Clinical trials: Hereditary Amyloidosis, Transthyretin-Related
Transthyretin Amyloidosis, Carpal Tunnel Syndrome
Systemic Transthyretin Amyloidosis: Carpal Tunnel Syndrome in a Portuguese Population
Systemic transthyretin amyloidosis is an aging-related disorder. It is usually associated with cardiac disease but also extends to other organs.
- Trial name
- CarPoS (NCT05409833)
- Testing
- Synovial biopsy and Cardiac scintigraphy with [99mTc]Tc-DPD
- Where
- 2 sites in Portugal
- Volunteers
- 100 people
- Sponsor
- Universidade do Porto
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
Biomarker for Transthyretin-Related Familial Amyloidotic Polyneuropathy (BioTRAP)
International, multicenter, observational, longitudinal study to identify biomarker/s for the development of a new MS-based biomarker for the early and sensitive diagnosis of Transthyretin-Related Familial Amyloidotic…
- Conditions
- Transthyretin Amyloidosis, Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin Amyloid Cardiopathy
- Trial name
- BioTRAP (NCT02713880)
- Where
- 2 sites in Germany, India
- Sponsor
- CENTOGENE GmbH Rostock
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.
Transthyretin Amyloidosis
Study of Cerebral MRI Anomalies in Mutated Transthyretin Amyloidosis Patients
Transthyretin amyloidosis (aTTR) initially described as a rare disease, became the most serious hereditary polyneuropathy of adult onset and family screening has made it possible to identify and follow up many asymptomatic…
- Trial name
- TTR-SNC (NCT05075798)
- Testing
- No intervention,
- Where
- France
- Volunteers
- 36 people
- Sponsor
- Centre Hospitalier Universitaire de Nīmes
Hereditary Transthyretin-mediated (ATTRv) Amyloidosis, Polyneuropathy
A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation
To evaluate the effectiveness of patisiran in patients with ATTRv amyloidosis with polyneuropathy who have a V122I or T60A mutation.
- Trial name
- NCT04201418
- Testing
- Patisiran
- Where
- 27 sites in United States
- Volunteers
- 67 people
- Sponsor
- Alnylam Pharmaceuticals
Amyloidosis, Hereditary, Transthyretin-Related, Microbial Colonization
Analysis of the Instestinal Microbiome of Patients With Transthyretin Amyloidosis
Amyloidosis is a serious systemic disease. Cardiac involvement has a great impact on prognosis and can occur in its three main forms: acquired monoclonal light chain, hereditary transthyretinal and senile form.
- Trial name
- AMIAT (NCT05196594)
- Testing
- collection of stools for genetic analysis
- Where
- Brazil
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- University of Sao Paulo General Hospital
Amyloidosis, Cardiomyopathy
Carpal Tunnel Syndrome and Amyloid Cardiomyopathy
The investigators will prospectively evaluate tissue samples obtained from patients undergoing carpal tunnel release surgery for amyloid in the soft tissue that is removed.
- Conditions
- Amyloidosis, Cardiomyopathy, Transthyretin Amyloidosis, Senile Systemic Amyloidosis
- Trial name
- NCT02792790
- Where
- United States
- Volunteers
- 96 people
- Sponsor
- The Cleveland Clinic
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
Amyloid, Amyloidosis
Screening for Systemic Amyloidosis Via the Ligamentum Flavum
The investigators will prospectively evaluate for the presence of amyloid deposits in ligamentum flavum (yellow ligament) tissue samples obtained from patients undergoing spinal stenosis surgery.
- Conditions
- Amyloid, Amyloidosis, Spinal Stenosis, Transthyretin Amyloidosis
- Trial name
- ALF (NCT03923920)
- Testing
- Biopsy
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- The Cleveland Clinic
Transthyretin Amyloidosis
The Effect of Tafamidis on Transthyretin Stabilization, Safety, Tolerability and Efficacy in Transthyretin Amyloid Polyneuropathy Patients
Transthyretin amyloid polyneuropathy (ATTR-PN) is a fatal illness resulting from autosomal dominantly inherited single-point mutations on the transthyretin gene.
- Trial name
- NCT04963985
- Testing
- Tafamidis Pill
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Peking University Third Hospital
Transthyretin Amyloidosis
ATTR-Cardiomyopathy Stabilization Following Tafamidis Therapy
The study will investigate the stabilization effects of Tafamidis utilizing cardiac imaging cardiac magnetic resonance imaging (CMR). The investigators propose to pursue the following specific aims: 1.
- Trial name
- NCT04513600
- Testing
- Cardiac MRI
- Where
- United States
- Volunteers
- 131 people
- Sponsor
- The Christ Hospital
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
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
Transthyretin Amyloidosis, Amyloidosis, Leptomeningeal, Transthyretin-Related
Short-term Effects of TOLCAPONE on Transthyretin Stability in Subjects With Leptomeningeal TTR Amyloidosis (ATTR)
The purpose of this study is to determine whether Tolcapone crosses from the blood stream into the fluid around the brain and stabilizes the protein that makes leptomeningeal amyloid.
- Trial name
- NCT03591757
- Testing
- Tolcapone
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Boston University
Transthyretin-mediated Amyloidosis (ATTR Amyloidosis)
A Safety and Tolerability Study of an Investigational Drug, ALN-TTRSC02, in Healthy Subjects
The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of ALN-TTRSC02 in healthy subjects.
- Trial name
- NCT02797847
- Testing
- ALN-TTRSC02, Sterile Normal Saline (0.9% NaCl)
- Where
- United Kingdom
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Alnylam Pharmaceuticals
Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC), Amyloidosis, Hereditary
ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)
The purpose of this study was to evaluate the safety and efficacy of revusiran (ALN-TTRSC) in patients with transthyretin (TTR) mediated Familial Amyloidotic Cardiomyopathy.
- Conditions
- Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC), Amyloidosis, Hereditary, Amyloid Neuropathies, Familial, Amyloid Neuropathies
- Trial name
- NCT02319005
- Testing
- Revusiran (ALN-TTRSC), Sterile Normal Saline (0.9% NaCl)
- Where
- 61 sites in United States, Belgium and 7 more
- Volunteers
- 206 people
- Sponsor
- Alnylam Pharmaceuticals
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
Transthyretin Amyloidosis
Safety, Efficacy and Pharmacokinetics of Doxycycline Plus Tauroursodeoxycholic Acid in Transthyretin Amyloidosis
This study is being conducted to explore the potential benefits of a twelve-month doxycycline (at the best tolerated dose of 200 mg/day) and tauroursodeoxycholic acid (750 mg/day) treatment on disease progression in patients…
- Trial name
- NCT01171859
- Testing
- Doxycycline + Tauroursodeoxycholic acid
- Where
- Italy
- Volunteers
- 40 people
- Sponsor
- Fondazione IRCCS Policlinico San Matteo di Pavia