Program for hereditary transthyretin amyloidosis
Part of Genetic & congenital, Hormones & metabolism clinical trials.
This trial is for people with a specific inherited form of amyloidosis caused by a change in the TTR gene. It offers a comprehensive care program to help manage this rare condition.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a confirmed genetic change (pathogenic variant) in the TTR gene.
- You must not have the non-inherited form of TTR amyloidosis (called wild-type).
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This study looks at people who carry certain TTR gene variants, which can cause a specific type of heart amyloid buildup. It also includes people with symptoms, to understand how the condition develops and how it may be monitored.
This study is observing people with amyloidosis or those who carry a specific gene mutation (transthyretin) to learn more about the condition. It does not test any new treatment, but may help researchers understand how the disease affects people.
This is an observational study (no study drug) for people with transthyretin (TTR)–mediated amyloidosis, including people who carry certain gene changes before symptoms begin. It may help researchers better understand how the disease affects health over time and what to measure in future treatments.
This study tests a structured psychological support program for people with hereditary transthyretin amyloidosis (ATTRv) with heart involvement, their caregivers, and those who carry the gene but have no symptoms yet. The goal is to see if the program is practical and well-accepted.
This study looks for “biomarkers” (measurable signs in blood or other tests) to understand inherited transthyretin amyloidosis, including in people who have not yet developed symptoms. Results may help researchers track the condition earlier and better understand how it changes over time.
This trial tests whether the drug tafamidis can help people with a specific type of nerve damage caused by abnormal protein buildup. It aims to slow or improve symptoms in adults with this condition.
Hear when a new Amyloidosis, Familial trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.