Amyloidosis happens when abnormal proteins fold incorrectly and build up in organs like the heart, kidneys, liver, and nerves. The most common types are AL amyloidosis (caused by plasma cells) and ATTR amyloidosis (caused by a liver protein called transthyretin). Treatment depends on type: AL amyloidosis is treated with chemotherapy-like drugs targeting plasma cells, while ATTR amyloidosis now has drugs that stop the liver from making the problem protein.
What's actually going on in research
Trials are testing combinations of plasma cell-targeting drugs for AL amyloidosis, including daratumumab paired with bortezomib or other agents. For ATTR amyloidosis, studies are comparing RNA-silencing drugs like patisiran and small molecules like tafamidis, and testing them earlier in disease. Researchers are also studying drugs that might break up amyloid deposits already formed in the heart and other organs.
Amyloid removal
Several drugs aim to clear amyloid deposits that have already formed, rather than just stopping new protein from accumulating. Early trials suggest some antibodies can reduce cardiac amyloid burden in ATTR and AL amyloidosis.
Earlier ATTR treatment
Studies are testing whether treating ATTR amyloidosis before severe heart damage occurs can prevent progression. Trials are enrolling people with early-stage disease or those who carry ATTR gene mutations but don't have symptoms yet.
Combination therapy for AL
Adding daratumumab to standard chemotherapy regimens has improved response rates in AL amyloidosis. Trials are now testing three- and four-drug combinations to deepen responses and help more people achieve remission.
What to know before you search
Eligibility typically depends on amyloidosis type, organs affected, severity of heart or kidney involvement, prior treatments, and genetic mutation status in ATTR cases.
What types of trials are currently open
- Treatment trials — Testing new drugs or combinations that target plasma cells in AL amyloidosis or stop transthyretin production in ATTR amyloidosis.
- Amyloid clearance trials — Testing antibodies and other agents designed to remove amyloid deposits from the heart, kidneys, or nerves.
- Early intervention trials — Treating people with early-stage disease or genetic mutations before organ damage becomes severe.
- Symptom and quality of life trials — Testing treatments for heart failure, neuropathy, fatigue, and other symptoms caused by amyloid deposits.
- Observational studies — Following people with amyloidosis to track how disease progresses and how well current treatments work in real-world settings.
Recently added Amyloidosis trials
Using Light Therapy for Mild Cognitive Impairment
The goal of this clinical trial is to test whether transcranial photobiomodulation (tPBM), a non-invasive brain stimulation technique using near-infrared light, can improve brain blood flow regulation (neurovascular coupling) and cognitive function in people with mild cognitive impairment (MCI). The main questions it aims to answer are: * Does tPBM enhance cognitive function and cerebral hemodynamic responses during memory and finger tapping tasks? * Does tPBM reduce oxidative stress, inflammation, and mitigate brain cell damage? * Is cognitive improvement linked to amyloid status, greater cerebral hemodynamic response, and lower levels of brain inflammation and oxidative stress? Researchers will compare an active tPBM treatment arm to a sham treatment arm to see if tPBM leads to measurable improvements in brain activity and cognitive function compared to no active stimulation. Participants will: * Receive a 20-minute-long active tPBM or sham stimulation session once per day, 6 times per week, for 12 weeks. * Complete questionnaires and an iPad-based cognitive testing protocol. * Complete memory and motor tasks while their brain activity is measured using non-invasive techniques: simultaneous functional near-infrared spectroscopy (fNIRS) and electroencephalography (EEG). Dynamic analysis of the vessels in the eye will also be performed based on eligibility. Transcranial Doppler (TCD) flowmetry is optionally performed. * Provide blood samples to test for biomarkers of inflammation, oxidative stress, and brain cell damage.
Concordance for Transthyretin Amyloidosis Between Synovial Biopsy and Anterior Carpal Ligament Biopsy
Transthyretin amyloidosis (ATTR) is an underdiagnosed condition that can present early as carpal tunnel syndrome, sometimes preceding cardiac involvement by several years. The recent emergence of new treatments underscores the importance of early diagnosis. Synovial biopsy performed during open carpal tunnel surgery is considered the gold standard for local screening, but it is less accessible, more difficult, and riskier to perform via endoscopy-a minimally invasive surgical technique that is currently the standard at our center and is becoming increasingly widespread. The anterior annular ligament of the carpus constitutes an alternative tissue to the synovium, easily accessible via endoscopy, safe, and standardizable. In this study, the systematic performance of an annular ligament biopsy in conjunction with a synovial biopsy during endoscopic carpal tunnel surgery would allow for the evaluation of the diagnostic concordance of the ligament biopsy by comparing it to the results obtained using the gold standard method. This strategy would also provide an opportunity to analyze the feasibility of systematic screening for ATTR on the ligament during endoscopic procedures. This approach could offer a practical and innovative method for the early identification of at-risk patients and facilitate appropriate and timely management of the condition.
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