100 Clinical Trials for Various Conditions
The MaesTTRo study aims to enroll a global cohort of patients with transthyretin (ATTR) amyloidosis to longitudinally observe the natural course of the disease and describe real-world treatment patterns and outcomes. In addition, information on the effectiveness of ATTR amyloidosis treatments, including eplontersen, which is a ligand-conjugated antisense oligonucleotide gene silencing treatment targeting activity against both the mutant and wild-type TTR protein, will be collected.
To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.
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 vutrisiran as part of routine clinical practice in the real-world clinical setting * Describe disease emergence/progression in pre-symptomatic carriers of a known disease-causing transthyretin (TTR) variant
To evaluate the effectiveness of patisiran in patients with ATTRv amyloidosis with polyneuropathy who have a V122I or T60A mutation.
The purpose of this study is to evaluate the efficacy and safety of patisiran in participants with ATTR amyloidosis with cardiomyopathy.
The purpose of the study is to evaluate the whitening potential of different mineral and chemical sunscreens across multi-cultural skin tones through instrumentation, imaging, and self-assessment. It also aimed to evaluate the relationship between self-perception and objective measurements of whitening.
The purpose of this study is to evaluate the proportion of ocular adverse events reported in subjects with dry eye disease (DED) between the intervention arm (0.003% AR-15512) and Refresh® Classic.
The purpose of this study is to evaluate the safety, reactogenicity, and immunogenicity in relation to the product attributes of mRNA-1083 vaccine when administered as a single intramuscular (IM) injection in adults ≥50 to \<65 years of age.
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 humanistic outcomes (for example Health Related Quality of Life (HRQoL), Neuropathy impairment score, activities of daily living (ADL) assessments) in patients with ATTR amyloidosis, and to better understand how the disease is presented.
The primary objective of this study is to access the efficacy of ALXN2220 in the treatment of adult participants with ATTR-CM by evaluating the difference between the ALXN2220 and placebo groups as assessed by the total occurrences of all-cause mortality (ACM) and cardiovascular (CV) clinical events.
This is an observational, retrospective non-inferiority study with a study sample from a large national database. A machine learning (ML) model will use a national database to predict the clinical diagnosis of ATTRwt-CM among HF patients. This study will include HF patients ≥50 years old.
The study design is a prospective registry including asymptomatic and symptomatic patients who carry a pathogenic TTR mutation. The study will enroll patients who meet the inclusion criteria and none of the exclusion criteria until 1000 patients are enrolled, at which point in time the study investigators will evaluate whether further patient accrual is meaningful.
This study will investigate 99mTc-p5+14, an amyloid-reactive synthetic peptide, p5+14, radiolabeled with technetium-99m, as a radiotracer for detecting paamyloid deposits in patients with AL or ATTR-associated systemic amyloidosis, notably with cardiac involvement.
The purpose of this research is to see if information from blood and data from smartwatches can be combined to help diagnose depression and determine if transitions between active depression and treated depression can be predicted.
The purpose of this study is to analyze various cosmetic endpoints, subjective assessments, and skin parameters such as hydration, transepidermal water loss, fine lines, and wrinkles with the use of a 3-part skin care regimen.
The purpose of this study is to evaluate the safety and tolerability of extended dosing with eplontersen in participants with ATTR-CM.
This is a single center, prospective cohort study that is evaluating the ability of 124I-evuzamitide PET scanning to detect potential therapeutic changes in subjects under treatment for ATTR after one year has elapsed since their original 124I-evuzamitide PET scan. Ten previously scanned subjects will re-consent to undergo another 124I-evuzamitide PET scan. Demographic, clinical and phenotypic data will be collected to characterize potential changes since their previous scans.
Recent studies have shown that transthyretin amyloidosis (ATTR) can sometimes cause a type of heart failure where the pumping function of the heart is normal, also known as Heart Failure with Preserved Ejection Fraction (HFpEF) or diastolic heart failure. In this single center diagnostic study, we will evaluate for ATTR in patients with HFpEF in order to to determine how frequently this occurs and how we can predict which heart failure patients may have TTR amyloidosis. Our goal is to identify amyloidosis in heart failure patients earlier so that they can start treatment.
A research team member will brush the inferior surface of the subjects' middle turbinate (nasal cavity) using a cytology brush to obtain the cells needed to perform our functional respiratory assays. An individual trained in phlebotomy will draw one 3 ml lavender top tube of blood to test c-reactive protein, calprotectin, and lactoferrin. They will also draw a 5 ml gold top serum separator tube of blood to test fibroblast growth factor-19. The participant will answer questions from the baseline survey and report their current medications interview-style with the research team member.
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 impairment who are being evaluated for Alzheimer's disease (AD) or other causes of cognitive decline. This study is designed to evaluate a novel use for 18F-Flutemetamol in cardiac amyloidosis.
Evaluation on the effects of smiling on first impressions and attractiveness with and without wearing a facial surgical mask.
The study will be performed with oral gels, with up to 55 study subjects who meet the inclusion and non-inclusion criteria required. Study subjects will be instructed to use the products during 12 weeks and they will be assessed and supervised throughout the study by clinic technicians, in order to check for possible adverse events. Efficacy documentation through digital photography, instrumental measurements with the devices Cutometer® MPA 580 and Tewameter®, image acquisition with Visia-CA® as well as self-assessment through questionnaires will be carried out.
The purpose of the study is to evaluate and compare different tools that are used to detect evidence of peripheral neuropathy in patients with TTRv.
The investigators are examining how weight distribution affects the way people walk, in terms of joint kinematics, kinetics, and muscle activity. The investigators are measuring these quantities while people walk while wearing a weighted belt. The investigators distribute the weights and walk for specified periods. They hypothesize that greater weight will have a greater effect on walking.
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.
Open-Label Extension and Safety Monitoring Study of Acoramidis (AG10) in Participants with Symptomatic Transthyretin Amyloid Cardiomyopathy Who Completed the Phase 3 ATTRibute-CM Trial (AG10-301)
The primary objective of this study is to evaluate the diagnostic performance of an algorithm in identifying patients with ATTR amyloidosis.
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. Utilize cardiac magnetic resonance to assess stabilization of ATTR after Tafamidis therapy based on extracellular volume mapping. 2. Investigate left ventricular myocardial mass, native T1, T2, and extracellular volume mapping after 12 month follow-up. 3. Utilize cardiac magnetic resonance feature tracking at baseline and at 12 month follow-up.
This study is a global, multi-center study designed to estimate the global prevalence of transthyretin amyloid cardiomyopathy (ATTR-CM) within a clinically at risk population \[participants with heart failure with preserved ejection fraction (HFpEF)\].
Neck pain, neck muscle weakness, and limited neck range of motion have been shown in individuals with sinus headaches. Individuals in this previous study self-diagnosed their sinus headaches. It is unknown whether or not individuals who have been diagnosed with sinus headaches according to diagnostic criteria also present with musculoskeletal impairments. The purpose of this study is to determine if musculoskeletal neck impairments are present in individuals diagnosed with headaches attributed to rhinosinusitis compared to people without headaches. Upper cervical range of motion can be measured using different tests. The side bend rotation test has been suggested as potentially more useful than the flexion rotation test in individuals with hyper-laxity, however, has not been examined in a symptomatic population. A secondary purpose is to compare the side bend rotation test to the cervical flexion rotation test in people with varying degrees of joint laxity.