All Clinical Trials

The goal of this clinical trial is to assess whether Lemborexant can improve sleep in patients with epilepsy. Read Less

The goal of this study is to evaluate the impact of a mobile app video game called Digital Vaccine for Type 1 Diabetes (DVx-T1D)™, proposed as a low-risk, non-invasive, digital therapeutic candidate for behavior change in children 6 to 12 years of age with Type 1 Diabetes (T1D). The investigators hypothesize that adoption of DVx-T1D™ by T1D patients will positively influence healthy dietary and physical activity behaviors by providing nutrition and lifestyle education through a playful, Artificial Intelligence (AI)-based, cartoon-style medium of mobile gaming, and result in improvements in their T1D control. The investigators propose to conduct a longitudinal randomized controlled trial (RCT) over a 3-month period with a study population of 80 T1D participants (40 each in two arms of the trial) of 6-12 years old. The investigators will quantify the impact of the mobile game app plus standard care vs. standard care (with no exposure to the app) on T1D control (assessed by glycosylated hemoglobin or HbA1c measured during clinic visits and continuous glucose monitoring (CGM) parameters measured at home), physical activity levels and food choices of children (measured using food logs and surveys). Game telemetry, food logs, clinical, anthropometric, demographic, and survey data will be collected to obtain adequately powered, theory-driven evidence of the value of game-based approaches delivered via mobile apps. Read Less

The main aim of this study is to collect real-world longitudinal data on participants with β-thalassemia treated with betibeglogene autotemcel (beti-cel) in the post marketing setting. To assess the long-term safety, including the risk of newly diagnosed malignancies, after treatment with beti-cel and evaluate the long-term effectiveness of treatment with beti-cel. Read Less

The goal of this clinical trial is to demonstrate the feasibility of bladder transplantation in patients with terminal bladder diseases who would benefit from a new bladder or a combined kidney and bladder transplant. The main questions it aims to answer are: * Is human bladder transplantation feasible and safe? * How will the new bladder function in terms of storage and emptying? Participants will undergo a bladder-only or combined kidney and bladder transplantation. They will then be followed for two years to evaluate the efficacy, safety, and functionality of the bladder transplant. Read Less

The objective of this project is to trans-create, conduct user testing of a previously developed and piloted mHealth app, and user acceptability with parents of children in the acute phase post-allogeneic hematopoietic stem cell transplant (HSCT). Nationwide Children's Hospital (NCH) will be the primary data collection site and Columbia University Irving Medical Center will be a secondary site. Read Less

WiTNNess is designed to accurately document the natural course and variation of muscle disease caused by pathogenic changes of the TNNT1 gene. The primary aim of the study is to specify meaningful outcome measures for future clinical trials. WiTNNess is open to children and adults worldwide. Participants can choose to include their information once (cross-sectional cohort) or every few months (prospective cohort). Read Less

The goal of this clinical trial is to assess the safety, toxicity, and antitumor activity of fourth ventricular infusions of nivolumab plus 5-azacytidine for recurrent ependymoma and nivolumab plus methotrexate for recurrent medulloblastoma and other CNS malignancies. Additionally, the study will explore immunologic responses to nivolumab. The hypothesis is that local administration of nivolumab, an immune checkpoint inhibitor, is safe and will lead to even more robust treatment responses when administered following 5-azacytidine in patients with recurrent ependymoma or methotrexate in patients with medulloblastoma or other CNS tumors. Read Less

B cell-depleting therapies, such as ocrelizumab, are among the most effective medications currently available for the treatment of multiple sclerosis (MS). This suggests that B cells play a very important role in MS. While B cells are rapidly eliminated from the blood of patients treated with medications like ocrelizumab, little is known about how effectively B cells are eliminated from lymph nodes, which are important sites of B cell activation. This study is being conducted to determine to what extent B cells are targeted in lymph nodes following ocrelizumab treatment, which may have important consequences for long-term MS outcomes. Read Less

The goal of this clinical trial is to assess changes in functional and psychosocial outcomes following a 12-week adaptive rock-climbing program for children with congenital upper limb differences. The main questions it aims to answer are * Assess functional outcomes following a 12-week adaptive rock-climbing program. * Assess psychosocial outcomes following a 12-week adaptive rock climbing program. * Assess barriers to participation in adaptive sports. Participants will complete 12-week adaptive rock climbing program. Read Less

This is a Phase Ib/II, open-label, single institution dose-escalation, safety, pharmacokinetics, pharmacodynamic and efficacy study. Read Less

This clinical trial will examine the effects of legal services on primary care outcomes for medically underserved communities. The aims of the study are: 1. To test the effectiveness and cost-benefits of a critical-time intervention Medical-Legal Partnership (CTI-MLP) on patient outcomes. 2. To determine the most efficient mechanisms for CTI-MLP delivery. 3. To develop innovative community engagement strategies for addressing health-harming legal needs within community health centers. Eligible patients will be asked to complete a questionnaire 4 times, first when they join the study and then at 3 months, 6 months, and 12 months. In the survey, they will be asked to provide information about themselves, their health care, aspects of their daily life, and hardships they face. They will also allow researchers to access their electronic health record information housed in the community-based organization and attorney notes. Patient information will be completely confidential and de-identified, meaning, the research team will not know the identity of the person who answered the questions. Participating community health centers will be randomized (assigned by chance) to provide basic legal information and referral to legal aid; or have an attorney on-site to provide legal aid to those who screen for legal needs. Read Less

This is a prospective clinical trial intended to capture echocardiographic images in patients clinically indicated for an echocardiographic examination using an additional Point of Care Ultrasound device . The images collected will be used to test and possibly validation an AI base Aortic Valve Stenosis algorithm Read Less