Clinical trials: Hereditary Transthyretin Amyloid Neuropathy
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
Transthyretin Amyloidosis, Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
TRAMmoniTTR Study Genetic Screening of an At-risk Population for hATTR and Monitoring of TTR Positive Subjects
National, multicenter, epidemiological, longitudinal protocol to investigate the hATTR prevalence in an at-risk population for Hereditary Transthyretin Amyloidosis (hATTR) and subjects diagnosed with hATTR, to monitor the…
- Conditions
- Transthyretin Amyloidosis, Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy, Polyneuropathies
- Trial name
- TRAMmoniTTR (NCT03237494)
- Where
- 84 sites in Austria, Germany and 1 more
- Volunteers
- 5,028 people
- Sponsor
- CENTOGENE GmbH Rostock
FAP, Familial Amyloid Polyneuropathy
Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy
The purpose of this study is to evaluate the efficacy and safety of inotersen given for 65 weeks in participants with Familial Amyloid Polyneuropathy (FAP).
- Conditions
- FAP, Familial Amyloid Polyneuropathy, TTR, Transthyretin
- Trial name
- NCT01737398
- Testing
- Inotersen
- Where
- 24 sites in United States, Argentina and 8 more
- Volunteers
- 173 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Polyneuropathy, Amyloid, Neuropathic Pain
Screening for the Transthyretin-Related Familial Amyloidotic Polyneuropathy (TTR FAP)
An International, multicenter, epidemiological observational study investigating the prevalence of Transthyretin-Related Familial Amyloidotic Polyneuropathy (TTR-FAP) in participants with small fiber polyneuropathy of no obvious…
- Conditions
- Polyneuropathy, Amyloid, Neuropathic Pain, Cardiac Failure, Orthostatic Hypotension
- Trial name
- TRAP2-1 (NCT01705626)
- Where
- 11 sites in Austria, Hungary and 4 more
- Volunteers
- 500 people
- Sponsor
- CENTOGENE GmbH Rostock
Amyloid Cardiomyopathy, Wild Type ATTR Amyloidosis
Association Between Lifetime Physical Activity and Exercise and the Development of Wild-type Transthyretin Amyloid Cardiomyopathy
The aim of this study is to investigate the association between increased lifetime physical activity and the development of wild-type transthyretin amyloid cardiomyopathy.
- Trial name
- NCT06261216
- Testing
- Interview
- Where
- Austria
- Volunteers
- 180 people, healthy volunteers welcome
- Sponsor
- Medical University of Graz
Aortic Stenosis, Transthyretin Amyloidosis
Prevalence of Transthyretin Cardiac Amyloidosis in Clinically Significant Aortic Stenosis
The purpose of this study is to determine the prevalence of transthyretin cardiac amyloidosis (TTR-CA) among patients with moderate and severe aortic stenosis in Southeast Minnesota using 99mTc-PYP single-photon positive emission…
- Conditions
- Aortic Stenosis, Transthyretin Amyloidosis, Transthyretin Amyloid Cardiomyopathy, Transthyretin Cardiac Amyloidosis
- Trial name
- NCT04899180
- Testing
- 99mTc-PYP
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Mayo Clinic
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)
To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.
- Trial name
- NCT06128629
- Testing
- NTLA-2001
- Where
- 138 sites in United States, Argentina and 24 more
- Volunteers
- 1,200 people
- Sponsor
- Intellia Therapeutics
Hereditary Transthyretin Amyloidosis, Carpal Tunnel Syndrome
Hereditary Transthyretin Amyloidosis Polyneuropathy in Patients With Carpal Tunnel Syndrome in Russia
This is a multicenter observational study consisting of retrospective and prospective phases. The retrospective phase will entail secondary data collection from electronic or paper medical records of patients who underwent…
- Conditions
- Hereditary Transthyretin Amyloidosis, Carpal Tunnel Syndrome, Polyneuropathy, Amyloidosis
- Trial name
- LOCUS (NCT06414746)
- Where
- 13 sites in Russia
- Volunteers
- 721 people
- Sponsor
- AstraZeneca
Wild-type ATTR Amyloidosis, Amyloidosis Cardiac
ATTR Amyloid Cardiomyopathy: Characterization of Extracellular Vesicles as Potential Disease Stratifiers and Prognostic Biomarkers
This study explores whether extracellular vesicles (EVs) tiny particles released into the bloodstream by cells can serve as early and minimally invasive biomarkers for transthyretin amyloid cardiomyopathy (ATTR-CM).
- Conditions
- Wild-type ATTR Amyloidosis, Amyloidosis Cardiac, Amyloidosis, Hereditary, Amyloidosis Transthyretin
- Trial name
- EV-ATTR (NCT07314268)
- Where
- Brazil
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- University of Sao Paulo General Hospital
Transthyretin Amyloidosis, Cardiomyopathy
Safety and Efficacy Study of Doxycycline/UrsoDeoxyCholicAcid on Disease Progression in ATTR Amyloidosis
The primary objective for this study is to evaluate the efficacy of doxycycline + ursodeoxycholic acid (UDCA) on disease progression in Transthyretin Amyloidosis (ATTR) subjects with cardiomyopathy with or without neuropathy.
- Trial name
- Dox/Urso (NCT02016365)
- Testing
- Doxycycline, Ursodeoxycholic acid
- Where
- 3 sites in Sweden
- Volunteers
- 55 people
- Sponsor
- Umeå University
Amyloid Neuropathies, Familial
Early and Systematic Screening in Chronic Neuropathy
TTR-FAP is a rare disabling inherited disorder that predominantly affects the peripheral nervous system and the heart. Due to an important phenotypic and genetic heterogeneity, the diagnosis is often delayed, preventing therefore…
- Trial name
- TTR-FAP (NCT03720275)
- Testing
- Systematic screening of TTR mutations
- Where
- France
- Volunteers
- 130 people
- Sponsor
- University Hospital, Bordeaux
Amyloidosis, Amyloidosis Cardiac
NatiOnal Referral cenTEr Study of Transthyretin Amyloid Cardiomyopathy(ATTR) Patients on Tafamidis
All ATTRwt patients on tafamidis 61 mg treatment will be clinically evaluated before treatment initiation and subsequently every six months for the eligibility to continue tafamidis treatment, according to Italian Medicines…
- Conditions
- Amyloidosis, Amyloidosis Cardiac, ATTR Amyloidosis Wild Type
- Trial name
- NOTE-ATTR (NCT06251778)
- Where
- Italy
- Volunteers
- 200 people
- Sponsor
- Paolo Milani
Amyloidosis, Trigger Finger
Cardiac Amyloidosis Screening at Trigger Finger Release
The investigators will prospectively evaluate for the presence of amyloid deposits in soft tissue samples obtained from patients undergoing trigger finger release surgery.
- Conditions
- Amyloidosis, Trigger Finger, Transthyretin Amyloidosis, Primary Amyloidosis of Light Chain Type
- Trial name
- CAST (NCT03886155)
- Testing
- Biopsy
- Where
- United States
- Volunteers
- 107 people
- Sponsor
- The Cleveland Clinic