The study…
Phase: PHASE2 • Sponsor: Institute of Hematology & Blood Diseases Hospital, China • Aim: Disease control
Last updated Jul 25, 2026 00:00 UTC
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New study tailors amyloidosis therapy based on genetic marker
Disease control Recruiting nowThis study tests a personalized approach for people with AL amyloidosis who have a specific genetic change called t(11;14). All 41 participants start with a standard three-drug combination. If their blood responds quickly within a week, they continue that treatment; if not, they …
Phase: NA • Sponsor: Jin Lu, MD • Aim: Disease control
Last updated Jul 09, 2026 00:00 UTC
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Experimental Dual-Target CAR-T therapy takes aim at Hard-to-Treat plasma cell cancers
Disease control Recruiting nowThis early-phase trial is testing a new type of gene therapy called CAR 70-BCMA dual-target CAR-T for people with relapsed or refractory plasma cell neoplasms, including multiple myeloma. The therapy involves taking a patient's own immune cells, re-engineering them to recognize a…
Phase: EARLY_PHASE1 • Sponsor: The General Hospital of Western Theater Command • Aim: Disease control
Last updated Jun 27, 2026 13:05 UTC
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Engineered immune cells take aim at rare Organ-Damaging disease
Disease control Recruiting nowThis early-phase trial tests a personalized cell therapy called BCMA-targeted CAR-T cells in 30 adults with relapsed or refractory light chain amyloidosis, a rare disease where abnormal proteins build up in organs. The treatment uses the patient's own immune cells, modified to at…
Phase: EARLY_PHASE1 • Sponsor: Beijing Boren Hospital • Aim: Disease control
Last updated Jun 27, 2026 13:02 UTC
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New antibody therapy aims to fight rare amyloidosis
Disease control Recruiting nowThis study tests a new drug called TQB2934 for people with systemic light chain amyloidosis, a rare disease where abnormal proteins damage organs. The drug is a bispecific antibody that helps the immune system target and destroy harmful cells. The trial will enroll 70 adults who …
Phase: PHASE1, PHASE2 • Sponsor: Shanghai Chia Tai Tianqing Pharmaceutical Technology Development Co., Ltd. • Aim: Disease control
Last updated Jun 27, 2026 12:30 UTC
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New hope for rare blood disease: experimental drug targets tough amyloidosis cases
Disease control Recruiting nowThis study tests an experimental drug called CM336 in 90 adults whose primary light-chain amyloidosis has come back or not responded to prior treatments. The drug is designed to help the immune system attack the abnormal cells causing the disease. The main goal is to see how many…
Phase: PHASE2 • Sponsor: Keymed Biosciences Co.Ltd • Aim: Disease control
Last updated Jun 27, 2026 08:00 UTC
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Can a radioactive peptide spot hidden amyloid in the heart?
Diagnosis Recruiting nowThis Phase 1 trial is testing a new radioactive tracer called 99mTc-p5+14 to see how it spreads through the body and how much radiation it delivers. The study includes healthy volunteers and people with AL or ATTR systemic amyloidosis, a condition where abnormal protein deposits …
Phase: PHASE1 • Sponsor: University of Tennessee Graduate School of Medicine • Aim: Diagnosis
Last updated Jul 26, 2026 00:00 UTC
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New scan could spot hidden amyloid in heart and organs
Diagnosis Recruiting nowThis study tests a radioactive imaging agent called Iodine-124 evuzumitide (AT-01) in 150 people with systemic amyloidosis. The goal is to see how well it detects amyloid deposits in the heart and other organs using PET/MRI or PET/CT scans. If accurate, it could become a new tool…
Phase: PHASE2 • Sponsor: Oregon Health and Science University • Aim: Diagnosis
Last updated Jun 27, 2026 12:07 UTC
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New PET tracer aims to spot hidden amyloid deposits
Diagnosis Recruiting nowThis study is testing a new imaging agent called [18F]FT8 to see if it can help doctors better see and diagnose amyloidosis, a disease where abnormal proteins build up in organs like the heart. The study will involve 20 patients with amyloidosis and 5 healthy volunteers. Research…
Sponsor: Tianjin Medical University • Aim: Diagnosis
Last updated Jun 27, 2026 07:58 UTC
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Heart scans may unlock hidden risks in minority communities
Knowledge-focused Recruiting nowThis study is looking at whether advanced heart imaging, especially cardiac MRI, can help identify the cause of cardiomyopathy and predict risks like death or heart failure in a diverse group of patients. The research focuses on adults in the Bronx, including many from Hispanic a…
Sponsor: Montefiore Medical Center • Aim: Knowledge-focused
Last updated Aug 12, 2026 00:00 UTC
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Nationwide registry aims to crack the code of a rare protein-clogging disease
Knowledge-focused Recruiting nowThis study is a national registry in Hungary that collects medical information from people diagnosed with systemic amyloidosis, a rare disease where abnormal proteins build up in organs. Researchers will look at patient records to understand how the disease starts, how it is diag…
Sponsor: Semmelweis University • Aim: Knowledge-focused
Last updated Jul 10, 2026 00:00 UTC
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App-Based diet tracking tested in rare disease
Knowledge-focused Recruiting nowThis pilot trial is testing whether amyloidosis patients can use the MyFitnessPal smartphone app to track their daily food and supplement intake for 8 weeks. The goal is to see if this low-cost method is feasible for studying malnutrition and weight loss in this condition. Forty …
Phase: NA • Sponsor: Barbara Ann Karmanos Cancer Institute • Aim: Knowledge-focused
Last updated Jun 28, 2026 00:00 UTC
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10,000 heart patients join online registry to unlock secrets of cardiomyopathy
Knowledge-focused Recruiting nowThis study is building a large online registry of 10,000 adults with cardiomyopathy or myocarditis, plus those with a strong family history. Researchers will track participants over time to see how many experience serious events like heart failure, dangerous heart rhythms, or dea…
Sponsor: Imperial College London • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:01 UTC
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AI could help predict sudden cardiac death in hereditary heart disease patients
Knowledge-focused Recruiting nowThis study aims to improve how doctors predict the risk of sudden cardiac death or heart failure in people with inherited heart diseases. Researchers will use artificial intelligence to analyze data from 1,000 participants, including medical history, ECGs, imaging, genetic tests,…
Sponsor: Nantes University Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:00 UTC
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Blood test may predict amyloidosis treatment success in days
Knowledge-focused Recruiting nowThis study looks at whether a rapid drop in a blood marker called dFLC can predict a complete response in people with AL amyloidosis treated with daratumumab-based therapy. Researchers will follow 50 patients to see if early changes in dFLC levels match up with later treatment su…
Sponsor: Peking University People's Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 11:00 UTC
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New biobank aims to unlock secrets of rare amyloidosis
Knowledge-focused Recruiting nowThis study is creating a biobank and data registry for people with amyloidosis, a rare disease where abnormal proteins build up in organs. Researchers will collect blood, urine, tissue samples, and medical information from 505 participants over time. The goal is to store these ma…
Sponsor: Universitaire Ziekenhuizen KU Leuven • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:09 UTC
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Can we predict who will die suddenly from heart problems?
Knowledge-focused Recruiting nowThis study follows 1500 people who already have an implantable cardioverter-defibrillator (ICD) to prevent sudden cardiac death. Researchers will track heart function, genetics, and blood markers to find better ways to predict who is at highest risk. The goal is to improve how do…
Sponsor: Johns Hopkins University • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:13 UTC
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Heart tissue analysis aims to unlock better diagnosis
Knowledge-focused Recruiting nowThis study looks at heart tissue samples from 216 adults who already need a biopsy as part of their care. Researchers want to find unique molecular patterns in different heart conditions, including after a heart transplant. The goal is to improve how these diseases are diagnosed …
Sponsor: University Hospital, Essen • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:12 UTC
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Scientists launch massive amyloid sample collection
Knowledge-focused Recruiting nowThis study is creating a large collection of blood, urine, and tissue samples from up to 10,000 people with amyloid diseases (like multiple myeloma). The goal is to store these samples and health data for future research to better understand these conditions. Participants do not …
Sponsor: Boston Medical Center • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:00 UTC
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New imaging method could unlock secrets of rare heart disease
Knowledge-focused Recruiting nowThis study is for people with light chain amyloidosis, a rare disease where abnormal proteins build up in the heart and other organs. Researchers will use special PET and MRI scans to take detailed pictures of these protein deposits in the heart. The goal is to better understand …
Phase: NA • Sponsor: Brigham and Women's Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:57 UTC