A phase III randomized multi-center study designed to compare the efficacy of Crenolanib with that of Midostaurin when administered following induction chemotherapy, consolidation chemotherapy and bone marrow transplantation in newly diagnosed AML subjects with FLT3 mutation. About 510 subjects will be randomized in a 1:1 ratio to receive either crenolanib in addition to standard first line treatment of AML (chemotherapy and if eligible, transplantation) (arm A) or midostaurin and standard treatment (arm B). Potentially eligible subjects will be registered and tested for the presence of FLT3 mutation. Once the FLT3 mutation status is confirmed and additional eligibility is established, subject will be randomized and enter into the treatment phase.
The purpose of this study is to look at a new cell therapy, MT-401, and determine the best dose for treating AML. The study will also look at the safety, tolerability, and effectiveness of this new therapy.
People with chronic blood cancers called myeloproliferative neoplasms (MPN) have a significantly increased risk of stroke, heart attack, and blood clots compared to the general population. This blood clot risk is only slightly reduced by current treatment. In this study, we are exploring blood tests that may help us learn why people with MPN clot and how current MPN treatment affects those blood tests. This is a blood draw-only study.
To determine the prevalence and severity of heavy menstrual bleeding in women with Hereditary Hemorrhagic Telangiectasia (HHT). This study will help us understand how to determine if this is a clinical feature of HHT.
We are developing a computer-based tool for people who have cancers of the blood, bone marrow, and lymph nodes. This tool will help us understand what is most important to patients when it comes to the results of their treatment. We are looking for healthy volunteers (those without cancer) to help us test the usability of this tool.
The purpose of this study is to learn more about brain AVMs (BAVMs) and bleeding in HHT. We plan to study risk factors for rupture of BAVMs and also for other bleeding and features of HHT disease, including primarily genetics and imaging characteristics of the BAVMs. We hope that knowledge about risk factors will help improve the care and management of HHT patients.
To have a well phenotyped cohort of people with sickle cell disease in order to examine risk factors for end organ disease through the creation of a multi-site registry and to develop a repository for the long-term storage of patient samples (whole blood, peripheral blood, serum) along with corresponding demographic and clinical information to allow for clinical and laboratory correlations.
The purpose of this study is to see if an investigational drug called pazopanib can improve nosebleeds and anemia in patients with hereditary hemorrhagic telangiectasia.
The purpose of this research study is to improve safety measures and reduce stressors associated with lumen hub-clave seizures when caring for patients with CVCs. The primary outcome of the pilot study is to document the types and frequency of workarounds employed, current disinfection techniques, and the stressors of those caring for and accessing CVCs.
To further the scientific knowledge base for the diagnosis, understanding, and management of sickle cell disease (SCD) by assembling data collected in routine clinical care and closed clinical trials. This umbrella Data Hub Protocol will permit the creation of a patient-level data repository obtained from individuals, or the records of individuals, with SCD thereby collecting key information and identifying gaps that will help advance SCD treatment and research.