AL amyloidosis
MONDO:0019438AL Amyloidosis is a plasma cell disorder characterized by the aggregation and deposition of insoluble amyloid fibrils derived from misfolding of monoclonal immunoglobulin light chains usually produced by a plasma cell tumor. It usually presents as primary systemic amyloidosis (PSA) with multiple organ involvement and less frequently as primary localized amyloidosis (PLA) restricted to a single organ.
Also known as: Light-chain amyloidosis, primary amyloidosis, Light chain amyloidosis, amyloidosis AL, amyloidosis primary systemic, primary AL amyloidosis, primary systemic AL amyloidosis, primary systemic amyloidosis
104 clinical trials for this condition and its sub-types.
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Broader categories
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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 Aug 02, 2026 00:00 UTC
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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: PHASE2 • Sponsor: Peking Union Medical College Hospital • Aim: Disease control
Last updated Jul 30, 2026 00:00 UTC
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Could a short course of steroids calm a dangerous heart rhythm?
Disease control ENROLLING_BY_INVITATIONThis trial tests whether an 8-week course of the immunosuppressive drug prednisone can reduce episodes of ventricular tachycardia (a potentially fatal fast heart rhythm) and improve heart function in people with non-ischemic cardiomyopathy and confirmed heart inflammation. Partic…
Phase: PHASE4 • Sponsor: Roderick Tung • Aim: Disease control
Last updated Jul 29, 2026 00:00 UTC
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New hope for rare blood disease: teclistamab trial targets AL amyloidosis
Disease control OngoingThis study tests a drug called teclistamab in 30 people with AL amyloidosis, a rare disease where abnormal proteins damage organs. Participants have already had other treatments. The goal is to see if teclistamab can reduce or eliminate the harmful proteins in the blood. The stud…
Phase: PHASE2 • Sponsor: European Myeloma Network B.V. • Aim: Disease control
Last updated Jul 22, 2026 00:00 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 Jul 16, 2026 00:00 UTC
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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: PHASE1, PHASE2 • Sponsor: Nexcella Inc. • Aim: Disease control
Last updated Jul 16, 2026 00:00 UTC
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Promising drug combo targets Hard-to-Treat amyloidosis
Disease control OngoingThis study tests a combination of two drugs, venetoclax and dexamethasone, in people with a rare blood disorder called AL amyloidosis that has come back or not improved after treatment. The trial includes 53 adults with a specific genetic marker (t(11;14)). The goal is to find th…
Phase: PHASE1, PHASE2 • Sponsor: Columbia University • Aim: Disease control
Last updated Jul 12, 2026 00:00 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 Jul 08, 2026 00:00 UTC
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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 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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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 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 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 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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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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Super MRI could spot hidden heart damage
Diagnosis OngoingThis study tests whether a new, more powerful 7 Tesla MRI can better detect scarring and swelling in the heart muscle of people with cardiomyopathy. Researchers will scan 13 adults aged 20-70 to see if the higher-resolution images improve diagnosis. The goal is to see if this adv…
Sponsor: University of Pennsylvania • Aim: Diagnosis
Last updated Jun 27, 2026 09:02 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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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: PHASE3 • Sponsor: Brigham and Women's Hospital • Aim: Knowledge-focused
Last updated Jul 08, 2026 00:00 UTC