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NCT ID: NCT05711992 Recruiting - Embryonal Tumor Clinical Trials

Rare Embryonal Tumors of the Central Nervous System: International Registry

Start date: February 1, 2023
Phase:
Study type: Observational

Central nervous system (CNS) tumors are the most common solid malignancies among children. Although some types of CNS tumors like medulloblastomas and low-grade gliomas are widespread and well-studied, there is a huge number of rare diseases that need further research. This international registry aims to establish a large multicenter database of pediatric and young adult patients with rare embryonal tumors of the central nervous system and describe the clinical presentations, diagnostics, treatment regimens, and outcomes. Embryonal tumors with multilayered rosettes (ETMR), FOXR2-activated CNS neuroblastoma, cribriform neuroepithelial tumor, and CNS tumor with BCOR internal tandem duplication are extremely rare embryonal tumors some of which were first described in the last edition of the World Health Organization (WHO) Classification of Tumors of the Central Nervous System. Objectives of the registry are 1) to evaluate prognostic factors, 2) to identify diagnostic and treatment gaps, 3) to investigate the characteristics and outcome of the disease with different treatment regimens, and 4) to generate data-based prospective diagnostic and treatment recommendations.

NCT ID: NCT05700149 Recruiting - Clinical trials for Nodal Marginal Zone Lymphoma

Integrated Molecular and Clinical Profiling to Improve Disease Characterization and Outcome Prediction in Nodal Marginal Zone Lymphoma

Start date: December 31, 2023
Phase:
Study type: Observational

International retrospective observational cohort study aimed to describe a molecular classification for NMZL.

NCT ID: NCT05605678 Recruiting - Hemophilia A Clinical Trials

Hemophilia Non-Interventional Study Prior to SerpinPC Intervention (PRESent-5)

Start date: December 9, 2022
Phase:
Study type: Observational

The primary objective of this study is to obtain prospective baseline documentation of annualized bleeding rates (ABRs) and treatment under standard-of-care (SOC) therapy among participants with hemophilia A or B. Participants in the study may be eligible to enroll in future planned interventional studies to be conducted by Sponsor.

NCT ID: NCT05430971 Recruiting - Clinical trials for Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)

Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) International Registry

Start date: July 1, 2022
Phase:
Study type: Observational [Patient Registry]

Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) is a very rare hematologic malignancy. Despite recent advances, at present there is no consensus on the optimal treatment of BPDCN. The optimal therapy of disease remains to be determined, and due to the rarity of cases, there is a need for international collaboration to collect data on BPDCN clinical presentations, diagnostics, treatment regimens and outcomes. Therefore, the objectives of this study are: (1) to build a large database of patients with BPDCN, (2) to investigate the characteristics and outcome of the disease with different treatment regimens, (3) to evaluate prognostic factors, and (4) to generate data-based prospective treatment recommendations.

NCT ID: NCT05257369 Recruiting - Jaundice, Neonatal Clinical Trials

Broad Band Emission LED Phototherapy Source Versus Narrow Band

Start date: January 1, 2022
Phase: N/A
Study type: Interventional

Phototherapy is the most frequently used treatment in neonatology when serum bilirubin levels exceed physiological limits. Light-emitting diodes (LEDs) are become routinely used for phototherapy in neonates with hyperbilirubinemia. Blue LED light with peak emission around 460 nm is regarded as the most suitable light sources for phototherapy and they recommended by most neonatal guidelines. However, the effectiveness of phototherapy with narrow-band LED light sources can be increased by expanding the spectral range of incident radiation within the absorption of bilirubin due to the strongly marked heterogeneity absorption properties of bilirubin in a different microenvironment. Longer wavelength light, such as green light, is expected to penetrate the infant's skin deeper. It is still controversial whether the use of green light has any advantage over blue light. The most effective and safest light source and the optimal method to evaluate phototherapy, however, remain unknown.The aim of this study was to compare, at equal light irradiance, the clinical efficacy of broad spectrum blue- green LED with blue narrow spectral band phototherapy device.

NCT ID: NCT05201638 Recruiting - Clinical trials for Multiple Sclerosis, Relapsing-Remitting

Study to Evaluate the Efficacy, Safety and Tolerability of IMU-838 in Patients With Relapsing Multiple Sclerosis

ENSURE-2
Start date: January 12, 2022
Phase: Phase 3
Study type: Interventional

Multi-Center, Randomized, Double-Blinded Phase 3 Study to Evaluate the Efficacy, Safety, and Tolerability of IMU-838 versus Placebo in Adults with Relapsing Multiple Sclerosis (ENSURE-2)

NCT ID: NCT05190991 Recruiting - Clinical trials for Familial Mediterranean Fever

Safety and Efficacy of RPH-104 Used to Prevent Recurrent Fever Attacks in Adult Patients With Colchicine Resistant or Colchicine Intolerant Familial Mediterranean Fever

Start date: October 5, 2021
Phase: Phase 2
Study type: Interventional

The primary purpose of this study is to assess the safety of the long-term treatment with RPH-104 at doses 80 mg or 160 mg once every 2 weeks in a population of patients with colchicine resistant or colchicine intolerant familial Mediterranean fever (FMF) who completed the core study, during which they received at least one dose of RPH-104. Long-term efficacy of RPH-104, the immunogenicity of the RPH-104, the pharmacokinetics of the RPH-104 and quality of life change in the population of patients receiving long-term treatment with RPH-104 will be assessed as well.

NCT ID: NCT05092776 Recruiting - Clinical trials for Familial Mediterranean Fever

Efficacy and Safety of RPH-104 for Resolution and Prevention of Recurring Attacks in Adult Subjects With Familial Mediterranean Fever With Resistance to or Intolerance of Colchicine

Start date: April 29, 2021
Phase: Phase 2
Study type: Interventional

The primary purpose of this study is to assess the efficacy and safety profiles of investigational product RPH-104 (R-Pharm Overseas, Inc., USA) for treatment of Familial Mediterranean Fever (FMF) in adult patients resistant/intolerant to colchicine (crFMF). Pharmacokinetic and pharmacodynamic parameters of RPH-104 single or multiple doses in this patient population will be assessed as well.

NCT ID: NCT05036161 Recruiting - Clinical trials for Predictive Value of Tests

ROX Index and ROX Vector to Predict Nasal High Flow / Continuous Positive Airway Pressure Failure in Neonates

Start date: September 6, 2021
Phase:
Study type: Observational

Nasal continuous positive airway pressure (CPAP) and Nasal High Flow (NHF) therapy are two primary therapies for the treatment of respiratory distress in newborns. However, a considerable number of infants, who are initially treated with CPAP and NHF, will develop worsening respiratory failure and eventually require intubation for mechanical ventilation and the administration of surfactant. Infants who fail noninvasive respiratory therapy may suffer the consequences of delayed intubation, surfactant administration and other adverse outcomes. The most challenging decisions in the management of respiratory distress after birth is to decide when to move from a noninvasive respiratory support to invasive mechanical ventilation and give surfactant to decrease pulmonary damage and improve outcomes. There are no clinically adequate predictors of early CPAP failure at the time of admission to the neonatal intensive care unit. Many measurements have been investigated for their ability to predict CPAP failure in infants such as fraction of inspired oxygen (FiO2), partial pressure of oxygen (PaO2), PaO2/FiO2 and the stable micro bubble test as soon as possible after birth. Roca and colleagues first established the ROX index to predict the success of NHF therapy in adults with pneumonia. The ROX index combines three common measurements: FiO2, peripheral oxygen saturation (SpO2) and respiratory rate. Combining the ROX values with the change in the respiratory rate and FiO2 can indicate whether escalation is required. It was proposed that XY plot of the key components of ROX may show the direction of changes in vector form. The investigators hypothesized that the ROX index and ROX vector can be used for predicting the failure of CPAP and NHF in neonates.

NCT ID: NCT04352634 Recruiting - Depression Clinical Trials

The Covid-19 HEalth caRe wOrkErS (HEROES) Study

HEROES
Start date: April 26, 2020
Phase:
Study type: Observational

Since December 2019 the world has been shaken with an enormous global threat: the Covid-19 pandemic. This new kind of coronavirus is generating an unprecedented impact both on the general population and on the healthcare systems in most countries. Health services are trying to expand their capacity to respond to the pandemic, taking actions such as increasing the number of beds; acquiring necessary equipment to provide intensive therapy (ventilators), and calling retired health professionals and health students so they can assist the overwhelmed health care workforce. Unfortunately, these organizational changes at health facilities, along with the fears and concerns of becoming ill with the virus or infecting their families, put an enormous emotional burden on workers in health services which may lead to negative outcomes on mental health in this population. Recent cross-sectional studies in China indicate that health service workers exposed to people with Covid-19 reported higher rates of depressive and anxious symptoms. This negative impact on mental health among health workers in China has also been informally reported in other countries where the Covid-19 pandemic has been devastating in its effects (such as Spain and Italy), as well as in countries where the pandemic is becoming a growing public health problem. This is particularly relevant in regions with fewer resources (Latin America, North Africa), where there are limited means and the response from the health system is usually insufficient. Moreover, it is necessary to study these negative effects longitudinally considering that some effects will appear over time (post-traumatic stress). The COVID-19 HEalth caRe wOrkErS (HEROES) study is a large, bottom-up, South-North initiative aimed to evaluate the impact of the COVID-19 pandemic on the mental health of health care workers (HCWs). HEROES encompasses a wide variety of academic institutions in 19 LMICs and 8 HICs, in partnership with the Pan American Health Organization (PAHO) and with support from the World Health Organization (WHO). The HEROES study is led by Dr. Rubén Alvarado at University of Chile, and Dr. Ezra Susser and Franco Mascayano at Columbia U Mailman School of Public Health.