Amyloid Neuropathies, Familial: Clinical Trials

58 recruiting, 53 completed, 36 unknown, 15 not yet recruiting. 43 Not applicable, 22 Phase 3, 13 Phase 2, 10 Phase 1, 9 Phase 4.

By phase and status

Phase	Trials	Status
Not applicable	43	Unknown 12, Recruiting 11, Completed 10, Not yet recruiting 7, Active not recruiting 1, Enrolling by invitation 1, Withdrawn 1
Phase 3	22	Completed 14, Recruiting 3, Active not recruiting 2, Withdrawn 2, Not yet recruiting 1
Phase 2	13	Completed 7, Recruiting 2, Enrolling by invitation 1, Not yet recruiting 1, Terminated 1, Unknown 1
Phase 1	10	Completed 5, Recruiting 3, Enrolling by invitation 1, Not yet recruiting 1
Phase 4	9	Recruiting 4, Completed 3, Terminated 2
Early Phase 1	4	Completed 2, Terminated 2

Follow this condition Literature Review of Amyloid Neuropathies, Familial trials Research Report on Amyloid Neuropathies, Familial

Trials newest first · largest first

Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.

Trial	Phase	Status	Sponsor	Enrollment	Start	Updated
To Evaluate the Long-term Safety and Tolerability of Acoramidis in Participants With Newly Diagnosed ATTR-CM (ACT-EARLY OLE) Acoramidis (AG10) - Amyloidosis in Transthyretin (TTR), Amyloidosis, Familial	Phase 3	Not yet recruiting	Eidos Therapeutics, a BridgeBio company	250	2026-11-01	2026-08-11
PET Imaging Study Using Evuzamitide to Detect Cardiac Amyloidosis in Patients With Inconclusive Nuclear Scans and Elevated TAD1 Levels Evuzamitide - Amyloidogenic Transthyretin (ATTR) Amyloidosis, Cardiac Amyloidosis		Not yet recruiting	Lorena Saelices	25	2026-10-01	2026-09-03
Right Ventricular Septal Biopsy to Detect Cardiac Amyloidosis ATTR-CM (Transthyretin Amyloid Cardiomyopathy), Heart Failure		Not yet recruiting	Geisinger Clinic	70	2026-10-01	2026-08-25
Safety and Efficacy Study of GEB-101 Injection in Subjects With TGFBI-related Corneal Dystrophy GEB-101 Injection - Hereditary Corneal Dystrophy, Corneal Abnormality	Phase 1 / Phase 2	Not yet recruiting	GenEditBio Limited	30	2026-10-01	2026-09-10
Impact of a Cardiac Rehabilitation Program on Patients With Cardiac Amyloidosis Cardiac Rehabilitation Program - Amyloidosis	Not applicable	Recruiting	University Hospital, Montpellier	40	2026-09-23	2026-10-08
Individualized Exercise Training in Cardiac Amyloidosis Exercise Intervention, Physical Activity Handout - Cardiac Amyloidosis	Not applicable	Not yet recruiting	Oregon Health and Science University	60	2026-09-15	2026-09-23
Prevalence of Cardiac Thrombi in Cardiac Amyloidosis Cardiac CT scan with contrast - Cardiac Amyloidosis	Not applicable	Recruiting	Centre Hospitalier Universitaire Dijon	200	2026-09-07	2026-09-17
Retrospective Identification of Scintigraphic Cardiac Amyloidosis (RISCA) Cardiac Amyloidosis, Transthyretin Amyloidosis		Not yet recruiting	University Hospital, Lille	57	2026-09-01	2026-05-15
Detection of Cardiac Amyloidosis Using Right Ventricular Sensing Amplitude in Patients With Left Ventricular Hypertrophy Cardiac amyloidosis screening during CIED implantation - Cardiac Amyloidosis, Left Ventricular Hypertrophy	Not applicable	Not yet recruiting	Assistance Publique - Hôpitaux de Paris	242	2026-09-01	2026-07-29
[64Cu]FBP8 PET for Early Detection of Intracardiac Thrombus in Amyloid Cardiomyopathy [64Cu]FBP8 PET/MR - Cardiac Amyloidosis	Phase 3	Recruiting	Brigham and Women's Hospital	20	2026-07-07	2026-07-21
AI-based Echocardiography for Detection of Cardiac Amyloidosis AI-based echocardiogram - Cardiac Amyloidosis, Heart Failure With Preserved Ejection Fraction (HFPEF)	Not applicable	Not yet recruiting	Germans Trias i Pujol Hospital	200	2026-07-01	2026-06-12
Early Screening for Cardiac Amyloid Using a History of Bilateral Carpal Tunnel Syndrome Technetium-99m Pyrophosphate (Tc-99m PYP) Scan - Bilateral Carpal Tunnel Syndrome (Diagnosis), Cardiac Amyloidosis	Not applicable	Not yet recruiting	London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's	139	2026-07-01	2026-05-12
Cardiac Magnetic Resonance and Echocardiography Study With Acoramidis Acoramidis (AG10) oral tablet - Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM), Amyloidosis	Phase 4	Recruiting	Eidos Therapeutics, a BridgeBio company	150	2026-07-01	2026-07-29
Screening, Characterization, and Longitudinal Follow-up of Patients With Cardiac Amyloidosis Amyloidosis Cardiac, Heart Failure		Not yet recruiting	Heinrich-Heine University, Duesseldorf	200	2026-06-15	2026-05-11
Fast Cardiac Bone SPECT-CZT in Transthyretin Cardiac Amyloidosis bone scintigraphy - Cardiac Amyloidosis		Not yet recruiting	Central Hospital, Nancy, France	200	2026-05-30	2026-05-26
Early Detection of Concealed Cardiac Amyloidosis Using AI-ECG and CT-Derived Extracellular Volume in Patients With Atrial Fibrillation AI-ECG analysis, CT-ECV analysis, Atrial fibrillation treatment - Atrial Fibrillation (AF), Cardiac Amyloidosis	Not applicable	Not yet recruiting	Samsung Medical Center	500	2026-05-01	2026-05-05
Hungarian National Systemic Amyloidosis Registry Systemic Amyloidosis, ATTR Amyloidosis		Recruiting	Semmelweis University	300	2026-03-08	2026-07-08
A Study Of Deep Learning For Echo Analysis, Tracking, And Evaluation Transthoracic Echocardiography (TTE) - Cardiomyopathy, Ischemic Heart Disease		Recruiting	Mayo Clinic	10040000	2026-02-26	2026-04-16
EFICAC-TTR Trial: Exercise and Nutritional Supplementation in Transthyretin Cardiac Amyloidosis Home-Based Multicomponent Exercise Program, Microcrystalline Cellulose Control Supplementation, Creatine and HMB Supplementation - Transthyretin Cardiac Amyloidosis	Not applicable	Completed	Universidad de Burgos	47	2026-02-03	2026-09-25
Increased Pacemaker Lower Rate in ATTR Cardiac Amyloidosis Increasing the pacemaker lower rate, Standard setting - Cardiac Amyloidosis, Pacemaker	Not applicable	Not yet recruiting	Region Skane	34	2026-02-01	2026-02-17

Showing 20 of 186. Sign in to see all of them.

Narrower conditions

Amyloidosis (50)  ·  Amyloidosis, Hereditary, Transthyretin-Related (29)  ·  Heart Failure (23)  ·  Cardiomyopathies (22)  ·  Alzheimer Disease (19)  ·  Amyloidosis, Familial (16)  ·  Polyneuropathies (15)  ·  Amyloid Neuropathies (11)  ·  Disease (10)  ·  Immunoglobulin Light-chain Amyloidosis (9)  ·  Aortic Valve Stenosis (8)  ·  Cardiomyopathy, Hypertrophic (8)  ·  Heart Diseases (6)  ·  Hypertrophy, Left Ventricular (6)

Broader conditions

Congenital, Hereditary, and Neonatal Diseases and Abnormalities (186)  ·  Nutritional and Metabolic Diseases (186)  ·  Proteostasis Deficiencies (185)  ·  Genetic Diseases, Inborn (184)  ·  Heredodegenerative Disorders, Nervous System (184)  ·  Metabolic Diseases (184)  ·  Metabolism, Inborn Errors (184)  ·  Nervous System Diseases (184)  ·  Neurodegenerative Diseases (184)  ·  Neuromuscular Diseases (184)  ·  Peripheral Nervous System Diseases (182)  ·  Amyloid Neuropathies (175)  ·  Amyloidosis, Familial (170)  ·  Amyloidosis (136)  ·  Cardiovascular Diseases (59)

Interventions

attruby (7)  ·  tafamidis (6)  ·  patisiran (5)  ·  4-(N-methylamino)-4'-(2-(2-(2-fluoroethoxy)ethoxy)ethoxy)stilbene (4)  ·  Echocardiography (3)  ·  Amyloid (2)  ·  Blood Specimen Collection (2)  ·  Exercise (2)  ·  Inotersen (2)  ·  NTLA-2001 (2)  ·  Tomography, Emission-Computed, Single-Photon (2)  ·  florbetapir (2)  ·  revusiran (2)  ·  2-phenyl-6-(2'-(4'-(ethoxycarbonyl)thiazolyl))thiazolo(3,2-b)(1,2,4)triazole (1)  ·  Adrenergic beta-Antagonists (1)

Sponsors

Alnylam Pharmaceuticals (9)  ·  Eidos Therapeutics, a BridgeBio company (9)  ·  Pfizer (7)  ·  Brigham and Women's Hospital (6)  ·  University Hospital Center of Martinique (5)  ·  University Hospital, Essen (5)  ·  Steen Hvitfeldt Poulsen (4)  ·  Assistance Publique - Hôpitaux de Paris (3)  ·  CENTOGENE GmbH Rostock (3)  ·  IRCCS Azienda Ospedaliero-Universitaria di Bologna (3)  ·  Intellia Therapeutics (3)  ·  Mayo Clinic (3)  ·  University of Texas Southwestern Medical Center (3)  ·  AstraZeneca (2)  ·  Cedars-Sinai Medical Center (2)

Countries

United States (58)  ·  France (34)  ·  Germany (28)  ·  Italy (27)  ·  United Kingdom (24)  ·  Spain (20)  ·  Brazil (15)  ·  Portugal (13)  ·  Sweden (13)  ·  Denmark (11)  ·  Belgium (10)  ·  Canada (10)  ·  China (10)  ·  Netherlands (10)  ·  South Korea (10)

Top investigators

Deniz Mutlu, MD (2)  ·  Jeff Packman (2)  ·  Mark McGovern, RN, CCRN (2)  ·  Peter Bauer, Prof. (2)  ·  Sharmila Dorbala, MD (2)  ·  Aiste Monika Jakstaite, MD (1)  ·  Alberto M. Marrra, Md,PhD (1)  ·  Amin Polzin, MD (1)  ·  Andrei Iagaru, MD (1)  ·  Andrew Stephens, MD, PhD (1)  ·  Angela Dispenzieri, MD (1)

Results publications

PMID 25803389, PMID 42389794, PMID 21732836, PMID 27494299, PMID 29972753, PMID 29972757, PMID 30558645, PMID 31353964, PMID 32107748, PMID 32524297, PMID 33070419, PMID 33825853, PMID 34355354, PMID 34396169, PMID 34923848, PMID 35799473, PMID 36715498, PMID 37434378, PMID 37943223, PMID 38548524, PMID 38932583, PMID 40488446, PMID 41205147, PMID 42658466, PMID 15044325, PMID 18242546, PMID 20103757, PMID 20150510, PMID 21427499, PMID 23478658, PMID 23644086, PMID 23974642, PMID 24368466, PMID 24841414, PMID 24928681, PMID 25190073, PMID 25950872, PMID 26253999, PMID 27206819, PMID 27339491, PMID 27511979, PMID 27618855, PMID 28393570, PMID 28611125, PMID 28893208, PMID 28987521, PMID 29052438, PMID 29115008, PMID 29389364, PMID 29596561

NIH funding

2018 (3), 2014 (2), 2015 (1), 2016 (1), 2017 (1), 2023 (1), 2025 (1), R01AG081582 (1), R01HL139671 (1), R01HL160892 (1), R01HL177670 (1), R01NS051306 (1), R24AG064025 (1)