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Lingual microcystic lymphatic malformations (LMLMs) are rare congenital vascular malformations, presenting as clusters of cysts filled with lymph fluid or blood. They are responsible for a heavy burden even with small well-limited lesions because of oozing, bleeding, infections, …

This phase I trial studies the best dose of sonidegib when given together with pembrolizumab and to see how well they work in treating patients with solid tumor that has spread to other places in the body (advanced). Sonidegib may stop the growth of tumor cells by blocking some o…

Regularly attending for Pap test cervical cancer screening in a clinic is often unfeasible and/or unacceptable to many women. This study evaluates if mailing and testing self-sampled kits for high-risk human papillomavirus (HPV) can cost-effectively increase screening participati…

This study is an open-label extension to evaluate the safety and tolerability of long-term dosing of Losmapimod in patients with FSHD1 who participated in the ReDux4 study.

This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of Cipaglucosidase Alfa/Miglustat treatment in enzyme replacement therapy (ERT)-experienced and ERT-naïve pediatric subjects with Pompe disease, aged 0 to \< 18 years

This Phase I/Il studies the side effects of endobronchial ultrasound guided interstitial photodynamic therapy work in treating patients with lung cancer that has spread to nearby tissues or lymph nodes. Photodynamic therapy consists of injecting a light sensitive drug called a ph…

The proposed study conducted in adult and pediatric patients aims to evaluate the safety and clinical activity of atezolizumab + cobimetinib in advanced/metastatic soft tissue sarcomas (up to 80 patients).

This study is being conducted to find out if the safety and tolerability of an experimental cell therapy is safe to administer to patients with a newly diagnosed glioblastoma multiforme (GBM) in combination with temozolomide (TMZ).

This study is being conducted to validate if photoacoustic imaging potentially reduces benign surgeries without compromising cancer detection sensitivity. The study will also explore whether using the photoacoustic imaging/ultrasound technique has any potential with early ovarian…

For several decades, allogeneic hematopoietic stem cell trans-plantation (allo-HSCT) has remained an important strategy in the management of patients with high-risk hematological malignancies. The acceptance of umbilical cord blood (UCBT) and haploidentical grafts (Haplo) as viab…

Heart transplantation (TC) is the standard treatment for terminal heart failure. Chronic kidney disease (CKD) is a common complication responsible for increased mortality and morbidity. The main risk factors for progression to CKD are advanced age, pre-transplantation CKD, degrad…

The purpose of this study is to determine the recommended phase 2 dose of the drug Vorinostat in children, adolescents and young adults following allogeneic blood or marrow transplant (BMT) and determine whether the addition of Vorinostat to the standard graft versus host disease…

In this multi-center study, the investigators plan to develop a simple blood-based test for early detection of Alzheimer's disease (AD). The test is based on a single injection of Pramlintide, an amylin analogue and FDA-approved drug currently used for treatment of diabetes. The …

INTRODUCTION Trauma-affected refugees are at high risk of developing mental health problems including post-traumatic stress disorder (PTSD) and depression. In addition to traumatic stress, refugees are furthermore subject to a range of post-migration stressors e.g. unemployment, …

The objective of this study is to evaluate the metabolic effects of a comprehensive wide-spectrum supplement for children with ASD to determine whether it physiologically targets mitochondrial pathways known to be abnormal in children with ASD.The intervention is a commonly used …

"SKILLZ," is a mixed methods evaluation of the Grassroots Soccer (GRS) SKILLZ Package based in Lusaka, Zambia. The package is made up of three football-based programs: (1) SKILLZ-Girl - implemented in schools as part of a 10-week program culminating in a tournament event; (2) SKI…

Major depressive disorder (MDD) is a leading cause of disability worldwide. Up to 50% of patients experience treatment resistant depression (TRD), which accounts for a vast majority of disease burden. Current medications for TRD have limited efficacy and can be associated with in…

Hardening of the blood vessels, called arterial stiffness, is a risk factor for future heart disease and its causes are unclear. The proposed study will 1) randomly assign adolescents at high risk of stiffening blood vessels to take a protein supplement called carnitine and study…

Randomized controlled trial to assess the effectiveness and patient perception of the benefit of the Sana Pain Reliever in individuals with chronic neuropathic pain.The study is fully remote with four study visits taking place over teleconferencing and the study devices mailed to…

The purpose of this study is to assess the safety and tolerability of BGB-A445 alone and in combination with tislelizumab in participants with advanced solid tumors; and to determine the maximum tolerated dose(s) (MTD) or maximum administered dose(s) (MAD) and recommended Phase 2…