Clinical trials: ATTR
ATTR Amyloidosis
OverTTuRe: Characteristics, Treatment Patterns and Outcomes of Patients With ATTR Amyloidosis
The overall aim of this observational study is to generate real-world evidence on the pre- and post-diagnosis disease journeys, including baseline characteristics, treatment patterns and selected clinical, economic, and…
- Trial name
- OverTTuRe (NCT06355934)
- Testing
- no intervention
- Where
- 29 sites in United States, Canada and 9 more
- Volunteers
- 52,121 people
- Sponsor
- AstraZeneca
Inflammatory Response
Relationship Between NLR and Prealbumin Levels With Diaphragm Thickness
A study to find the relationship between neutrophil to lymphocyte ratio (NLR) with diaphragm thickness of critical patients in ICU.
- Trial name
- NCT04014439
- Testing
- Ratio of Neutrophil to Lymphocyte, Level of Prealbumin Serum
- Where
- Indonesia
- Volunteers
- 30 people
- Sponsor
- Universitas Indonesia
Chronic Cholestasis, Severe Malnutrition
Prealbumin and IGF-1 Levels in Pediatric Chronic Cholestasis With Severe Malnutrition After Nutrition Therapy
The goal of this clinical trial is to learn if a high-energy liquid diet improves the nutritional status of children with chronic cholestasis (a liver condition that blocks bile flow).
- Conditions
- Chronic Cholestasis, Severe Malnutrition, Pediatric Cholestasis
- Trial name
- NCT07569003
- Testing
- Nutrition Therapy
- Where
- Indonesia
- Volunteers
- 15 people
- Sponsor
- Universitas Indonesia
Transthyretin Amyloidosis
The Eplontersen Pregnancy and Lactation Outcomes Study
The overall objective of this DPSS is to describe the occurrence of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant associated with exposure to…
- Trial name
- EPPRO (NCT07205666)
- Where
- Germany
- Volunteers
- 10 people
- Sponsor
- AstraZeneca
Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of ALXN2220 in Adults With Transthyretin Amyloid Cardiomyopathy
The purpose of Study ALXN2220-ATTRCM-302 is to evaluate the long-term safety, tolerability and efficacy of ALXN2220 in adult participants with ATTR-CM treated with ALXN2220 in addition to SoC who have completed Study…
- Trial name
- LTSAFE-ALXN (NCT07789483)
- Testing
- ALXN2220
- Where
- 195 sites in United States, Argentina and 25 more
- Volunteers
- 1,180 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Symptomatic Transthyretin Amyloid Cardiomyopathy
A Phase 3 Study of ALXN2060 in Japanese Participants With Symptomatic ATTR-CM
This prospective study is designed to evaluate the efficacy, safety, and tolerability of ALXN2060 (also known as AG10), as well as to establish its pharmacokinetic and pharmacodynamic profile in Japanese participants with…
- Trial name
- NCT04622046
- Testing
- ALXN2060
- Where
- 11 sites in Japan
- Volunteers
- 25 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
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
Hereditary Transthyretin-mediated (hATTR) Amyloidosis, Polyneuropathy
Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program
The purpose of this study is to collect and evaluate pregnancy outcomes, pregnancy complications, and fetal/neonatal/infant outcomes in women exposed to patisiran-LNP.
- Trial name
- NCT05040373
- Where
- 7 sites in United States, France and 5 more
- Volunteers
- 10 people
- Sponsor
- Alnylam Pharmaceuticals
Transthyretin-Mediated Amyloidosis, ATTR Amyloidosis
ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)
The purpose of this study is to: Describe epidemiological and clinical characteristics, natural history and real-world clinical management of ATTR amyloidosis patients Characterize the safety and effectiveness of patisiran and…
- Trial name
- ConTTRibute (NCT04561518)
- Where
- 40 sites in United States, Brazil and 10 more
- Volunteers
- 1,500 people
- Sponsor
- Alnylam Pharmaceuticals
Amyloidosis, Amyloid
Monitoring of Early Disease Progression in Hereditary Transthyretin Amyloidosis
This study measures circulating, misfolded ATTR oligomers in asymptomatic ATTRm amyloidosis genetic carriers longitudinally over five years.
- Conditions
- Amyloidosis, Amyloid, Amyloid Neuropathies, Familial, Amyloid Cardiomyopathy
- Trial name
- MED-hATTR (NCT03431896)
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- The Cleveland Clinic
Hereditary Amyloidosis, Transthyretin-Related, Asymptomatic Carrier State
Observational Study of Neurofilament Light Chain (NfL) as a Biomarker in Asymptomatic Carriers of the Transthyretin (TTR) Variants and Patients With Hereditary Transthyretin-mediated (hATTR) Amyloidosis With Polyneuropathy
This is a single-center observational study evaluating the potential value of NfL as a biomarker for diagnosis, detection of disease onset, monitoring of disease progression, and treatment response in asymptomatic carriers of TTR…
- Trial name
- NCT06360289
- Testing
- Standard of Care
- Where
- France
- Volunteers
- 346 people, healthy volunteers welcome
- Sponsor
- Alnylam Pharmaceuticals
Amyloidosis
Identification of Clinically Significant Markers of ATTRv in Pre-symptomatic Mutation Carriers.
The objective of this prospective observational study is to periodically monitor pre-symptomatic subjects carrying a mutation of Transthyretin (TTR), identified in the context of a family screening of affected proband, through…
- Trial name
- NCT05754099
- Where
- Italy
- Volunteers
- 20 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Hereditary Transthyretin-mediated Amyloidosis With Polyneropathy, Pregnancy
TEGSEDI Pregnancy Surveillance Program
This is a worldwide safety surveillance study of pregnancy outcomes in women with hATTR-PN who may have been exposed or were not exposed to TEGSEDI prior to or during the pregnancy and of pediatric outcomes up to 1 year of age.
- Trial name
- NCT04270058
- Testing
- inotersen
- Volunteers
- 20 people
- Sponsor
- Akcea Therapeutics
Aortic Stenosis Symptomatic, Amyloidosis Cardiac
ATTR Amyloidosis in Elderly Patients With Aortic Stenosis
Severe aortic stenosis is defined with a mean transvalvular pressure gradient (MTPG) > 40mmHg and a calculated aortic valve area of < 1cm2.
- Trial name
- NCT04061213
- Testing
- Tc-99m-DPD scintigraphy
- Where
- Switzerland
- Volunteers
- 489 people
- Sponsor
- Insel Gruppe AG, University Hospital Bern
Obesity
Circulating Levels of Pealbumin in Relation to Overweight or Obesity
The study was aimed to investigating the relationship between circulating level of prealbumin and parathormone (PTH), triglycerides and triiodothyronine (FT3) in overweight and obese subjects (BMI≥25 Kg/m2)
- Trial name
- NCT04333446
- Where
- Italy
- Volunteers
- 84 people, healthy volunteers welcome
- Sponsor
- Azienda Ospedaliera Specializzata in Gastroenterologia Saverio de Bellis
Transthyretin Familial Amyloid Polyneuropathy
Vyndaqel Drug Use Investigation (Regulatory Post Marketing Commitment Plan)
The purpose of this study is to understand safety (e.g., occurrence of adverse drug reactions [ADRs]) and efficacy data on the long-term use of Vyndaqel Capsules (hereinafter referred to as Vyndaqel) in all patients who received…
- Trial name
- NCT02146378
- Testing
- Vyndaqel
- Volunteers
- 500 people
- Sponsor
- Pfizer
Amyloidosis Cardiac
Multimodality Cardiac Imaging for Disease Progression in ATTR-CM
The goal of this clinical trial is to investigate whether new imaging techniques can help us to better understand the cardiac amyloidosis.
- Trial name
- FAPI-ATTR (NCT07112066)
- Testing
- FAPI tracer
- Where
- Switzerland
- Volunteers
- 50 people
- Sponsor
- Dominik Benz
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
Amyloidogenic Transthyretin (ATTR) Amyloidosis, Cardiac Amyloidosis
PET Imaging Study Using Evuzamitide to Detect Cardiac Amyloidosis in Patients With Inconclusive Nuclear Scans and Elevated TAD1 Levels
The goal of this study is to learn whether PET-CT imaging using evuzamitide can help diagnose transthyretin cardiac amyloidosis (ATTR-CA) in patients whose standard nuclear imaging results are unclear but who have elevated TAD1…
- Trial name
- TRACE (NCT07538518)
- Testing
- Evuzamitide
- Where
- 3 sites in United States
- Volunteers
- 25 people
- Sponsor
- Lorena Saelices
Alpha-Thalassemia, Beta-Thalassemia
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases.
- Conditions
- Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis
- Trial name
- NCT06539169
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- xCures