Purpose: Evaluate the efficacy of prophylactic Emicizumab administered on a scheduled basis to prevent bleeds in patients with acquired hemophilia A (AHA).
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].
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.
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.
Evaluate night time hemoglobin desaturation (NHD), as part of routine clinical care, in subjects with sickle cell disease (SCD
We want to better understand how and why the dosing of and response to hydroxyurea is different for different patients.
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.
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.