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.
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.
Purpose: Evaluate the efficacy of prophylactic Emicizumab administered on a scheduled basis to prevent bleeds in patients with acquired hemophilia A (AHA).
Study the effectiveness and safety of Epeleuton
To study individuals with sickle cell trait (SCT) and examine if their levels of hemoglobin S have any influence on red blood cells, clotting or kidney function.
To evaluate the safety, tolerability, and efficacy of GBT021601 in adult participants with SCD to determine an optimal dose.
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].
To evaluate the safety of GBT021601 and its effect on sickle cell complications
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 goal of this study is to uncover the frequency of low oxygen levels during sleep in pregnant women that have severe sickle cell disease. These women have a high risk of complications during pregnancy, and low oxygen levels might contribute to the complications. Therefore, finding and treating low oxygen can be impactful. The study will also look at blood cells and assess how low oxygen affects them.