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Filter by:Wild-type transthyretin cardiac amyloidosis (ATTRwt) is a deposition disorder in which one of the proteins of the body misfolds and accumulates at various places in the body, including the heart, leading to both mechanical and cellular damage. The gradual development of the disease will ultimately lead to heart failure and death The protein which deposits in the heart of patients, damages both the heart mechanically as the myocardium becomes rigid and hypertrophic over time but also at the cellular level. Cell damage can be observed by elevated blood tests for cell damage (Troponin) and during exercise tests that show patients' hearts burning oxygen inefficiently when exposed to physical stress compared with the hearts of healthy individuals . No one has, however, intimately studied this cellular damage. Vastarel® (Trimetazidine, TMZ) is an already known drug for the treatment of chest pain. The mechanism of action indicates that it may have an effect on patients with cardiac amyloidosis. The study aims to investigate the effects of TMZ on the mitochondrial function, myocardial performance, and invasive hemodynamics in patients with ATTRwt with a randomized, double-blinded, crossover-trial.
The study aims to investigate the safety, tolerability, efficacy, pharmacodynamics (PD), pharmacokinetics (PK), and immunogenicity of efgartigimod compared to placebo in participants with post-COVID-19 postural orthostatic tachycardia syndrome (POTS) (post-COVID-19 POTS).
Hypermobility Spectrum Disorder and hypermobile Ehlers-Danlos Syndrome (HSD/hEDS) is under-recognized and poorly understood and its management is therefore not clear. The goal of this study is to better understand pain and its impact on function in the daily activities of adolescents with Hypermobility Spectrum Disorder and/or hypermobile Ehlers-Danlos Syndrome. This study will explore the presence of the pain sensitivity status after physical exercise as well as movement behaviour in adolescents with HSD/hEDS compared to a healthy control group.
Hereditary hemorrhagic telangiectasia (HHT) or Osler-Weber-Rendu syndrome patients are carriers of a heterozygous mutation of the activin receptor-like kinase 1 (ACVRL1), Endoglin (ENG) or Mothers against decapentaplegic homolog 4 (SMAD4) gene. HHT involves the Bone Morphogenetic Protein 9 (BMP9)/Activin receptor-Like Kinase 1 (ALK1)-endoglin signalling pathway. BMP9 is a growth factor that binds to ALK1 receptor and to endoglin its co-receptors and physiologically activates Smad signaling pathway. Endothelial cells in HHT patients display half expression of functional ALK1 receptors or endoglin co-receptors or of the transcription factor SMAD4, which should lead to effects on the functions of these cells. The identification of differences in gene expression between endothelial cells from HHT patients and healthy donors will allow the identification of new functions or new target pathways for therapy. Circulating endothelial cells are rare in the bloodstream in adults, but are present in greater quantities in cord blood.
To evaluate the effects of oral administration rifampicin or itraconazole capsules on the pharmacokinetics of ASK120067 and its metabolite CCB4580030 in healthy subjects. To evaluate the safety of ASK120067 tablets or combination with rifampicin capsules or itraconazole capsules in healthy subjects.
The goal of this clinical trial is to evaluate the efficacy and safety of CTx-1301 in adults with ADHD in a laboratory classroom setting.
Patients in the severe stages of Chronic limb-threatening ischemia (CLTI) are prone to amputation and death, leading to poor quality of life and a great socioeconomic burden. There is an urgent need to develop an effective therapeutic strategy to treat this disease. In this context, autologous bone marrow mononuclear cells (BM-MNC) and allogeneic mesenchymal stem cells derived from different sources have emerged as promising therapeutic approaches for this condition.
Hypertensive disorders of pregnancy (HDP) are major causes of maternal morbidity and mortality globally and are associated with peripartum and future cardiovascular disease, including stroke, heart failure, and myocardial infarction. About 1 out of every 10 maternal deaths in Sub-Saharan Africa are associated with HDP, and most of these deaths are preventable with timely, implementation of evidence-based strategies, including postpartum blood pressure (BP) monitoring, treatment of elevated BP and comprehensive postpartum follow up as recommended by the American College of Obstetricians and Gynecologists (ACOG) and the World Health Organization (WHO). This study aims to contextualize, implement, and evaluate the feasibility, fidelity, and acceptability of: 1) postpartum remote blood pressure monitoring and 2) NTproBNP testing for postpartum risk stratification in women with HDP at the University of Abuja Teaching Hospital in the Federal Capital Territory of Nigeria
The purpose of this clinical trial is to evaluate the safety, tolerability and pharmacokinetics (PK) profile of TNM001 injection in healthy preterm and term infants. The main questions it aims to answer are: - the safety and tolerability of TNM001 injection - the pharmacokinetic (PK) profile of TNM001
The goal of this pilot study is to evaluate the prospective performance of an image-based, smartphone-adaptable artificial intelligence electrocardiogram (AI-ECG) strategy to predict and detect left ventricular systolic dysfunction (LVSD) in a real-world setting.