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.
The purpose is to evaluate coagulation activation and the impact of iron status in patients with Hereditary Hemorrhagic Telangiectasia (HHT). Participants on this study will include patients with HHT, patients with iron deficiency +/- anemia but without HHT, and healthy volunteers The study team will procure single and/or serial blood samples from participants and assess various markers of hemostasis and activation of coagulation, and host iron status.
The primary objective of the study is to evaluate the safety and feasibility of treating patients with a history of iTTP with efgartigimod, an FcRn inhibitor that reduces IgG autoantibodies, including those responsible for ADAMTS13 depletion.
We want to test adults with sickle cell disease, using both the conventional in-person Montreal Cognitive Assessment (MOCA) and, within 2-4 months, the abbreviated 'MOCA-blind', which can be given remotely. In this way we can begin to evaluate whether these tests are sensitive to similar degrees of impairment when given remotely compared with in-person in people with sickle cell disease (SCD).
Study the effectiveness and safety of Epeleuton
Purpose: Evaluate the efficacy of prophylactic Emicizumab administered on a scheduled basis to prevent bleeds in patients with acquired hemophilia A (AHA).
This study will evaluate the use of MMUD PBSC in adults and explore the safety and efficacy of MMUD BM in pediatric recipients with hematological malignancies who may lack other donor options.
This study is being done to answer the following question: Do two different anti-cancer drugs, paclitaxel (PTX) and pegylated liposomal doxorubicin (PLD) have similar effects on treating KS in PLWH in sub-Saharan Africa? We are doing this study because we want to find out if one of these approaches, PTX or PLD, is better than the other for KS. Both drugs may be given as part of the usual approach for your KS. The usual approach is defined as care most people get for KS.
The main purpose of this clinical research study is to assess the safety, tolerability, efficacy (the ability of the study drug to produce the desired anti cancer effect), PK] and PD of GHF009X2101 the study drug in patients with relapsed or refractory hematologic malignancies including AML, CLL, SLL or other lymphomas [including other leukemia].