AMYLOIDOSIS, FAMILIAL
Clinical trials for AMYLOIDOSIS, FAMILIAL explained in plain language.
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Gene-Editing shot aims to halt rare nerve disease in phase 3 trial
Disease control Recruiting nowThis phase 3 trial tests a single dose of NTLA-2001, a gene-editing therapy, in 60 adults with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN), a rare genetic disease that damages nerves. Participants are randomly assigned to receive either the active treatmen…
Matched conditions: AMYLOIDOSIS, FAMILIAL
Phase: PHASE3 • Sponsor: Intellia Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 12:08 UTC
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Hidden heart risk: study aims to catch deadly protein buildup before symptoms start
Knowledge-focused Recruiting nowThis study is looking for early signs of heart amyloidosis in Black adults who carry a specific gene change (V122I TTR) that raises their risk. Researchers will use heart MRI scans and blood tests to detect protein buildup before symptoms appear. The goal is to find ways to diagn…
Matched conditions: AMYLOIDOSIS, FAMILIAL
Sponsor: University of Texas Southwestern Medical Center • Aim: Knowledge-focused
Last updated Jun 28, 2026 00:00 UTC
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New study aims to unravel mysteries of rare genetic disease
Knowledge-focused Recruiting nowThis observational study will follow 20 adults with a confirmed genetic mutation for hereditary transthyretin amyloidosis (ATTR), a rare and serious condition that causes abnormal protein buildup in organs. Researchers will conduct thorough medical exams, heart tests, and neurolo…
Matched conditions: AMYLOIDOSIS, FAMILIAL
Sponsor: Hospital de Alta Complejidad en Red • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:00 UTC