Mar 17th, 2023 - PrismaLung+ is indicated to provide extracorporeal carbon dioxide removal (ECCO2R) as a stand-alone therapy or in combination with continuous renal replacement therapy (CRRT). This study is intended for patients with increased levels of Carbon Dioxide (CO2) in their blood and ≥ 18 years old, and will treat patients with mild or moderate acute respiratory distress syndrome (ARDS) using PrismaLun...

Mar 17th, 2023 - Food protein induced enterocolitis syndrome (FPIES), is a non-IgE mediated food allergy (FA) which seems to expand, and occurring in infancy. Prevalence of FPIES is unknown. In 2011, Katz published cumulative incidence of cow 'milk FPIES of 3 per 1000 new-borns, from prospective birth cohort in Israel. The offending food depend on the country, probably in relation to eating habits. Cow's milk (...

Mar 14th, 2023 - The overall objective of this randomized clinical trial is to test the effectiveness of a novel personalized approach to the surgical treatment of OSA in children with Down syndrome (DS). DS is a common disorder, affecting 1 in 691 births. The estimated prevalence of obstructive sleep apnea (OSA) in children with DS ranges from 45-83%, compared to 1-6% in the general pediatric population. Untre...

Mar 10th, 2023 - Background: Children with obstructive sleep apnea syndrome (OSAS) have apneas and disturbed sleep, which causes daytime symptoms such as neurobehavioral problems. OSAS is a significant cause of morbidity among children with an incidence of 1-3%, and a peak prevalence at 2-5 years of age. If left untreated it can cause severe complications including failure to thrive, cardiovascular complication...

Mar 10th, 2023 - Acquired AA patients will receive the experimental regimen of fludarabine with dose-reduced cyclophosphamide, with results in this prospective single arm experimental group evaluated in the context of our institutional historical experience using HD Cy regimens as well as published outcomes using both fludarabine and high-dose cyclophosphamide-based regimens for MRD-BMT in aplastic anemia. iBMF...

Mar 10th, 2023 - Up to 90% of known traumatic brain injuries are classified as mild, with many more injuries likely going undetected. While each of these individuals experiences somatic, cognitive, and/or affective symptoms acutely following the injury, an estimated 10-15% will continue to experience these symptoms for months up to a year post-injury. The persistence of symptoms for three months or greater is t...

Mar 10th, 2023 - PRIMARY OBJECTIVES: I. To evaluate the tolerability and feasibility of MLN4924 (pevonedistat) added to the 3-drug backbone of azacitidine (aza), fludarabine phosphate (fludarabine), and cytarabine re-induction for pediatric patients with recurrent/refractory acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). II. To define and describe the toxicities of MLN4924 (pevonedistat) when ...

Mar 10th, 2023 - The investigators welcome anyone (with or without a family history) with unexplained, non syndromic FSGS, nephrotic syndrome, or proteinuria to join the study. Participation involves a saliva (or blood if it is preferable) sample and urine sample (if applicable). There is no cost to participate and the study can be done from home in most cases.

Mar 10th, 2023 - Evaluation of the Efficacy and Safety of Oral Azacitidine plus Best Supportive care versus Placebo and Best Supportive care in subjects with red blood cell (RBC) transfusion-dependent anemia and thrombocytopenia due to International Prognostic Scoring System (IPSS) lower risk myelodysplastic syndromes (MDS).

Mar 10th, 2023 - PRIMARY OBJECTIVES: I. To establish recommended phase 2 dose (RP2D) of pomalidomide after liposome-encapsulated daunorubicin-cytarabine (daunorubicin and cytarabine liposome) induction. II. To compare the rate of overall complete response (CR)/complete response with incomplete hematologic recovery (CRi) with daunorubicin and cytarabine liposome + pomalidomide versus daunorubicin and cytarabine ...

Mar 10th, 2023 - This research is being done to find out if bone marrow transplantation (BMT) followed by chemotherapy will help people with aplastic anemia who have failed other treatments. You have a severe, life threatening disease (severe aplastic anemia) in your bone marrow. Your disease has come back or not responded after receiving one or more immunosuppressive treatments. High dose chemotherapy followed...

Mar 10th, 2023 - The overall objective of this study is to prospectively determine the epidemiology of children with PARDS and describe its management in Asia. The first specific aim is to determine the prevalence of PARDS among PICU admissions. This will be achieved by establishing a systematic manner of screening patients and including them in this study. The second specific aim is to describe the use of pulm...

Mar 10th, 2023 - R3R01 is investigational small molecule designed to decrease fat levels in certain cells in the kidney and therefore may improve kidney function and reduce damage in the kidney. This is a single arm open-label study enrolling patients in three cohorts. Cohort 1 will include 5 adult (≥18 y/o) patients from Cohorts 2 and 3 (including at least one patient from Cohort 2 and at least one patient fro...

Mar 10th, 2023 - During the first 7 days of the patient on mechanical ventilation (MV), measurements will be taken. For this, the patient will be in the supine position (DS) or the prone position (DP). The patient will be on a dose of analgesics, under sedation and muscle relaxation and will not present any respiratory stimulus. Data from MV will also be recorded (prior to recruitment assessment): tidal volume ...

Mar 10th, 2023 - This is an investigator-initiated open-label multi-institutional phase I study of venetoclax combination therapy in both myeloid and lymphoid hematologic malignancies. This study is designed as a basket trial with three separate cohorts. All cohorts include a dose finding portion (Part I) followed by a dose expansion at the Recommended Phase II dose (RP2D, Part II). This research study is looki...

Mar 10th, 2023 - Background: RASopathies are a group of neurodevelopmental genetic conditions caused by mutations affecting components within the RAS-map kinase (RAS-MAPK) cellular signaling pathway. Caregivers of children with a RASopathy are faced with numerous challenges related to the physical, cognitive, and behavioral symptoms associated with their child s condition. The use of virtual interventions has b...

Mar 10th, 2023 - Background: Myelodysplastic syndromes (MDS) are heterogenous stem cell disorders characterized by ineffective hematopoiesis resulting in cellular dysplasia, peripheral cytopenias, and increased risk for transformation to acute myeloid leukemia (AML). There are limited treatments options, all of which have unimpressive response rates and limited response durations, with the only potential cure b...

Mar 10th, 2023 - Background: The myelodysplastic syndromes (MDS) are a group of clonal bone marrow neoplasms characterized by ineffective hematopoiesis, cytopenia, and high risk for transformation to acute myeloid leukemia (AML). MDS is primarily a disease of the elderly, with about 80% of participants being older than 65-years of age; with 10,000 new diagnoses per year in the U.S. The only curative treatment f...

Mar 10th, 2023 - Study Description: We hypothesize that the use of advanced, computationally-based analytic techniques can provide insights into the causes, manifestations, and mechanisms of genetic diseases. To address this hypothesis, we will collect phenotypic and biologic data relevant to genetic conditions and will study computational tools that analyze these data. Objectives: Primary Objectives: To collec...

Mar 10th, 2023 - Recent published case reports and clinical experience of the investigators indicate Down Syndrome Regression Disorder (DSRD) may be successfully treated with immune-modulating therapies, in addition to current pharmacologic options. This study is a multidimensional clinical trial designed to advance the understanding of the etiology of DSRD and to evaluate the safety and efficacy of three disti...