Amyloidosis was once almost uniformly fatal, but transthyretin (ATTR) and AL amyloidosis now have multiple effective treatments. Gene-silencing drugs that lower amyloid production are extending life dramatically, and a new oral stabilizer for ATTR is changing the outlook for cardiac amyloidosis.
What's actually going on in research
Trials are testing gene-silencing drugs (siRNA and antisense), stabilizer pills like acoramidis and tafamidis, antibodies that clear amyloid already in tissues, and treatments for AL amyloidosis using daratumumab-based regimens. Researchers are also evaluating earlier diagnosis through cardiac imaging and biomarkers.
Gene-silencing drugs
siRNA and antisense drugs lower amyloid protein production by 80 percent or more. They are extending life in ATTR amyloidosis and are now being tested earlier and in more forms.
Amyloid-clearing antibodies
New antibodies aim to remove amyloid already deposited in the heart and other organs. Early trial results suggest they may reverse some damage, not just slow it.
Better detection
A simple bone scan (PYP) can diagnose cardiac ATTR amyloidosis without biopsy. Trials are studying screening in older adults with unexplained heart failure.
What to know before you search
Eligibility often depends on amyloidosis type (ATTR wild-type, hereditary, or AL), organs involved, stage, and prior treatments.
What types of trials are currently open
- Treatment trials — Testing gene-silencing drugs, stabilizers, and antibodies for ATTR or AL amyloidosis.
- Cardiac amyloidosis trials — Testing treatments specifically for amyloid affecting the heart.
- AL amyloidosis trials — Testing daratumumab-based and other regimens for AL amyloidosis.
- Screening trials — Testing strategies to find amyloidosis earlier, especially in older adults with heart failure.
- Observational studies — Following people with amyloidosis to understand progression and treatment response.
Recently added Amyloidosis trials
[64Cu]FBP8 PET for Early Detection of Intracardiac Thrombus in Amyloid Cardiomyopathy
The primary goal of this pilot study is to determine whether \[64Cu\]FBP8, a novel fibrin-binding positron emission tomography (PET) probe, can identify intracardiac thrombi when paired with simultaneous hybrid cardiac PET/MRI in twenty (20) individuals with transthyretin or light chain cardiac amyloidosis and atrial fibrillation (AF) or atrial flutter (AF). The primary hypothesis of this study is that \[64Cu\]FBP8 PET/MRI can identify intracardiac thrombi in \>90% of subjects with confirmed intracardiac thrombi based on transesophageal echocardiogram (TEE). In secondary analyses, the investigators will seek to determine associations between intracardiac thrombi and left atrial function and left ventricular amyloid burden.
Comparison of the Effects of Oral Hygiene Regimens on Clinical, Immunomodulatory, and Microbial Outcomes and Oral Tolerance in People With Gingivitis
This study is a six-week, double-blind, randomized, parallel-group controlled clinical study. The objective of this study is to evaluate the changes to the oral microbiome, inflammatory mediators, gingival health indices and to assess oral tolerance after 4 weeks of twice daily use of differing oral hygiene regimens including mouthwash compared to a control group. A follow-up assessment will be completed 2 weeks after cessation of treatments.
Find Amyloidosis trials matched specifically to you
Answer 3 quick questions and we'll show you trials that fit your situation.