Amyloidosis occurs when misfolded proteins clump in organs, disrupting normal function. AL amyloidosis, the most common form, often affects the heart and kidneys. Treatment depends on the type: AL amyloidosis typically requires chemotherapy to stop the bone marrow cells making the abnormal protein, while ATTR amyloidosis now has RNA-targeting drugs that reduce production of the misfolded protein.
What's actually going on in research
Trials are testing new ways to silence or degrade the proteins that form amyloid, drugs to remove existing amyloid deposits from organs, and combinations that address both production and clearance. Research focuses heavily on preserving heart and kidney function, improving quality of life, and extending survival. Several RNA-targeting therapies approved since 2018 have changed the outlook for ATTR amyloidosis, and similar approaches are being tested for AL forms.
Amyloid removal therapies
New antibodies aim to clear existing amyloid deposits from the heart and other organs, potentially reversing damage rather than just stopping new buildup. Early results show heart wall thickness decreasing in some patients.
Gene silencing drugs
RNA-targeting drugs like patisiran and inotersen reduce production of the TTR protein that misfolds in hereditary ATTR amyloidosis. Newer versions require less frequent dosing and are being tested in broader patient groups.
AL amyloidosis therapies
Trials are testing targeted antibodies that bind to the abnormal light chains in AL amyloidosis before they form deposits. Other studies combine existing chemotherapy with newer drugs to achieve faster, deeper responses.
What to know before you search
Eligibility typically depends on amyloidosis type (AL, ATTR, or other), which organs are affected, severity of heart or kidney involvement, and prior treatments received.
What types of trials are currently open
- Amyloid-targeting trials — Testing drugs that directly remove or prevent formation of amyloid deposits in organs like the heart and nerves.
- Gene therapy trials — For hereditary ATTR amyloidosis, testing one-time treatments that turn off the gene making the misfolded protein.
- Combination trials — Testing whether combining a drug that stops amyloid production with one that removes existing deposits works better than either alone.
- Cardiac function trials — Studying treatments specifically for people whose amyloidosis has affected the heart, measuring survival and heart function improvements.
- Quality of life studies — Following people with amyloidosis to understand symptom burden, treatment side effects, and how new therapies affect daily function.
Recently added Amyloidosis trials
CMN LIFU for Enhanced Amyloid Reduction (CLEART Study)
The goal of this clinical trial is to learn if a wearable ultrasound device, called ATTN201, is safe and easy to use. The study is for adults ages 65 and older who have mild cognitive impairment (MCI) and a buildup of a protein called amyloid in the brain. Amyloid buildup is an early sign of Alzheimer's disease. The ATTN201 device sends low-intensity sound waves to a deep area of the brain that helps control sleep. The aim is to boost deep-sleep brain waves. These brain waves may help the brain clear away waste, including amyloid. The main questions it aims to answer are: 1. What, if any, issues do participants have when using the ATTN201 device? 2. Can participants complete the full study schedule? Researchers will compare focused ultrasound to unfocused ultrasound. Unfocused ultrasound is a look-alike treatment. It feels and sounds the same but is not aimed at the brain target. The comparison will show if the focused treatment changes sleep brain waves, blood markers of Alzheimer's disease, or memory and thinking. Participants will: * Visit the study site for memory and thinking tests, blood draws, a health check, and MRI scans of the brain * Record their sleep at home for at least 5 days with a sleep diary, a watch-like sensor, and a headband that measures brain waves * Be assigned by chance to focused or unfocused ultrasound * Take part in 10 nap sessions over 2 weeks while wearing the device, at the study site or at home * Return for a final visit to repeat the first tests, and may return for an optional visit 3 months later
Receive low-dose radiation or sham therapy twice weekly for 3 weeks
This randomized, sham-controlled, exploratory clinical trial will evaluate the efficacy and safety of HeLaXON, an investigational low-dose radiation therapy device, in patients with mild Alzheimer's disease dementia. A total of 30 participants will be randomly assigned in a 1:1 ratio to receive either low-dose whole-brain radiation therapy or a sham procedure using the same device. Participants in the treatment group will receive 0.04 Gy per session twice weekly for 3 weeks, for a total of 6 sessions and a cumulative dose of 0.24 Gy. Participants in the sham group will undergo the same device procedures without radiation delivery. Participants and outcome assessors will remain blinded to treatment assignment. Changes in cognitive function, dementia severity, daily functioning, and amyloid burden will be evaluated at 6 and 12 months after treatment. Safety will be assessed throughout the study. Exploratory blood biomarkers, including plasma Aβ42/40 ratio, p-tau217, and Aβ oligomer, will also be evaluated.
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