Recruiting Studies
Advanced SearchData: ClinicalTrials.gov · Source of record for eligibility and locations
Top Cities for Hereditary Transthyretin Amyloidosis Clinical Trials
Hereditary Transthyretin Amyloidosis clinical trials are recruiting across 10 cities. Here are the cities with the most active studies:
Hereditary Transthyretin Amyloidosis Trial Phases
2 research organizations are sponsoring hereditary transthyretin amyloidosis trials.
About Hereditary Transthyretin Amyloidosis
Hereditary transthyretin amyloidosis (hATTR) is a rare, progressive, and fatal disease caused by misfolded transthyretin protein deposits in nerves and organs. It causes polyneuropathy and cardiomyopathy. Treatment includes TTR stabilizers (tafamidis) and gene-silencing therapies (patisiran, inotersen).
Clinical trials are advancing new treatments for hereditary transthyretin amyloidosis. Currently, 3 studies are recruiting a combined 52,256 participants across the United States. Research is being conducted by 2 organizations including AstraZeneca, Alnylam Pharmaceuticals.
2026 Hereditary Transthyretin Amyloidosis Research Landscape
As of July 2026, the hereditary transthyretin amyloidosis clinical trial landscape includes 3 actively recruiting studies across 10 cities in the United States. These studies are collectively seeking 52,256 participants, with an average enrollment target of 17,419 per study.
The research spans multiple phases of development: 1 study is in PHASE3. The presence of Phase 3 trials indicates that some hereditary transthyretin amyloidosis treatments are in late-stage testing and may be approaching regulatory review.
Research is being led by 2 different organizations, including AstraZeneca, Alnylam Pharmaceuticals.
Geographically, hereditary transthyretin amyloidosis trials are most concentrated in Eden Prairie, Minnesota (1 trial); Aurora, Colorado (1 trial); Chicago, Illinois (1 trial); Baltimore, Maryland (1 trial); Boston, Massachusetts (1 trial) and 5 other cities.
Featured Hereditary Transthyretin Amyloidosis Studies
Highlighted recruiting studies for hereditary transthyretin amyloidosis, selected by enrollment size and research scope.
OverTTuRe: Characteristics, Treatment Patterns and Outcomes of Patients With ATTR Amyloidosis
The overall aim of this observational study is to generate real-world evidence on the pre- and post-diagnosis disease journeys, including baseline characteristics, treatment patterns and selected clinical, economic, and humanistic outcomes (for example Health Related Quality of Life (HRQoL), Neuropathy impairment score, activities of daily living (ADL) assessments) in patients with ATTR amyloidosi...
TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy
The purpose of this study is to: * Determine the efficacy of nucresiran in patients with hATTR-PN by evaluating the effect on neurologic impairment, quality of life, nutritional status, disability, and gait speed * Demonstrate superiority of nucresiran compared to in-study vutrisiran with respect to serum transthyretin (TTR) levels
Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program
The purpose of this study is to collect and evaluate pregnancy outcomes, pregnancy complications, and fetal/neonatal/infant outcomes in women exposed to patisiran-LNP.
Frequently Asked Questions About Hereditary Transthyretin Amyloidosis Clinical Trials
Are there hereditary transthyretin amyloidosis clinical trials near me?
Yes, there are 3 hereditary transthyretin amyloidosis clinical trials currently recruiting across 10+ cities in the United States, including Eden Prairie, Minnesota; Aurora, Colorado; Chicago, Illinois. Browse the studies above to find one at a location convenient for you.
How do I join a hereditary transthyretin amyloidosis clinical trial?
To join a hereditary transthyretin amyloidosis clinical trial: 1) Browse the available studies on this page, 2) Click on a study that interests you, 3) Check the study locations to find a site near you, 4) Review the eligibility criteria, and 5) Contact the study site or complete the eligibility form. The process is free and you can withdraw at any time.
Are hereditary transthyretin amyloidosis clinical trials free?
Yes, participation in hereditary transthyretin amyloidosis clinical trials is free. Study-related treatments, medical tests, and doctor visits are provided at no cost to participants. Many studies also offer compensation for your time and travel expenses.
What types of hereditary transthyretin amyloidosis treatments are being studied?
Current hereditary transthyretin amyloidosis clinical trials are testing a range of approaches across PHASE3 (1 study). These include new drugs, combination therapies, medical devices, and other interventions sponsored by 2 research organizations.
Is it safe to participate in hereditary transthyretin amyloidosis clinical trials?
Clinical trials are carefully regulated by the FDA and institutional review boards (IRBs). All trials must follow strict safety protocols, and participants receive close medical monitoring throughout the study. You can withdraw from a trial at any time without penalty.
Data updated July 15, 2026 from ClinicalTrials.gov
About This Data
Data: ClinicalTrials.gov · Source of record for eligibility and locations
Clinical trial information on this page is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health (NIH) and National Library of Medicine (NLM). Study data is refreshed every hour to ensure accuracy.
Medical Disclaimer: The information provided on this page is for informational purposes only and does not constitute medical advice. Always consult with a qualified healthcare provider before making decisions about clinical trial participation or changes to your treatment plan.
Page reviewed by the HelloStudys Research Team · Last updated July 15, 2026 · Data from ClinicalTrials.gov