Clinical trials: Hereditary Amyloidosis, Transthyretin-Related
Polyneuropathies, Wild Type ATTR Amyloidosis
Spectrum of Peripheral and Autonomic Neuropathies in Patients With aTTRwt Amyloidosis and Response to Patisiran Therapy
To evaluate the efficacy and safety of patisiran in patients with wtATTR amyloidosis and symptomatic polyneuropathy by evaluating the effect on neurologic impairment and quality of life.
- Conditions
- Polyneuropathies, Wild Type ATTR Amyloidosis, Wild-Type Transthyretin-Related (ATTR)Amyloidosis, Wild-Type Transthyretin Cardiac Amyloidosis
- Trial name
- NCT05023889
- Testing
- patisiran
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Austin Neuromuscular Center
Amyloidosis, Hereditary, Transthyretin-Related
Exercise in Hereditary ATTR (ATTRv) Amyloidosis
This study aims to explore the effects of a 12-week exercise intervention on the physical and mental health of people living with Hereditary ATTR (ATTRv) Amyloidosis, a rare genetic disease caused by mutations in the…
- Trial name
- NCT07033715
- Testing
- Exercise
- Where
- Portugal
- Volunteers
- 42 people
- Sponsor
- University of Maia
Familial Amyloid Polyneuropathy, Familial Amyloidosis
The Effect of Diflunisal on Familial Amyloidosis
The purpose of this study is to determine if diflunisal can prevent progressive lower leg nerve damage in patients with familial amyloidosis polyneuropathy.
- Trial name
- NCT00294671
- Testing
- diflunisal
- Where
- 8 sites in United States, Italy and 3 more
- Volunteers
- 130 people
- Sponsor
- Boston University
Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
A Study to Learn About the Use of Acoramidis in Patients With a Heart Condition Called Transthyretin Amyloid Cardiomyopathy (ATTR-CM) in a Real-world Setting
Transthyretin Amyloid Cardiomyopathy (ATTR-CM) is a serious and life-threatening condition where a protein called transthyretin (TTR) misfolds and builds up as amyloid fibrils in the heart muscle.
- Trial name
- ACO-REAL (NCT07235462)
- Testing
- Acoramidis (356 mg film-coated tablets)
- Where
- 17 sites in Austria, Belgium and 15 more
- Volunteers
- 2,000 people
- Sponsor
- Bayer
Amyloidosis, Transthyretin Amyloidosis
Characterizing Iodine-124 Evuzumitide (AT-01) in Systemic Amyloidosis
This is a single center prospective study evaluating 124I-evuzumitide in patients with systemic amyloidosis. The purpose of this study is to 1) Establish the diagnostic accuracy of 124I-evuzumitide in cardiac amyloidosis 2)…
- Trial name
- NCT05758493
- Testing
- 124I-Evuzamitide
- Where
- United States
- Volunteers
- 150 people
- Sponsor
- Oregon Health and Science University
Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
Efficacy and Safety of AG10 in Subjects with Transthyretin Amyloid Polyneurophathy
See updated study design under NCT04882735. Phase 3 efficacy and safety of AG10 compared with placebo in subjects with symptomatic Transthyretin Amyloid Polyneuropathy (ATTR-PN)
- Trial name
- ATTRibute-PN (NCT04418024)
- Testing
- AG10
- Where
- United States
- Sponsor
- Eidos Therapeutics, a BridgeBio company
Amyloidosis, Amyloid Cardiomyopathy
Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Cardiomyopathy
Phase 3 efficacy and safety study to evaluate acoramidis (AG10) HCl 800 mg administered orally twice a day compared to placebo in subjects with symptomatic Transthyretin Amyloid Cardiomyopathy (ATTR-CM).
- Conditions
- Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis, Cardiomyopathies
- Trial name
- ATTRibute-CM (NCT03860935)
- Testing
- acoramidis
- Where
- 104 sites in United States, Australia and 16 more
- Volunteers
- 632 people
- Sponsor
- Eidos Therapeutics, a BridgeBio company
Hereditary Transthyretin Amyloidosis
hATTR Polyneuropathy in Russia
A multicenter observational retrosPective Registry of patIents with transthyretin aMyloid polynEuropathy (hATTR-PN) and chRonic idiopathic axonal polyneuropathy (CIAP) in the population of the Russian Federation (PRIMER) There…
- Trial name
- PRIMER (NCT06365593)
- Where
- 3 sites in Russia
- Volunteers
- 100 people
- Sponsor
- AstraZeneca
Cardiomyopathies, Primary
Parametric Cardiac 18F-flutemetamol PET Imaging in ATTR Cardiomyopathy
18F-Flutemetamol (Vizamyl) is a radioactive diagnostic agent indicated and FDA-approved for Positron Emission Tomography (PET) imaging of the brain to estimate β-amyloid neuritic plaque density in adult patients with cognitive…
- Trial name
- NCT05374564
- Testing
- (18F)Flutemetamol
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Yale University
Familial Amyloid Polyneuropathy, ATTR-PN
An Extension of Study Fx-005 Evaluating Long-Term Safety And Clinical Outcomes Of Fx-1006A In Patients With Transthyretin Amyloid Polyneuropathy
This study is designed to determine the long-term safety and tolerability of Fx-1006A as well as the effects of Fx-1006A on clinical outcomes in patients with ATTR-PN.
- Trial name
- NCT00791492
- Testing
- Fx-1006A
- Where
- 11 sites in Argentina, Brazil and 4 more
- Volunteers
- 86 people
- Sponsor
- Pfizer
Amyloidosis, Hereditary, Amyloidosis Cardiac
Subclinical Transthyretin Cardiac Amyloidosis in V122I TTR Carriers
Approximately 1.5 million of the 44 million Blacks in the United States are carriers of the valine-to-isoleucine substitution at position 122 (V122I) in the transthyretin (TTR) protein.
- Conditions
- Amyloidosis, Hereditary, Amyloidosis Cardiac, Amyloidosis, Familial, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy
- Trial name
- NCT05489549
- Where
- 3 sites in United States
- Volunteers
- 500 people
- Sponsor
- University of Texas Southwestern Medical Center
Transthyretin Amyloidosis
Evaluation of a Claims-based Algorithm for the Identification of Transthyretin-mediated Amyloidosis (ATTR) Amyloidosis in Medical Records
The primary objective of this study is to evaluate the diagnostic performance of an algorithm in identifying patients with ATTR amyloidosis.
- Trial name
- NCT04569903
- Testing
- Computer algorithm for ATTR
- Where
- United States
- Sponsor
- Yale University
Amyloid Cardiomyopathy, Transthyretin-Related
Single and Multiple Ascending Dose Study Assessing the Safety, Tolerability, PK and PD of AG10
This is a single center, prospective, randomized, placebo-controlled study of AG10 in healthy adult subjects
- Trial name
- NCT03294707
- Testing
- AG10 oral tablet
- Where
- United States
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- Eidos Therapeutics, a BridgeBio company
Amyloidosis; Systemic
A Study to Characterize the Biodistribution of 124I-Labeled AT-03 in Patients With Systemic Amyloidosis
This is a single center Phase 1 study is to evaluate the biodistribution of radiolabeled AT-03 in patients with systemic amyloidosis.
- Trial name
- AT03-001 (NCT05201911)
- Testing
- 124I-AT03
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Attralus, Inc.
Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy, hATTR-PN
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…
- Trial name
- TRITON-PN (NCT07223203)
- Testing
- Nucresiran, Vutrisiran
- Where
- 61 sites in United States, Argentina and 20 more
- Volunteers
- 125 people
- Sponsor
- Alnylam Pharmaceuticals
Familial Amyloid Polyneuropathy
Safety and Efficacy Study of Fx-1006A in Patients With Familial Amyloidosis
This study will examine whether Fx-1006A is effective in halting the progression of Familial Amyloid Polyneuropathy (FAP). Deposition of TTR amyloid is associated with a variety of human diseases.
- Trial name
- NCT00409175
- Testing
- Fx-1006A
- Where
- 10 sites in United States, Argentina and 7 more
- Volunteers
- 128 people
- Sponsor
- Pfizer
Transthyretin Amyloidosis, Transthyretin Cardiac Amyloidosis
Prevalence of wtATTR-CM After Carpal Tunnel Release Surgery
Patients above the age of 60 will be recruited at their carpal tunnel release surgery. Biopsies will be taken from the wrist and examined for the presence of amyloid protein.
- Conditions
- Transthyretin Amyloidosis, Transthyretin Cardiac Amyloidosis, Wild-Type Transthyretin-Related (ATTR)Amyloidosis
- Trial name
- CACTuS2 (NCT04276220)
- Testing
- Tenosynovial biopsy
- Where
- Denmark
- Volunteers
- 109 people
- Sponsor
- Steen Hvitfeldt Poulsen
Transthyretin Amyloidosis
Patient-Reported Outcome Measures in Wild-Type and Variant Cardiac Transthyretin Amyloidosis
Specific, standardized, comprehensive, universally accepted Patient-Reported Outcome Measures (PROMs) are currently lacking for variant and wild-type cardiac amyloid transthyretin amyloidosis (v-ATTR/wt-ATTR).
- Trial name
- ITALY (NCT04563286)
- Testing
- questionnaire on life quality
- Where
- 5 sites in Italy
- Volunteers
- 250 people
- Sponsor
- University of Messina
Amyloidosis; Heart (Manifestation), Senile Cardiac Amyloidosis
Doxycycline and TUDCA in Patients With Transthyretin Amyloid Cardiomyopathy
The objective of the study is to determine whether the combination of the bile acid TUDCA, and doxycycline will slow the progression of familial and senile amyloidosis.
- Trial name
- NCT01855360
- Testing
- Tauroursodeoxycholic Acid and Doxycycline
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Brigham and Women's Hospital
Transthyretin Amyloidosis
The Regional Scintigraphic DPD Uptake in Cardiac Transthyretin Amyloidosis.
Cardiac transthyretin (ATTR) amyloidosis is an infiltrative cardiomyopathy with an inexorably progressive clinical course and poor prognosis.
- Trial name
- AMYLOIDOZA (NCT05814380)
- Testing
- 99mTc-DPD scintigraphy, Collection of blood samples and echocardiography
- Where
- Poland
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Katarzyna Holcman