Transthyretin Amyloidosis Clinical Trials

7 recruiting

Transthyretin Amyloidosis Trials at a Glance

14 actively recruiting trials for transthyretin amyloidosis are listed on ClinicalTrialsFinder across 6 cities in 39 countries. The largest study group is Not Applicable with 3 trials, with the heaviest enrollment activity in Boston, Pavia, and Lisbon. Lead sponsors running transthyretin amyloidosis studies include Alnylam Pharmaceuticals, AstraZeneca, and Akcea Therapeutics.

Browse transthyretin amyloidosis trials by phase

Treatments under study

About Transthyretin Amyloidosis Clinical Trials

Looking for clinical trials for Transthyretin Amyloidosis? There are currently 7 studies actively recruiting participants. Clinical trials offer access to new treatments before they are widely available, and every approved therapy in use today was first tested through a clinical trial.

Below you can browse trials, sign up for alerts when new Transthyretin Amyloidosis trials open, and view eligibility criteria for each study. Each listing includes the study phase, locations, and enrollment details.

Frequently Asked Questions

Common questions about Transthyretin Amyloidosis clinical trials

A clinical trial is a carefully designed research study that tests new medical treatments, drugs, devices, or approaches in human volunteers. Every approved medication and treatment available today was proven safe and effective through clinical trials.

All clinical trials are reviewed and approved by Institutional Review Boards (IRBs) — independent committees that evaluate patient safety. Trials follow strict protocols, and your health is monitored closely throughout. You can withdraw at any time.

Not necessarily. Many trials compare the new treatment against the current standard of care, meaning all participants receive active treatment. When placebos are used, they are typically combined with standard treatment, not given alone. The trial description will always specify the design.

Under the Affordable Care Act, most private insurers are required to cover routine patient care costs during a clinical trial. The sponsor typically covers the investigational treatment itself. Medicare also covers routine costs for qualifying trials.

Yes. Participation is completely voluntary. You can withdraw at any time, for any reason, without it affecting your access to standard medical care.

Each trial has specific eligibility criteria — including age, diagnosis, disease stage, prior treatments, and general health. Browse the trials listed above and check their eligibility sections. You can also contact the trial site directly to discuss your situation.

Showing 114 of 14 trials

Recruiting

The Eplontersen Pregnancy and Lactation Outcomes Study

Transthyretin Amyloidosis
AstraZeneca10 enrolled1 locationNCT07205666
Recruiting

DemonsTTRate: A Global, Observational, Multicenter, Long-term Study of Patients With ATTR-CM in a Real-World Setting

Transthyretin Amyloidosis With Cardiomyopathy
Alnylam Pharmaceuticals2,000 enrolled4 locationsNCT07358078
Recruiting
Phase 3

TRITON-CM: A Study to Evaluate Nucresiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

Transthyretin Amyloidosis With Cardiomyopathy
Alnylam Pharmaceuticals1,250 enrolled207 locationsNCT07052903
Recruiting
Phase 2

Characterizing Iodine-124 Evuzumitide (AT-01) in Systemic Amyloidosis

AmyloidosisTransthyretin Amyloidosis
Oregon Health and Science University150 enrolled1 locationNCT05758493
Recruiting
Phase 3

Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant

CardiomyopathiesAmyloidosisHeart Diseases+3 more
Eidos Therapeutics, a BridgeBio company582 enrolled98 locationsNCT06563895
Recruiting
Phase 3

MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)

Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Intellia Therapeutics1,200 enrolled132 locationsNCT06128629
Recruiting

Non-interventional Study of Patients With Transthyretin (ATTR) Amyloidosis

Transthyretin AmyloidosisATTR-CMATTRv-PN+3 more
AstraZeneca1,850 enrolled53 locationsNCT06465810
Recruiting

A Non-interventional Cohort Safety Study of Patients With hATTR-PN

Hereditary Transthyretin Amyloidosis With Polyneuropthy
Akcea Therapeutics240 enrolled26 locationsNCT04850105
Recruiting

Phenotyping and Characterization of wtATTR-CM (TRACE 1)

Quality of LifeTransthyretin Amyloid CardiomyopathyTransthyretin Amyloidosis+1 more
Steen Hvitfeldt Poulsen120 enrolled1 locationNCT06291805
Recruiting
Not Applicable

An Interventional Pilot Study to Investigate the Feasibility and Acceptance of a Structured Psychological Support Program for Patients, Caregivers, and Presymptomatic Carriers in Hereditary Transthyretin Amyloidosis With Cardiomyopathy

Hereditary Transthyretin Amyloidosis
Fondazione IRCCS Policlinico San Matteo di Pavia150 enrolled1 locationNCT06907186
Recruiting
Not Applicable

Prevalence and Prediction of ATTR in Ambulatory Patients With HFpEF

Heart FailureAmyloidosisHeart Failure, Diastolic+1 more
Massachusetts General Hospital515 enrolled1 locationNCT05577819
Recruiting
Not Applicable

Exploring Biomarkers in Hereditary Transthyretin Amyloidosis

Hereditary Transthyretin Amyloidosis
Fondazione Policlinico Universitario Agostino Gemelli IRCCS80 enrolled1 locationNCT05929209
Recruiting
Phase 1Phase 2

A Phase I/IIa,Open-label, Single Ascending Dose and Dose-expansion Clinical Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of YOLT-201 in Patients With Transthyretin Amyloidosis Polyneuropathy (ATTR-PN) or Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)

Transthyretin Amyloidosis PolyneuropathyTransthyrexin Amyloidosis Cardiomyopathy
YolTech Therapeutics Co., Ltd31 enrolled3 locationsNCT06539208
Recruiting

New Biomarkers and Plasma Prothrombotic Potential in Cardiac Transthyretin Amyloidosis

Transthyretin Amyloidosis
John Paul II Hospital, Krakow100 enrolled1 locationNCT06345235